| Study Title | Brief Summary | Conditions | Sex | Age | Locations |
|---|---|---|---|---|---|
| Nephropathy in Patients With Sickle Cell Disease | There are some diseases that give rise to diverse renal manifestations as does sickle cell disease | Sickle Cell Nephropathy | ALL | CHILD, ADULT | |
| Biological, Genetic and Environmental Involved in the Complications of Sickle Cell Disease | The objective of the study is to refine our knowledge on the physiopathology of the symptoms and the complications for the patients affected by a drepanocytic syndrome. The establishment of risk factors and indicators of severity will allow to target better the patients requiring an adequate strategy in order to prevent the installation of some complications or to limit their worsening. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Erasme Hospital, Brussels, 1070, Belgium |
| Sickle Cell Disease Obstetric Multi-Disciplinary Care Programme | To implement an effective but low-cost strategy to decrease SCD maternal and perinatal mortality in Ghana. The objectives are to 1) assess the impact of a multidisciplinary SCD-obstetric team for decreasing mortality across three hospital sites in Ghana. 2) assess the implementation fidelity for 2a) preventing and 2b) treating acute chest syndrome in pregnant women with SCD admitted to the hospital. 3) standardize an ultrasound protocol for the prospective monitoring of fetal growth among pregnant women with SCD. | Sickle Cell Disease|Pregnancy Related | FEMALE | ADULT | Greater Accra Regional Hospital, Accra, Ghana|Korle Bu Teaching Hospital, Accra, Ghana|Tamale Teaching Hospital, Tamale, Ghana |
| Patient and Public Involvement and Engagement in Research With Children and Young People With Sickle Cell Disorder and Their Families | Aim: To co-produce resources for inclusive and equitable Patient and Public Involvement and Engagement in research on life-limiting conditions, with children and young people with sickle cell disorder and their families. Methods: Workshops with a) members of a patient advocacy organisation (Sickle Cell Society n=5) b): i) Children and young people (10-18 years) with sickle cell disorder (n=15) and ii) their siblings (10-18 years, n=10) and iii) their parents (n=15), c) Researchers form the Cicely Saunders Institute Outputs: Resources that enable children and young people with sickle cell disorder and their families to engage in research | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | King's College London, London, SE5 9RS, United Kingdom |
| Screening of Coexistence Between Sickle Cell Anaemia and G6PD Deficiency | G6PD deficiency may have a subtle effect on the severity of hemolysis and also worsen the degree of anaemia in SCD when the two disorders coexist. Therefore, selective decision should be taken in patients in whom the two conditions coexist in the choice of drug and in the treatment of infections.The prevalence of the G-6-PD deficiency is high in SCD patients, but does not differ from that observed among non-SCD subjects .However, the G-6-PD deficiency appears to worsen the clinical features of SCD, there were more hospitalizations, major vaso-occlusive crises among G-6-PD deficient sickle cell patients. | Sickle Cell Disease and G6PD Deficiency | ALL | CHILD | |
| Sleep and Pain in Sickle Cell Disease | This is a study testing the effects of behavioral sleep interventions on pain and brain function in sickle cell disease. | Sickle Cell Disease|Sleep Disturbance|Pain | ALL | ADULT, OLDER_ADULT | Johns Hopkins, Baltimore, Maryland, 21224, United States |
| Study of Vitamin B12 Metabolism in Children with Sickle Cell Disease Exposed to MEOPA | Short description of the protocol intended for the lay public. Include a brief statement of the study hypothesis (Limit : 5000 characters) The sickle cells anemia is a monogenic disease linked to the presence of Hemoglobin S due to a mutation in the Hemoglobin Beta chain. The lack of circulating oxygen induces a polymerization of the Hemoglobin S which change the red cell conformation into sickle. Those cells interact and causes vaso-occlusive crisis (CVO). The MEOPA is a medical gas used as an antalgic and a sedative especially in sickle cells disease patients. The nitrous oxide, oxide the cobalt ion in the vitamin B12 which inactivate it irreversibly creating a functional deficiency. During the metabolism of vitamin B12, homocysteine is transformed in methionine which is used in to form the myelin sheath and helped in producing DNA. Numerous studies already shown that the longer the exposition to MEOPA is the greater the functional deficiency of vitamin B12 occur. A few studies shown a symptomatic deficiency of vitamin B12 due to the exposition of MEOPA in sickle cells patient but there is no explanation on the necessary amount of exposure or if some patients are more at risk. When there is a deficiency of vitamin B12 the symptoms can go from a simple orthostatic hypotension to a combined spinal sclerosis. The participation to the study will be proposed to every patient hospitalized for a CVO in the follow up of the emergency room visit or directly in pediatric reanimation. During a usual blood test, a small amount of blood (4mL) will be collected in addition to dose the Vitamin B12, the vitamin B9, the homocysteine, and the methionine. A small amount of urine will also be collected to dose the methylmalonic acid, all those elements are a part of the metabolism of B12 vitamin. The same sample will be taken on the day of departure of the hospital. During the hospitalization the pain management, a daily neurological exam, and the exposition to the MEOPA will be assessed meticulously. An appointment will take place at 7 days and at one month after the hospital departure to evaluate the possible neurological defect. Each patient can only be included once. | Sickle Cells Patients | ALL | CHILD, ADULT | Chu Reims, Reims, 51092, France |
| Sickle-cell Disease Registry of the GPOH | Sickle cell disease is one of the most common hereditary diseases. Most severe complications can be avoided if the disease is detected early and treated appropriately. The sickle cell disease registry of the Society for Paediatric Oncology/Haematology aims at describing the epidemiology of sickle cell disease in German-speaking central Europe. Patients with sickle cell disease will be characterized clinically and genetically and treatment will be documented with the aim to find predictors of the course of disease. In addition, the registry results should provide a solid evidence base to incorporate sickle cell disease into routine newborn screening and to update the national guidelines for the management of patients suffering from sickle cell disease in Germany. A consortium of five university hospitals (Berlin, Frankfurt, Hamburg, Heidelberg, Ulm) has been mandated by the Society for Paediatric Oncology/Haematology to implement this registry. The number of participating centers is constantly increasing and new centers that take care of either pediatric or adult patients with sickle cell disease are encouraged to support the registry. For further information please refer to: http://www.sichelzellkrankheit.info/ | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Center for Child and Adolescent Medicine, University Medical Center Heidelberg, Heidelberg, BW, 69124, Germany |
| Red Cell Half Life Determination in Patients With and Without Sickle Cell Disease | Background: Sickle cell disease (SCD) is an inherited blood disorder. It results from a single genetic change (mutation) in red blood cells (RBCs). RBCs are the cells that carry oxygen to the body. In people with SCD, some RBCs are abnormal and die early. This leaves a shortage of healthy RBCs. Researchers want to learn more about how long RBCs live in the human body. Objective: To study how long RBCs live in people with and without SCD. Eligibility: People age 18 and older who either have SCD, had SCD but were cured with a bone marrow transplant, have the sickle cell trait (SCT), or are a healthy volunteer without SCD or SCT Design: Participants will be screened with a medical history and physical exam. They will give a blood sample. Participants will have a small amount of blood drawn from a vein. In the laboratory, the blood will be mixed with a vitamin called biotin. Biotin sticks to the outside of RBCs without changing their function, shape, or overall lifetime. This process is known as biotin labeling of RBCs. The biotin labeled RBCs will be returned to the participant via vein injection. Participants will give frequent blood samples. Their RBCs will be studied to see how many biotin labeled RBCs remain over time. This shows how long the RBCs live. Participants will give blood samples until no biotin labeled RBCs can be detected. During the study visits, participants will report any major changes to their health. Participation lasts for up to 6 months. | Sickle Cell Disease|Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Sickle Cell Disease, Neurocognitive Disorders, Social Participation | This project will promote the development of transdisciplinary analyses. Neuropsychological disorders will be explored with the usual appropriate tests done by psychologists and neuropsychologists regularly involved in the management of sickle cell disease affected children. For the social sciences' component, various methods will be used: Measure of the Life habits (MHAVIE), Measure of Environmental Quality (MQE) and semi-guided interviews will complete the collection of qualitative data. The expected results concern the identification of the barriers or facilitators the sickle cell patients might face in their social participation, whether they are affected or not by neurological disorders. | Sickle Cell Disease | ALL | CHILD | CHU de la Guadeloupe, Pointe-à-Pitre, France, 97139, Guadeloupe |
| The Relationship of Platelet Counts With Sickle Cell Anemia in the Eastern Region of Saudi Arabia | This study aims to evaluate the relationship between platelet counts and sickle cell disease in the eastern region of the Kingdom of Saudi Arabia. | Sickle Cell Anaemia|SCD | ALL | CHILD, ADULT, OLDER_ADULT | Khalid Ali Majrashi, Dammam, Saudi Arabia |
| Siplizumab for Sickle Cell Disease Transplant | The purpose of this study is to find out whether siplizumab is safe and effective for patients with SCD undergoing an allogeneic transplant and to prevent development of Graft versus Host Disease (GVHD) and graft failure. The main goals of this study are : * To determine if acute GVHD occurs and how severe the acute GVHD is in subjects receiving the study drug * To determine if graft failure occurs in subjects receiving the study drugs In this study, participants will receive 5 infusions of the study drug, siplizumab, while getting a stem cell transplant for SCD. Before siplizumab infusion, participants will be given medications to reduce the risks of allergic reaction to the drug. | Anemia, Sickle Cell | ALL | ADULT | Columbia University Irving Medical Center, New York, New York, 10032, United States |
| Transfusion in Sickle Cell Disease: Screening of Sickle Cell Disease Trait in Blood Donors | Bearers of the sickle cell allele (S) are currently eligible for blood donations in Belgium. As blood donors are not tested for this allele, their heterozygous status is unknown. However, guidelines recommend to transfuse sickle cell patients with blood that is negative for the 'S' hemoglobin. To the investigator's knowledge, no study has been conducted to evaluate the impact of transfusion with blood originating from heterozygous donors on the transfusion performance and the improvement of clinical status of the sickle cell disease patients. | Sickle Cell Disease|Sickle Cell Trait | ALL | CHILD, ADULT, OLDER_ADULT | CHU Brugmann, Brussels, 1020, Belgium|HUDERF, Brussel, 1020, Belgium |
| Sickle Cell Disease, Hemechip | Sickle cell disease is very common in Nigeria. Early diagnosis is important to prevent or reduce serious complications from the disease and to enable children stay healthy. To this end, the investigators would like to test a new, simple and quick device called the HemeChip to determine if it can detect whether or not someone has sickle cell disease. The investigators will compare the results obtained with the HemeChip with a standard method of diagnosing sickle cell disease known as Isoelectric focusing (IEF) or High Performance Liquid Chromatography (HPLC).If the investigators show that the new device can differentiate between children who have sickle cell disease and those who don't as successfully as the IEF or HPLC, they estimate a sharp increase in the use of this device in many countries especially in Africa due to its lower cost | Sickle Cell Disease | ALL | CHILD | University of Nebraska Medical Center, Omaha, Nebraska, 68198, United States|University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, 27599, United States|Case Western Reserve University, Cleveland, Ohio, 44106, United States|University Hospitals Cleveland Medical Center, Cleveland, Ohio, 44106, United States|Aminu Kano Teaching Hospital, Kano, Nigeria|Hasiya Bayero Pediatric Hospital, Kano, Nigeria|Murtala Mohammed Specialist Hospital, Kano, Nigeria |
| Venous Thrombosis Biomarkers in Sickle Cell Disease and Sickle Cell Trait | Background: Venous thromboembolism (VTE) includes the abnormal clotting of blood in a deep vein of the upper or lower limbs (deep vein thrombosis) that may travel to and block a blood vessel in the lung (pulmonary embolism). Some people with sickle cell disease (SCD)-a red blood cell disorder-seem to be at greater risk for developing these blood clots. Researchers want to study the blood of people with SCD and VTE as well as healthy people to develop better treatments to prevent blood clots. Objective: To study blood clotting in SCD because it is the most common cause of vascular death after a heart attack or stroke. Eligibility: People ages 18-80 who have SCD (with or without a history of blood clots) or the trait for SCD, and healthy volunteers Design: Participants will be screened with medical history, physical exam, and medical records review. They will give blood samples. Participants will have phone calls either every 3 months or once a year, for 2 years. They will give updates on their health. They may give additional medical records. The phone calls may last up to 30 minutes. If participants have a VTE or pain crisis episode, they may visit the Clinical Center. These visits may last up to 4 hours. They will repeat the screening tests and give blood samples. Some participants may be invited to take part in blood studies. After 2 years, some participants will have a follow-up visit at the Clinical Center. Participation will last for about 2 years. | Sickle Cell Disease|Venous Thrombosis|Sickle Cell Trait|Hypercoagulable State|Venous Thromboembolism | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Vitamin D and Bisphosphonates in the Treatment of Sickle Cell Disease | Sickle cell disease (SCD) is a worldwide distributed hereditary red cell disorder, which affects approximately 75,000 individuals in the United States and almost 20,000- 25,000 subjects in Europe, this latter mainly related to the immigration fluxes from endemic areas such as Sub-Saharian Africa to European countries. Studies of global burden disease have pointed out the invalidating impact of SCD on patient quality of life. This requires the development of new therapeutic options to treat sickle cell related acute and chronic complications. SCD is caused by a point mutation in the β-globin gene resulting in the synthesis of pathological hemoglobin S (HbS). HbS displays peculiar biochemical characteristics, polymerizing when deoxygenated with associated reduction in cell ion and water content (cell dehydration), increased red cell density and further acceleration of HbS polymerization. Pathophysiological studies have shown that dense, dehydrated red cells play a central role in acute and chronic clinical manifestations of SCD, in which intravascular sickling in capillaries and small vessels leads to vaso-occlusion and impaired blood flow with ischemic/reperfusion injury. In microcirculation, vaso-occlusive events (VOC) result from a complex and still partially known scenario, involving the interactions between different cell types, including dense red cells, reticulocytes, abnormally activated endothelial cells, leukocytes, platelets and plasma factors. Target organs, such as bone or lung, are involved in both acute and chronic clinical manifestations of SCD, related to their peculiar anatomic organization mainly characterized by sluggish circulation and relative local hypoxia. VOCs combined with marrow hyperplasia and inflammation has been suggested to contribute to the development of sickle bone disease (SBD). Recently, it has been proposed a possible role of vitamin D deficiency in SBD, which appears to be subordinated to the primary defect in bone homeostasis. In a humanized mouse model for SCD, we recently reported that SBD is due to imbalance between osteoblast/osteoclast activity induced by recurrent VOCs. In addition, we show that zoledronic acid prevents bone impairment related to SCD, reducing osteoclast activity and improving osteoblast performance. | Sickle Cells Disease | ALL | ADULT | Ospedali Galliera - S.S.D. Microcitemia, anemie congenite e dismetabolismo del ferro, Genova, 16128, Italy|Ospedali Galliera - S.S.D. Ortogeriatria per intensità di cure, Genova, 16128, Italy|Università degli Studi di Verona, Verona, 37129, Italy |
| Hematopoietic Stem Cell Transplant for Sickle Cell Disease | This is a study of patients with sickle cell disease. It aims to find out if people with sickle cell disease can be cured by changing their immune system before they have blood stem cell transplants. Doctors will give patients a new drug (fludarabine) to see if this drug changes patients immune system and reduces the patient's cells (host) from rejecting donor cells (graft) after the patient gets a Hematopoietic (blood) stem cell transplant. | Sickle Cell Disease|Sickle Cell Anemia|SCD | ALL | ADULT, OLDER_ADULT | University Hospitals Seidman Cancer Center, Case Comprehensive Cancer Center, Cleveland, Ohio, 44106, United States |
| Sickle Cell Disease Biofluid Chip Technology (SCD BioChip) | 'Sickle-shaped' anemia was first clinically described in the US in 1910, and the mutated heritable sickle hemoglobin molecule was identified in 1949. The pathophysiology of SCD is a consequence of abnormal polymerization of sickle hemoglobin (HbS) and its effects on red cell membrane properties, shape, and density, and subsequent critical changes in inflammatory cell and endothelial cell function. Our goal is to understand the impact of CMA abnormalities in SCD, by interrogating a number of recognized interactions in a range of clinical phenotypes. To date, correlative studies in SCD, by us and others, have range between clinical reports, based on tests, interventions, and chart review of individuals or groups of individuals and, at the other extreme, identification of functional gene polymorphisms based on population studies. The investigators wish to augment these studies through a systematic examination of cellular membrane properties and activation status. Of hematologic disorders, SCD may be unusually susceptible to such an examination. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | University Hospitals Case Medical Center, Cleveland, Ohio, 44106, United States |
| Cardiovascular Complications of Sickle Cell Disease | In this research study, we are using heart imaging exams and blood testing, in order to gain an improved understanding of the pulmonary (lung) hypertension and cardiovascular (heart) complications that often occur in sickle cell patients. Information gathered from the healthy volunteers that participate in this study will be compared to information from the sickle cell patients in this study in order to help further our understanding. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of Chicago Medical Center, Chicago, Illinois, 60430, United States |
| Evaluation of Spectra Optia Red Blood Cell Exchange in Sickle Cell Patients | The purpose of this study is to evaluate the performance of the Spectra Optia system red blood cell exchange (RBCx) protocols (exchange and depletion/exchange) in study participants with sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Children's of Alabama, Birmingham, Alabama, 35233, United States|Children's Hospital and Research Center at Oakland, Oakland, California, 94609, United States|University of Colorado at Denver, Aurora, Colorado, 80045, United States|Kosair Children's Hospital, Louisville, Kentucky, 40202, United States|Johns Hopkins Medical, Baltimore, Maryland, 21205, United States |
| Molecular Phenotyping of Asthma in Sickle Cell Disease | Asthma and sickle cell disease each are serious medical problems. People with asthma have difficulty breathing, wheeze (a whistling noise when breathing), cough, produce sputum or phlegm, and have inflammation (swelling, irritation, redness) and narrowing of the bronchial tubes. When a person has both asthma and sickle cell disease together, more serious medical problems can occur such as having acute chest syndrome and pain episodes more often. It is sometimes hard to diagnose asthma in a person with sickle cell disease because sickle cell disease can also cause lung problems. The purpose of this study is to see if the investigators can better understand asthma when it occurs in a person who has sickle cell disease. The investigators will do this by taking a blood, urine, and saliva sample. The blood and urine samples will be analyzed for chemicals and DNA (genes). Certain genes can cause patients to have sickle cell disease or asthma. The investigators will use the saliva sample for future studies to compare the results from the blood testing with saliva. The investigator's long-term goal is to make sure people who have asthma and sickle cell disease are getting the best asthma treatments. The investigator's hypothesis is that the analysis of the blood, urine and saliva using a method called, metabolomics, may identify a unique asthma signature in children with sickle cell disease which may lead to targeted treatments. | Sickle Cell Disease|Asthma | ALL | CHILD, ADULT | Nemours Children's Clinic, Wilmington, Delaware, 19803, United States|Nemours Children's Clinic, Jacksonville, Florida, 32207, United States|Nemours Children's Clinic, Orlando, Florida, 32827, United States |
| Priapism in Boys and Men With Sickle Cell Disease - Demographics, Characteristics and Prevalence | Priapism, a prolonged erection of the penis, is a medical issue that often affects men with sickle cell disease. The purpose of this study is to collect demographic and clinical information on priapism by interviewing men with sickle cell disease. | Priapism|Anemia, Sickle Cell | MALE | CHILD, ADULT, OLDER_ADULT | University of South Alabama, Mobile, Alabama, 36688-0002, United States|University of California, Davis, Davis, California, 95616, United States|Children's Hospital and Research Center at Oakland & Summit Medical Center, Oakland, California, 94609, United States|Kaiser Permanente, Oakland, Oakland, California, 94611, United States|University of California, San Francisco, San Francisco, California, 94143, United States|University of Colorado Health Sciences Center, Denver, Colorado, 80206, United States|University of Florida, Gainesville, Florida, 32610-0383, United States|Kosair Children's Hospital, Louisville, Kentucky, 40202, United States|Brigham and Women's Hospital at Harvard Medical School, Boston, Massachusetts, 02115, United States|Children's Hospital of Boston, Boston, Massachusetts, 02115, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|Wayne State University, Detroit, Michigan, 48202, United States|Children's Hospital Montefiore, Bronx, New York, 10467-2940, United States|Montefiore Medical Center and Children's Hospital at Montefiore, Bronx, New York, 10467, United States|University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, 27599, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States|University of Cincinnati Hospital, Cincinnati, Ohio, 45267, United States|Ohio State University, Columbus, Ohio, 43210, United States|University of Oklahoma Health Sciences Center, Oklahoma City, Oklahoma, 73104, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Thomas Jefferson University Hospital, Philadelphia, Pennsylvania, 19107, United States|St. Christopher's Hospital for Children, Philadelphia, Pennsylvania, 19134, United States|St. Jude Children's Hospital, Memphis, Tennessee, 38105, United States|University of Texas Parkland Health & Hospital System, Dallas, Texas, 75390-8852, United States|University of Texas Children's Medical Center of Dallas, Dallas, Texas, 75390, United States|University of Texas Medical Branch at Galveston, Galveston, Texas, 77555, United States |
| Evaluation of the Impact of Red Blood Cell Exchange on Thrombo-inflammation in Sickle Cell Disease | Sickle cell disease is the most common inherited blood disorder worldwide. It is a hemoglobinopathy characterized by chronic hemolysis, endotheliopathy, coagulation activation, and chronic inflammation. It is a multisystemic disease leading to acute (vaso-occlusive crisis, acute chest syndrome, stroke...) and chronic complications with multiorgan damage. Thrombo-inflammation is defined by the cooperation and interaction between hemostasis and the innate immune system. The platelet represents the cornerstone of this phenomenon, being at the interface of these two systems. In sickle cell disease, platelets are activated and release cytokines, leading to a pro-coagulant and pro-inflammatory state. Transfusion, whether occasional or chronic, is a major sickle cell disease treatment. It is common to distinguish simple transfusion from exchange transfusion. The latter involves replacing a given volume of sickle red blood cells with healthy red blood cells. Exchange transfusion allows avoiding an excessive increase in hemoglobin. The decrease of hemoglobin S under 30% achieved by red blood cell exchange reduces the risk of stroke by more than 90% in children with cerebral vasculopathy. Moreover, transfusion can be used in acute complications such as vaso-occlusive crisis and acute chest syndrome. Despite this efficacy, a subgroup of patients is not totally protected against acute and chronic complications. The persistence of chronic inflammation is suggested. To date, it is not known if red blood cell exchange can reduce the thrombo-inflammatory dynamic in sickle cell disease. The aim of this study is to evaluate the impact of red blood cell exchange on thrombo-inflammatory parameters in 20 adult sickle cell patients (10 patients on manual exchange and 10 patients on automatized exchange). | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Oncopole - Toulouse Hospital, Toulouse, 31059, France |
| Prevalence of Osteoporosis in Sickle Cell Disease | Sickle cell disease is the most common single-gene disease in the world. Its prevalence is increasing in France, with patients' life expectancy increasing into developed countries. It mainly affects populations originating from sub-Saharan Africa. Among the chronic bone complications associated with sickle cell disease, osteoporosis has previously been highlighted but remains a poorly known complication in this very particular context. A dedicated evaluation of osteoporosis and associated risk factors in sickle cell disease patients living in France may enable better bone management of these patients in the future, as this problem, specific to their disease, is likely to become more frequent as their life expectancy increases. This is a prospective interventional and monocentric study whose objective is to describe the prevalence of osteoporosis in black patients with sickle cell disease in France | Sickle Cell Disease | ALL | ADULT | Hôpital Edouard Herriot, Lyon, 69437, France |
| Sleep Apnea in Sickle Cell Disease | Despite the fact that obstructive sleep apnoea (OSA) is highly prevalent in the sickle cell population, studies focusing on the associations of the two diseases and their common pathophysiological mechanisms are scarce. OSA is one of the most common conditions responsible for hemoglobin desaturation. The nocturnal hemoglobin desaturation occurring in some sickle cell disease (SCD) patients with OSA could trigger hemoglobin S polymerization and red blood cell (RBC) sickling, leading to further blood rheological alterations, hence increasing the risks for VOC. Moreover, OSA has been demonstrated to increase oxidative stress and inflammation in non Sickle Cell Disease (SCD) patients, which, in SCD patients, could increase the risk for complications. Finally, OSA is accompanied by impaired vascular function and autonomic nervous system dysfunction in the general population. Indeed, the presence of OSA in SCD could increase the clinical severity of patients and the frequency of VOC. | Sickle Cell Disease | ALL | CHILD, ADULT | Hôpital Edouard Herriot, Lyon, 69003, France|Centre Léon Berard, Lyon, 69008, France|Hôpital de la Croix Rousse, Lyon, 69317, France |
| Hydroxyurea and EPO in Sickle Cell Disease | The proposed study is a Phase 1/2 multi-center study evaluating the safety and efficacy of erythropoietin (EPO) in combination with hydroxyurea in the treatment of chronic anemia in patients with sickle cell disease (SCD). | Anemia, Sickle Cell|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | UPMC, Pittsburgh, Pennsylvania, 15213, United States|Lagos University Teaching Hospital, Lagos, 102215, Nigeria |
| Hydroxyurea Treatment for Adult Sickle Cell Anemia Patients in Kinshasa | The goal of this clinical trial is to evaluate the efficacy of hydroxyurea (HU) in improving disease severity in adult patients with sickle cell anemia in Kinshasa (Democratic Republic of Congo). This study aims to: * assess the safety and efficacy of HU treatment in the Congolese environment; * assess the reversibility of chronic cardiac lesions. Participants will take hydroxyurea for two years. The effects of the treatment will be evaluated periodically by clinical evaluation, biological tests, and echocardiographic exploration. | Sickle-Cell Anaemia | ALL | ADULT | University of Kinshasa, Kinshasa, Congo, The Democratic Republic of the |
| Immunogenicity Study of an Anti-pneumococcal Vaccination Strategy in Patients With Sickle Cells Disease | Streptococcus pneumoniae is the major cause of bacterial infection in patients with sickle cells disease. The 23-valent pneumococcal polysaccharide vaccine (PSV) is supposed to be poorly immunogenic in these patients. We want to evaluate whether a prime with a 13-valent pneumococcal conjugate vaccine (PCV), able to induce immunologic memory, would improve the immune response against SP polysaccharides (SPP). Primary objective: To evaluate and compare the specific antibody response to a prime-boost vaccine strategy combining PCV prime at W0 followed by the administration of PSV boost at W4, to the administration of PSV alone at W4 in patients with sickle cells disease. Secondary objectives: Evaluation and comparison of the specific antibody response to the thirteen pneumococcal serotypes shared by the PCV and PSV vaccines, 4 weeks after the single PSV vaccination for patients from Group 1 or 4 weeks after the boost PSV vaccination for patients from group 2. Evaluation of the duration of the specific antibody response at W24 and 96. Evaluation of the T CD4 lymphocyte response to the CRM 197 protein. Safety of the vaccines. Study Design: Randomised, monocentric, controlled phase II study of the immunological efficacy of a prime boost strategy combining the sequential administration of the PCV and PSV, compared to the administration of the PSV alone. 180 adults patients with sickle cells disease will be included. The primary endpoint : proportion of responders at W8 to at least 10 of thirteen serotypes. Secondary endpoints : Proportion of responders at W8 according to 4 categories of responders: 5-7; 3-4; 2-1 and 0. Evaluation of the pneumococcal opsonophagocytic activity (OPA) at baseline and W8 for each serotype, defined as the proportion of patients with OPA \> 1:8 geometric mean of the specific antibody titers proportion of patients who experienced an increase of specific antibody levels 1 g/ml. Evaluation of the priming effect of the PCV vaccine in the group 1. Duration of the specific antibody responses at week 24 and W96. CD4 T lymphocyte responses to the CRM 197 protein (proliferative and cytokine production) at weeks 0, 8 and 12. Safety of the vaccines frequency of Streptococcus pneumoniae infections. Statistical Considerations: With a sample size of 180 patients, and a randomization ration of 1:1, the study will have a power of at least 90% to show a difference of 25% category between the group receiving PCV and PSV vs the group receiving PSV alone (two-sided type I error = 5%). The primary comparison between both groups will be performed using a Chi2 test for independent groups or a Fisher exact test where appropriate. | Invasive Pneumococcal Infections|Sickle Cells Disease | ALL | ADULT, OLDER_ADULT | Henri Mondor Hospital, Creteil, 94010, France |
| COVID-19 Vaccination Hesitancy in Adults With Sickle Cell Disease | The goal of this clinical trial is to test an COVID-19 vaccination information video in adults with sickle cell disease. The main questions it aims to answer are why are some adults with sickle cell disease hesitant to receive COVID-19 vaccination and whether a COVID-19 vaccination information video tailored for people with sickle cell disease will reduce vaccine hesitancy. Participants will complete a brief survey before and after watching a short video with information on vaccine safety, efficacy, and the greater impact of COVID-19 infection on people with sickle cell disease. | Sickle Cell Disease|COVID-19 Vaccine|Vaccine Hesitancy | ALL | ADULT, OLDER_ADULT | Duke Adult Sickle Cell Program, Durham, North Carolina, 27710, United States |
| Iron Mediated Vascular Disease in Sickle Cell Anemia Patients | The purpose of this research study is to determine the frequency and severity of iron overload in patients with Sickle Cell Anemia and its relationship to blood vessel function. The investigators hypothesize that intermittent transfusions that these patients receive during hospitalizations produces significant iron overload and impairs blood vessel relaxation. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital Los Angeles, Los Angeles, California, 90027, United States |
| CHOICES3: Sickle Cell Disease Parenting CHOICES | The study will use web-based data collection (SCKnowIQ) and intervention delivery strategies enhanced by nudges and tailored boosters in a sample of 430 adult men and women, aged 18-45 yr with SCD (Sickle Cell Disease) or SCT (Sickle Cell Trait), at-risk, and planning within 2 years to have a child free of SCD. | Sickle Cell Disease|Sickle Cell Trait | ALL | ADULT | University of Florida, Gainesville, Florida, 32611, United States |
| Preventing Sickle Cell Kidney Disease | Untreated hypertension and renal injury are risk factors for increased morbidity and mortality in sickle cell disease, yet early markers of progressive disease have not been identified and therapies to prevent the development of adverse cardiovascular outcomes have not been defined. Circadian blood pressure, as defined by 24 hour blood pressure monitoring, is more accurate than clinic blood pressure in defining secondary hypertension and abnormal nocturnal blood pressured dipping and nocturnal hypertension have been linked to progressive renal disease in other diseases. Methodology/Aims: A randomized feasibility trial of losartan will be conducted among adolescent HbSS and SB0 thalassemia patients (11-19 years) with abnormal nocturnal blood pressure dipping. During this six month feasibility trial, two dosing strategies of losartan (titrated to keep clinic BP \<95th percentile vs. \<75th percentile) will be analyzed for safety and effect on restoring normal circadian blood pressure. A prospective cohort study among HbSS and SB0 thalassemia patients (6-19 years) will also be conducted to evaluate the incidence of hypertension and role of monitoring potential biomarkers of kidney injury and hypertension. Cohort participants will undergo annual evaluations of hypertension(24 hour blood pressure monitoring for participants ≥ 11yrs, clinic BP in all participants) and markers of kidney injury/hypertension. Expected Results: At the completion of the feasibility trial, vital background information will be obtained to design a definitive multicenter trial of hypertension in sickle cell disease. At the completion of the cohort study, the incidence of pediatric hypertension will be identified and the role for monitoring blood and urine biomarkers will be better understood. As therapy for patients with renal failure is dismal, it is imperative that SCD patients at risk are identified early and that therapeutic trials are conducted that prevent progression. | Anemia, Sickle Cell|Sickle Cell Disease|Kidney Disease|Hypertension|Proteinuria | ALL | CHILD, ADULT | University of Alabama at Birmingham, Birmingham, Alabama, 35223, United States |
| RH Genotype Matched RBC Transfusions | To determine the feasibility and efficacy of matching donor red cells by RH genotype for a cohort of chronically transfused patients with SCD. | Sickle Cells Disease | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States |
| A Sickle CEll Disease ComplicatioN Trial | The objective of this study is to assess the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in sickle cell disease (SCD) subjects receiving SC411 compared to those subjects receiving placebo. | Sickle Cell Disease | ALL | CHILD | |
| Evaluation of Sickle Cell Liver Disease | Background: - Sickle cell disease changes the shape of red cells. This makes them more likely to break down as they get stuck in small blood vessels. This leads to low red cell count and also damage to small blood vessels that supply many organs. One of the affected organs is the liver. Sickle cell disease and its treatment through blood transfusion can lead to significant liver damage. This disease also can cause the liver to regrow abnormally after damage. This can cause high blood pressure in the liver. Researchers want to know if curing sickle cell disease with a stem cell transplant improves liver damage. Objectives: - To explore specific factors that improve or worsen sickle cell liver disease after a stem cell transplant. Eligibility: - Adults ages 18 and older with sickle cell liver disease. Design: * Participation will take approximately 7 days over 2 years. * Visit 1: participants will be screened with medical history and review of current treatment regimen. * Visit 2: participants will return to the clinic for explanation of the study and physical exam. They will also have blood and urine tests, and scans of the liver. * All participants will have a 2-night stay at the clinic. They will have a liver biopsy and a test of liver pressure. They will be sedated and a tube will be inserted in a vein in their neck. * Participants who have a stem cell transplant will have a second biopsy about 24 months later. * Over the 2-year study period, participants will have blood drawn 2-4 times and stool samples collected 2 times. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| Uganda Sickle Surveillance Study (US-3) | It is estimated that over 250,000 babies are born with sickle cell disease (SCD) annually in sub-Saharan Africa, and only 10% - 50% of them survive beyond five years of age. Data describing the magnitude of the sickle cell problem are lacking in most African countries. The available data on prevalence were mainly from older studies and small numbers of hospitalized patients. In Uganda, approximately 25,000 children are born with SCD but 70-80% die before their 5th birthday. Lehmann and Raper found 'sicklaemia' prevalence of 0.8% and 45% in the Sebei and Bambaa ethnic groups, respectively. A recent study found a SCT and SCD prevalence of 3% - 19% and 0% - 3%, respectively but this study addressed only 5 of Uganda's 111 districts and used a small convenience sample of children aged 6 - 60 months. The objective of this study is to determine the prevalence and map out the burden of SCT and SCD in Uganda. | Sickle Cell Disease | ALL | CHILD | Makerere University, Kampala, Uganda|Minister of Health, Kampala, Uganda|National Coordinator EID Program, Kampala, Uganda|Sickle Cell Clinic Department of Pediatrics & Child Health Mulago Hospital, Kampala, Uganda |
| Improving Disease Knowledge in Adolescents With Sickle Cell Disease | Increased knowledge about an illness can increase self-management among those afflicted. In order to facilitate people with sickle cell disease living a longer and healthier life, they should be taught to manage their illness.An adolescent with a chronic illness has many unique challenges, in addition to maneuvering the turbulent adolescence period itself. It has been that better knowledge and more positive perceptions of their illness equate not only to better control of their illness but also better quality of life.Studies have also shown the benefits of self-management: when patients are responsible for managing their own illness, their clinical outcomes and quality of life improve and they become less dependent on health care services. In this study we aim to examine if knowledge, and any changes in knowledge, will each have any association with Quality of Life (QOL) and their perceptions of their illness (IP). We also seek to investigate the effects of an educational booklet, as well as an intervention including the educational booklet with formal counselling on their knowledge, QOL and IPs. | Sickle Cell Disease|Quality of Life | ALL | CHILD, ADULT | Sickle Cell Unit, University of West Indies, Mona Campus, Kingston 7, Jamaica |
| Isoquercetin in Sickle Cell Anemia | This research study is being done to assess the safety and effectiveness of isoquercetin to reduce levels of soluble P-Selectin in patients with sickle cell disease. Isoquercetin is a naturally occurring flavonoid-or vitamin. You will find quercetin and isoquercetin in fruits and vegetables. The names of the study drug involved in this study are/is: - Isoquercetin | Sickle Cell Disease|Sickle Cell-Beta0-Thalassemia | ALL | ADULT | |
| Stem Cell Gene Therapy for Sickle Cell Disease | This Phase I clinical trial will assess the safety and initial evidence for efficacy of an autologous transplant of lentiviral vector modified peripheral blood for adults with severe sickle cell disease. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of California, Los Angeles (UCLA), Los Angeles, California, 90095, United States |
| Support Strategies for Parents During the First Year Following Their Child's Diagnosis of Sickle Cell Disorder | Background: Sickle cell disorder (SCD), the commonest genetic (faulty gene inherited from both parents) condition in the UK, affects mainly underserved groups. Babies with SCD must start treatments soon after birth to prevent them becoming unwell. Stigma, fear and inequalities can make it difficult for parents to accept their child's diagnosis and access appropriate treatment and support. Aim: Develop strategies to improve support for parents during their child's first year of life following a SCD diagnosis to encourage early engagement with health services. Method: Comprises two stages: (i) Determine why parents choose to engage with support or not (ii) Use this information to co-design strategies to ensure greater accessibility of support for parents during their child's first year of life. Patient and Public Involvement: We are working with Sickle Cell Society and parents of children with SCD. Dissemination: Findings will be shared with support groups, charities, health professionals and academics. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | |
| A Blood Stem Cell Transplant for Sickle Cell Disease | Blood stem cells can produce red blood cells (which carry oxygen), white blood cells of the immune system (which fight infections) and platelets (which help the blood clot). Patients with sickle cell disease produce abnormal red blood cells. A blood stem cell transplant from a donor is a treatment option for patients with severe sickle cell disease. The donor can be healthy or have the sickle cell trait. The blood stem cell transplant will be given to the patient as an intravenous infusion (IV). The donor blood stem cells will then make normal red blood cells - as well as other types of blood cells - in the patient. When blood cells from two people co-exist in the patient, this is called mixed chimerism. Most children are successfully treated with blood stem cells from a sibling (brother/sister) who completely shares their tissue type (full-matched donor). However, transplant is not an option for patients who (1) have serious medical problems, and/or (2) do not have a full-matched donor. Most patients will have a relative who shares half of their tissue type (e.g. parent, child, and brother/sister) and can be a donor (half-matched or haploidentical donor). Adult patients with severe sickle cell disease were successfully treated with a half-matched transplant in a clinical study. Researchers would like to make half-matched transplant an option for more patients by (1) improving transplant success and (2) reducing transplanted-related complications. This research transplant is being tested in this Pilot study for the first time. It is different from a standard transplant because: 1. Half-matched related donors will be used, and 2. A new combination of drugs (chemotherapy) that does not completely wipe out the bone marrow cells (non-myeloablative treatment) will be used to prepare the patient for transplant, and 3. Most of the donor CD4+ T cells (a type of immune cells) will be removed (depleted) before giving the blood stem cell transplant to the patient to improve transplant outcomes. It is hoped that the research transplant: 1. Will reverse sickle cell disease and improve patient quality of life, 2. Will reduce side effects and help the patient recover faster from the transplant, 3. Help the patient keep the transplant longer and 4. Reduce serious transplant-related complications. | Sickle Cell Disease|Sickle Cell Disorder|Hemoglobinopathies|Thalassemia|Anemia, Sickle Cell | ALL | ADULT | City of Hope Medical Center, Duarte, California, 91010, United States |
| Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease | This study is being done to determine the safety and outcome (long-term control) of a high-dose chemotherapy regimen followed by an infusion of CD34 selected (immune cells) stem cells from a partially matched adult family member donor, called haploidentical stem cell transplantation, in high-risk sickle cell disease patients. Funding Source - FDA OOPD | Sickle Cell Disease | ALL | CHILD, ADULT | University of California Los Angeles (UCLA), Los Angeles, California, 90095, United States|Children's Hospital and Research Center Oakland, Oakland, California, 94609, United States|Lurie Children's Hospital, Chicago, Illinois, 60611-2605, United States|Washington University/St. Louis Children's Hospital, Saint Louis, Missouri, 63110, United States|New York Medical College, Valhalla, New York, 10595, United States|Medical College of Wisconsin/Children's Hospital of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| COVID-19 Vaccine Response in Sickle Cell Disease | The purpose of this study is to assess the antibody response to COVID-19 vaccination in a cohort of patients with sickle cell disease (SCD) and to assess vaccine and SCD related complications around the time of vaccination. | Sickle Cell Disease|COVID-19 | ALL | CHILD, ADULT, OLDER_ADULT | UCSF Benioff Children's Hospital Oakland, Oakland, California, 94609, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States|Johns Hopkins University, Baltimore, Maryland, 21205, United States|Montefiore Hospital, Bronx, New York, 10467, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|Prisma Health - Upstate, Greenville, South Carolina, 29601, United States|University of Texas Southwestern Medical Center, Dallas, Texas, 75390, United States|Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| Cutaneous Hydration Assessment in SCD | This study will validate the diagnostic accuracy of a cutaneous hydration sensor. This sensor will also be evaluated for its feasibility as a point-of-care device for the assessment of hydration status and its potential to guide hydration therapy in patients with sickle cell disease (SCD). | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | UPMC Sickle Cell Clinic, Pittsburgh, Pennsylvania, 15213, United States |
| Gene Editing For Sickle Cell Disease | This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD. Primary Objective * To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD. Secondary Objective * To assess the efficacy autologous infusion of CRISPR/Cas9 genome-edited CD34+ HSPCs into patients with severe SCD. | Sickle Cell Disease | ALL | ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Liver Fibrosis in Sickle Cell Disease | Patients with sickle cell disease many have a number of systemic complications, including liver problems. Some of these liver problems lead to liver fibrosis/cirrhosis, secondary to chronic blood transfusions. The purpose of this study is to investigate FibroScan readings in patients with sickle cell disease and iron overload secondary to blood transfusions, and to correlate the FibroScan results with Ferriscan. A comparison with the results of FibroScan to patients with Sickle cell without known liver disease, who have never been on chronic transfusions and with normal liver function profiles will also be made.The primary hypothesis is that the results of FibroScan will correlate with the results of Ferriscan and liver biopsy. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | University of Miami, Miami, Florida, 33136, United States |
| Gerofit Exercise Intervention for Older Adults With Sickle Cell Disease (SICKLE-FIT Study) | The purpose of this study to assess the feasibility, acceptability, and safety of a personalized exercise training program adapted from Gerofit to improve physical health and quality of life for adults with SCD | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Duke University, Durham, North Carolina, 27710, United States |
| Sickle Cell Disease (SCD) Decision Aid | The purpose of this study is to gather decision making needs information from caregivers and patients with sickle cell disease (SCD) in order to develop a web-based decision aid tool. Study subjects will participate in interviews defining treatment decision making needs during which investigators will ask information about their SCD. Notes taken from these interviews will allow the research team to better understand current practice related to clinical practice and allow for better refinement of the decision aid tool. An additional group of participants will be asked to review the web-based Sickle Cell Decision Aid. Participants will be asked to describe thoughts about the site, including but not limited to ease of navigation, content and construction. This study will provide information for the conduct of a randomized controlled trial for the use of a web based decision aid to give patients with sickle cell disease and parent/legal guardian of children with sickle cell disease accurate information about risks and benefits of therapies and enable them to make decisions based on their individual values and preferences. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | |
| Sickle Cell Disease (SCD) Bone Pain Study | A prospective study to determine how low bone mineral density and/or vertebral compression fractures associate with pain in adults with sickle cell disease | Sickle Cell Disease|Sickle Cell Anemia|Low Bone Density|Osteoporosis|Osteopenia|Vertebral Fracture|Vertebral Compression|Osteonecrosis|Ischemic Necrosis|Avascular Necrosis | ALL | ADULT, OLDER_ADULT | UC Davis Comprehensive Cancer Center, Sacramento, California, 95817, United States |
| HEMAGO Monitoring and Impact on the Occurrence of Complications During Pregnancy in Sickle Cell Patients | Sickle cell disease is the most common monosemic genetic disease in the world and affects approximately 32,000 people in France. In recent years, improvements in the management of this pathology have led to a steady increase in the life expectancy of sickle cell patients. Women with sickle cell disease are increasingly likely to have one or more pregnancies. Several studies have shown that these pregnancies are marked by maternal morbidity due to acute sickling crises, thromboembolism, infection, chronic end-organ dysfunction, or pre-eclampsia (PE), while neonatal outcomes may be intrauterine growth retardation (IUGR), preterm delivery, small infants for gestational age, stillbirth, and neonatal death. Consequently, monitoring pregnancies in sickle cell patients represents a complex medical challenge due to the risk of potentially serious maternal and fetal complications. Multidisciplinary follow-up is then crucial to monitor and manage these possible complications. The double hematologist-obstetrician consultation program (HEMAGO) offers complete and adapted close follow-up, in order to minimize the risks for the mother and the unborn child. This study aims to evaluate the impact of this HEMAGO monitoring on the various maternal and obstetric complications by comparing the outcome of pregnancies of patients who were followed before the implementation of this device to patients who were followed in the frame their pregnancy through this program. | Sickle Cell Disease and Pregnancy | FEMALE | ADULT, OLDER_ADULT | Service de gynécologie-obstétrique de l'Hôpital Femme Mère Enfant, Bron, 69500, France|Croix-Rousse Hospital - Service de Gynécologie-Obstétrique, Lyon, 69003, France|Service de gynécologie-obstétrique de l'Hôpital de Lyon Sud, Lyon, France |
| Effectiveness of a Computerized Tool (PAINRelieveIt) to Help Manage Pain Related to Sickle Cell Disease | Sickle cell disease (SCD) is a blood disorder that is characterized by intense, painful episodes known as sickle cell crises. This study will evaluate the effectiveness of PAINRelieveIt, a three-part computer-based pain management tool, in treating adults with SCD. | Anemia, Sickle Cell|Hemoglobin SC Disease | ALL | ADULT, OLDER_ADULT | University of Illinois at Chicago Sickle Cell Center and Medical Center, Chicago, Illinois, 60612, United States |
| Stem Cell Transplantation for Sickle Cell Anemia | This protocol will be investigating the use of stem cell transplantation, in related donors, to cure sickle cell disease. Sickle cell disease is a recessive disorder caused by a point mutation that results in the substitution of valine for glutamic acid at the sixth position in the B-chain of hemoglobin. This leads to sickling of the red blood cells under many conditions, such as hypoxia, dehydration, and hyperthermia. The sickling leads to vaso-occlusion, which causes irreversible damage in almost all systems in the body, including the central nervous system (CNS), lungs, heart, bones, eyes, liver, and kidneys. | Sickle Cell Disease | ALL | CHILD, ADULT | Hackensack University Medical Center, Hackensack, New Jersey, 07601, United States |
| Inflammation, Platelets and Sickle Cell Disease | Sickle cell disease (SCD) is an autosomal recessive genetic disorder linked to a single mutation on beta-globin chains. This leads to red blood cell deformation and chronic hemolysis which can result in vaso-occlusive events, anemia and vasculopathy. Pathophysiology is incompletely understood, and beyond red blood cell's abnormalities this involves hemostasis and innate immunity. The aim of our study is to describe the mechanisms of thrombo-inflammation during the vaso-occlusive crisis (VOC) in adults with sickle cell disease. | Platelet Activation|Thromboinflammation|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | IUCT-Oncopole University Hospital, Toulouse, 31500, France |
| Allograft for Sickle Cell Disease and Thalassemia | The design of the study incorporates the following features: 1. This is a phase II study to determine the safety and therapeutic potential of a new transplant approach (disease-free survival, graft versus myeloma effect) and to evaluate its toxicity profile (immediate toxicity, graft-versus-host disease, graft rejection, mortality) in a patient population with severe congenital anemias. 2. The patient cohort to be studied: Those patients with severe sickle cell disease and thalassemia who have risk factors for high mortality and morbidity related to their disease 3. Transplant Conditioning Regimen - Immunosuppression without myeloablation: Patients will receive conditioning sufficient to allow donor lympho-hematopoietic engraftment without complete marrow ablation. If the graft is rejected, the patient will reconstitute autologous marrow function. We will use a combination of low dose irradiation, Alemtuzumab (Campath®), and sirolimus. 4. Peripheral blood hematopoietic progenitor cell (PBPC) transplant: An unmanipulated peripheral blood stem cell collection from a filgrastim (G-CSF) stimulated HLA-matched donor should improve the chance of engraftment because of the high stem cell dose (5 x 106/kg CD34+ cells) and the presence of donor lymphocytes. To reduce the risk of GVHD, patients will receive sirolimus before and after the transplant. The sirolimus will be tapered as necessary to minimize any graft versus host disease while still maintaining adequate chimerism. | Sickle Cell Disease and Thalassemia | ALL | ADULT | UT Southwestern Medical Center, Dallas, Texas, 75390, United States |
| Gene Transfer for Patients With Sickle Cell Disease | The purpose of this Phase 1/2 study is to determine the feasibility and safety of stem cell collection and gamma-globin gene transfer, and success of gene correction in subjects with sickle cell disease | Anemia, Sickle Cell | ALL | ADULT | Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States |
| Microvascular Blood Flow in Sickle Cell Anemia | Sickle cell disease (SCD) is an inherited blood disorder that causes the red blood cells to change their shape from a round shape to a half-moon/crescent or sickled shape. Sickle-shaped cells can cause problems by getting stuck in blood vessels, blocking blood flow, and can cause inflammation and injury to important body parts. There are no specific treatments that improve this condition and promote blood flow hindered by sickle cell blockages. Another big challenge in managing sickle cell disease is that there are no good measures to determine changes and improvements in blood flow. Contrast-enhanced ultrasound is a technique currently used to detect blood flow in the heart, muscles, and other organs. It is extremely sensitive and can detect blood flow in the smallest of blood vessels. It would be very useful in helping healthcare providers know whether treatment strategies are improving blood flow during sickle cell blockages. The hypothesis is that contrast-enhanced ultrasound will be a feasible tool for determining changes in blood flow of subjects with sickle cell disease. | Sickle Cell Disease|Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | The University of Illinois, Chicago, Illinois, 60607, United States|Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| Sickle Cell Disease - Stroke Prevention in Nigeria Trial | Given large absolute numbers of individuals with sickle cell disease in Nigeria, hydroxyurea therapy for all individuals with sickle cell disease may not be initially feasible; however, a targeted strategy of hydroxyurea use for primary prevention of strokes is an alternative to the standard therapy (observation) for high-risk individuals. The investigators propose a feasibility study, Sickle Cell Disease - Stroke Prevention in Nigeria (SPIN) Trial, to determine whether hydroxyurea can be used for primary prevention of strokes in Nigerian children with sickle cell anemia. | Sickle Cell Anemia|Sickle Cell Disease|Stroke | ALL | CHILD | Aminu Kano Teaching Hospital, Kano, P.MB. 3452, Nigeria |
| Aspirin Prophylaxis in Sickle Cell Disease | Neurologic complications secondary to cerebrovascular damage are prevalent in children with sickle cell disease. These patients experience both clinically overt cerebrovascular accidents and "silent infarctions" demonstrated by magnetic resonance imaging (MRI). They are also at risk for neurocognitive abnormalities.We hypothesize that daily, low-dose aspirin therapy will safely diminish the incidence and progression of cognitive deficits as well as the predisposition to overt and silent stroke in children with homozygous sickle cell disease (Hgb SS) or hemoglobin S Beta Zero Thalassemia (Hgb SB-0 Thal). In order to optimize the design of a future trial to test this hypothesis, we propose a pilot study to test the safety and tolerability of aspirin in young children with sickle cell disease. | Sickle Cell Disease | ALL | CHILD | |
| Moya Moya Syndrome With or Withtout Sickle Cell Disease | Moya Moya disease or syndrome ar characterized by a progressive or occlusion of the intracranial carotid arteries and their mainproximal branches, followed by the development of fragile neovessels at the base of the skull, leding to a high risk of both ischemic and hemorragic stroke over time. Moya Moya syndrome are associated to a variety of disease, which main frequent is sickle cell disease (SCD). Among patients with SCD who had suffered from at least one ischemic stroke, the prevalence of moya moya syndrome was estimated up to 43%. In general, therapeutic strategies in Moya Moya to prevent first ever ou recurrent stroke can be divided into conservative medical treatment and surgical revascularisation (direct bypass, indirect bypass or combined bypass). The aim of this study is to compare prognosis of patients with Moya Moya syndrome associated with sickle cell disease or not. The investigators retrospectiveluy analysed medical chart from 2010 to 2021 of patients with Moya Moya disease or syndrome at two French university hospitals (including a center of the french West Indies where prevalence of sickle cell disease is high). The diagnosis was based on angiography or MRI records showing uni- or bilateral stenosis of distal intracranial internal carotide arteries or middle cerebral arteries associated wirh classic collateral network. Main endpoint will be comparison of a composite outcome defined as time from Moya Moya diagnosis to first or recurrent stroke or bad prognosis achivement (defined by modified Rankin score \>2) | Moya Moya Disease | ALL | CHILD, ADULT, OLDER_ADULT | Uhmontpellier, Montpellier, 34295, France |
| Nonmyeloablative Stem Cell Transplant in Children with Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor | The aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with sickle cell disease (SCD) who have a matched related major ABO-incompatible donor. The nonmyeloablative regimen will use alemtuzumab, total body irradiation (TBI) and sirolimus for immune suppression. This study will expand the access of HSCT for patients with SCD who are currently not eligible because of donor restrictions. | Sickle Cell Disease|Stem Cell Transplant Complications|Red Blood Cell Disorder|Pure Red Cell Aplasia | ALL | CHILD, ADULT | Alberta Children's Hospital, Calgary, Alberta, T3B 6A8, Canada |
| Predictors of Pain in Sickle Cell Disease | Sickle cell disease is a painful inherited disorder that affects approximately 100,000 people in the United States, and more than half of these individuals develop chronic or persistent pain that is often severe and disabling. The factors that predict whether an individual with sickle cell disease will develop severe, disabling pain are unclear. The goal of this project is to identify the factors that predict severe pain outcomes in individuals living with sickle cell disease in order to improve pain management strategies and guide future studies of non-opioid therapies for treatment of their pain. Participants who agree to enroll in this study will be asked to participate in a virtual and then an in-person study visit for their full initial study assessment. They will answer survey questions during the virtual visit, and will be asked to complete several types of standard testing to understand how their body handles pain during the in-person visit. After completing the virtual and in-person sessions, participants will receive text or electronic medical record messages with brief survey (will take less than 8 minutes to complete) on their pain experiences every three months until the study is completed (or up to 48 months for people who are enrolled at the beginning of the study). | Sickle Cell Disease|Chronic Pain | ALL | CHILD, ADULT | Duke University Medical Center, Durham, North Carolina, 27710, United States |
| Obesity in Pediatric Sickle Cell Disease: A New Phenomenon | The objective of this project is to determine the prevalence of hypertension, hyperlipidemia and hyperglycemia in the pediatric population with sickle cell disease who are obese in Mississippi compared to those pediatric patients with sickle cell disease who are not overweight/obese. The pediatric hematology department at the University of Mississippi Medical Center (UMMC) has a relatively large population of patients with sickle cell disease who are overweight and obese. This is a paradoxical trend since high-energy expenditure of the body to produce new red blood cells usually results in underweight to normal weight patients. From our previous chart review, the investigators found our pediatric patients with sickle cell disease to have similar rates of overweight and obesity to that of state and national levels. The metrics our team will measure include: blood pressure, blood cholesterol levels and blood glucose levels. The investigators expect to find higher rates of hypertension, high cholesterol and high glucose levels in the overweight and obese patients with SCD compared to that of underweight and normal weight. Our ultimate goal for follow up projects will be to determine the baseline risk of hypertension, hyperlipidemia and hyperglycemia in this population so we can then determine effective, sustainable interventions for weight and the co-morbidities that come with increasing weight status. Our goal would also be to educate the patient and families on these interventions and provide them with resources, which could lead to an overall improvement in health and patients quality of life. | Sickle Cell Disease|Obesity, Childhood|Hypertension|Hyperlipidemia in Children|Hyperglycemia | ALL | CHILD, ADULT | University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States |
| A Multi-Center Study of Riociguat in Patients With Sickle Cell Diseases | The proposed study is a Phase 2 multi-center, randomized, double-blind, placebo-controlled, parallel groups study aimed to evaluate the safety, tolerability and the efficacy of riociguat compared with placebo in patients with sickle cell disease (SCD). | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | UCSF Benioff Children's Hospital Oakland, Oakland, California, 94609, United States|Howard University, Washington, District of Columbia, 20060, United States|University of Miami, Miami, Florida, 33136, United States|Emory University School of Medicine, Atlanta, Georgia, 30329, United States|University of Illinois, Chicago, Chicago, Illinois, 60612, United States|Indiana University, Indianapolis, Indiana, 46202, United States|Tulane University, New Orleans, Louisiana, 70112, United States|Johns Hopkins University, Baltimore, Maryland, 21205, United States|Boston University Medical Center, Boston, Massachusetts, 02118, United States|Albert Einstein University/ Montefiore Medical Center, Bronx, New York, 10467, United States|New York Presbyterian Brooklyn Methodist Hospital, Brooklyn, New York, 11225, United States|UNC Comprehensive Sickle Cell Center, Chapel Hill, North Carolina, 27599-7305, United States|Duke University, Durham, North Carolina, 27710, United States|East Carolina University, Greenville, North Carolina, 27834, United States|Ohio State University, Columbus, Ohio, 43210, United States|UPMC Division of Hematology and Oncology, Pittsburgh, Pennsylvania, 15233, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|University of Tennessee Health Science Center, Memphis, Tennessee, 38163, United States|University of Texas Southwestern, Dallas, Texas, 75390, United States|Virginia Commonwealth University Medical Center, Richmond, Virginia, 23298, United States |
| Testing the Sickle Cell Caregiver Collaboration for Child Development (SCCCD) Intervention | Sickle cell disease affects 100,000 people and 2,000 newborns each year; 50% of these children have a developmental deficit (\>2 SD) before the age of 3. Early identification of developmental deficit supports timely intervention, but children with sickle cell disease are grossly underdiagnosed and undertreated. The goal of the proposed study is to determine the incidence and severity of developmental deficit at 9, 18 and 30 months of age among children with sickle cell disease and test a 12-month, home-based caregiver intervention with this disproportionately affected population. | Sickle Cell Disease | ALL | CHILD | Washington University School of Medicine, Saint Louis, Missouri, 63108, United States |
| Sleep Respiratory Disorders Evaluation in Sickle Cell Disease Children | In Sickle cell disease children, sleep respiratory abnormalities are risk factors for vaso-occlusive complications, as well as cerebral vasculopathy. A 18 months follow-up children with sickle cell disease evaluating sleep respiratory problems frequency and etiology, as well as their influence on sickle cell disease complications. | Sickle Cell Disease|Respiration Disorders | ALL | CHILD | Assistance Publique - Hôpitaux de Paris, Paris, 75019, France |
| Codeine in Sickle Cell Disease | The objective of this study is to determine if a subject's genetic make-up would affect the treatment response to codeine in subjects with sickle cell disease. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of Illinois Medical Center, Chicago, Illinois, 60612, United States |
| Early Life Exposures Among Children with Sickle Cell Disease | This study is being conducted to determine the relationship between early childhood exposures, such as Adverse Childhood Experiences, Social Determinants of Health and nutrition/breastfeeding, among children with sickle cell disease, and behavioral interventions aimed to reshape psychological resilience and lifestyle factors towards positive health outcomes. | Sickle Cell Disease|Adverse Childhood Experiences|Breastfeeding | FEMALE | ADULT | University of Alabama at Birmingham, Birmingham, Alabama, 35233, United States |
| Enhancing Use of Hydroxyurea In Sickle Cell Disease Using Patient Navigators | Multi-phase, patient navigator-based program in the Richmond and Tidewater regions of Virginia to demonstrate: 1. the feasibility of using patient navigators to improve the percentage of children and adult (age 15 and older) patients with sickle cell disease (SCD) in SCD specialty care 2. the efficacy of using patient navigators to improve hydroxyurea (HU) (re-)initiation and adherence among adult patients with SCD eligible for HU (Patient navigators may also be known as public health workers.) | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Virginia Commonwealth University, Richmond, Virginia, 23298, United States |
| Sickle Cell Disease and Endothelial Progenitor Cells (EPCs) | Sickle Cell Disease (SCD) is the most prevalent genetic disease of haemoglobin.The underlying abnormality in the red blood cell (RBC) of SCD is the presence of abnormal sickle cell hemoglobin (HbS), which, when deoxygenated, becomes relatively insoluble, forms aggregates with other hemoglobin molecules within the RBC and causes rigid deformation of the cell. Acute pain vaso-occlusive crisis, strokes and acute chest syndrome are the main acute complications, sometimes life-threatening, often leading to organic and functional squeal. Although the common SS form of SCD is a unique gene disorder, the range of the clinical severity is remarkably wide and striking, suggesting that clinical polymorphism is due to modifier genes and environmental factors.Most of the research efforts have been focused on the biology of haemoglobin and of the red cells. Meanwhile, the complex pathophysiology of SCD is undoubtedly influenced by the many physiologic functions of the vascular wall. In line with this hypothesis are several reports of increased circulating levels of endothelium-derived surface molecules in SCD patients suggesting marked endothelial stress in SCD. Similarly, other processes that involve the endothelium, such as leukocyte adhesion and activation, may play a role in vascular occlusion. This accumulation of data raises the unanswered question of the mechanisms of endothelium maintenance and regeneration in SCD. Through these mechanisms, it is likely that function or dysfunction of the vascular endothelium contributes to the overall vascular pathobiology of this disease, which includes recurrent vaso-occlusions, stroke, leg ulcers, chronic organ ischemic damages, and neovascularizing retinopathy that affect nearly one-half (48%) of the surviving patients by the fifth decade.Thus, our groups have combined their respective clinical and biological expertises to test the hypothesis that SCD is a condition of specific endothelial stress and dysfunction upon chronic and Paracystic abnormal interactions with circulating cells and abnormal oxygen delivery to tissues. Specifically, we hypothesize that chronic endothelial stress with detachment of activated endothelial cells require increases mobilisation of the Endothelial Progenitor Cells (EPCs) that maintain endothelial homeostasis to avoid major thromboembolic events and vasospasm. Inappropriate mobilisation or maturation of the EPCs in SCD may participate to the severity of the disease. | Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | Hopital Tenon, Assistance Publique Hôpitaux de Paris, Paris, 75020, France |
| Vitamin D3 in Patients With Sickle Cell Disease | There are approximately 90,000 individuals in the United States with sickle cell disease (SCD). Studies have shown that up to 98 percent of patients with Sickle Cell Disease have a vitamin D deficiency, defined as a 25-hydroxyvitamin D level (25(OH)D) less than or equal to 20 ng/mL. As a result, of low bone density, patients may develop osteonecrosis, chronic inflammation and related pain. This study will be coordinated with patients' regularly scheduled visits for medical care and will require patients to submit blood sample at the start of the study and at 3, 6, 9, AND 12 month visits. Patients will also be scheduled for a bone density measurement (DXA scan) at the start of the study and after 12 months of supplementation to assess for any bone re-mineralization. Thus, the main purpose of this study is to find the amount of nutritional vitamin D that needs to be taken by patients with sickle cell disease in order to correct vitamin D deficiency. The study will also test whether vitamin D supplements improve bone health and reduce inflammation. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Icahn School of Medicine at Mount Sinai, New York, New York, 10029, United States |
| Ketamine Sickle Cell Disease | Sickle cell disease (SCD) often results in acute vaso-occlusive crisis (VOC), an obstruction of blood vessels resulting in ischemic injury and pain. The pain experienced during these episodes is due to a wide range of pathophysiological processes. Though recent studies have begun to unravel the underlying mechanisms of these processes, literature focused on pain management for sickle cell disease is scarce. Opioids and non-steroidal anti-inflammatory drugs (NSAIDs) remain the predominate treatment for VOC. However, the efficacy of these treatments has come into question. A large sub-set of patients with SCD report continued pain despite treatment with opioids. Tolerance and opioid-induced hyperalgesia (OIH) may be responsible for unresponsiveness to opioid-centric treatment modalities. New classes of drugs are being tested to prevent and treat acute pain associated with SCD, but in the meantime physicians are looking to existing therapies to bridge the gap. The N-methyl-d-aspartate (NMDA) receptor has been implicated in both tolerance and OIH. As a NMDA receptor agonist, ketamine has been shown to modulate opioid tolerance and OIH in animal models and clinical settings. Ketamine utilized as a low dose continuous infusion could benefit patients with SCD related pain that are unresponsive to opioid analgesics. Based on limited studies of adjuvant ketamine use for pain management, low-dose ketamine continuous infusion appears safe. Further clinical investigations are warranted to fully support the use of low-dose ketamine infusion in patients with SCD-related pain. | SC Disease|Pain, Chronic | ALL | ADULT, OLDER_ADULT | |
| Contraception in Women With Sickle Cell Disease | Sickle cell anemia is a homozygous genetic disease with high prevalence in Brazil. There are changes in conformation and physicochemical properties of red cells that generate varied clinical manifestations among which is chronic hemolytic anemia, cardiovascular diseases, fever, splenic sequestration and usually painful crises. Women with sickle cell anemia have high maternal-fetal and neonatal morbidity and mortality. During pregnancy, there is intensification of maternal anemia, episodes of painful crises; and also, more obstetric risks, such as pre-eclampsia, thromboembolism and hemorrhage. Thus, there is the need for adequate reproductive family planning for this population conducted mainly through hormonal contraception. The World Health Organization recommends that all contraceptive methods may be prescribed for people with sickle cell anemia women, being the progestogen-only contraceptive methods the most indicated due to no changes in venous or arterial thrombosis. Nevertheless, there is need for further scientific evidence as the best contraceptive choice among women with sickle cell anemia in relation to safety, adhesion and reduction of pain crises. The objective of this study is to evaluate the clinical effect through safety of etonogestrel-releasing contraceptive implant in women with sickle cell anemia during twelve months. | Sickle Cell Disease | FEMALE | ADULT | Bahiana School of Medicne and Public Health, Salvador, Bahia, Brazil|University of Sao Paulo, Ribeirao Preto, Sao Paulo, 14049-900, Brazil |
| Transfusion Alternatives Pre-operatively in Sickle Cell Disease (TAPS) | TAPS is a sequential trial which aims to investigate whether the administration of a blood transfusion pre-operatively to patients with sickle cell disease (HB SS or Hb SB0 thal)having low or medium risk elective surgery increases or decreases the overall rate of peri-operative complications. The proportion of patients with peri-operative complications in two randomised groups of transfused and untransfused patients will be compared. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | NBS/MRC Clinical Studies Unit, National Blood Service, Cambridge, Cambridgeshire, CB2 2PT, United Kingdom |
| VO2max & HRQoL in Children With Sickle Cell Disease | Sickle cell disease is the most common inherited genetic disorder, accounting for 300,000 births worldwide per year. It is caused by an autosomal recessive mutation of the β-globin gene, responsible for an abnormal hemoglobin, the main protein in red blood cells, responsible for transporting oxygen from the lungs to the tissues. The abnormal hemoglobin, known as "Sickle" or S, deforms the red blood cell, causing chronic hemolytic anemia, organ damage (heart, spleen, etc.) and vaso-occlusive crises. Therapeutic progress and specialised patient follow-up have considerably improved the vital and functional prognosis of children and adolescents with sickle cell disease. Physical fitness, measured during a cardiorespiratory exercise test (CPET), is used to determine maximal oxygen uptake (VO2max). Patients with sickle cell disease have a multifactorial limitation of exercise tolerance, which may affect their physical fitness. Authors have shown that VO2max is impaired in children and adolescents with sickle cell disease, independently of their baseline hemoglobin level. Yet VO2max is a key determinant of health-related quality of life (HRQoL) in patients being monitored for a chronic disease. In the past, our team has contributed to the assessment of HRQoL in several groups of pediatric patients suffering from chronic disease (congenital heart disease, PAH). To date, the link between impaired physical fitness and HRQoL has not been demonstrated in sickle cell children. The pathophysiological determinants of reduced physical capacity and exercise tolerance in sickle cell patients have also not been fully elucidated. Studying these factors will enable us to propose appropriate treatment in the future, with the aim of improving physical fitness and HRQoL in children and adolescents with sickle cell disease. | Sickle Cell|Children | ALL | CHILD | Pediatric and Congenital Cardiology Department, Arnaud De Villeneuve University Hospital, Montpellier, Occitanie, 34295, France |
| Gene Transfer for Sickle Cell Disease | A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new genetic material in order to fix or replace the patient's disease gene, with the goal of curing the disease. The procedure is similar to a bone marrow transplant, in that the patient's malfunctioning blood stem cells are reduced or eliminated using chemotherapy, but it is different because instead of using a different person's (donor) blood stem cells for the transplant, the patient's own blood stem cells are given back after the new genetic material has been introduced into those cells. This approach has the advantage of eliminating any risk of GVHD, reducing the risk of graft rejection, and may also allow less chemotherapy to be utilized for the conditioning portion of the transplant procedure. The method used to introduce the gene into the patient's own blood stem cells is to engineer and use a modified version of a virus (called a 'vector') that efficiently inserts the "correcting" genetic material into the cells. The vector is a specialized biological medicine that has been formulated for use in human beings. The investigators have recently discovered a gene that is very important in the control of fetal hemoglobin expression. Increasing the expression of this gene in sickle cell patients could increase the amount of fetal hemoglobin while simultaneously reducing the amount of sickle hemoglobin in their blood, and therefore potentially cure the condition. In summary, the advantages of a gene therapy approach include: 1) it can be used even if the patient does not have a matched donor available; 2) it may allow a reduction in the amount of chemotherapy required to prepare the patient for the transplant; and 3) it will avoid the strong medicines often required to prevent and treat GVHD and rejection. The goal is to test whether this approach is safe, and whether using gene therapy to change the expression of this particular gene will lead to increased fetal hemoglobin production in people with sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT | UCLA - Mattel Children's Hospital, Los Angeles, California, 90095, United States|Boston Children's Hospital, Boston, Massachusetts, 02115, United States |
| A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients With Sickling Disorders | Sickle cell anemia is a genetic disorder that results from a single nucleotide substitution in codon 6 of the beta-globin gene which, in the homozygous state, produces an abnormal hemoglobin that is prone to polymer formation when deoxygenated. The polymerized hemoglobin leads to impaired deformability and sickling of red blood cells which subsequently lodge in end-arterioles producing the classic and most prominent feature of the disorder, repeated vasoocclusive crises. Despite knowledge of the precise genetic defect for decades, only recently has there been therapeutic impact based upon this knowledge when a clear benefit from treatment with hydroxyurea, a cell cycle-specific agent administered to induce production of fetal hemoglobin (HbF) by stimulating gamma-globin synthesis, was reported in patients with sickle cell disease (SCD). The reduction in the frequency and severity of vasoocclusive crises seen has been attributed to the increase in HbF levels in responsive patients. While the majority of patients demonstrate a rise in HbF, not all such patients benefit from treatment. Given these results, alternative agents that also stimulate the production of HbF warrant investigation in the treatment of SCD. Recombinant-methionyl human stem cell factor (SCF) is a hematopoietic growth factor with activity on immature hematopoietic progenitor cells. SCF stimulates the production of HbF in vitro and in vivo, and this effect is attainable without the myelosuppression associated with hydroxyurea. In this phase I/II trial, we will administer SCF in a dose escalating fashion to patients with sickling disorders. Parameters to be measured are HbF levels, F cell levels, peripheral blood CD34 levels, frequency, duration, and severity of vasoocclusive crises, and toxicity. | Hemoglobin SC Disease|Sickle Cell Anemia | ALL | CHILD, ADULT, OLDER_ADULT | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), Bethesda, Maryland, 20892, United States |
| Red Blood Cell Survival in Sickle Cell Disease | This is a single-arm, mechanistic clinical trial to measure predictors of senescence and the in vivo survival of transfused red blood cells (RBCs) in individuals with sickle cell disease (SCD) receiving chronic transfusion therapy (CTT). Chronic transfusion in patients with SCD is a common treatment. The efficacy of RBC transfusion therapy to treat or prevent complications of SCD may be hampered by variable survival of the transfused donor RBC. The overall aim is to see how long RBC survive in SCD patients who are chronically transfused. When a study participant has a regular blood transfusion the researchers will label a small portion of the RBCs that are transfused with biotin. The participant will return at Day 1, weekly for 3 months and monthly for 3 months to measure how long those RBCs survive. An optional sub-study using INTERCEPT RBCs will mirror the main study but will use INTERCEPT RBCs that have biotinylated for 1 RBC unit. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Hughes Spalding Children's Hospital, Atlanta, Georgia, 30303, United States|Childrens Healthcare of Atlanta, Atlanta, Georgia, 30322, United States|Grady Health System, Atlanta, Georgia, 30322, United States |
| Treatment of Sickle Cell Anemia With Stem Cell Transplant | This is a clinical research trial in which a novel preparatory regimen was developed for bone marrow transplant (BMT) which eliminates the primary obstacle to transplant, the lack of a matched sibling donor. It is believed this regimen is sufficiently efficacious and sufficiently gentle to apply to patients with sickle cell anemia and related disorders. It is proposed to characterize the efficacy and toxicity of this regimen in high risk patients with sickle cell anemia using criteria for patient selection that have been accepted in prior BMT trials in patients with sickle cell disease, specifically only the subset of patients whose prior clinical behavior indicates that they are at high risk for serious morbidity and early mortality. In addition, it is proposed to characterize the pathophysiology of a consistent febrile response seen in the haploidentical BMT regimen the investigators have developed at Thomas Jefferson University (TJU). The primary goal of this study is to determine the response rate to a reduced intensity conditioning regimen which consists of fludarabine, cytarabine, low dose total body irradiation and cyclophosphamide in patients with severe sickle cell anemia. | Sickle Cell Anemia|Sickle Cell-hemoglobin C Disease|Sickle Cell-β0-thalassemia | ALL | ADULT | Thomas Jefferson University, Philadelphia, Pennsylvania, 19107, United States |
| Blood Sampling for Research Related to Sickle Cell Disease | This study will collect representative blood samples from healthy children and adults and from children and adults who have unique red blood cell features that are related to sickle cell disease. Sickle cell disease is a blood disease that limits the ability of red blood cells to carry oxygen throughout the body. The purpose of the study is to collect a variety of blood samples that may then be used to investigate advances and potential new drug treatments for sickle cell disease. Volunteers must be at least 18 years of old. Samples will be taken both from healthy volunteers and from volunteers who have unique red blood cell features that are related to sickle cell disease. Candidates will be screened with a medical history. During the study, participants will undergo a one- to two-hour outpatient procedure at the National Institutes of Health Clinical Center. Once researchers have explained the study and obtained the participant s consent, participants will donate 8 cc (approximately 2 teaspoons) of blood. Because repeat testing helps researchers validate study findings, participants who have the unique red blood cell features mentioned above may also be asked if they are willing to return and donate another 2 cc to 8 cc of blood for additional studies. The amount of blood drawn will not exceed 50 ml with any eight-week period for adults or 7 cc within any six-week period for children. | Sickle Cell Trait|Sickle Cell Disease|Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Preventing Stroke Triggers in Children With Sickle Cell Anaemia in Mulago Hospital, Kampala (PREST ): a Randomized Control Trial | Sickle cell anaemia (SCA) is a common hereditary haemoglobin disorder in Africa. World wide it is estimated that about 300,000 newborns are born every year. Of which 75% of them live in Sub-saharan Africa (SSA). In Uganda, about 15,000 babies are born with sickle cell disease per year. In Uganda, the stroke prevalence was found to be 6.2% in children admitted to the National referral hospital in Kampala. Notable between 21 to 30% of these children presented with co-morbidities such as anaemia, bacteraemia and painfull crisis. Stroke in SCA is mediated by several mechanism such as cellular adhesions, inflammatory markers, hemolysis associated oxidative stress and hemostatic activation. Stroke in SCA is primarily a large vessel stroke and the mechanisim state above lead to a narrowing of the lumen of the cerebral arteries Arterial ischaemic stroke which occurs frequently in children with SCA has been associated with bacterial infections. Recent studies have shown that minor infections such as flu like infections can play a critical role in the trigger of stroke in children. Our hypothesis is that viral flu infections is a key trigger for the risk of stroke in children with SCA. Our objective is to prevent the occurrence of flu illnesses in children with SCA thereby reducing the risk for stroke in our population of children with SCA. Methods: A randomized controlled double blinded study Study site: The study will be conducted at the Sickle Cell Clinic (SCC), Mulago Hospital. Inclusion criteria: will be ;age between 2 years and 12 years;All children whose parents will have consented and those above 7years will have to assent. Exclusion criteria: all children with previous strokes; children who have acute illness and are not clinically stable; any child with previous documented adverse event following immunization (AEFI). Sample Size: Using Open EPI calculator for cohort studies we calculated a total sample size of 136 participant to achieve our objective. Using a 95% confidence interval, power of 80% and an unexposed outcome of 25% (4) using a ratio of 1:1. Each arm will have 68 participants. With anticipated 10% loss to follow up a total sample size of 150 with each arm having 75 participants. Study utility: Globally, stroke triggers have been recently identified independent of the existing risk factors such as high cerebral velocity speeds on TCDs. Flues like illnesses have been reported to be stroke triggers in children with arterial ischaemic strokes worldwide.This study may influence the role of influenza vaccination in the prevention of stroke triggers in children with sickle cell anaemia. It will also add to the existing modalities which have helped to reduce the incidence of stroke amongst this high risk group of children with | Preventing Stroke in Sickle Cell Anaemia | ALL | CHILD | Department of paediatrics and child Health,Makerere university, Kampala, P O 7072, Uganda |
| Sickle Cell Anemia WE CARE | This mixed-methods study aims to understand the implementation of a previously tested, efficacious social determinants of health (SDoH) screening and referral intervention in the outpatient pediatric hematology setting; qualitatively assess possible mechanisms for such interventions on improving child health; and obtain population-specific empirical estimates to plan a large-scale clinical trial. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Boston Medical Center, Boston, Massachusetts, 02118, United States |
| Peripheral Arterial Tonometry and Neurocognition in Sickle Cell Disease | This study will examine sleep disordered breathing and sleep quality in participants (ages 12-18) diagnosed with sickle cell disease of any genotype. We will utilize remote peripheral arterial tonometry (PAT) and questionnaires to evaluate difficulties with sleep. PAT assessments will occur remotely in the homes of participants. Neurocognitive, behavioral, and neuroimaging evaluations will occur on the same day as a routine clinic visit. Primary Objective: Evaluate the relationship between nocturnal oxyhemoglobin saturation (SpO2) and neurocognitive functioning (working memory and verbal comprehension) in children (ages 12-18) diagnosed with sickle cell disease controlling for age, genotype, and social vulnerability. Secondary Objective: Assess differences in white matter integrity, silent cerebral infarcts, neuroinflammation, and functional connectivity among children (ages 12-18) diagnosed with sickle cell disease with and without sleep disordered breathing after controlling for age. Assess differences in self- and caregiver-reported mood and pain severity among children (ages 12-18) diagnosed with sickle cell disease with and without sleep disordered breathing after controlling for age. Exploratory Objectives: Explore the relationship between nocturnal oxyhemoglobin saturation (SpO2) and neurocognitive functioning (attention, processing speed, verbal memory, visual memory, motor dexterity) in children (ages 12-18) diagnosed with sickle cell disease controlling for age, genotype, and social vulnerability. Assess the feasibility of an ultraportable ring oximeter (BodimetricsCircul+ Ring) in children (ages 12-18) diagnosed with sickle cell disease. Assess the concordance between the Circul+Ring with the WatchPAT in children (ages 12-18) diagnosed with sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| iCanCope With Sickle Cell Pain | The project will test a tailored web and smartphone-based application (iCanCope with SCD) to improve pain self-management and functioning in youth (aged 12-18) with sickle cell disease. The program will include goal setting, peer-based social support, and pain self-management training. The investigators will determine initial program effectiveness through a pilot three-site randomized controlled trial in 160 youth randomized to treatment compared to attention control. | Sickle Cell Disease | ALL | CHILD, ADULT | Connecticut Children's Medical Center, Hartford, Connecticut, 06106, United States|Emory University, Atlanta, Georgia, 30322, United States|Seattle Children's Hospital, Seattle, Washington, 98105, United States|The Hospital for Sick Children, Toronto, Ontario, Canada |
| The Role of Endothelin-1 in Sickle Cell Disease | The primary goal of the study is to determine the safety and tolerability of ambrisentan. It is also expected that ambrisentan will improve blood flow in the lungs, decrease inflammation, and reduce pain in sickle cell patients. An additional goal is to evaluate the use of select biomarkers in evaluating sickle nephropathy. | Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | Augusta University, Augusta, Georgia, 30912, United States |
| Glucose Metabolism in Sickle Cell Disease | The purpose of the study is to better understand how the body handles sugars glucose and fats, such as cholesterol and triglycerides in sickle cell disease, and what puts certain persons at risk to develop diabetes. This understanding may help us to find new treatments to control blood sugar and prevent diabetes in people with and without sickle cell disease (SCD). In this research, DNA and RNA will be isolated from blood cells. DNA will be used to find genes that cause or protect from diabetes, high cholesterol and high triglyceride, and RNA will be used for studies designed to find out how genes are doing their job of eventually producing proteins. | Sickle Cell Disease|Diabetes Mellitus | ALL | ADULT, OLDER_ADULT | University of Illinois at Chicago, Chicago, Illinois, 60612, United States |
| Hepcidin Levels in Sickle Cell Disease (SCD) | The investigators propose that patients with HbSβ-thalassemia have lower levels of hepcidin and higher levels of GDF-15 than HbSS patients during the non-crisis, "steady states." In addition, the investigators propose that when controlled for RBC transfusion, patients with HbSβ-thalassemia will have higher levels of storage iron (based on serum ferritin). Participants: Total number of subjects is 42 - 21 subjects with HbSS, and 21 subjects with HbSβ-thalassemia ). Procedures (methods): Eligible subjects with documented SCD (HbSS, HbS-β 0-thalassemia or HbS-β+-thalassemia) followed at the University of North Carolina (UNC) Comprehensive Sickle Cell Program will be evaluated in this single-center, prospective, cross-sectional study. The patients will be screened for eligibility at the time of a routine sickle cell clinic visit. Patients' data will be obtained in person at the time of evaluation and through review of their medical records. Investigators will obtain information on SCD-related clinical complications and obtain an estimate of the number of lifetime RBC transfusions. Blood samples will be obtained for laboratory tests. Plasma samples for hepcidin, growth differentiation factor 15 (GDF -15), and high-sensitivity CRP will be stored at -80 degrees Celsius until analysis. Other routine laboratory studies including complete blood count (CBC) with differential and reticulocyte count, serum iron profile and ferritin, and liver function tests will be performed at the clinical laboratories of UNC Hospitals.The subjects will have 30 ml. of blood drawn for this research study. Females of child bearing potential will have a urine pregnancy test at the time of the study. | Sickle Cell Anemia|Sickle - Beta Thalassemia | ALL | ADULT, OLDER_ADULT | |
| Natural History of Sickle Cell Disease | This study is not a treatment protocol and no experimental treatments are involved. Study participants may be seen as needed for clinical, translational and basic research studies, or as medically indicated. Subjects will receive their general medical care outside the NIH and will be seen at our clinic or at CNHS with varying frequency. Subjects may be seen for multiple visits. Subjects may be asked to return for additional testing as needed. Clinical care for patients with sickle cell disease will be provided as appropriate through the Sickle Cell Clinic and the inpatient clinical center. | Pain Crisis | ALL | CHILD, ADULT, OLDER_ADULT | Childrens National Health Center, Washington, District of Columbia, 20010, United States|Suburban Hospital, Bethesda, Maryland, 20814, United States|National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Telemedicine for Children With Sickle Cell Disease | The purpose of this study is to learn more about how the use of two different types of telemedicine (distance medical care) can address barriers to receiving comprehensive sickle cell care, and whether care can be improved. Aim 1: Adapt two telemedicine models (i.e., hub-and-spoke; direct-to-consumer) for use with children with SCD using caregiver input from our preliminary K12 work. Aim 2: Demonstrate the feasibility of the telemedicine models developed in Aim 1 as the models undergo successive stakeholder refinement during use in actual clinical care. Aim 3: Evaluate the effectiveness of the refined models from Aim 2 in a pre/post study by assessing (a) process of care measures, (b) provider satisfaction, (c) caregiver/patient-centered outcomes, and (d) clinical outcomes and healthcare utilization. | Anemia, Sickle Cell | ALL | CHILD, ADULT, OLDER_ADULT | Indiana University, Indianapolis, Indiana, 46202, United States |
| Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease | Background: Peripheral blood stem cell transplantation procedures are used for people with sickle cell disease. Researchers want to improve the success and reduce the complications for these procedures. This might allow more people to have a transplant. Objective: To see if a new transplant regime is effective, safe and well tolerated in people with sickle cell disease. Eligibility: Adults at least 18 years old with sickle cell disease and certain complications. A relative who is a half tissue match. Design: Participants will be screened with medical history, physical exam, and blood tests. Recipients will also have: * Heart, lung, and mental health tests * Chest x-rays * Bone marrow taken from the pelvic bone * Eyes and teeth checked Recipients will have a large central line inserted into a vein for up to 6 months. Donors will have their veins tested and have an IV inserted for 1 day or on rare occasions 2 days. Donors will get a drug to activate bone marrow. It will be injected for about 6 days. Donors will have at least 1 five-hour procedure where bone marrow stem cells will be collected. Blood will be taken from a vein in one arm or in rare cases from a groin vein and put through a machine. Some blood will be saved and the rest will be returned. Stem cells will be taken from the saved blood in a lab and frozen until ready to give to the recipient. Recipients will have: * Stems cells collected and frozen * Hygiene lessons * Bone density scans * Low-dose radiation * Drugs for their immune system * Donor cells infused through their central line * Transfusions After about 30 days, recipients will leave the hospital. They must stay near NIH for 3 months after the transplant and have frequent visits. After returning home, they will have 8 visits over 5 years, then be contacted yearly. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Ketorolac Versus Ibuprofen to Treat Painful Episodes of Sickle Cell Disease | The purpose of this study is to compare ketorolac, a potent, non-steroidal anti-inflammatory drug (NSAID), with ibuprofen, a commonly used NSAID, for the treatment of the painful crisis of sickle cell disease (SCD). | Hematologic Diseases|Anemia, Sickle Cell | ALL | CHILD, ADULT | University of Texas Southwestern Medical Center, Dallas, Texas, 75390, United States |
| Cerebrovascular Involvement in Sickle Cell Disease - Comprehensive Sickle Cell Center | To continue studies on the two major neurological complications of sickle cell disease (SCD): namely, stroke and chronic encephalopathy. | Anemia, Sickle Cell|Blood Disease|Cerebrovascular Accident | MALE | CHILD, ADULT, OLDER_ADULT | |
| Sickle Cell Disease: A Retrospective Chart Review | This study is a retrospective chart review of sickle cell patients and will include patients whom have received blood transfusions and those whom have not. Of the transfused patients, it will also include those whom have received chelation therapy and those whom have not. | Sickle Cell Anemia | ALL | CHILD, ADULT, OLDER_ADULT | Tulane University, New Orleans, Louisiana, 70112, United States|Universit of Tennessee, Memphis, Tennessee, 38163, United States |
| Dihydroartemisinin-Piperaquine or Sulphadoxine-Pyrimethamine for the Chemoprevention of Malaria in Sickle Cell Anaemia | Sickle Cell Anaemia (SCA) is an inherited disease that makes the body produce red blood cells with abnormal sickle-shaped cells. The sickle-shaped cells are rigid, not flexible and break up easily resulting in anaemia. The abnormal cells also stick to the vessel walls, causing a blockage that slows or stops the flow of blood. When this happens, oxygen cannot reach nearby tissues. The lack of oxygen can cause attacks of sudden, severe pain, called pain crises, stroke or damage to important organs such as the spleen. All of these can lead to death. These attacks can occur without warning and are often started and made worse by infections such as malaria. Therefore, in many countries in Africa where malaria is common, children with SCA are given malaria medicines to prevent the infection. However, many of the medicines do not work effectively, are too difficult to take or they have side effects, resulting in poor adherence. The aim of this study is to find safe, acceptable and effective medicines for malaria prevention in children with SCA in eastern and southern Africa. The investigators propose to conduct a study to find out whether giving weekly doses of dihydroartemisinin-piperaquine, also called DP, is safe, more effective, acceptable and cost-effective than the current strategy of monthly sulphadoxine-pyrimethamine (SP) to prevent malaria in children with sickle cell anaemia. Overall, 548 children aged 6 months to 15 years will be chosen randomly to receive either weekly DP or monthly SP for about 18 months. To test if the study medicine is effective, the study will compare the case burden of malaria. The investigators will also monitor every child for any type of illness, blood transfusions and other complications of sickle cell anaemia and admissions to the hospital. In addition, the study will evaluate the impact of DP on the development of resistance by malaria parasites. The study will also include nested safety studies on the effect of DP on the heart. All study participants will receive all the other usual care and treatments, including patient education on home care, and daily penicillin if younger than 5 years. If proven safe and efficacious, chemoprophylaxis with DP may decrease the incidence of malaria in children with SCA, prevent ill-health and deaths, and improve wellbeing. | Sickle Cell Anemia in Children|Malaria | ALL | CHILD | Queen Elizabeth Hospital, Blantyre, Malawi|Jinja Regional Referral hospital, Jinja, Uganda|Kitgum General Hospital, Kitgum, Uganda |
| SMILES: Study of Montelukast in Sickle Cell Disease | Sickle cell disease (SCD) is a genetic blood condition causing long term health problems including pain and brain problems which affect quality of life. These may be made worse if patients have low night-time oxygen levels when the upper airways close repeatedly during the night (obstructive sleep apnoea). This is associated with increased pain, poorer concentration and increased kidney problems. Montelukast, widely used in the treatment of Asthma, has been shown to improve symptoms of obstructive sleep apnoea in patients without sickle cell anaemia. Investigators think this treatment could be useful in patients with sickle cell disease too. Early intervention with Montelukast could help prevent deterioration in concentration and thinking skills. The aim of this trial is to see whether young children with sickle cell disease randomised (randomise: the same as tossing a coin and not knowing whether it will come up heads or tails) to Montelukast treatment have better thinking skills compared with people randomised to placebo (tablet with no active medical ingredients - i.e. "sugar pill"). This means that the child could be on Montelukast treatment or he/she might be on placebo tablets. | Anemia, Sickle Cell|Sleep-Disordered Breathing | ALL | CHILD | Great Ormond Street Hospital NHS Foundation Trust, London, WC1N 1EH, United Kingdom |
| Acupressure in Patients With Sickle Cell Disease | The proposed research is to determine the clinical efficacy and neurobiological mechanisms of acupressure analgesia in patients with sickle cell disease (SCD). | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | University of California, Irvine, Irvine, California, 92868, United States|Indiana University School of Medicine, Indianapolis, Indiana, 46075, United States |
| Enablers and Barriers to Hydroxyurea Use for Sickle Cell Disease Jamaica | Sickle cell disease (SCD) is associated with a lifetime of medical and socio-behavioural complications that require coordination of care from multidisciplinary teams. Access to adequate care for SCD is important as inadequate access can contribute to increased acute care utilization, disjointed care delivery, and earlier mortality for many SCD patients. Hydroxyurea (HU) is the first drug approved for the treatment of SCD and improves many adverse outcomes of SCD and yet its use remains sub-optimal. This mixed-methods study aims to identify the barriers and enablers that SCD patients, caregivers of children (under age 18 years), and health care providers (including physicians, nurses and pharmacists) identify for health care access and HU utilization. The findings may guide development and implementation of strategies to improve access to SCD healthcare and HU uptake which may result in significant benefits to patients, families and the healthcare system including possible reduction in healthcare utilization. Participants will be recruited from the Sickle Cell Unit, Kingston and from all four Jamaican regional health authorities. Questionnaires and interview guides for provider and patient/caregiver assessments are adapted, with permission, from the Sickle Cell Disease Implementation Consortium tools. The study will also examine data on HU usage from the National Health Fund of Jamaica since its addition of SCD to its list of chronic illnesses in 2015. All data collected will be de-identified and maintained in a secure database, with access limited to key personnel. There is minimal risk to participants. Participants will be selected only because of the specific problem under investigation, and not because of easy availability, diminished autonomy, or social bias. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Sickle Cell Unit - Caribbean Institute for Health Research, Kingston, Saint Andrew, Kingston 7, Jamaica |
| Evaluation of the Hemostatic Potential in Sickle Cell Disease Patients | Sickle cell disease is a genetic disorder caused by a point mutation on the amino acid sequence of the β chain of hemoglobin. The most expressive and most frequent complication of the disease is vaso-occlusive crisis, dominated by a painful syndrome. In addition to vaso-occlusive crises, many more chronic biological disturbances are observed in sickle cell patients.Sickle cell disease is considered nowadays as a hypercoagulable state. However, the approach used so far to the measure of clotting in sickle cell disease was segmented in the sense that the various components of the hemostatic balance were studied separately.The thrombin generation test is a functional test which explores the coagulation globally, integrating both pro players that anticoagulants actors in the system. The investigators already used this test to demonstrate that the hemostatic potential was high in a cohort of affected children compared to control children of the same age. This test will be used to characterize the hemostatic potential of adult sickle cell patients followed at the CHU Brugmann Hospital. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | CHU Brugmann Hospital, Brussels, 1020, Belgium |
| Kidney Function in Sickle Cell Anemia | This is a prospective clinical cohort study that involves a baseline study visit followed by up to 3 annual follow-up study visits for a total follow-up of 36-48 months to evaluate the age- and sex-adjusted rate of change in kidney function, and to identify biomarkers of endothelial function, metabolomic profiles and clinical characteristics for the worsening of kidney function and for a rapid decline in kidney function. "Funding Source - FDA OOPD" | Sickle Cell Disease|Kidney Failure, Chronic | ALL | ADULT, OLDER_ADULT | University of North Carolina-Chapel Hill, Chapel Hill, North Carolina, 27599, United States|Ohio State Adult Sickle Cell Program, Columbus, Ohio, 43210, United States|UTHSC Center for Sickle Cell Disease, Memphis, Tennessee, 38163, United States |
| L-Arginine and Sickle Cell Disease | One of the main problems in sickle cell disease is the decreased bioavailability of nitric oxide and arginine. This study was designed to assess if treating sickle cell disease patients with L-arginine would improve pulmonary arterial pressure and other aspects. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, 90035-001, Brazil |
| Cognitive Rehabilitation in Sickle Cell Disease | The majority of school-age children with sickle cell disease (SCD) experience neurocognitive deficits, even in the absence of stroke. In particular, deficits in attention and working memory have emerged as two of the most common neurocognitive sequelae of SCD. Thus, the goal of the present proposal is to address feasibility and compliance of a novel computerized cognitive training program, Cogmed. Pilot data will also be collected to establish preliminary efficacy. Twenty-four children meeting initial age and diagnostic criteria will be identified and approached about participation by their attending physician during regularly-scheduled SCD clinic visits. Baseline assessments will include a brief measure of intellectual functioning, a brief cognitive testing battery evaluating processing speed and working memory, in addition to questionnaires regarding behavior and quality of life. Children will then be randomized to the computerized CT program Cogmed (n=12) or a waitlist control (n=12). Participants enrolled in the computerized CT program will be asked to complete 25-sessions of Cogmed over a five to eight week period (3 to 5 sessions per week). Following completion of the program, children and their parents will be asked to return to clinic for a follow-up visit. After a five to eight-week waiting period, children in the waitlist condition will also be asked to return to clinic for a second visit. Following this assessment, participants initially enrolled in the waitlist will be offered an opportunity to participant in the intervention. If interested, they will follow the same intervention protocol described above. These children will return to clinic for a third visit following completion of the intervention. Compliance rate and its confidence interval will be calculated for the overall study population. A t-test for binomial proportion with continuity correction will be used to examine whether the compliance rate is lower than the target. Participants' change in criterion outcomes will be evaluated (i.e., those neurocognitive measures such as attention, executive functioning and working memory, that are most closely related to the trained tasks). | Sickle Cell Disease|Cognitive Impairment | ALL | CHILD | Duke Child and Family Study Center, Durham, North Carolina, 27705, United States |
| Exploring Adherence Monitoring in Sickle Cell Disease | Despite the well-documented benefits of hydroxyurea (HU) therapy in decreasing morbidity and mortality in youth with Sickle cell disease (SCD), pediatric HU adherence rates range as low as 49% and lead to discontinuation of HU regimens in 8-20%. In addition, treatment non-adherence may lead to unnecessary increases in medication dosage resulting from erroneous assumption that a patient is non-responsive to treatment (versus non-adherent to the regimen as prescribed). Given the detrimental effects of non-adherence, assessment of and intervention for HU non-adherence is essential to improving health outcomes in the pediatric SCD population. Electronic adherence monitoring is widely considered the "gold standard" in objective adherence measurement. These monitors provide continuous, real- time records of medication adherence and reveal problematic behavior patterns, including underdosing, overdosing, delayed dosing, "drug holidays," and "white coat" adherence. Overall, electronic adherence measures are considered valid, reliable, and accurate, with clear advantages over pharmacy refill records, physician estimates and self-report measures. The primary purpose of this pilot study is to determine the use of the AdhereTech as a feasible and valid measure of HU adherence in pediatric SCD. Primary Objective Estimate the association between HU adherence as measured by the AdhereTech device to a) caregiver-report, b) youth-report, c) lab values, d) pill- count, and e) Medication Possession Ratio (MPR) adherence measures Secondary Objectives Estimate the rate of consent to the study, the rate of AdhereTech device use, the rate of AdhereTech device failure, and the perceived acceptability of using the AdhereTech device, as reported by caregivers and youth | Sickle Cell Disease | ALL | CHILD | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Prasugrel Versus Placebo in Adult Sickle Cell Disease | The purpose of this trial is to assess the safety of Prasugrel in adult patients with sickle cell disease (SCD) by monitoring the rate and severity of hemorrhagic events requiring medical intervention compared to placebo for 30 days. | Sickle Cell Anemia | ALL | ADULT | For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Birmingham, Alabama, 35205, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Little Rock, Arkansas, 72211, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Sacramento, California, 95817, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Daytona Beach, Florida, 32117, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Augusta, Georgia, 30912, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Indianapolis, Indiana, 46260, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Baltimore, Maryland, 21205, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Boston, Massachusetts, 02118, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Jackson, Mississippi, 39216, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Chapel Hill, North Carolina, 27599, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Greenville, North Carolina, 27834, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Jenkintown, Pennsylvania, 19046, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Pittsburgh, Pennsylvania, 15224, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Houston, Texas, 77002, United States |
| A Low-Interventional Study of an Electronic Sickle Cell Disease Patient Reported Outcomes in Sickle Cell Participants | The purpose of this study is to learn more about painful crisis in people with Sickle Cell Disease. For this reason, Pfizer is conducting a study to understand how people with Sickle Cell Disease feel on a daily basis, treat these painful episodes including if they treat themselves at home or go to a doctor's office/emergency room, as well as the types of medications that are taken during these episodes. | Sickle Cell Disease|Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | Sanguine Biosciences, Woburn, Massachusetts, 01801, United States |
| Adapting the FACETS Program to Sickle Cell Disease | Adaptation of a fatigue management program combining the principles of cognitive-behavioral therapy and energy conservation strategies (FACETS program) for a population of adult patients with sickle cell disease (Drépa-FACETS program). | Fatigue Symptom|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | |
| Stem Cell Transplant in Sickle Cell Disease and Thalassemia | The primary purpose of this study is to see if giving lower doses of chemotherapy (moderately ablative) will result in successful bone marrow replacement without as severe side-effects but with permanent control of the disease. Patients will receive a chemotherapy regimen with busulfan, fludarabine, and alemtuzumab followed by an infusion of stem cells, either from a family-related or cord-blood matched donor. | Sickle Cell Disease|Beta Thalassemia | ALL | CHILD, ADULT | Morgan Stanley Children's Hospital, New York-Presbyterian, Columbia University, New York, New York, 10032, United States |
| Cohort Study of Sickle Cell Patients Evaluating Analgesic Strategies and Their Consequences | To date, there is no comprehensive data among adults with sickle cell disease concerning the prevalence of consumption of level 2 and 3 analgesics at home and the use of cannabis or CBD for analgesic use. Preliminary data shows a very high prevalence of an opioid use disorder (OUD) in these patients, above 50%, but more precise additional studies are necessary to objectify these results and implement appropriate multidisciplinary care. The general objective of this project is to establish an exhaustive collection on the addiction of sickle cell patients followed in a sickle cell reference center (hôpital Henri Mondor, hôpital Necker-Enfants Malades and hôpital européen Georges-Pompidou, Assistance Publique-Hôpitaux de Paris). The study investigative team proposes to set up a prospective observational study using a questionnaire. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Hôpital Henri Mondor, Créteil, 94010, France|Hôpital européen Georges-Pompidou, Paris, 75015, France|Hôpital Necker-Enfants Malades, Paris, 75015, France |
| The Efficacy of Jobelyn (Sorghum Bicolor Extract)in the Treatment of Sickle Cell Anemia | The primary objective is to determine if there is a significant increase in the haematocrit value of patients on Jobelyn and standard therapy compared to those on standard therapy alone. | Sickle Cell Anemia | ALL | CHILD, ADULT | Lagos State University Teaching Hospital, Ikeja, Lagos, 100001, Nigeria |
| Imatinib for Pain in Sickle Cell Anemia | In this protocol, the investigators propose to evaluate the biochemical effects of imatinib on sickle red blood cells (RBCs). Patients will be administered imatinib mesylate orally following the guidelines previously established for use of imatinib in other disorders. The biochemical effects of imatinib on sickle RBCs will be examined, including changes in their levels of band 3 tyrosine phosphorylation and the abundances of RBC-derived microparticles in their blood. In addition, the patients will be monitored for symptoms of sickle cell disease (SCD). The investigators expect band 3 tyrosine phosphorylation to decrease dramatically in patients treated with imatinib. The investigators also anticipate a reduction in the numbers of RBC-derived microparticles in circulation (quantitated by assaying the number of glycophorin A positive microparticles in peripheral blood samples by flow cytometry. Most importantly, the investigators expect to see a reduction in the frequency of vaso-occlusive crises, and possibly acute chest syndrome and utilization of opioids. The study duration is planned as 6 months in order to provide adequate time for potential change in the primary endpoints (e.g. percent irreversibly sickled cells). | Sickle Cell Disease | ALL | ADULT | Riley Hospital for Children at IU Health, Indianapolis, Indiana, 46202, United States|Cincinnati Children's Hospital, Cincinnati, Ohio, 45229, United States |
| Music Therapy in Sickle Cell Transition Study | The purpose of this study is to investigate the effects of the BEATS Music Therapy Program on the self-efficacy, trust, knowledge, and adherence of young adult patients with sickle cell disease during transition. Primary Hypotheses: Compared to baseline, young adult patients with sickle cell disease who receive the music therapy interventions will report: 1. Higher sickle cell self-efficacy as measured by the Sickle Cell Self Efficacy Scale (SCSES), 2. Higher trust in health care providers as measured by the Wake Forest Trust in the Medical Profession Scale, and 3. Higher sickle cell disease knowledge as measured by the Seidman Sickle Cell Knowledge Quiz. Secondary Hypotheses 1. Compared to the one year prior to the study period, young adults with sickle cell disease who receive the music therapy interventions will have a higher rate of adherence to clinic appointments during the one-year study period. Additional Questions 1. Do music therapy interventions influence attendance to scheduled blood transfusions? 2. Do music therapy interventions influence the rate of hospital utilization as measured by Emergency Department visits, Acute Care Clinic visits, and admissions during the study period compared to the previous year? 3. Do music therapy interventions influence adherence to hydroxyurea therapy for patients receiving hydroxyurea as measured by change in mean corpuscular volume (MCV) during the study period? 4. Do music therapy interventions influence adherence to iron chelation therapy for patients receiving iron chelation therapy as measured by ferritin count during the study period? | Sickle Cell Disease | ALL | ADULT | University Hospitals Seidman Cancer Center, Cleveland, Ohio, 44106, United States |
| Minocycline in Neurocognitive Outcomes - Sickle Cell Disease | Sickle cell disease (SCD) is a common, inherited blood disorder that primarily affects people of African Ancestry. It has a lot of complications including neurological complications. The neurological complications of SCD are particularly devastating and lead to cognitive decline even in the absence of overt brain injury. In such cases, it is thought that inflammation in the brain maybe partly responsible for the cognitive decline. The main reasons for this research study are to see 1) how safe and 2) how well minocycline works to try to stop/reverse cognitive decline in people with SCD. People with SCD are at risk for changes in their brain over time that can cause problems with learning, memory, and attention. Part of the reason for this is inflammation within the brain. Minocycline may be able to stop these brain changes by stopping this brain inflammation. Minocycline is a second-generation tetracycline antibiotic that has been shown to both inhibit neuroinflammation and improve cognitive function in a variety of neurodegenerative and psychiatric disorders but has not yet been studied in SCD. We are proposing here, a pilot double-blinded, randomized controlled trial to examine the tolerability and early efficacy of minocycline in adults with SCD at two dosing regimens (200 mg and 300 mg daily) versus placebo over one year. Participants will undergo a neuropsychological exam using the NIH Toolbox Cognition Battery at both study enrollment and exit (after one year) to assess for changes/stability of cognition. Participants will receive monthly phone calls/text messages to assess for adverse events and will be seen every three months for pill counts and routine laboratory monitoring. The primary outcome will be a comparison of adverse events across the two dosing strategies versus placebo. Early evidence for cognitive benefit will also be assessed from the results of the NIH Toolbox. | Sickle Cell Disease|Cognitive Impairment|Cognitive Decline|Cognitive Change|Cognitive Dysfunction|Cognitive Deficit|Neuroinflammatory Response | ALL | ADULT, OLDER_ADULT | |
| Heart Disease in Sickle Cell Anemia | This study will explore what may cause people with sickle cell anemia to have heart problems and an increased risk of sudden death. People 18 years of age and older with sickle cell anemia may be eligible for this study. Candidates are screened with a medical history and physical examination, electrocardiogram (EKG), echocardiogram (heart ultrasound), and blood tests. Participants undergo the following tests and procedures: * Holter monitoring: The patient wears a small, battery-operated device to record heart rate and rhythm over 24 to 48 hours. * QRST surface mapping: An EKG using 64 electrodes is done at rest and during exercise to provide a detailed look at the heart and its conduction system. * Chest x-rays are taken to examine the lungs. * Bicycle exercise echocardiography test: Blood pressure, pulse, heart rhythm and oxygen use are monitored while the patient exercises on a stationary bicycle. Ultrasound pictures are also obtained during the exercise. * Echocardiogram: A heart ultrasound is done to check how well the heart is pumping blood. * Pulmonary artery catheterization: A catheter (plastic tube) is inserted into a vein and advanced to the chambers of the heart, through the heart valve and into the lung artery. The pressures in the heart and lung blood vessels are measured while the patient is resting and during exercise, with the bed tilted up and down, and after giving 500 mls of fluid into a vein. * Blood tests are done to measure a hormone called brain natriuretic peptide, which can increase with the development of heart failure, and nitrite, a substance that can affect blood vessel dilation. Some blood is stored to test for inflammatory markers and for possible future gene and protein analysis. * Cardiac magnetic resonance imaging (cMRI): The patient lies in a donut-shaped magnet while pictures of the heart are obtained using a magnetic field and radio waves. Earplugs are worn to muffle the loud sounds that occur with electrical switching of the magnetic fields. A contrast agent called gadolinium may be injected to enhance the quality of the images. * Invasive electrocardiographic (reveal) monitoring: This procedure permits study of the heart rhythms over a long time period. A small device is placed just under the skin on the left side of the chest. It can be left in for up to 14 months to monitor the heartbeat continuously during this time. | Sickle Cell Anemia|Pulmonary Hypertension | ALL | ADULT | Suburban Hospital, Bethesda, Maryland, 20814, United States|National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| A 3-year, Prospective, Non-interventional, Multicenter Registry in Sickle Cell Disease (SCD) Patients | A long term observational study in sickle cell disease will enhance the understanding of the disease patterns, current transfusion practices, treatments and outcomes in sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Hematology Oncology Services of Arkansas, Little Rock, Arkansas, 72205, United States|Children's Hospital Oakland (CHO), Oakland, California, 94609, United States|Children's Hospital of Orange County Sickle Cell Disease Center, Orange, California, 92668, United States|Rady Children's Hopsital / UC San Diego University Hospital Sickle Cell Disease Center, San Diego, California, 92123, United States|Hematology Oncology, P.C., Stamford, Connecticut, 06902, United States|Howard University, Washington, District of Columbia, 20060, United States|Broward Oncology Associates, P.A., Fort Lauderdale, Florida, 33308, United States|Miami Children's Hospital, Miami, Florida, 33155, United States|Innovative Medical Research, Miami, Florida, 33179, United States|M.D. Anderson Cancer Center Orlando, Orlando, Florida, 32806, United States|All Children's Hospital, Saint Petersburg, Florida, 33701, United States|Central Tampa Hematology and Oncology Associates, Tampa, Florida, 33607, United States|St. Joseph Children's Hospital, Tampa, Florida, 33607, United States|University of Illinois at Chicago, Dept of Pediatrics, Chicago, Illinois, 60612, United States|Abington Hematology Oncology Associates, Inc, Oak Lawn, Illinois, 60453, United States|Tulane University School of Medicine, New Orleans, Louisiana, 70112, United States|LSU Department of Pediatrics Children's Hospital, New Orleans, Louisiana, 70118, United States|Johns Hopkins University Division of Hematology, Baltimore, Maryland, 21205, United States|Oncology-Hematology Associates, P.A., Clinton, Maryland, 20735, United States|Children's Hospital Boston - Harvard, Boston, Massachusetts, 02115, United States|Boston Comprehensive Sickle Cell Center, Boston, Massachusetts, 02118, United States|West Michigan Cancer Center, Kalamazoo, Michigan, 49007-3731, United States|University of Missouri Child Health, Division of Pediatric Hematology/Oncology, Columbia, Missouri, 65212, United States|Children's Mercy Hospital, Kansas City, Missouri, 64108, United States|Research Medical Center, Kansas City, Missouri, 64132, United States|Newark Beth Israel Medical Center, Newark, New Jersey, 07112, United States|Maimonides Medical Center, Brooklyn, New York, 11209, United States|New York Methodist Hospital Dept of Med/Sickle Cell Program, Brooklyn, New York, 11215, United States|Interfaith Medical Center-Comprehensive Sickle Cell Program, Brooklyn, New York, 11238, United States|Cohen Children's Medical Center of NY (CCMC), New Hyde Park, New York, 11040, United States|Center for Children's Cancer & Blood Disorders, Syracuse, New York, 13210, United States|Presbyterian Hospital Pediatric Sickle Cell Program, Charlotte, North Carolina, 28204, United States|Ohio Region VI Sickle Cell Program, Akron, Ohio, 44308, United States|Cincinnati Children's Medical Center, Cincinnati, Ohio, 45229-3039, United States|The Children's Medical Center of Dayton, Dayton, Ohio, 45404, United States|Medical University of SC-Pediatric Sickle Cell Center, Charleston, South Carolina, 29425, United States|M. Francisco Gonzalez, M.D., P.A., Columbia, South Carolina, 29203, United States|Diggs-Kraus Sickle Cell Center, Memphis, Tennessee, 38103, United States|Meharry Comprehensive Sickle Cell Center, Nashville, Tennessee, 37208, United States|Sickle Cell Disease and Hemoglobinopathy Clinic Children's Hospital at Vanderbilt, Nashville, Tennessee, 37232-6310, United States|Baylor College of Medicine-Texas Children's Hospital, Houston, Texas, 77030-2399, United States|Peninsula Cancer Institute, Newport News, Virginia, 23601, United States |
| Primary Prevention of Stroke in Children With Sickle Cell Anaemia in Nigeria in the Community | The overall goal of this feasibility study is to establish a standard of care stroke prevention program for children with sickle cell anemia in a community hospital by task shifting stroke detection and transcranial Doppler ultrasound screening to nurses. In Nigeria, approximately 150,000 children with sickle cell anemia (SCA) are born annually, accounting for more than half of the total births with SCA worldwide. In comparison, only 1,700 children with SCA are born in the United States annually. An estimated 11% of unscreened and untreated children at increase of strokes with SCA will have at least one stroke by 17 years of age. In high-income countries, evidence-based practices (EBP) for primary stroke prevention in children with SCA involves screening for abnormal transcranial Doppler ultrasound (TCD) velocity (\>200cm/s) coupled with regular blood transfusion therapy for at least one year followed by treatment with hydroxyurea is considered standard care. This strategy decreases the risk of stroke by 92%. Due to safety and availability, regular blood transfusion is not a viable option for primary stroke prevention in most low-income settings, including Nigeria, where \~50% of the 300,000 children with SCA are born. Among each birth cohort, 15,000 children will have stroke annually in Nigeria. The American Society of Hematology (ASH) Central Nervous System Guidelines recommends moderate dose hydroxyurea (20mg/kg) to children with SCA with abnormal TCD measurements, living in resource-constrained settings where regular blood transfusions are not readily available. Our team has demonstrated in a previous trial the feasibility of primary stroke prevention with hydroxyurea in Kano, Nigeria. In 2016, as part of capacity building objective of Stroke Prevention Trial in Nigeria (1R01NS094041-SPRING) at Barau Dikko Teaching Hospital in Kaduna, TCD screening was adopted as standard of care. Before the trial, no TCD screening was done at our trial site in Kaduna. Now, as standard care, physicians at the teaching hospital do TCD screening, however, only 5.4% (1,101/20,040) of the eligible children with SCA living in Kaduna, Nigeria were reached. Clearly, for there to be an appreciable impact on decreasing the stroke rates in children with SCA living in Nigeria and elsewhere, applying the ASH guidelines and a better implementation strategy to increase the TCD reach (proportion of children eligible for TCD screening that are screened) is necessary. Therefore, objective of this physician-mentored application is to conduct an Effectiveness-Implementation Feasibility Trial is to test the test the hypothesis that the task-shifted site for primary stroke prevention team in a community hospital will have a non-inferior effectiveness in identifying children with abnormal TCD measurements when compared to primary stroke prevention team in a teaching hospital in Kaduna, Nigeria. the investigators will conduct i) a needs assessment at the community hospital to identify barriers and facilitators to the intervention, ii) Build capacity for stroke detection and TCD screening and iii) Compare the effectiveness of a physician-based stroke prevention program in a teaching hospital to a task-shifted stroke prevention in a community hospital. | Sickle Cell Disease|Stroke | ALL | CHILD | Barau Dikko Teaching Hospital/Kaduna State University, Kaduna, Nigeria |
| Study of Erythrocyte Parameters and Hypercoagulability in Sickle Cell Disease (SCD-TGA) | Sickle cell disease (SCD) is an inherited haemoglobinopathy disorder caused by mutations in HBB gene with amino-acid substitution on β globin chain. The consequence is synthesis of altered haemoglobin S (HbS) which polymerises in red blood cell (RBC) at deoxygenated state. SCD is associated with chronic haemolytic anaemia, vaso-occlusive crisis (VOC) leading to frequent hospitalisation. The aim of the study was to to investigate whether a combination of routine laboratory biomarkers of haemolysis could be used to predict VOC development in confirmed SCD patients. | Sickle Cell Disease|Vaso-occlusive Crisis | ALL | ADULT, OLDER_ADULT | Rouen university Hospital, Rouen, 76000, France |
| European Sickle Cell Disease Cohort - Hydroxyurea | In the context of the Risk Management Plan (RMP), as requested from Addmedica by the EMEA, to collect information about long-term safety of Siklos® (hydroxycarbamide) when used in patients with Sickle Cell Disease. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | CHU d'Amiens - Picardie - Site sud, Amiens, France|Centre Hospitalier Robert Ballanger, pédiatrie et néonatalogie, Aulnay-sous-Bois, 93602, France|Hôpital Avicenne; médecine interne, Bobigny, 93009, France|Hôpital Jean Verdier; médecine interne, Bondy, 93143, France|Hôpital Jean Verdier; pédiatrie, Bondy, 93143, France|CHU Bordeaux, urgences pédiatriques, Bordeaux, 33000, France|Hôpital Saint-André, Bordeaux, France|CHU Estaing, pédiatrie, Clermont Ferrand, 63003, France|Hôpital Louis Mourier; pédiatrie, Colombes, 92700, France|Hôpital Louis Mourier; service de pédiatrie, Colombes, 92700, France|Centre hospitalier intercommunal de Créteil, Créteil, 94010, France|Hôpital Henri Mondor, centre de la Drépanocytose, Créteil, 94010, France|Centre Hospitalier de Gonesse, médecine interne, Gonesse, 95500, France|Centre Hospitalier de Gonesse, pédiatrie, Gonesse, 95500, France|CHU de Grenoble, hôpital Couple Enfant, Grenoble, 38700, France|CHU de Grenoble, Grenoble, France|CHU de Limoges, Limoges, France|Hôpital Mère-Enfant, Limoges, France|Hôpital Edouard Herriot, hémovigilance, Lyon, 69003, France|Institut d'Hématologie et d'Oncologie Pédiatrique, Lyon, 69373, France|CHU Timone, Marseille, 13385, France|Hôpital de la Conception, médecine interne, Marseille, 13385, France|Centre Hospitalier Intercommunal André Grégoire, Montreuil-sous-Bois, 93100, France|CHU Nantes, Hôpital Hôtel Dieu, Nantes, 44093, France|Hôpital Trousseau; pédiatrie générale, Paris, 75012, France|Hôpital Européen Georges Pompidou, Paris, 75015, France|Hôpital Necker enfants, maladies infectieuses et tropicales, Paris, 75015, France|Hôpital Necker; biothérapie, Paris, 75015, France|Hôpital Necker; maladies infectieuses et tropicales, Paris, 75015, France|Hôpital Robert Debré, immunologie-hématologie pédiatrique, Paris, 75935, France|CHR Reims, American Memorial Hospital, Reims, 51100, France|CHU Rennes, Hôpital Pontchaillou, Rennes, 35033, France|CHU Rennes, hôpital Sud, Rennes, 35203, France|CHU Charles Nicolle, Rouen, 76000, France|CHU de Rouen - Hôpital Charles Nicolle, Rouen, 76031, France|Centre Hospitalier Delafontaine, médecine interne, Saint-Denis, 93205, France|Centre Hospitalier Delafontaine, pédiatrie, Saint-Denis, 93205, France|CHRU Strasbourg, Hôpital de Hautepierre, Strasbourg, 67098, France|CHU Toulouse; hôpital des enfants, Toulouse, 31059, France|Institut Universitaire du cancer; médecine interne, Toulouse, 31059, France|CHU Nancy, Brabois enfants, Vandoeuvre-lès-Nancy, 54511, France|CH André Rosemon, Cayenne, 97306, French Guiana|CMCK, Kourou, 97387, French Guiana|CH Ouest Guyanais, Saint Laurent du Maroni, 97320, French Guiana|Charité-Universitätsmedizin, Berlin, Berlin, Germany|Pädiatrie Klinikum Delmenhorst, Delmenhorst, Germany|Heinrich-Heine Universität Düsseldorf, Düsseldorf, Germany|University of Freiburg, Freiburg, Germany|Universitätsklinikum Hamburg-Eppendorf, Hamburg, Germany|München, München, Germany|Laiko General Hospital, Athens, Greece|CH Basse-Terre, adultes, Basse-Terre, 97100, Guadeloupe|CH Basse-Terre, enfants, Basse-Terre, 97100, Guadeloupe|CHU Pointe à Pitre, Pointe-à-Pitre, 97159, Guadeloupe|Clinica Pediatrica AOU, Napoli, Italy|Azienda Ospedaliera-University of Padova, Padova, Italy|Azienda Ospedaliera Universitaria Integrata (AOUI Verona), Verona, Italy|CHU Fort de France, Fort de France, 97200, Martinique|Hôpital Pierre Zobda-Quitman, Fort de France, Martinique|CH Lamentin, Le Lamentin, 97232, Martinique |
| Sickle Cell Clinical Research and Intervention Program | Despite the important work of previous sickle cell disease (SCD) cohort studies, there remain many understudied areas that require investigation. An important knowledge deficit is the slow but progressive process of chronic end-organ dysfunction. The majority of organ dysfunction becomes apparent in the young adult years, but comprehensive assessment of adults and understanding of predictors of adulthood organ dysfunction are insufficient. Similarly, the role of disease-modifying therapies, such as hydroxyurea, in preventing organ dysfunction later in life is not clear. Extended follow-up of patients through the transition into adulthood is imperative to understand the long-term implications of pediatric sickle cell care. This observational study will collect data in a systematic fashion at participants' regular clinic visits (in-person or remote) to answer the objectives described below. In addition to primary study objectives, SCCRIP participants will be eligible to participate in a sub-study, which will investigate genetically determined responses to Hydroxyurea (HU) via a pharmacokinetic study (PK). This one time study will involve blood collection at timed intervals proceeding a dose of HU. Defining the basis for this inter-individual variability will allow the identification of poor HU responders prior to initiation of therapy and the seeking of alternative treatments which seek to optimize disease treatment by accounting for individual variability in genes, environment, and lifestyle. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital of Illinois at OSF-Saint Francis Medical Center, Peoria, Illinois, 61637, United States|Our Lady of the Lake Regional Medical Center, Baton Rouge, Louisiana, 70808, United States|Novant Health Hemby Children's Hospital, Charlotte, North Carolina, 28204, United States|Regional One Health, Diggs-Kraus Sickle Cell Center, Memphis, Tennessee, 38103, United States|Methodist Adult Comprehensive Sickle Cell Center, Memphis, Tennessee, 38104, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| CS-206 in Patients With Sickle Cell Disease | The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 injection in treating sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT | The First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi, China |
| Cerebrovascular Reserve Measurements in Sickle Cell Disease | The primary aim of this study is to evaluate MRI-based cerebrovascular reserve (CVR) measurements in adult patients with Sickle Cell Disease (SCD). The primary objective is to assess whether there is a correlation between CVR and silent cerebral infarcts (SCIs). | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Academic Medical Center, Amsterdam, Netherlands |
| Stem Cell Transplant in Patients With Severe Sickle Cell Disease | This is a prospective pilot study of matched-related donor allogeneic stem cell transplantation in adults with severe sickle cell disease using a matched-sibling PBSC graft with a non-myeloablative conditioning regimen (Alemtuzumab). | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | UPMC Hillman Cancer Center, Pittsburgh, Pennsylvania, 15232, United States |
| Examining Cognitive Function and Brain Abnormalities in Adults With Sickle Cell Disease - Pilot Intervention Study | Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause intense pain episodes and may lead to organ failure. Preliminary studies have shown that adults with SCD may have brain abnormalities that contribute to problems with cognitive functioning, including attention and memory difficulties. This study will use brain magnetic resonance imaging (MRI) and neuropsychological testing to examine the differences in cognitive functioning in adults with SCD who are treated for anemia with monthly blood transfusions for 6 months versus adults with SCD who receive usual care for 6 months. | Anemia, Sickle Cell | ALL | ADULT | Children's Hospital & Research Center at Oakland, Oakland, California, 94609, United States|Howard University, Washington, District of Columbia, 20001, United States|Memorial Cancer Institute, Hollywood, Florida, 33021, United States|University of Miami Miller School of Medicine, Miami, Florida, 33136, United States|Medical College of Georgia, Augusta, Georgia, 30912, United States|Johns Hopkins, Baltimore, Maryland, 94117, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|Karmanos Cancer Institute at Wayne State University, Detroit, Michigan, 48201, United States|Washington University, St. Louis, Missouri, 63110, United States|University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, 27514, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|Cincinnati Children's Hospital, Cincinnati, Ohio, 45229-3039, United States|University of Cincinnati Medical Center, Cincinnati, Ohio, 45267, United States|University of Texas Medical Branch, Galveston, Texas, 77555, United States |
| Characteristics of Patients With Sickle Cell Disease | This was a retrospective descriptive analysis of health care claims data using the IQVIA open source medical and pharmacy claims databases. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Novartis Investigative Site, East Hanover, New Jersey, 07936-1080, United States |
| Risk Factors for Allo-immunization in Sickle Cell Disease | Sickle cell patients have a high prevalence of alloimmunization. This high rate of alloimmunization can be partially explained by the existence of an antigenic difference between the predominantly Caucasian donor population and the sickle cell patients of African origin. Genetic and environmental risk factors have also been described. The main risk factors that have been shown in retrospective or cross-sectional studies are some HLA alleles, the age of the patient, the number of leukocyte-depleted erythrocyte concentrates (CED) transfused, the number of transfusion episodes, the age of the CEDs, the existence of an inflammatory event at the time of transfusion and the presence of anti-erythrocyte autoantibodies.There is also evidence of an impaired TH response but the underlying immunological mechanism is not fully understood. The aim of this study is to study the prevalence and the risk factors for anti-erythrocyte alloimmunization in pediatric and adult patients with Sickle Cell Disease (with a SS genotype) who are being followed at Queen Fabiola University Children's Hospital (HUDERF) and at the CHU Brugmann Hospital. The identification of risk factors would allow the investigators to improve, or at least adapt, their transfusion policy to certain clinical or immuno-haematological situations. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | CHU Brugmann, Brussels, 1020, Belgium|HUDERF, Brussel, 1020, Belgium |
| Losartan for Sickle Cell Kidney Disease | Sickle cell nephropathy (SCN) is a progressive complication of sickle cell disease (SCD) that begins in childhood and results in renal (kidney) failure and early mortality in nearly 12% of adults with hemoglobin SS (HbSS). The potential for prevention and reversal of kidney damage in SCD is not known. Albuminuria is a commonly used biomarker of glomerular damage; however the correlations of albuminuria with specific measurements of glomerular function and pathophysiology have not been determined. The investigators hypothesize that in patients with persistent albuminuria despite treatment of SCD with hydroxyurea, losartan will reverse kidney dysfunction in early stage nephropathy and ameliorate progressive kidney dysfunction in more advanced nephropathy. The primary aim is to study the acute and longer-term effects of losartan (study drug) on specific glomerular functions in children and adults with SCD who have persistent albuminuria. Research glomerular function tests will be done at study entry (prior to taking losartan), 1 month, and 1 to 2 years after starting losartan therapy (participants may take losartan for up to 24 months). In addition, participants are seen each month in clinic and assessed by their regular clinical team. The second aim is to assess the correlation of changes in albuminuria after 1 month of losartan with changes in direct measurements of glomerular function at 12-24 months, thus determining if the magnitude of the initial decrease in albuminuria in response to losartan predicts sustained improvements in renal function. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Grady Health Systems, Atlanta, Georgia, 30303, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States |
| RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs) | Rare Anaemia Disorders (RADs) is a group of rare diseases characterized for presenting anaemia as the main clinical manifestation. Different medical entities classified as RADs by ORPHA classification are most of them chronic life threating disorders with many unmet needs for their proper clinical management creating an impact on European health systems. RADs present diagnostic challenges and their appropriate management requires from specialised multidisciplinary teams in Centers of expertise. Although there are some examples of well-established national registries on RADs in EU, the lack of recommendations for Rare disease registries implementation and the lack of standards for interoperability has led to the fragmentation or unavailability of data on prevalence, survival, main clinical manifestations or treatments in most of the European countries. | Sickle Cell Disease|Thalassemia|Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder|Anemia Due to Membrane Defect|CDA|Sideroblastic Anemia|Constitutional Aplastic Anemia|Iron Metabolism Disorders|Hereditary Anemia | ALL | CHILD, ADULT, OLDER_ADULT | Vall d'hebron Research Institute - Vall d'Hebron Research Institute - University Hospital Vall d'Hebrón (VHIR/HUVH), Barcelona, Catalunya, 08035, Spain |
| Ketamine Infusion for Acute Sickle Cell Crisis in the Emergency Department | Pain associated with sickle cell disease is a common emergency department visit. It is also frequently associated with a high emergency department recidivism rate for pain control and admissions to the hospital. Opiates are considered the first line therapy for acute flares and to manage chronic pain. This often times leads to a stigma of being "opiate seekers" or "frequent fliers". With this study, we wish to explore whether adding ketamine to standard acute opiate therapy (morphine or dilaudid) will decrease subsequent repeat doses of opiates while improving the patient's perception of pain. In addition, we will be exploring whether ketamine as an adjuvant therapy can help reduce hospital admissions for the management of acute sickle cell crisis pain. | Pain|Sickle Cell Disorder | ALL | ADULT, OLDER_ADULT | The Brooklyn Hospital Center, Brooklyn, New York, 11201, United States |
| Nano-rheological Biomarkers for Patients With Sickle Cell Disease (SCD) Versus Control Subjects (Other Constitutional Red Blood Cell Diseases and Healthy Subjects) | Numerous pathologies (sickle cell disease, thalassemia, spherocytosis, etc.) lead to changes in the rheological properties of the blood, in particular via alterations in the deformability of red blood cells. These alterations lead to circulatory complications of which an emblematic example is the sickle cell crisis which manifests itself by microcirculatory occlusions. Several authors suggest that the deformability of erythrocytes is a key parameter for the diagnosis and monitoring of patients. Numerous studies, especially in vitro, show that the mechanical properties of the red blood cell significantly influence its dynamics in flow (blood viscosity, distribution in capillary networks). Moreover, concerning the specific problem of vaso-occlusion, the proportion of the most rigid red blood cells is a determining factor of the probability of occlusion more than the average value of this rigidity which can hide great disparities. There is no clinically usable test to assess the alteration of the fine rheology of the red blood cell in a patient. Functional tests such as ektacytometry require heavy equipment and teams of specialized biologists; this technique is therefore only available in 3 biological reference centers in France. "Mechanical phenotyping" seems to be a potentially simpler and more accessible technique, and has already shown promising prospects in other nosological settings than red blood cell pathologies. Today, there is no specific marker of sickle cell vaso-occlusive crisis, nor marker of severity, that would be useful for pathophysiological understanding but also for clinical management. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | |
| SCD Stem Cell Mobilization and Apheresis Using Motixafortide | This study is being done to see if the study drug, motixafortide, is safe in participants with sickle cell disease (SCD). Investigators also want to see if the drug will help the body increase the number of stem cells that can be collected for possible future transplant use. PRIMARY OBJECTIVE * To characterize the safety and tolerability of motixafortide in participants with SCD as determined by the incidence of adverse events (AEs). SECONDARY OBJECTIVES * To characterize the efficacy of a single dose (Part A) or two doses (Part B) of motixafortide for hematopoietic stem cell (HSC) mobilization and apheresis collection in participants with SCD as determined by the yield of CD34+ cells (CD34+ cells/kg). * To measure the mobilization effects of single-day (Part A) or daily dosing (Part B) dosing with motixafortide in the peripheral blood in participants with SCD as determined by peak peripheral blood CD34+ counts * To recommend a phase 2 dosing strategy based on safety, efficacy, and mobilization effects | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Pilates Physical Activity in Sickle Cell Disease | Sickle cell anemia (SCA) is a life-threatening hereditary hemoglobinopathy characterized by hemoglobin (Hb) polymerization that affects many people worldwide.Reduced physical capacity is common in people with SCA. Pilates is a form of physical activity that recently used in clinicial practice | Sickle Cell Disease | ALL | ADULT | Faculty of Physical Therapy Cairo University, Giza, Dokki, Egypt |
| Epidemiology and Pathophysiological Mechanisms of HTAP in SS and SC Children in Martinique and Guadeloupe. | pulmonary arterial hypertension (PAH) has been reported with a prevalence of approximately 30% in adult sickle cell disease (SCD) patients, with an increased mortality in SCD patients with PAH, compared with those without PAH. The identification of several hemolysis biomarkers such as lactate dehydrogenase, bilirubin, reticulocytes or hemoglobin level, has clearly documented a link between hemolysis and PAH. However, other physiopathological mechanisms may be involved to explain PAH in these patients, such as pulmonary thromboembolism, pulmonary fibrosis or left heart diastolic and / or systolic dysfunction. The investigators suggest studying HTAP in patient's presenting the most frequent both drepanocytic syndromes, SS and SC and homogeneous in their medical coverage and the association between HTAP risk and specific SCD complications. | Sickle Cell Disease | ALL | CHILD | Hospital University Center of Martinique, Fort-de-France, 97261, Martinique |
| Pumilio1 (PUM1) Expression, Sickle Cell Anemia, β-thalassemia Intermedia | 1. To study the expression pattern of PUM1 gene in patients with sickle cell anemia and β-thalassemia intermedia. 2. To detect PUM1 protein levels in sickle cell anemia and β-thalassemia intermedia patients. 3. To correlate PUM1 gene expression pattern and protein levels with HbF levels in sickle cell anemia and β-thalassemia intermedia patients. | Sickle Cell Disease, Beta Thalassemia Intermedia | ALL | CHILD, ADULT, OLDER_ADULT | |
| Sickle Cell Uric Acid (SCUA) - Cohort Repository | The purpose of this research is to study the causes of Sickle Cell kidney disease, as well as to collect and store samples and information about people with Sickle Cell Disease. | Chronic Kidney Diseases|Sickle Cell Disease | ALL | CHILD, ADULT | Virginia Commonwealth University, Richmond, Virginia, 23298, United States |
| Quantitative MRI for Patients With Sickle Cell Disease Undergoing Hematopoietic Cell Transplant | The primary purpose of this research is to determine if it is feasible to perform serial magnetic resonance imaging (MRI) to evaluate the amount of bone marrow cells (also called cellularity) and iron stores before and after bone marrow transplantation for severe sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | University of Michigan, Ann Arbor, Michigan, 48109, United States |
| BEATS 2: Music Therapy in Sickle Cell | The purpose of this study is to investigate the effects of the BEATS music therapy program on the self-efficacy, trust, knowledge, and adherence of young adult patients with SCD. Primary Hypotheses: Compared to baseline, young adult patients with SCD who receive the music therapy interventions will report: Higher sickle cell self-efficacy as measured by the Sickle Cell Self Efficacy Scale (SCSES), Higher trust in health care providers as measured by the Wake Forest Trust in the Medical Profession Scale, and Higher SCD knowledge as measured by the Seidman Sickle Cell Knowledge Quiz. Secondary Hypotheses Compared to the one year prior to the study period, young adults with SCD who receive the music therapy interventions will have a higher rate of adherence to clinic appointments during the one-year study period. Additional Questions Do music therapy interventions influence the rate of hospital utilization as measured by ED visits, Acute Care Clinic (ACC) visits, and admissions during the study period compared to the previous year? Do music therapy interventions influence adherence to hydroxyurea therapy for patients receiving hydroxyurea as measured by change in mean corpuscular volume (MCV) during the study period? Do music therapy interventions influence adherence to iron chelation therapy for patients receiving iron chelation therapy as measured by ferritin count during the study period? Does the schedule of music therapy interventions in this study improve outcomes more significantly than the schedule of music therapy interventions from \[IRB# 03-15-30\]? | Sickle Cell Disease | ALL | ADULT | University Hospitals Seidman Cancer Center, Cleveland, Ohio, 44106, United States |
| Propanolol and Red Cell Adhesion Non-asthmatic Children Sickle Cell Disease | Propanolol is a beta blocker which has been found to inhibit the ability of epinephrine to upregulate sickle red cell adhesion to laminin and endothelial cells in vitro. The purpose of this pilot study is to administer one dose of propanolol to children with sickle cell disease and to measure pre and post dose red cell adhesion. The hypothesis is that a single dose of propanolol will decrease red cell adhesion to laminin and endothelial cells as compared to baseline. | Sickle Cell Disease | ALL | CHILD | University of Miami, Miami, Florida, 33136, United States |
| Dense Red Blood Cells in Sickle Cell Children | Quantitative and prognostic evaluation of dense red blood cells in sickle cell children: preliminary single center study from the Creteil pediatric cohort. | Sickle Cell Disease | ALL | CHILD, ADULT | CHI Creteil, Créteil, 94000, France |
| Hemoglobin Desaturation in Sickle Cell Disease | As part of routine care for SCD, some people are found to have low oxygen levels (≤ 88%) while sleeping, at rest, or with exercise. Testing is done with a small portable device positioned on the finger that measures oxygen levels during sleep, at rest, or following exercise. The investigators start oxygen treatment for people with low levels of oxygen. As a part of this study, the investigators will find out if any changes in cell "stickiness" occur with low oxygen levels (at rest, at night, or with exertion) and if cells become "less sticky" with oxygen treatment. Study subjects will be seen before testing and 2 months after testing. In some cases (people with low oxygen levels during testing), study subjects will have been prescribed oxygen, and the investigators will test the effects of that treatment on the stickiness of red cells. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University Hospitals Cleveland Medical Center, Cleveland, Ohio, 44106, United States |
| A Phase III Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemia | The purpose of this research is to evaluate the effects of L-glutamine as a therapy for Sickle Cell Anemia or Sickle ß0 Thalassemia as evaluated by the number of occurrences of sickle cell crises. | Sickle Cell Anemia|Sickle ß0-Thalassemia | ALL | CHILD, ADULT, OLDER_ADULT | University of South Alabama Medical Center, Mobile, Alabama, 36617, United States|Phoenix Children's Hospital Center for Cancer and Blood Disorders, Phoenix, Arizona, 85016, United States|Kaiser Permanente, Inglewood, California, 90301, United States|Children's Hospital & Research Center at Oakland, Oakland, California, 94609, United States|Children's Hospital of Orange County, Orange, California, 92868, United States|Harbor-UCLA Medical Center, Torrance, California, 90509, United States|University of Denver School of Medicine Sickle Cell Treatment & Research Center, Aurora, Colorado, 80045, United States|Howard University Hospital & Howard University, Washington, District of Columbia, 20060, United States|University of Florida, Gainesville, Florida, 32610-0296, United States|All Children's Hospital, Saint Petersburg, Florida, 33701, United States|Children's Healthcare of Atlanta at Egleston/Emory University, Atlanta, Georgia, 30322, United States|University of Illinois at Chicago, Chicago, Illinois, 60612, United States|University of Louisville School of Medicine, Louisville, Kentucky, 40202, United States|Sickle Cell Center of S. Louisiana, Tulane University School of Medicine, New Orleans, Louisiana, 70112, United States|Johns Hopkins University, Baltimore, Maryland, 21205, United States|Boston University Medical Center, Boston, Massachusetts, 02118, United States|Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States|Children's Mercy Hospitals and Clinics, Kansas City, Missouri, 64108, United States|Children's Specialty Center of Nevada, Las Vegas, Nevada, 89109, United States|Comprehensive Cancer Center of Nevada, Las Vegas, Nevada, 89109, United States|Cooper University Hospital, Camden, New Jersey, 08103, United States|Bronx Lebanon Hospital, Bronx, New York, 11203, United States|The Brooklyn Hospital Center, Brooklyn, New York, 11201, United States|SUNY - Downstate Medical Center, Brooklyn, New York, 11203, United States|Brookdale University Hospital and Medical Center, Brooklyn, New York, 11212, United States|New York Methodist Hospital - SC/Thalassemia Program, Brooklyn, New York, 11215, United States|Interfaith Medical Center, Brooklyn, New York, 11238, United States|Presbyterian Blume Pediatric Hematology-Oncology Clinic, Charlotte, North Carolina, 28204, United States|University of Tennessee Cancer Institute, Memphis, Tennessee, 38104, United States|Virginia Commonwealth University, Richmond, Virginia, 23298-0306, United States |
| Prevention Of Morbidity In Sickle Cell Disease Pilot Phase | The hypothesis is that in sickle cell anaemia, nocturnal oxyhaemoglobin desaturation, is associated with low processing speed index, and this morbidity can be reduced with overnight auto Continuous Positive Airways Pressure and/or oxygen supplementation. | Sickle Cell Anaemia | ALL | CHILD | Neuroscience Unit, Institute of Child Health, London, WC1N 1EH, United Kingdom|Kings College hospital, London, WC2R 2LS, United Kingdom |
| Living With Sickle Cell Disease in the COVID-19 Pandemic | Background: Sickle cell disease (SCD) is a chronic illness. It affects about 100,000 people in the United States. People with SCD have red blood cells that are sickle-shaped and impaired in their function. This results in a lifetime of complications that affect every organ system. People with SCD also are at greater risk for respiratory infections and lung problems. Researchers want to study how this population s stress, anxiety, fear, pain, sleep, and health care use are being affected by the COVID-19 pandemic. Objective: To study the extent and impact of life changes induced by the COVID-19 pandemic on people living with SCD in the U.S. Eligibility: People age 18 and older with SCD who live in the U.S. Design: Participants will complete a survey online. The questions will focus on the following: Medical history Mental and physical health Demographics Stress Resilience Health care use COVID-19 Beliefs about medical mistrust and participation in research. At the end of the survey, participants will be asked if they would like to take the survey again in the future. If they reply "yes," then they will be contacted by the study team in 6-9 months to take the survey again. They may complete the survey again in 6-8 months, 12-15 months, and 18-21 months. The survey should take less than 40 minutes to complete. Participants' data will be coded to protect their privacy. The coded data may be shared with other researchers. | Isolation|Anxiety|Health Care Utilization|Sickle Cell Disease|Pain | ALL | ADULT, OLDER_ADULT | National Human Genome Research Institute (NHGRI), Bethesda, Maryland, 20892, United States |
| Collection and Storage of Umbilical Cord Stem Cells for Treatment of Sickle Cell Disease | This study will determine the best ways to collect, process and store umbilical cord blood from babies with sickle cell disease, sickle cell trait and unaffected babies. Sickle cell disease is an abnormality of the hemoglobin in red blood cells that causes the cells to change shape and clump together, preventing their normal flow in the bloodstream. This impairs blood flow to various organs, and the resulting oxygen deprivation causes organ damage. Cord blood is rich in stem cells (cells produced in the bone marrow that mature to different types of blood cells), which may prove useful in new sickle cell therapies. However, cord blood from babies with sickle cell trait, sickle cell disease and normal babies may act differently under laboratory conditions, so it is important to learn how best to work with blood from all three groups of babies for future use in possible treatments. Pregnant women between 18 and 45 years of age who are at risk of having an infant with sickle cell disease and normal volunteers who are pregnant and not at risk for this disease may be eligible for this study. Potential participants will be counseled about donating her infant s blood in order to make an informed choice. All women who participate in the study will provide a medical history and have blood collected from the umbilical cord and placenta (afterbirth) after the baby s delivery. The blood will be tested for various infectious diseases, processed, frozen and stored for research purposes. In addition, blood from women with babies at risk for sickle cell disease will be tested for the presence of the sickle cell gene, tissue typed, and used for research as follows: * Sickle cell disease - If cord blood tests show the baby has sickle cell disease, the blood will be frozen for an indefinite period of time for possible use in future treatment of the child. This treatment could include stem cell transplantation or gene therapy, treatments are not currently considered routine for sickle cell disease. * Sickle cell trait or normal hemoglobin - If cord blood tests show the baby has sickle cell trait or is unaffected, the blood will be processed and stored for up to 3 years, during which time it may possibly be used to treat a currently living or future sibling with sickle cell disease. After 3 years, the participant may agree to either have the blood discarded, given to research or moved to another facility for continued storage at the participant s expense, if there is a storage fee. Alternatively, if there is no anticipated future need for the collected blood, or if it does not meet standards needed for future treatment, it will be used in NIH-approved research studies. Participants and their family doctor or the baby s pediatrician will be contacted twice a year for information about changes in the baby s health. Participants may also be asked permission to perform new tests developed by researchers. | Sickle Cell Disease|Sickle Cell Trait | ALL | ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Autonomic Nervous System and Sickle Cell Disease | Sickle cell disease (SCD) children and adults with asthma have an increased rate of vaso-occlusive crisis, acute chest syndrome episodes, and premature mortality when compared to those without asthma. We hypothesised that either asthma diagnosis and/or bronchodilator treatment may aggravate SCD via their modulating effect on autonomic nervous system. | Heart Rate Variability (ANS Function) | ALL | CHILD | Robert Debre Hospital, Paris, 75019, France |
| Functional Neuroimaging of Pain Using EEG and fMRI | The purpose of this research is to use non-invasive imaging technologies to study how the human brain processes pain. The investigators will use contact heat to induce pain and record data scalp EEG and functional magnetic resonance imaging (fMRI). What the investigators learn from this study will help us gain insights in pain management with broad socioeconomic impacts | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Biomedical Engineering Department, Minneapolis, Minnesota, 55413, United States |
| Decitabine for High-Risk Sickle Cell Disease | Background: * In sickle cell disease (SCD), the proteins in the red blood cells that carry oxygen do not behave normally. In parts of the body where there are low levels of oxygen or where oxygen is used more, the sickle hemoglobin proteins may change shape and stick together. This causes the red cells to clump, which reduces blood flow. This leads to even lower oxygen levels and causes damage and/or pain. * One way to stop the red blood cells from sticking together is to increase the levels of fetal (baby or good ) hemoglobin. The good hemoglobin then takes the place of the sickle hemoglobin. * Hydroxyurea is the only approved drug for SCD. But hydroxyurea works in only about two-thirds of people with SCD. Even in those cases it sometimes stops working over time. * Researchers are interested in testing decitabine. The drug may help to increase fetal hemoglobin levels. But it has not yet been approved to treat SCD. Objectives: - To test the safety and effectiveness of decitabine in increasing fetal hemoglobin levels and improving the symptoms of sickle cell disease. Eligibility: - People at least 18 years of age who have sickle cell disease that has not improved after at least 6 months of hydroxyurea therapy. Those who cannot take hydroxyurea because of side effects may also participate. Design: * Participants will be screened with a physical exam and medical history. They will also have blood and urine tests, a lung function test, and other tests as required. * Participants will receive decitabine injections up to twice a week for 1 year. Depending on the response to treatments, the dose will remain the same or be reduced to once a week. * Participants will be monitored with frequent blood tests and other studies as directed by the study doctors. * After the study is completed, participants will go back to their usual sickle cell care. If decitabine has improved a participant's SCD, treatment may be continued under regular health coverage insurance if this can be arranged. | Neutropenia|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| Neuropathic Pain in Jamaicans With Sickle Cell Disease | Pain is the most common component of the morbidity seen in sickle cell disease (SCD), and may be acute or chronic. It is most commonly acute and a result of the hallmark vaso-occlusive episodes of the disease. Many patients however suffer from chronic pain - defined as pain lasting over three months- with neuropathic pain being a component of chronic pain. Neuropathic pain significantly contributes to the chronicity and morbidity of pain in SCD patients, and is an inadequately managed complication. There is a paucity of literature covering this area, and it has never been examined in the Jamaican population. The main objective of this study is to determine the epidemiology of pain among Jamaicans with SCD, and determine the prevalence of chronic and neuropathic pain among these patients. A second objective is to validate, using gold-standard measures, screening tools to determine neuropathic pain among the study population. This cross-sectional study will investigate the prevalence of neuropathic pain and complications in a sample of persons with SCD in Jamaica aged 14 years and older, with a validation sub-study to be conducted on a random 20 percent of the sample. With improved diagnosis of neuropathic pain, clinicians may potentially improve the management of pain in SCD, as clinicians should be able to direct our treatment toward medications and non-pharmacological methods of pain relief that are more specific for neuropathic pain. All data will be de-identified and maintained in a secure database, with access limited to key personnel. There is very minimal risk to participants. | Sickle Cell Disease|Neuropathic Pain | ALL | CHILD, ADULT, OLDER_ADULT | Caribbean Institute for Health Research, Kingston, Kingston 7, Jamaica |
| TRF-1101 Assessment in Sickle Cell Disease | This study is designed to assess the safety, tolerability, and activity of TRF-1101 on microvascular blood flow, vascular endothelial injury, and vasoocclusive pain associated with sickle cell disease. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Howard University, Washington, District of Columbia, 20060, United States|Medical College of Georgia, Augusta, Georgia, 30912, United States|University of Illinios Medical Center, Chicago, Illinois, 60612, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|Wayne State University Medical Center, Detroit, Michigan, 48201, United States|University of North Carolina, Chapel Hill, North Carolina, 27599, United States |
| L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia | The purpose of this research is to evaluate the effects of L-glutamine as a therapy for sickle cell anemia and sickle ß0-thalassemia. as evaluated by the number of occurrences of sickle cell crises. | Sickle Cell Anemia|Thalassemia | ALL | CHILD, ADULT, OLDER_ADULT | Kaiser Permanente, Bellflower, California, 90706, United States|Harbor-UCLA Medical Center, Torrance, California, 90502, United States|Grady Memorial Hospital, Atlanta, Georgia, 30303, United States|University of Medicine and Dentistry, New Jersey, New Brunswick, New Jersey, 08903, United States|Jacobi Medical Center, Bronx, New York, 10461, United States |
| Evaluation of the AMICUS RBCx System in Sickle Cell Patients | The purpose of this study is to evaluate the performance of the AMICUS Red Blood Cell Exchange (RBCx) System (Exchange and Depletion/Exchange procedures) in patients with sickle cell disease. | Sickle Cell Anemia | ALL | CHILD, ADULT, OLDER_ADULT | Phoenix Children's Hospital, Phoenix, Arizona, 85016, United States|Barbara Ann Karmanos Cancer Institute, Detroit, Michigan, 48201, United States|Washington University, Saint Louis, Missouri, 63110, United States|The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|University of Texas Southwestern Medical Center, Dallas, Texas, 75390, United States|BloodCenter of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| N-Acetylcysteine in Patients With Sickle Cell Disease | The primary aim of this study is to evaluate the effect of the drug N-Acetylcysteine on the frequency of pain in daily life in patients with Sickle Cell Disease (SCD). Pain is an invalidating hallmark of this disease and has a considerable impact on the Quality of Life of patients and the medical health care system. Oxidative stress is hypothesized to play a central role in its pathophysiology. In pilot studies the administration of N-Acetylcysteine (NAC) resulted in a reduction of oxidative stress. Moreover, administration of NAC seemed to decrease hospitalization for painful crises in a small pilot study in patients with SCD. This study will be performed as a multicenter, randomized, controlled trial where patients will be treated with either NAC or placebo for a period of 6 months. The investigators expect that NAC can reduce the frequency of pain in patients with SCD, thereby improving their quality of life and participation in society. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | CHU Brugmann, Brussels, Belgium|CHU St. Pierre, Brussels, Belgium|Hôpital Erasme, Brussels, Belgium|Hôpital Universitaire Des Enfants Reine Fabiola (HUDERF), Brussels, Belgium|UCL St. Luc, Brussels, Belgium|CHR de la Citadelle, Liège, Belgium|Academic Medical Center, Amsterdam, 1105 AZ, Netherlands|University Medical Center Groningen, Groningen, 9713 GZ, Netherlands|Erasmus Medical Center, Rotterdam, 3015 AA, Netherlands|Haga Hospital, The Hague, 2545 CH, Netherlands|Guys' & St. Thomas Hospital, London, United Kingdom |
| Inflammatory Response to Hydroxyurea Therapy in Sickle Cell Disease | In sickle cell disease (SCD), polymerisation of haemoglobin S and the resulting shape change of the red blood cells (RBC) lead to vascular occlusion and severe painful crises. Permanent inflammatory state and abnormal RBC adhesion to the endothelium trigger these phenomenon. Hydroxyurea (HU) is the only drug that has been shown to reduce clinical severity of SCD, and this was initially attributed to the stimulation of foetal haemoglobin (HbF). However, the clinical response does not correlate consistently with the degree and time of HbF increment, suggesting that HU clinical benefits may involve other mechanisms such as the induction of natural anti-inflammatory response via the hypothalami-pituitary-adrenal axis. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Hopital Louis Mourier, Colombes, 92701, France |
| A Pilot Study of Chronic Red Blood Cell Transfusion in Sickle Cell Disease-Associated Pulmonary Hypertension | Pulmonary hypertension, a complication associated with an increased risk of death, is common in patients with sickle cell disease. Despite its frequency, there remains no standard treatment for this complication in patients with sickle cell disease. In this small study, the investigators will evaluate the effect of monthly transfusion of red blood cells to patients with sickle cell disease-associated pulmonary hypertension. The investigators speculate that by increasing the hemoglobin level and decreasing the amount of sickle red blood cells, these patients would experience improvements in their PHT. | Pulmonary Hypertension|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | |
| Abatacept for GVHD Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease | To assess the tolerability of the costimulation blocking agent abatacept (CTLA4-Ig) when added to the standard graft versus host disease (GVHD) prophylaxis regimen of a calcineurin inhibitor and methotrexate in patients receiving early alemtuzumab followed by fludarabine, thiotepa, melphalan, and alemtuzumab for conditioning. | Sickle Cell Disease|Graft Versus Host Disease | ALL | CHILD, ADULT | Children's National Medical Center, Washington, District of Columbia, 20010, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States|Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States|Floating Hospital for Children at Tufts Medical Center, Boston, Massachusetts, 02111, United States|Columbia University Irving Medical Center, New York, New York, 10032, United States|North Carolina Cancer Hospital, Chapel Hill, North Carolina, 27514, United States|Nationwide Children's Hospital, Columbus, Ohio, 43205, United States |
| Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease | The goal of this clinical research study is to find out about the safety and effects of a drug called panobinostat when given to adults with sickle cell disease. Panobinostat is a pan histone deacetylase (HDAC) inhibitor. HDAC inhibitors have been shown to significantly increase hemoglobin F induction, which is well documented to improve outcomes in sickle cell disease. HDAC inhibitors are also known to potently inhibit cell-specific inflammation, which is a primary contributor to the debilitating effects of sickle cell disease. Given the relevance of these mechanisms of action in SCD, panobinostat may prove to contribute significantly to the management of SCD patients, a population in critical need of further effective treatment options. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Augusta University, Augusta, Georgia, 30912, United States |
| Community Health Workers and mHealth for Sickle Cell Disease Care | This study will compare the effectiveness of two self-management support interventions-Community Health Workers (CHW) and mobile health (mHealth)-versus enhanced usual care to improve health-related quality of life and acute care use for transitioning youth with sickle cell disease (SCD), and identify and quantify mediators and moderators of intervention treatment effects. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Connecticut Children's Medical Center, Hartford, Connecticut, 06106, United States|Cohen's Children's Medical Center, New Hyde Park, New York, 11040, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|St. Christophers Hospital for Children, Philadelphia, Pennsylvania, 19134, United States |
| Carbon Monoxide Levels and Sickle Cell Disease Severity | Background: - Some people with sickle cell disease have different health problems than others. This may be related to how easily and frequently the red blood cells break apart in the blood. Researchers want to test breath and blood samples from people with sickle cell disease to look for very small amounts of carbon monoxide, which is produced when red blood cells break apart. They will compare these results with breath samples from healthy volunteers. Studying different levels of carbon monoxide may help predict what health problems a person with sickle cell disease may get. It may also provide more information on possible treatments. Objectives: - To study breath carbon monoxide levels and their possible relation to the severity of sickle cell disease. Eligibility: * Individuals at least 18 years of age with sickle cell disease. * Healthy volunteers who are matched for age, sex, and race with the sickle cell disease group. Design: * Participants will be screened with a medical history. * Participants with sickle cell disease will provide a blood sample and have a heart function test. They will also breathe into a bag to provide an exhaled breath sample. * Healthy volunteers will provide an exhaled breath sample. * No treatment or care will be provided as part of this study. | Sickle Cell Disease|Sickle Cell Anemia|Anemia, Sickle Cell | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| The Feasibility of the PAINReportIt Guided Relaxation Intervention-Outpatient | Our goal is to improve the self-management of pain and stress in adult outpatients with sickle cell disease (SCD) by determining the feasibility of a self-managed guided relaxation (GR) stress reduction intervention using a tablet-based mobile device. Currently, opioid analgesics are primarily used to treat SCD pain while self-managed behavioral modalities such as GR, are rarely used. Little is known about the effects or mechanisms of GR on pain and stress, in adults with SCD. Emerging evidence from the hypothalamic pituitary adrenal (HPA) axis theory offer insights for understanding the mechanisms. Adding GR as a supplement to analgesic therapies will address the paucity of self-management strategies for controlling pain in SCD. GR is a simple and cost-effective non-drug intervention that could reduce pain and stress in outpatients with SCD. GR is an intervention where outpatients with SCD are directed to listen to and view audio-visual recordings while they visualize themselves being immersed in that scene. | Sickle Cell Disease|Stress|Pain | ALL | ADULT, OLDER_ADULT | University of Florida Hematology Clinic-Medical Plaza, Gainesville, Florida, 32610, United States |
| The Spleen in Sickle Cell Anemia and Sickle Cell Thalassemia | The spleen in Sickle Cell Anemia and Sickle Cell Thalassemia is usually enlarged in the first years of life but the immune protection provided is considered insufficient. In homozygous Sickle cell patients the spleen usually developed recurrent infarcts and after the first decade of age become fibrotic. Acute splenic sequestration is also frequent in those patients and this is considered as an indication for splenectomy. In comparison in Sickle cell thalassemia patients, hypersplenism is more frequent. The purpose of this study is to compare the clinical and laboratory issues related to the spleen in two groups of Sickle cell patients. | Sickle Cell Anemia|Thalassemia | ALL | CHILD, ADULT | Pediatric Hematology Unit and Pediatric Dpt B - HaEmek Medical Center, Afula, 18101, Israel |
| Therapeutic Application of Intravascular Nitrite for Sickle Cell Disease | This study examines ways in which nitric oxide (NO), an important molecule that controls how blood flows through the body's vessels, might be restored with a compound called sodium nitrite. It is hoped that the result will reverse the effect of decreased flow of blood due to sickled cells-that is, cells that have changed into the shape of a crescent or sickle. Sickle cell disease is the most common genetic disease affecting African Americans. About 8% of that population has the sickle cell trait. The changed cells can become attached to blood vessels, decreasing blood flow to vital organs. There can be the loss of needed proteins, including hemoglobin, that deliver oxygen throughout the body. Adults at least 18 years of age who have the SS form of sickle cell disease or S-beta-thalassemia, are in either a steady state or crisis, give informed and written consent for participation, and have had a negative pregnancy test may be eligible for this study. Adults with any other disease that puts them at risk for reduced circulation are not eligible. Women who are breastfeeding are not eligible. Participants will undergo a medical history, including family medical history, and a detailed physical evaluation, to take about 1 hour. There will be a collection of blood; echocardiogram, which involves taking a picture of the heart and its four chambers; and measurement of exhaled carbon monoxide, carbon dioxide, and NO. A procedure called orthogonal polarization spectral imaging will be performed. A small object the size of a Popsicle stick will be placed under the tongue or on a fingertip. This procedure presents a picture of blood flow and how the red blood cells appear as they circulate through blood vessels. The study will be conducted in the Vascular Laboratory/Cardiovascular Floor or Intensive Care and will last about 4 hours. During the study, patients will lie in an adjustable reclining bed and chair. Small tubes will be placed in the artery and vein of the forearm at the inside of the elbow. A small pressure cuff will be applied to the wrist and a larger one to the upper arm. Both cuffs will be inflated with air. A strain gauge, resembling a rubber band, will go around the widest part of the forearm. When the pressure cuffs fill with air, blood will flow into the arm, and information from the strain gauge will be recorded. Between administrations of each medicine, there will be 30-minute rests. Normal saline will be put into the small tube in the artery. Measurements of the blood flow in the forearm will be taken, and a small blood sample will be taken to measure blood counts, proteins, and other natural body chemicals. Then a medicine called sodium nitroprusside, which causes blood vessels to expand and increase blood flow, will be placed into the forearm. It will be given at three different doses for 3 minutes each, with measurements recorded after each dose. Then a medicine called L-NMMA will be placed into the forearm. L-NMMA generally decreases local blood flow by preventing nitric oxide from being produced in the cells lining the blood vessels. It will be given at two different doses for 5 minutes each, with blood flow measured after each dose. Next, nitrite will be placed in the forearm at three different doses for 5 minutes each. Before and after nitrite is given, the researchers will measure the amount of the NO, carbon monoxide, and carbon dioxide that the patients breathe out. Then the procedure for administering normal saline, sodium nitroprusside, and L-NMMA will be repeated, as will a blood test. This study will not have a direct benefit for participants. However, it is hoped that the information gained from the study will help to develop treatment options for patients with sickle cell disease. | Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| Home-Based Intervention for Chronic Pain in Adults With Sickle Cell Disease | This project will evaluate AppliedVR's EaseVRx - a multi-modal, skills-based, 8-week, virtual reality, home intervention - in an exploratory randomized controlled trial for self-management of chronic pain among Black, young adults (ages 18-50) with sickle cell disease. | Sickle Cell Disease | ALL | ADULT | Grady Memorial Hospital - Outpatient Sickle Cell Clinic, Atlanta, Georgia, 30303, United States |
| Kidney Transplantation in Patients With Sickle Cell Disease | The purpose of this research is to better characterize the components and mechanisms of the immune systems of persons with sickle cell disease who have had a kidney transplant and are immunosuppressed. If we can improve our scientific understanding of the fundamental mechanisms involved in patient outcomes, we can potentially maximize the benefits that we seek from transplantation in sickle cell patients with end stage renal disease. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Mason Outpatient Tranplant Clinic Emory University, Atlanta, Georgia, 30302, United States |
| Androgen Regulation of Priapism in Sickle Cell Disease | It is believed that when androgen (testosterone) levels are below normal there is a disturbance of normal bodily functioning that is associated with priapism in some men. Conversely, it is believed that testosterone replacement will improve the condition of priapism when the testosterone levels are brought to normal. In turn, this will also improve psychological well being in men with sickle cell disease (SCD). | Priapism|Sickle Cell Disease|Hypogonadism | MALE | ADULT | Johns Hopkins University School of Medicine, Johns Hopkins Hospital, Baltimore, Maryland, 21287, United States |
| The Feasibility of the PAINReportIt Guided Relaxation Intervention-INPATIENT | The goal of this research study is to improve the self-management of pain, stress, and cognitive/affective symptoms that may result in adult inpatients with sickle cell disease (SCD) by determining the feasibility of a self-management guided relaxation (GR) stress reduction intervention using a tablet-based mobile device. Currently, opioid analgesics are primarily used to treat SCD pain while self-managed behavioral modalities such as GR, are rarely used, particularly, in inpatient settings. Little is known about the effects or mechanisms of GR on pain, stress, and cognitive/affective symptoms in adults with SCD hospitalized with pain. Emerging evidence from the hypothalamic pituitary adrenal (HPA) axis theory offer insights for understanding the mechanisms. Adding GR as a supplement to analgesic therapies will address the dearth of self-management strategies for controlling pain in SCD. GR is a simple and cost-effective non-drug intervention that could reduce pain and stress in inpatients with SCD. GR is an intervention where inpatients with SCD are directed to listen to and view audio-visual recordings while they visualize themselves being immersed in that scenario. | Sickle Cell Disease|Stress|Pain | ALL | ADULT, OLDER_ADULT | University of Florida, Gainesville, Florida, 32610, United States |
| Safety, Tolerability and Pharmacokinetics of FTX-6058 | This is a study to evaluate the safety, tolerability and pharmacokinetics of FTX-6058 in healthy adult subjects and adult subjects with sickle cell disease (SCD). | Healthy Adult Subjects|Sickle Cell Disease | ALL | ADULT | Atlanta Center for Medical Research - Sickle Cell Subjects Only, Atlanta, Georgia, 30331, United States|Altasciences Clinical Kansas, Inc. - Healthy Adults Subjects Only, Overland Park, Kansas, 66212, United States |
| Microvessels and Heart Problems in Sickle Cell Disease | Background: - Small blood vessels (microvessels) in many different organs are affected by diseases such as diabetes and atherosclerosis. These microvessels may also be abnormal in people who have sickle cell disease. Stiffness of the red blood cells leads to problems in the microvessels of the heart and kidneys. However, these problems may not be detected until these organs are severely affected. Researchers want to study problems with microvessels in people with and without sickle cell disease. Objectives: - To study how microvessels in the heart and other organs are affected by sickle cell disease. Eligibility: * Individuals at least 18 years of age who have sickle cell disease. * Healthy volunteers at least 18 years of age. Design: * Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. * All participants will have about 3 to 4 hours of testing for the study. Participants with sickle cell disease who are having a pain crisis at the time they enter the study may be asked to have the testing again when the crisis is over. The repeat testing will occur at least 4 weeks after the pain crisis ends. * All participants will have the following tests: * Blood draws to check kidney and liver function, and other blood tests * Measure of blood flow in the brachial (upper arm) artery * Heart ultrasound * Ultrasound scans of arm muscles to study blood flow * Ultrasound scans after taking vasodilators to increase blood flow * Healthy volunteers will also have a magnetic resonance imaging scan. It will show blood flow in the heart. This scan will involve another dose of a vasodilator. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| Evaluation of Low-cost Techniques for Detecting Sickle Cell Disease and β-thalassemia in Nepal and Canada | Sickle cell disease (SCD) is an inherited blood disorder associated with acute illness and organ damage. In high resource settings, early screening and treatment greatly improve quality of life. In low resource settings, however, mortality rate for children is high (50-90%). Low-cost and accurate screening techniques are critical to reducing the burden of the disease, especially in remote/rural settings. The most common and severe form of SCD is sickle cell anemia (SCA), caused by the inheritance of genes causing abnormal forms of hemoglobin (called sickle hemoglobin or hemoglobin S) from both parents. The asymptomatic or carrier form of the disease, known as sickle cell trait (SCT), is caused by the inheritance of only one variant gene from one of the parents. In areas such as Nepal, β-thalassemia (another inherited blood disorder) and SCD are both prevalent, and some combinations of these diseases lead to severe symptoms. The purpose of this study is to determine the accuracy of low-cost point-of-care techniques for screening and detecting sickle cell disease, sickle cell trait, and β-thalassaemia, which will subsequently inform on feasible solutions for detecting the disease in rural, remote, or low-resource settings. One of the goals of the study is to evaluate the feasibility of techniques, such as the sickling test with low-cost microscopy and machine learning, HbS solubility test, commercial lateral-flow assays (HemoTypeSC and Sickle SCAN), and the Gazelle Hb variant test, to supplement or replace gold standard tests (HPLC or electrophoresis), which are expensive, require highly trained personnel, and are not easily accessible in remote/rural settings. The investigators hypothesize that: 1. an automated sickling test (standard sickling test enhanced using low-cost microscopy and machine learning) has a higher overall accuracy than conventional screening techniques (solubility and sickling tests) to detect hemoglobin S in blood samples 2. the automated sickling test can additionally classify SCD, SCT and healthy individuals with a sensitivity greater than 90%, based on morphology changes of red blood cells, unlike conventional sickling or solubility tests that do not distinguish between SCD and SCT cases 3. Gazelle diagnostic device can detect β-thalassaemia and SCD/SCT with an overall accuracy greater than 90%, compared with HPLC as the reference test | Sickle Cell Disease|Sickle Cell Trait|Beta-Thalassemia|Sickle Cell-Beta Thalassemia|Sickle Cell-SS Disease | ALL | CHILD, ADULT, OLDER_ADULT | BC Children's Hospital, Vancouver, British Columbia, V6H 3N1, Canada|St. Paul's Hospital, Vancouver, British Columbia, V6Z 1Y6, Canada|Mount Sagarmatha Polyclinic and Diagnostic Center, Nepalgunj, Banke, Nepal |
| Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease (BMT CTN 0601) | Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause organ damage, stroke, and intense pain episodes. A blood stem cell transplant is a treatment option for someone with a severe form of the disease. Prior to undergoing a transplant, people typically receive a conditioning regimen of high doses of chemotherapy and other medications to prepare the body to accept the transplant. A conditioning regimen that uses lower doses of chemotherapy and medications may be safer for transplant recipients. This study will evaluate the safety and effectiveness of blood stem cell transplants, using bone marrow from unrelated donors, in children with severe SCD who receive a reduced intensity conditioning regimen prior to the transplant. | Sickle Cell Disease | ALL | CHILD, ADULT | University of Alabama at Birmingham, Birmingham, Alabama, 35294, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States|University of Miami, Miami, Florida, 33136, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States|Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, 60614, United States|Children's Hospital of New Orleans/LSUMC CCOP, New Orleans, Louisiana, 70118, United States|University of Michigan Medical Center, Ann Arbor, Michigan, 48105, United States|University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States|Children's Mercy Hospital and Clinics, Kansas City, Missouri, 64108, United States|Washington University, St. Louis Children's Hospital, Saint Louis, Missouri, 63110, United States|Cohen Children's Hospital, New Hyde Park, New York, 11040, United States|Columbia University Medical Center, New York, New York, 10032, United States|University of North Carolina Hospital at Chapel Hill, Chapel Hill, North Carolina, 27599, United States|University Hospitals of Cleveland, Cleveland, Ohio, 44106, United States|Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, 15213, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|Baylor College of Medicine/The Methodist Hospital, Houston, Texas, 77030, United States|Virginia Commonwealth University, Richmond, Virginia, 23298, United States|Medical College of Wisconsin, Milwaukee, Wisconsin, 53211, United States |
| Serum Brain Natriuretic Peptide Levels and Pulmonary Hypertension in Pediatric Sickle Cell Patients | The purpose of this study is 1. To determine if Brain natriuretic peptide levels correlates with elevated tricuspid regurgitation flow velocity levels in pediatric patients with sickle cell disease 2. To determine the role of age, gender, steady state hemoglobin and disease type on Brain natriuretic peptide levels and pulmonary hypertension | Pulmonary Hypertension | ALL | CHILD, ADULT | Akron Children's Hospital Sickle Cell Clinic, Akron, Ohio, 44308, United States |
| Predictors and Outcomes in Patients With Sickle Cell Disease | Children with sickle cell disease (SCD) are living longer with the advent of medical advances such as prophylactic penicillin, chronic transfusion, and hydroxyurea. Despite greater longevity in SCD, the period following the transition from pediatric to adult care is critical; youth aged 18-30 years are at high risk for mortality and have high rates of healthcare utilization, leading to high healthcare costs. As such, health care transition (HCT) programs have been created to prepare patients for adult-centered care and subsequently, improve health outcomes. However, very few programs have been evaluated for effectiveness in achieving optimal health outcomes in SCD. This paucity of program evaluation is attributed to a lack of identifiable predictors and outcomes. Researchers at St. Jude Children's Research Hospital want to identify factors and patterns of successful HCT. This information will be used to develop approaches to best evaluate HCT interventions and identify areas of improvement of HCT programming. PRIMARY OBJECTIVE: Describe hospital utilization, treatment adherence, and health-related quality of life in a cohort of patients with sickle cell disease (SCD) who will transfer to adult care during the study period. SECONDARY OBJECTIVE: Examine the associations between various factors and health care transition (HCT) outcomes. | Sickle Cell Disease | ALL | CHILD, ADULT | Methodist Adult Comprehensive Sickle Cell Center, Memphis, Tennessee, 38104, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Exploratory Study on Global Reflexology in Sickle Cell Disease | Sickle cell disease (or sickle cell anemia) is the most common genetic disease in France with 586 children screened in 2019. This chronic disease is characterized by the presence of abnormal Hemoglobin (Hb) S and a deformation of the red blood cells which take the elongated shape of a sickle and become more rigid and more fragile. Sickle cell disease manifests itself among other things by very painful vaso-occlusive crises (VOC) and for some chronic pain. Their management is an emergency and often requires hospitalization. Despite analgesic treatment, some patients have persistent pain. In 2013, a childcare assistant trained in Canadian global reflexology EMC offered reflexology sessions to 12 sickle cell patients. She observed a relief in all patients with a decrease in the pain score in 8 of them. These sessions seem to show us a double interest: the reduction of the child\'s pain and the emergence of a technique that can be used by paramedics in the context of their own role. The investgators hypothesize that global reflexology is an effective and acceptable complementary technique for pain management in addition to the usual analgesic management in sickle cell children under 18 years of age in CVO. In order to verify our hypothesis, the investigators propose to explore the practice of Canadian global reflexology as an innovative therapeutic option complementary to drug treatments in the hospital management of sickle cell children. | Sickle Cell Disease (SCD) | ALL | CHILD, ADULT | Hôpital Femme Mère Enfant, Bron, 69500, France |
| A Study of HQK-1001 in Patients With Sickle Cell Disease | The purpose of this study is to evaluate the safety and tolerability of three dose levels of HQK-1001 administered once daily for 26 weeks in subjects with sickle cell disease. | Sickle Cell Disease|Sickle Cell Anemia|Sickle Cell Disorders|Hemoglobin S Disease|Sickling Disorder Due to Hemoglobin S | ALL | CHILD, ADULT | Children's Hospital and Research Center - Oakland, Oakland, California, 94609, United States|University of Miami Miller School of Medicine - Dept of Pediatrics, Miami, Florida, 33101, United States|Georgia Health Sciences University - Adult SIckle Cell Center, Augusta, Georgia, 30912, United States|University of Illinois at Chicago - Dept of Pediatrics, Chicago, Illinois, 60612, United States|LSU Health Sciences Center - Feist Weiller Cancer Center, Shreveport, Louisiana, 71103, United States|Tufts Medical Center, Boston, Massachusetts, 02111, United States|University of North Carolina at Chapel Hill - Comprehensive Sickle Cell Program, Chapel Hill, North Carolina, 27599, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States|Univerisity of Texas Southwestern Medical Center at Dallas - Pediatric Hematology Oncology, Dallas, Texas, 75390-9063, United States|The Hospital for Sick Children, Toronto, Ontario, MSG 1X8, Canada|University Health Network Toronto General Hospital, Toronto, Ontario, MSG 2C4, Canada|Abu El Reesh Pediatric University Hospital, Cairo, Egypt|University of the West Indies - Sickle Cell Unit, Mona, Kingston, Jamaica|American University of Beirut Medical Center, Beirut, Lebanon|Rafik Hariri University Hospital, Beirut, Lebanon|Chronic Care Center, Hazmieh, Lebanon |
| The Link Between Anemia and Deficits in Memory and Attention in Individuals With Sickle Cell Disease | Sickle cell disease is an inherited blood disorder that affects red blood cells (RBCs). People with sickle cell disease frequently experience anemia, or a low number of RBCs. RBCs are responsible for carrying oxygen to the brain and other body tissues that need oxygen to function properly. The purpose of this study is to determine what changes, which were possibly caused by anemia, exist in the brains of individuals with sickle cell disease. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of California San Francisco, San Francisco, California, 94121, United States |
| TMLI and Alemtuzumab for Treatment of Sickle Cell Disease | This phase I trial tests the safety and effectiveness of total marrow and lymphoid irradiation (TMLI) and alemtuzumab as a conditioning regimen in patients with sickle cell disease. Conditioning regimens are treatments used to prepare a patient for stem cell transplantation. A stem cell transplant is a procedure in which a person receives blood stem cells, which make any type of blood cell. A conditioning regimen may include chemotherapy, monoclonal antibody therapy, and radiation to the entire body. It helps make room in the patient's bone marrow for new blood stem cells to grow, and helps prevent the patient's body from rejecting the transplanted cells. Alemtuzumab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread. Graft-versus-host disease (GVHD) is a complication that may occur after hematopoietic cell transplantation (HCT) in which donated cells view the recipient's cells as foreign and attack them. Giving TMLI and alemtuzumab may help reduce organ damage that can be caused by radiation and decrease the risk of GVHD. | Sickle Cell Disease | ALL | CHILD, ADULT | City of Hope Medical Center, Duarte, California, 91010, United States |
| Mobile-Directly Observed Therapy on Adherence to Hydroxyurea | To examine the effect of mobile-directly observed therapy (mDOT) on adherence to HU (mDOT-HuA) adults with SCA at Muhimbili National Hospital (MNH) in Tanzania. | Sickle Cell Anaemia | ALL | ADULT, OLDER_ADULT | Muhimbili University of Health and Allied Sciences, Dar es Salaam, 11101, Tanzania |
| State Of The Art Functional Imaging In Sickle Cell Disease | Sickle cell anemia (SCA) is a serious blood disease with blood vessel changes leading to brain injury and stroke. Studies show about 11% of patients with SCA will develop obvious stroke before age 20 years, with children less than 10 years of age especially vulnerable. The main objective of the SCDMR4\[State Of The Art Functional Imaging In Sickle Cell Disease\] trial is to compare the gray matter cerebral blood flow, measured by MRI,\[magnetic resonance imaging\] ASL \[Arterial Spin Labeling\] perfusion before treatment begins and after the appropriate hydroxyurea dosage is reached (\~ one year). Other important objectives of the SCDMR4 trial include describing the effect of hydroxyurea therapy and transfusion therapy on the functional MRI response, diffusion tensor imaging of white matter, brain function, and transcranial Doppler blood velocities. | Sickle Cell Anemia | ALL | CHILD, ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Evaluating Thromboelastography (TEG) and ETP in Sickle Adults | The primary aim of this study is to investigate the reported enhanced coagulation status (prothrombotic status) in patients with sickle cell disease using 2 laboratory tests; thromboelastography (TEG) and Endogenous Thrombin Potential (ETP), and comparing the results to healthy race matched controls to ascertain if there is a significant difference. Race matching of the control participants is being carried out due to the well reported racial differences in coagulation parameters that exist in healthy individuals. The investigators are aiming to study the clotting state in sickle patients on regular transfusion therapy and those on hydroxycarbamide, both treatments offered to sickle patients to ameliorate the condition. The study will assess the reported prothrombotic state using TEG and ETP. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Guys and St Thomas NHS Foundation Trust, London, SE1, United Kingdom |
| SCRIPT: Sickle Cell Risk in Pregnancy Tool | Sickle Cell Disease (SCD), common in persons of Black ancestry, affects the shape of hemoglobin, the oxygen-carrying part of red blood cells (RBC). It is characterized by many complications, the most dreaded of which are related to pregnancy - affecting both the mother and unborn child. Compared to those without SCD, people with SCD have more adverse pregnancy outcomes (APO): 6x maternal mortality, 2x preeclampsia \& preterm birth, 4x risk of having a baby not growing well in the womb \& stillbirth. There is also greater need for access to care (7x higher hospitalization often multiple times lasting days to months). Yet up to 30% of SCD pregnancies are uncomplicated. Treatments in pregnancy are limited and carry risks. A method to distinguish pregnancies at high-risk of APO that may benefit from these potentially risky treatments, from those likely to be uncomplicated, is urgently needed. To meet this need, the investigators developed a calculator to estimate pregnancy complication risk, using single-centre data. Its accuracy and precision will now be evaluated with international information from several centers by testing the calculator, and adjusting it as needed, using already available pregnancy-data from study centres in several countries. Those age \>16 years, who have a confirmed SCD genotype, pregnancy with one baby, and pregnancy care and birth at a participating study centre will be included. Pregnancy care for the participants will be up to their doctors, with no changes based on the study. SCRIPT - the new tool - will guide future care by predicting who may benefit from specific treatments, reducing harm to low-risk individuals \& will allow selection of high-risk patients for a future trials to determine whether currently available and novel treatments in well-selected patients can improve APO sufficiently to balance treatment-related harms. | Sickle Cell Disease|Pregnancy, High Risk|Pregnancy Complications | FEMALE | CHILD, ADULT | Johns Hopkins University, Baltimore, Maryland, 21218, United States|Providence Health Care, Vancouver, British Columbia, V5Z 1M9, Canada|Centre hospitalier de l'Université de Montréal, Montréal, Quebec, H2X 0C1, Canada |
| A Dose-Finding Study of AG-348 in Sickle Cell Disease | Background: Sickle Cell Disease (SCD) is an inherited blood disorder. People with SCD have abnormal hemoglobin in their red blood cells. Researchers are investigating the safety and efficacy of an investigational medicine called AG-348 (mitapivat sulfate) to determine if it will help people with SCD. Objective: To test the tolerability and safety of AG-348 in people with SCD. Eligibility: People ages 18 and older with SCD. Design: Participants will have 8 visits over approximately 14 weeks. At the first visit participants will be screened with a medical history, a physical exam, blood and urine testing, and an EKG. During the following 5 visits, participants will stay at the clinic for 1 night each. Participants will take study drug in increasing doses up to visit 6, after which the drug will be tapered off. All visits will include physical exam, blood, and urine tests. The last visit will occur 4 weeks after stopping the drug. Participants will provide DNA from the blood samples they provide. The DNA will be tested for an inherited gene that can cause differences in response to the study drug. Researchers may also test other genes to see if they can find any genes that interact with SCD. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Oral GSK4172239D Compared With Placebo in Sickle Cell Disease Participants Aged 18 to 50 Years | This will be a first time in human (FTIH) study in sickle cell diseases (SCD) participants. The FTIH study is planned to evaluate the safety, tolerability, and pharmacokinetics of GSK4172239D. The study will be composed of 3 periods for all participants (Screening, Treatment, and Follow up). Participants will be screened and, prior to first dose on Day 1, will be randomized to receive either GSK4172239D or placebo. GSK4172239D is a prodrug that is converted in vivo into GSK4106401. This study will be a single dose, dose-escalation study. The initial dosing for all cohorts will be staggered so that 2 participants will be dosed as sentinel participants. Provided there are no safety concerns in 48 hours (h), the remaining 6 participants scheduled for the cohort may be dosed. One selected cohort of participants will also receive an additional single dose of GSK4172239D (or matching placebo) under fed (high calorie and high fat) conditions after a washout period of a minimum of 20 days or 5 half-lives, whichever is longer, designated as the Food Effect Cohort. | Hematologic Diseases|Anaemia, Sickle Cell | ALL | ADULT | GSK Investigational Site, Los Angeles, California, 90095, United States|GSK Investigational Site, Miami, Florida, 33165, United States|GSK Investigational Site, Miami, Florida, 33186, United States|GSK Investigational Site, Tamarac, Florida, 33321, United States|GSK Investigational Site, Atlanta, Georgia, 30315, United States|GSK Investigational Site, Columbus, Georgia, 31904, United States|GSK Investigational Site, Douglasville, Georgia, 30134, United States|GSK Investigational Site, Las Vegas, Nevada, 89113, United States|GSK Investigational Site, Greenville, North Carolina, 27834, United States|GSK Investigational Site, Raleigh, North Carolina, 27617, United States|GSK Investigational Site, Houston, Texas, 77030, United States |
| RElated Haplo-DonoR Haematopoietic stEm Cell Transplantation for Adults With Severe Sickle Cell Disease | The purpose of this clinical trial is to evaluate the clinical and cost effectiveness of Haploidentical Stem Cell Transplantation (SCT) for adults with severe sickle cell disease (SCD), who have failed other therapies or are intolerant of existing therapies or require chronic transfusions to prevent on-going complications of SCD. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | King's College Hospital, London, United Kingdom |
| Alendronate for Osteonecrosis in Adults With Sickle Cell Disease | A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell disease and osteonecrosis | Sickle Cell Disease|Sickle Cell Anemia|Osteonecrosis|Ischemic Necrosis|Avascular Necrosis | ALL | ADULT, OLDER_ADULT | UC Davis Comprehensive Cancer Center, Sacramento, California, 95817, United States |
| Pilot and Feasibility Trial of Plerixafor for Hematopoietic Stem Cell (HSC) Mobilization in Patients With Sickle Cell Disease Pilot and Feasibility Trial of Plerixafor for Hematopoietic Stem Cell (HSC) Mobilization in Patients With Sickle Cell Disease | Sickle cell disease (SCD) is one of the most common genetic diseases in the world. In North America, an estimated 2600 babies are born with SCD each year1, and approximately 70,000 to 100,000 individuals of all ages are affected in the United States2. The clinical manifestations of SCD include acute events, such as recurrent debilitating painful crises, as well as life-threatening pulmonary, cardiovascular, renal, and neurologic complications. The only established curative treatment for SCD patients is allogeneic hematopoietic stem cell transplant (HSCT). Unfortunately, access to this intervention is limited by availability of suitable matched donors, and HSCT is associated with significant morbidity and mortality. For patients who cannot undergo HSCT, treatment of SCD has been limited to one FDA-approved medication, hydroxyurea, and supportive symptomatic care. After decades with very few novel therapeutic options for SCD patients, autologous cell-based genetic therapies, including lentiviral-based gene therapy as well as gene editing, now offer the possibility of innovative curative approaches for patients lacking a matched donor for hematopoietic stem cell transplantation. Gene therapy for sickle cell disease is increasingly promising, and there are currently open clinical trials at several centers that employ a gene addition strategy. Options for autologous HSC collection include bone marrow harvest or peripheral blood HSC mobilization. Bone marrow (BM) harvest is an invasive procedure requiring anesthesia, which is associated with sickle cell-related morbidities, and may not achieve goal CD34+ cell dose, necessitating repeated procedures scheduled over multiple months. In most gene therapy trials, HSCs are obtained through peripheral collection after mobilization with granulocyte colony-stimulating factor (G-CSF) followed by peripheral blood (PB) apheresis. However, this approach is contraindicated in SCD because G-CSF has been reported to cause severe adverse effects in sickle cell patients. Even with doses sometimes smaller than standard, G-CSF has been shown to result in vaso-occlusive crises, severe acute chest syndrome, and in one report, massive splenomegaly and death. Alternative options for mobilization are needed. Plerixafor has been compared to G-CSF in a sickle cell mouse model, and results showed effective mobilization of HSC subsets, without neutrophil or endothelial activation, and with lower total WBC and neutrophil counts compared to G-CSF-treated mice. Plerixafor use has not yet been documented in sickle cell patients. One other trial is currently open to test plerixafor in SCD patients (NCT02193191) but no results have yet been reported. Based on pre-clinical data, the mechanism of action of plerixafor, as well strategies the investigator will employ to mitigate risk, the investigator anticipates that it will be well-tolerated in the SCD patient population. | Sickle Cell Disease Without Crisis | ALL | ADULT | Boston Childrens Hospital, Boston, Massachusetts, 02115, United States |
| Simvastatin (Zocor) Therapy in Sickle Cell Disease | Recent clinical and experimental data indicate that statins have effects beyond cholesterol lowering that may be beneficial in sickle cell disease by protecting the vascular endothelium. Statins have been shown to attenuate endothelial dysfunction through their anti-inflammatory, anti-oxidant and anti-thrombotic properties. This phase I/II dose-escalating trial is designed to assess the safety and potential clinical efficacy of oral simvastatin (Zocor)in adolescents and adults with sickle cell disease (SCD). | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital and Research Center Oakland, Oakland, California, 94609, United States |
| The Implementation of the Automated Erythrocytapheresis in Egyptian Sickle Cell Disease Center | Improvements of health infrastructure, preventive care and clinical treatment have reduced the morbidity and mortality of sickle cell disease (SCD). However, SCD is still an increasing national health problem, with increase longevity the chronic effect of sustained hemolysis and episodic vaso-occlusive events and the recurrent episodes of ischemic reperfusion injury drive the development of progressive end organ complications and cardiovascular, pulmonary, neurological and renal systems are most commonly affected. Today there is hope for a cure using hematopoietic stem cell transplantation (HSCT). However, at present; the procedure is infrequently performed and very expensive. In this research we will assess the effect of implementation of the automated erythrocytapheresis in the outcome of sickle cell disease in single Egyptian center. | Sickle Cell Disease | ALL | CHILD, ADULT | Faculty of Medicine, Ain Shams University, Cairo, Non-US, 11556, Egypt |
| Omega-3 Fatty Acids in Sickle Cell Disease | The purpose of this study is to determine the safety of a new formulation of the omega-3 fatty acids Docosahexaenoic Acid (DHA) and Eicosapentaenoic Acid (EPA) and to assess whether it decreases inflammation and inflammatory pain in children and young adults with Sickle Cell Disease. | Sickle Cell Disease | ALL | CHILD, ADULT | Nemours/Alfred I duPont Hospital for Children, Wilmington, Delaware, 19899, United States |
| Sickle Cell Hemoglobinopathies and Bone Health | This research study has two purposes. The first purpose is to determine whether having sickle cell trait (SCT) is a risk factor for the development of bone thinning at an earlier age than expected. Nearly 10% of African Americans (AA) carry sickle cell trait and most of them are unaware of it. African Americans are less likely to develop thin bones than whites, but if they sustain a bone fracture, they are more likely to die from it. We believe having sickle cell trait may lead to bone thinning and predispose a subset of African Americans to dangerously thin bones. The second purpose is to try to understand why individuals with sickle cell disease (SCD) have thinner bones than healthy individuals do. Doctors have already discovered that people with sickle cell disease have very thin bones, but they have not determined why. Our study will try to identify whether the bone thinning is from the body not making enough bone or from the body losing bone once it is made. | Sickle Cell Disease|Sickle Cell Trait | FEMALE | ADULT | UConn Health, Farmington, Connecticut, 06032, United States |
| Peripheral Blood Stem Cell Collection for Sickle Cell Disease (SCD) Patients | The constitution of blood relies upon hematopoietic stem cells (HSCs), which stay in the bone marrow and differentiate to all lineages of peripheral blood cells. HSC transplantation is the only curative option currently available for sickle cell disease (SCD) patients either via allogeneic HSC transplantation or HSC-targeted gene therapy. Granulocyte-colony stimulating factor (G-CSF)- mobilized HSCs are frequently utilized in the adult setting of HSC transplantation because of the faster hematologic recovery as compared to bone marrow. As an autologous HSC source for gene therapy, bone marrow harvest has been generally employed since G-CSF has been prohibitive in SCD patients due to granulocyte stimulation and the associated reports of vaso-occlusive crises, multi-organ failure, and death. However, when bone marrow harvest is used, the amounts of collected cells are limited and anesthesia is required. In order to obtain HSCs in large numbers without anesthesia, patients will undergo mobilization followed by large volume apheresis. Plerixafor is an alternative treatment for mobilization without direct stimulation to granulocytes, and it is theoretically applicable for SCD patients. The primary endpoint of this study is to obtain sufficient amounts of HSCs collected from the peripheral blood in SCD patients after plerixafor mobilization with an acceptable safety profile. The harvested products will be stored as backup for patients undergoing gene therapy as well as allogeneic HSC transplantation. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Muscle Function and Its Biological and Physiological Determinants in Sickle Cell Disease | Background : Sickle cell patients have profound remodeling of their muscle microcirculation networks with signs of amyotrophy. However, the consequences of these muscle alterations on the functional status of muscles are unknown. In addition, whether the poor physical fitness of sickle cell patients can be attributed, at least partly, to an hypothetical muscle dysfunction has never been tested. Purpose : this study will compare the muscle function of legs between sickle cell patients (SS and SC genotypes) and healthy individuals (AA genotype) before, during and after a short localized muscle endurance exercise. Abstract : Very recently, a study reported large differences between the muscle microcirculation networks of sickle cell patients compared to healthy individuals with decreased capillary density and higher proportion of large capillaries in the former population. In addition, the same study showed signs of amyotrophy in sickle cell patients. However, the muscle function of sickle cell patients has not been investigated and one may suggest that muscle dysfunction could participate in the decrease of physical fitness, in association with the hematological and hemorheological disorders, already reported in this population. The hypothesis is that muscle fatigue during a short localized muscle endurance exercise should be higher in sickle cell patients compared to healthy individuals, due to a greater recruitment of glycolytic fibers and a faster decrease of muscle oxygenation during exercise. | Sickle Cell Disease | ALL | CHILD, ADULT | Hôpital Edouard Herriot, Lyon, 69003, France |
| MACS Study - Microparticles and Coagulation in Sickle Cell Disease | Sickle cell disease (SCD) is an inherited disorder of the red blood cell. It is now the commonest genetic disorder in the UK and of childhood stroke, with up to 40% of children having a stroke (clinical or picked up on a scan) by school age. Patients are prone to develop acute crises necessitating hospital admission and resulting in long-term complications. Such events result in considerable morbidity, disability and mortality with its consequent burden on patients, families, the health service and society as a whole. Doctors have very little ability to predict who will get ill and when and so it is very difficult to known when and how to administer treatments. Furthermore there is very little in the way of treatments available and the mainstay of prevention is a chronic blood transfusion programme which is expensive, requires time off work and school and can be fraught with complications. This, in a population who is frequently educationally and socially disadvantaged at the outset. Recent evidence in sickle cell disease and other diseases that have similar underlying processes, points towards the importance of microparticles (circulating broken pieces of cells) and the coagulation system as being important. By comparing levels of these particles and molecules in patients with those found in healthy volunteers and with other measures known to be important, this study hopes to identify their role so as to improve the management of these patients and potentially to lead the way for new therapies. Participants will be required to donate a small amount of blood (1 teaspoon in the very young, two in older children and adults). The investigators aim to take this sample where possible when people are having a blood test in any case. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Whittington Hospital NHS Trust, London, N19 5NF, United Kingdom|University College London Hospitals NHS Foundation Trust, London, NW1 2PJ, United Kingdom|Guy's and St Thomas' NHS Foundation Trust, London, SE1 7EH, United Kingdom |
| Families Taking Control (FTC): Family-based Problem-solving Intervention for Children With Sickle Cell Disease | This study aims to develop an effective, brief, family-based intervention targeting quality of life and school functioning for youth with sickle cell disease. Utilizing a randomized, delayed control group intervention methodology, the present study will systematically document the effectiveness of a family-based, one-day intervention plus booster phone calls to improve quality of life and increase school functioning for children with sickle cell disease transitioning to school and their families. | Sickle Cell Disease | ALL | CHILD | |
| Sickle Cell Disease and CardiovAscular Risk - Red Cell Exchange Trial (SCD-CARRE) | The SCD-CARRE trial is a Phase 3, prospective, randomized, multicenter, controlled, parallel two-arm study aimed to determine if automated exchange blood transfusion and standard of care administered to high mortality risk adult SCD patients reduces the total number of episodes of clinical worsening of SCD requiring acute health care encounters (non-elective infusion center/ER/hospital visits) or resulting in death over 12 months as compared with standard of care. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of Alabama, Tuscaloosa, Alabama, 35401, United States|UCSF Benioff Children's Hospital Oakland, Oakland, California, 94609, United States|Howard University Center for Sickle Cell Disease, Washington, District of Columbia, 20060, United States|Emory University, Atlanta, Georgia, 30322, United States|University of Illinois at Chicago, Chicago, Illinois, 60607, United States|University of Maryland, Baltimore, Maryland, 21201, United States|Johns Hopkins University, Baltimore, Maryland, 21206, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|Washington University-St. Louis, Saint Louis, Missouri, 63110, United States|Icahn School of Medicine at Mount Sinai, New York, New York, 10029, United States|Montefiore Medical Center, New York, New York, 10461, United States|University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, 27599, United States|Atrium Health, Charlotte, North Carolina, 28204, United States|Duke University, Durham, North Carolina, 27708, United States|East Carolina University, Greenville, North Carolina, 27834, United States|University Hospitals Cleveland Medical Center, Cleveland, Ohio, 44106, United States|Ohio State University, Columbus, Ohio, 43210, United States|University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, 15232, United States|University of Texas Health Science Center at Houston, Houston, Texas, 77030, United States|Virginia Commonwealth University, Richmond, Virginia, 23284, United States|Hemorio, Rio De Janeiro, Brazil|Kremlin-Bicêtre, Créteil, France|Henri Mondor Hopital, Paris, France |
| A Pilot Study of N-acetylcysteine in Patients With Sickle Cell Disease | Part 1: A pilot study in patients with homozygous S (HbSS) or hemoglobin S with beta zero thalassemia(HbS-βo thalassemia), with the aim of examining the effect of intravenous NAC treatment on plasma VWF parameters and measures of redox and RBC function. Part 2: A pilot study in patients with sickle cell disease admitted to the hospital in vaso-occlusive crisis to determine the effects of NAC infusions on plasma VWF parameters and measures of redox and RBC function, and on measures of pain and hospital length of stay. | Sickle Cell Disease|Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | University of Washington, Seattle, Washington, 98106, United States |
| The Role of Endothelin 1 as a Marker of Renal Impairment in Sickle Cell Disease | Sickle cell disease (SCD) refers to a group of hemoglobinopathies that include mutations in the gene encoding the beta subunit of hemoglobin. Within the umbrella of SCD, many subgroups exist, namely sickle cell anemia (SCA), hemoglobin SC disease (HbSC), and hemoglobin sickle-beta-thalassemia (beta-thalassemia positive or beta-thalassemia negative). Several other minor variants within the group of SCDs also, albeit not as common as the varieties mentioned above. It is essential to mention the sickle cell trait (HbAS), which carries a heterozygous mutation and seldom presents clinical signs or symptoms. Sickle cell anemia is the most common form of SCD | Sickle Cell Disease | ALL | CHILD, ADULT | Sohag university Hospital, Sohag, Egypt |
| Acupuncture for Pain in Sickle Cell Disease | Sickle cell disease (SCD) is the most common genetic disorder in the United States affecting approximately 100,000 individuals primarily of African ancestry. Pain is the most common complication of SCD. Currently, the mainstay therapy for pain in SCD is opioids. The CDC recommends using non-opioid, non-pharmacologic therapies for pain. There is a growing body of literature to support the use of various integrative therapies for pain. Acupuncture therapy is a non-pharmacological Chinese medicine approach which has been used in many non-SCD conditions associated with pain. Proposed study will test acceptability and feasibility of use of acupuncture in SCD patients hospitalized for pain. It is hypothesized that the use of acupuncture as an adjuvant therapy will be acceptable to SCD patients admitted for pain control. Its impact on opioid use and circulating cytokines and neuropeptides will also be determined. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's National Health System, Washington, District of Columbia, 20010, United States |
| Antioxidant Therapy to Reduce Inflammation in Sickle Cell Disease | The purpose of this study is to determine whether alpha-lipoic acid and acetyl-L-carnitine will lower systemic inflammation in patients with Sickle Cell Disease by reducing oxidative stress, which will result in a decrease in the frequency of vaso-occlusive pain episodes and improve their quality of life. | Anemia, Sickle Cell | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital & Research Center Oakland, Oakland, California, 94609, United States |
| Effectiveness of Laying-on-of-hands for Sickle Cell Disease | The objective of this study is to evaluate the effectiveness of 1-year administration of laying-on-of-hands on the morbidity and mortality of patients with sickle cell disease in Africa. | Sickle Cell Disease|Pain Crisis Recurrent|Anemia|Infection|Death | ALL | CHILD, ADULT | The national medical center for sickle cell disease, Kinshasa, Congo, The Democratic Republic of the |
| Phase 1 Study of Zoledronic Acid in Sickle Cell Disease | The long-term goal of this study is to learn if Zoledronic Acid can prevent or reduce pain in sickle cell disease. The goal of this study is to learn about the safety of Zoledronic Acid in persons with sickle cell disease who experience chronic pain requiring medical treatment or use of narcotics. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Virginia Commonwealth University, Richmond, Virginia, 23298, United States |
| Carbon Monoxide Measurement to Screen for Sickle Cell Disease | Modify the design of the CoSense device (Model C20112, currently cleared by the FDA for ETCO (end-tidal carbon monoxide) monitoring to improve accuracy and consistency under temperature conditions encountered in countries with high prevalence of SCD (Sickle Cell Disease). | Sickle Cell Anemia | ALL | CHILD, ADULT | |
| iPeer2Peer Program for Youth With Sickle Cell Disease | The iPeer2Peer Sickle Cell Disease (SCD) study matches youth (12-18 years of age) with SCD to a mentor (trained young adult) who has learned to manage their SCD well, transitioned to adult care, and can support youth participants emotionally and socially. Participants will be randomly assigned one of two groups, either (1) The intervention group: Study group participants are matched with a mentor for 15 weeks, and are expected to have up to ten calls with one another; (2) The control group: This study group will be on a 15 week waitlist to receive a mentor. This study will first assess the feasibility of conducting this research with youth with SCD. Also, this study will assess the preliminary effectiveness of peer mentorship by comparing various health outcomes of the two study groups post-intervention. | Sickle Cell Disease | ALL | CHILD, ADULT | Connecticut Children's Medical Center, Hartford, Connecticut, 06106, United States|Children's Hospital of Eastern Ontario (CHEO), Ottawa, Ontario, Canada|The Hospital for Sick Children, Toronto, Ontario, Canada|Jim Pattison, Saskatoon, Saskatchewan, Canada |
| The Effect of Rivaroxaban in Sickle Cell Disease | The primary study hypothesis is that inhibition of factor Xa with rivaroxaban will reduce inflammation, coagulation and endothelial cell activation, and improve microvascular blood flow in patients with sickle cell disease (SCD) during the non-crisis, steady state. To test this hypothesis, this study will evaluate the effects of rivaroxaban on: * plasma markers of inflammation; * plasma markers of endothelial activation; * plasma markers of thrombin generation; and * microvascular blood flow assessed using laser Doppler velocimetry (LDV) of post-occlusive reactive hyperemia (PORH). In a cross-over design, subjects will receive rivaroxaban 20 mg/day and placebo for 4 weeks each, separated by a 2-week washout phase. | Sickle Cell Anemia|Sickle Cell-Beta0-Thalassemia | ALL | ADULT, OLDER_ADULT | University of North Carolina - Chapel Hill, Chapel Hill, North Carolina, 27599, United States |
| Exercise Capacity in Pediatric Sickle Cell Anemia | The purpose of this study is to use comprehensive exercise testing to examine causes of exercise limitation in children and young adults with sickle cell anemia. | Sickle Cell Anemia | ALL | CHILD, ADULT | Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States |
| Lipid Balance in Adult Sickle Cell Patients | This study aims to describe and/or searches for, in cohorts of adult sickle cell anemia (SCA) and SC sickle cell patients living in the French West Indies and followed by SCD Reference and Competence Centers: 1-lipids profiles and associations at steady state with occurrence of sickle cell disease (SCD) complications, 2-lipids profile evolution during and after prospective acute complications (vasoocclusive crises (VOC) and priapism), 3-lipids profile variation (inter /intra individuals) during 4 prospective years, 4- Genetic primary modulators of SCD complications, 5- insulin resistance (HOMA), free fatty acids and glycerol dosages, 6- lipids enzymes, lipidome and functionality of HDL in sub-groups of SCD population. | Sickle Cell Disease|Dyslipidemia|Complication | ALL | ADULT, OLDER_ADULT | Unité Transversale de la Drépanocytose, Pointe-à-Pitre, Guadeloupe, 97159, France|Centre de Référence de la Drépanocytose, Le Lamentin, Martinique, 97292, France |
| Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease | This is a Phase II, single arm, multi-center trial. It is designed to estimate the efficacy and toxicity of hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD) who have high risk features. The primary goal of this multi-center Phase II study is to determine the safety and feasibility of a conditioning regimen consisting of busulfan (Bu)/ fludarabine (Flu)/ anti-thymocyte globulin (ATG) in adult patients with severe SCD. A two-component design will be used for this study. The first component will be restricted to patients who have an HLA-identical sibling donor. Five patients will be transplanted during the first component of the study. If no more than 2 of the first 5 patients experience unacceptable toxicity, including death, within the first six months after transplantation, then the safety of the regimen will be considered promising in adult SCD patients. The second component will include patients who have a related or an unrelated human leukocyte antigen (HLA) matched donor. Up to 15 additional patients will be transplanted in this component of the study which will evaluate the safety and feasibility of unrelated donor hematopoietic cell transplantation (HCT) in adults with SCD. Data related to study endpoints for 1 year after transplantation will be collected; however, participating centers will be encouraged to conduct long-term follow-up evaluations of patients according to standard institutional guidelines. The purpose of this pilot safety trial is to see if this approach is feasible and meets accrual goals lending support to the development of a subsequent full scale investigation of HCT and comparing outcomes in a transplantation cohort to a control cohort of adults eligible for, but unwilling or unable to receive HCT treated by supportive therapy with a primary endpoint of five years survival for this full scale comparative trial. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's Hospital of Oakland, Oakland, California, 94609, United States|Children's National Medical Center, Washington, D.C., District of Columbia, 20010, United States|University of Miami, Miami, Florida, 33136, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States|Emory University, Atlanta, Georgia, 30322, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|Chidren's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, 15224, United States|Virginia Commonwealth University, Richmond, Virginia, 23298, United States |
| Niacin to Improve Blood Flow in People With Sickle Cell Disease | This study will determine whether niacin can improve blood flow in people with sickle cell disease, in which abnormal red blood cells interfere with blood flow to cause the disease symptoms. Niacin, a drug that has been used to increase HDL (good cholesterol) levels, improves blood flow in people without sickle cell disease. This study will see if it can do the same in people with the disease. Patients with sickle cell disease between 18 and 65 years of age may be eligible for this study. Candidates are screened with a medical history, physical examination, blood tests, echocardiogram and 6-minute walk test of exercise capacity. Participants have the following baseline blood flow studies: * Flow-mediated dilation (FMD): An ultrasound picture of the artery in the forearm is obtained. A blood pressure cuff is then placed on the upper arm and inflated for 5 minutes. After the pressure cuff is released, the ultrasound is repeated. * Peripheral artery tonometry (PAT): A sensor is placed on the subject s finger. The sensor puts pressure on the finger and measures blood flow. * Standard forearm blood flow test: Small tubes are placed in the artery of the forearm at the inside of the elbow. Saline is infused into one tube. Pressure cuffs are applied to the wrist and upper arm. A strain gauge (rubber band device) is placed around the forearm. When the cuffs are inflated, blood flows into the arm, stretching the strain gauge, and the flow measurement is recorded. Blood samples are collected from the tube in the artery to measure blood counts, proteins and other chemicals. At various times, small doses of the following drugs are administered through the tube in the vein: * Sodium nitroprusside causes blood vessels to dilate and increases blood flow to the heart. * Acetylcholine causes blood vessels to dilate and slows heart rate. * LNMMA decreases blood flow by blocking the production of nitric oxide. Blood flow is measured after each dose of the different drugs. There are rest periods between injections of the different drugs. Pictures of the forearm are taken during the studies using an infrared camera and computer. -Drug Treatment. Participants are assigned to take three 4-week courses of niacin or placebo. They return to the Clinical Center at the following intervals from the time they start the test drug for followup: * Weeks 2, 6 and 10: Brief medical history, review of medication side effects and blood tests. * Weeks 4 and 8: Physical examination, brief medical history, review of medication side effects and blood tests, repeat FMD and PAT blood flow studies and 6-minute walk test. * Week 12: Same as weeks 4 and 8 plus standard blood flow studies and echocardiogram. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Howard University Hospital, Washington, District of Columbia, 20060, United States|National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| Improving Self-Management in Adolescents With Sickle Cell Disease | The objective of this study is to determine the feasibility and acceptability of SCThrive, a an innovative, technology-enhanced, group self-management intervention that uses a mixed in-person and online format and supported by a tailored mHealth tool, iManage. The study will also evaluate the initial efficacy of SCThrive for increasing behavioral activation (BA) in adolescents with Sickle Cell Disease (SCD) ages 13 to 21. The investigators hypothesize that participants in the SCThrive group will show greater BA (primary outcome) at post-treatment than the attention control group, and that participants in the SCThrive group will continue to show significantly greater BA at the six week follow-up compared to the attention control group. Investigators will also explore whether SCThrive is associated with greater improvements in self-management behaviors and quality of life (secondary outcome) compared to attention control at the six-week follow-up assessment. | Sickle Cell Disease | ALL | CHILD, ADULT | Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States |
| Hydroxyurea in the Treatment of Sickle Cell Disease | This is a retrospective cohort study of Sickle Cell Disease (SCD) patients attending 32 treatment centers across Italy. The aim of this study will be to report the Italian experience with the use of hydroxyurea in a large cohort of SCD patients and to evaluate the benefits and safety of this intervention for the prevention and management of a wide range of clinical morbidities | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Ospedali Civili Riuniti di Sciacca, Sciacca, Agrigento, 92019, Italy|Ospedale Vittorio Emanuele III, Gela, Caltanisetta, 93012, Italy|Azienda Ospedaliero-Universitaria, Bari, Italy|U.O. Oncoematologia Pediatrica Ospedali Civili, Brescia, 25100, Italy|Ospedale "A. Perrino", Brindisi, 72100, Italy|Azienda Ospedaliera Universitaria Di Cagliari, Cagliari, 09121, Italy|Ospedale Vittorio Emanuele, Catania, 95123, Italy|ARNAS "Garibaldi", Catania, 95124, Italy|University of Catania, Catania, 95124, Italy|A.O. "Pugliese-Ciaccio", Catanzaro, 88100, Italy|Osp.San Giovanni Di Dio, Crotone, Italy|Azienda Ospedaliero Universitaria Meyer, Firenze, 50139, Italy|E.O. Ospedali Galliera, Genova, 16128, Italy|Università degli Studi di Milano, Milano, 20122, Italy|Azienda Ospedaliero-Universitaria di Modena - Policlinico, Modena, 41124, Italy|Clinica pediatrica Monza S. Gerardo, Monza, 20052, Italy|Azienda Ospedaliera San Gerardo di Monza, Monza, 20900, Italy|Aorn A. Cardarelli, Napoli, Italy|Clinica di Onco-Ematologia Pediatrica, Università di Padova, Padova, 35128, Italy|A.R.N.A.S. "Civico", Palermo, 90100, Italy|Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello, Palermo, 90146, Italy|Azienda Ospedaliero-Universitaria di Parma, Parma, 43126, Italy|Policlinico San Matteo, Pavia, 27100, Italy|Centro Emofilia e Medicina Trasfusionale - Pres. Ospedaliero, Ravenna, 48121, Italy|A. O. Bianchi Melacrino Morelli, Reggio Calabria, 89100, Italy|Ospedale S. Eugenio - FF UOSD DH Talassemici, Roma, 00144, Italy|Università Cattolica del Sacro Cuore - Policlinico A.Gemelli, Roma, 00168, Italy|U.O.S. Talassemia P.O. Umberto I°, Siracusa, 96100, Italy|Università degli Suidi di Torino, Torino, 90146, Italy|Policlinico G.B. Rossi, Verona, 37122, Italy |
| Study of Biological Markers in Children With Sickle Cell Disease | Sickle cell disease is associated with significant morbi-mortality hence the interest in an early and targeted care. At present, there is no plasmatic marker able to identify infants at higher risk of developping severe complications later in life. However, recent studies have demonstrated a correlation between certain complications of the disease and biomarkers of the endothelial dysfunction characterizing it. Investigators prospectively followed a cohort of children diagnosed with SCD through the universal neonatal screening using inflammatory and haemostatic plasmatic markers to study their annual evolution. Investigators then will evaluate potential associations between these biological markers and the occurrence of SCD related complications. A secondary objective of this study is to evaluate the repercussions of therapeutic intervention on these markers. . | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | CHU Saint Pierre, Brussels, 1000, Belgium|Hôpital Universitaire Des Enfants Reine Fabiola, Brussels, 1020, Belgium|HIS - Site Etterbeek-Ixelles, Brussels, 1050, Belgium |
| T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias | The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting. | Sickle Cell Anemia|Beta-thalassemia Major|Diamond-blackfan Anemia | ALL | CHILD, ADULT | Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, 15224, United States |
| Reduced Intensity Transplantation for Severe Sickle Cell Disease | This study is being done to test a transplant method that may have fewer side effects (or less toxic, less harmful) than conventional high dose chemotherapy conditioning-based transplants for children and young adults with Sickle Cell Disease (SCD). Patients less than or equal to 25 years old with SCD who would likely benefit from allogeneic hematopoietic cell transplantation (HCT) will be included in this study. Patients with a suitable HLA matched sibling donor (MSD) will be enrolled on the MSD arm while patients without an eligible MSD who have a suitable haploidentical (HAPLO) donor available will be enrolled on the HAPLO arm of the study. Primary Objective To assess the donor T-cell chimerism at 1-year post transplant in each respective arm (MSD, HAPLO) of the trial. Secondary Objectives * Assess the overall survival and 1-year, 2-year and 3-year post-transplant graft versus host disease (GVHD)-free SCD-free survival. * Estimate the primary and secondary graft rejection rate at 1-year, 2-year and 3-year post- transplant. * Estimate the incidence and severity of acute and chronic (GVHD). * Estimate the incidence of SCD recurrence after transplant * Assess the neutrophil and platelet recovery kinetics post-transplant. Exploratory Objectives * Record immune reconstitution parameters, including chimerism analysis, quantitative lymphocyte subsets, T cell receptor excision circle (TREC) analysis, V-beta spectratyping, and lymphocyte phenotype and function. * Conduct longitudinal examination of impact of HCT on patient health-related quality of life (HRQL) and adjustment, and parental adjustment. * Examine impact of HCT on patient cognitive and academic function. * Determine factors that influenced the decision to undergo HCT, explore perceptions of the HCT experience, and assess decisional satisfaction/regret. * Develop and evaluate an objective/quantitative imaging biomarker to assess organ (liver and heart) function/disease status and changes following HCT. * Develop and evaluate an objective/quantitative imaging biomarker to determine cerebral blood flow and oxygen extraction fraction following HCT. | Sickle Cell Disease | ALL | CHILD, ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Clinical and Laboratory Assessment of Iron Overload in Sickle Cell Anemia and Sickle Cell Thalassemia | Iron overload is well study in Thalassemia patients and it's not only related to blood transfusions, since intestinal iron absorption is also increased in those patients. Sickle cell patients didn't developed significant clinical symptoms and signs of iron overload in spite frequent transfusions. The purpose of this study is to assess the iron overload in Sickle cell anemia and Sickle cell Thalassemia patients using clinical parameters and laboratory studies including Non Transferrin Binding Iron, Labile Iron and Hepcidin, in order to determine the cardiac and liver iron. | Sickle Cell Anemia|Sickle Cell Thalassemia | ALL | CHILD, ADULT, OLDER_ADULT | Pediatric Hematology Unit - HaEmek Medical Center, Afula, 18101, Israel |
| Study of Hydroxyurea to Treat Sickle Cell Disease | The aim of this single-center observational study was to evaluate quality of life, clinical effectiveness, and satisfaction in pediatric and young adult patients with sickle cell disease receiving hydroxyurea. | Sickle Cell Disease | ALL | CHILD, ADULT | Selma Unal, Mersin, 33169, Turkey |
| The Role of Red Cell Characteristics, Angiogenesis, Viscosity and Oxygenation in the Pathophysiology of Sickle Cell Related Retinopathy | Objective: to gain insight in the pathogenesis, to identify biomarkers to recognize patients at risk for proliferative SCR and to investigate its associations with clinical and laboratory characteristics. Endpoints: The investigators will determine the difference in the above named parameters between patients with and without PSCR Study design: This case control study will include adult sickle cell disease patients with the HbSS or HbSC genotype. For both genotypes, 20 patients without sickle cell retinopathy (SCR) and 20 patients with PSCR will be included, resulting in a total of 80 patients. Venous blood samples and retinal imaging scans will be collected for each included patient. | Sickle Cell Retinopathy|Sickle Cell Disease|Sickle Cell SC Disease|Sickle Cell-SS Disease | ALL | ADULT, OLDER_ADULT | Amsterdam UMC, Amsterdam, Noord-Holland, 1105AZ, Netherlands |
| Sickle Cell Children's Exercise Study (SuCCESs) | The Sickle Cell Children's Exercise Study (SuCCESs) will explore the feasibility and effects of a moderate intensity strengthening, balance, speed, and agility intervention program in children with sickle cell disease. | Sickle Cell Disease | ALL | CHILD | University of Maryland School of Medicine Dept. of Physical Therapy & Rehabilitation Science, Baltimore, Maryland, 21201-1082, United States|University of Maryland, Baltimore, Baltimore, Maryland, 21201, United States |
| Diastolic Dysfunction in Sickle Cell Disease During Vaso-occlusive Crisis | This study is designed to investigate the presence and absence of abnormal heart muscle contractions during sickle cell crises. You will be asked to do echocardiography during and after your admission to the hospital. We will compare tow pictures and study the differences. | Sickle Cell | ALL | CHILD, ADULT, OLDER_ADULT | King Abdulaziz Hospital, Al-Ahsa, EP, 31982, Saudi Arabia |
| Haploidentical Hematopoietic Stem Cell Transplantation (HSCT) for Patients With Severe Sickle Cell Disease | The purpose of this study is to develop a safe and curative stem cell transplant approach to treating sickle cell disease by assessing the safety of haploidentical hematopoietic stem cell transplantation using αβ+ T-cell depletion for children and adolescents with severe sickle cell disease (SCD). | Sickle Cell Disease | ALL | CHILD, ADULT | The University of Chicago, Chicago, Illinois, 60637, United States |
| School Readiness Intervention for Preschool Children With Sickle Cell Disease | The study participant is being asked to take part in this clinical trial, a type of research study, because the participant is a young child with sickle cell disease or the caregiver of a child with sickle cell disease. This study is being done to test a school readiness program for children with sickle cell disease (ages 3.5-6,5 years old). Primary Objective Assess feasibility and acceptability of an adapted school readiness intervention among preschool children (ages 3.5-6.5) diagnosed with sickle cell disease. Secondary Objectives Objective 1: Measure preliminary efficacy of the adapted school readiness intervention compared to routine care among preschool children ages (3.5-6.5) diagnosed with sickle cell disease. Objective 2: Examine implementation factors (i.e., barriers and facilitators) during post-intervention. | Sickle Cell Disease | ALL | CHILD | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling | Background: Sickle cell disease (SCD) is an inherited disorder of the blood. SCD causes red blood cells (RBCs) to die early. This can lead to a shortage of healthy cells. SCD and other blood disorders can be managed with drugs or cured with a bone marrow transplant. Researchers want to know how long RBCs survive in people with SCD and other blood disorders before and after treatment compared to those who had a bone marrow transplant. Objective: To learn how long RBCs survive in the body in people with SCD and other blood disorders compared to those whose disease was cured with a bone marrow transplant. Eligibility: People aged 18 years or older with SCD or another inherited blood disorder. People whose SCD or blood disorder was cured with a bone marrow transplant are also needed. Design: Participants will be screened. They will have a physical exam with blood and urine tests. Participants will have about 7 tablespoons of blood drawn. In the lab, this blood will be mixed with a vitamin called biotin. Biotin sticks to the outside of RBCs. This process is called "biotin labeling of RBCs." The next day, the participant s own biotin-labeled RBCs will be returned to their bloodstream. Participants will return regularly to have smaller blood samples (about 2 teaspoons) drawn. These samples will be tested to detect the percentage of cells that have biotin labels. These visits may be every 2 weeks, 4 weeks, or some other interval. Participants will continue this schedule for up to 20 weeks or until biotin can no longer be detected. | Sickle Cell Disease|Thalassemia|Hemoglobinopathy | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Relationship Between Biological Phenotype, Clinical Severity of Sickle Cell Disease, and Blood Coagulation | Sickle cell disease is characterized by chronic hemolytic anemia and blood rheological alterations. In addition, blood coagulation abnormalities have been reported in patients with sickle cell disease and hemolysis-derived products could be involved. The investigators hypothesized that patients with sickle cell disease and severe hemolysis (Lactate Dehydrogenase level \> 484 IU/L) could have an increased risk of hypercoagulable state and subsequent thromboembolic complications. | Sickle Cell Disease (SCD) | ALL | CHILD, ADULT, OLDER_ADULT | Insitut Hématologique et Oncologique Pédiatrique (IHOPe), Lyon, 69008, France|Service de Médecine Interne - Hôpital Edouard Herriot, Lyon, 69008, France |
| GenoMed4ALL: Improving SCD Classification and Prognosis by AI | GenoMed4All 'Genomics and Personalized Medicine for all though Artificial Intelligence in Haematological Diseases' aims to advance on individual SCD patients' disease characterisation and to improve the monitoring of patients' health status, optimise clinical therapy guidance and ultimately improved health outcomes by the identification of biomarkers and the development of individual (risk) models in SCD. Genomed4All supports the pooling of genomic, clinical data and other "-omics" health through a secure and privacy respectful data sharing platform based on the novel Federated Learning scheme, to advance research in personalised medicine in haematological diseases thanks to advanced Artificial Intelligence (AI) models and standardised interoperable sharing of cross-border data, without needing to directly share any sensitive clinical patients' data. The SCD Use case will gather multi-modal clinical and -OMICs data from 1,000 SCD patients in 4 EU-MS: France, Italy, Spain and The Netherlands. In close collaboration with the European Reference Network on Rare Hematological Diseases (ERN-EuroBloodNet, GA101157011), GENOMED4ALL involves multiple clinical partners from the network, while leveraging on healthcare information and repositories that will be gathered incorporating interoperability standards as promoted by ERN-EuroBloodNet central registry, the European Rare Blood Disorders Platform. | Sickle Cell Disorders | ALL | CHILD, ADULT, OLDER_ADULT | APHP Henri Mondor, Créteil, 94000, France|APHP Necker, Paris, 75015, France|Azienda Ospedale Università Padova, Padova, 35121, Italy|UMC Utrecht, Utrecht, 3584, Netherlands|Hospital Universitari Vall d'Hebron Research Institute, Barcelona, 08035, Spain |
| Transfusion in Sickle Cell Disease: Risk Factors for Alloimmunization | Sickle cell patients have a high prevalence of alloimmunization. This high rate of alloimmunization can be partially explained by the existence of an antigenic difference between the predominantly Caucasian donor population and the sickle cell patients of African origin. Genetic and environmental risk factors have also been described. The main risk factors that have been shown in retrospective or cross-sectional studies are some HLA alleles, the age of the patient, the number of leukocyte-depleted erythrocyte concentrates (CED) transfused, the number of transfusion episodes, the age of the CEDs, the existence of an inflammatory event at the time of transfusion and the presence of anti-erythrocyte autoantibodies.There is also evidence of an impaired TH response but the underlying immunological mechanism is not fully understood. The aim of this study is to study the prevalence and the risk factors for anti-erythrocyte alloimmunization and to try to understand the immunological mechanisms. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | CHU Brugmann, Brussels, 1020, Belgium|HUDERF, Brussel, 1020, Belgium |
| Oxygen Therapy and Pregnancy in Sickle Cell Disease | The purpose of this study is to assess the efficiency of the preventive oxygen therapy on the occurrence of vaso-occlusive complications, which last more than 24 hours and require hospitalisation, in women with sickle cell disease. | Sickle Cell Disease | FEMALE | ADULT | Hôpital Necker Enfants-Malades (Public Hospitals of Paris), Paris, 75015, France |
| Prevention of Malnutrition in Children with Sickle Cell Disease | The investigators are studying how to prevent malnutrition in children with sickle cell disease (SCD) in northern Nigeria. Community health workers will teach mothers about nutritious local foods that might help children grow better than the usual advice from doctors. The investigators will enroll 148 children with SCD aged 6 to 18 months old. The investigators will determine if their weight and diet improve and check for low vitamin A levels. This information will help us find better ways to improve growth for children with SCD. | Sickle Cell Disease|Malnutrition | ALL | CHILD | Vanderbilt University Medical Center, Nashville, Tennessee, 37203, United States |
| Heart Arteries and Sickle Cell Disease / Coeur Artères DREpanocytose | The CADRE study is a multinational observational cohort of patients with sickle-cell disease (SCD) in five west and central sub-Saharan African countries. The aim of this project is to describe the incidence and assess the predictive factors of SCD-related micro- and macro-vascular complications in sub-Saharan Africa. | Sickle Cell Anemia|Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Central Hospital of Yaounde, Yaounde, Cameroon|Centre mère et enfant / fondation Chantal Biya, Yaounde, Cameroon|Centre Pasteur du Cameroun, Yaounde, Cameroon|Pediatrics unit, Centre Hospitalier d'Essos, Yaounde, Cameroon|Centre hospitalier Monkole, Kinshasa, Congo, The Democratic Republic of the|Hematology Unit, CHU Yopougon, Abidjan, Côte D'Ivoire|Institut de cardiologie, Abidjan, Côte D'Ivoire|CIRMF, Libreville, Gabon|Cardiology Unit, Centre gyneco-obstretrique, Bamako, Mali|Centre de Recherche et de Lutte contre la Drepanocytose, Bamako, Mali|Centre hospitalier d'enfants Albert Royer, Dakar, Senegal|Centre hospitalo-universotaire de Fann, Cardiology department, Dakar, Senegal|Centre national de transfusion sanguine, Dakar, Senegal |
| QST-Pupillometry in Sickle Cell Disease Patients | There has been little progress for effective treatment of pain in sickle cell disease (SCD) patients. Many organizations have recognized that understanding the causes and reducing the burden of pain in SCD is critical in order to improve the quality of life in SCD patients. As patients with SCD face the challenge of living with both acute and chronic pain which is often improperly treated, our translational and interdisciplinary project aims to identify objective measures of pain sensitivity and its biochemical and genetic correlates. We hypothesize that SCD patients will have decreased tolerance to thermal and electrical stimuli. | SCD With Severe Phenotype (HbSS, HbSβ0 Thalassemia, HbSOARab) | ALL | CHILD, ADULT | Children's National Health System, Washington, District of Columbia, 20010, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States |
| Nitric Oxide and Sickle Cell Pain | Background: - Sickle cell disease often causes crises, with episodes of pain. Many people with sickle cell disease also have pain between crises. Inflammation is an important part of sickle cell pain. It may be related to levels of nitric oxide. Nitric oxide is a gas in the body that helps relax blood vessels and may be related to the pain from sickle cell disease. Researchers want to study the relationship between blood levels of nitric oxide and pain in people with sickle cell disease. Researchers also want to study how certain genes express themselves related to sickle cell pain. Objectives: - To collect blood samples and other genetic expression information to study sickle cell pain and its relation to nitric oxide levels in the blood. Eligibility: * People at least 18 years of age who have sickle cell disease. * Healthy volunteers at least 18 years of age. Design: * This study requires a screening visit and four study visits scheduled 1 week apart. Each visit will last about 1 hour. * Participants will be screened with a medical history and physical exam. They will complete questionnaires about pain levels (if any). They will also provide blood samples for genetic and other testing. * Participants will have a breath test to see how much nitric oxide they exhale. They will also have a test of their ability to detect small changes in temperature and touch. * Participants will keep a diary to record daily pain levels and pain medicines taken. They will write down what they eat to track foods that contain nitrates (such as meats like ham and bacon and vegetables like beets and spinach). * At each of the four study visit, participants will bring the pain diary, provide blood samples, and have breath nitric oxide tests. | Sickle Cell Disease|Pain | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| Integrative Medicine in Pain Management in Sickle Cell Disease | The proposed research is to determine the clinical efficacy and neurobiological mechanisms of acupuncture analgesia in patients with sickle cell disease. | Sickle Cell Disease|Pain|Acupuncture|Quantitative Sensory Testing|Magnetic Resonance Imaging|Circulating Biomarkers|Electroencephalography|Functional Near-infrared Spectroscopy | ALL | CHILD, ADULT, OLDER_ADULT | Indiana University School of Medicine, Indianapolis, Indiana, 46075, United States |
| Peripheral Blood Stem Cell Collection From Patients With Sickle Cell Disease (SCD) Using Plerixafor | With recent advances in gene editing, gene therapy is becoming a viable curative treatment option for sickle cell disease. In order to do genetic manipulation, investigators need to collect hematopoietic stem cells from patients with sickle cell disease. In this study, investigators want to study the safety and feasibility of collecting peripheral blood stem cells from pediatric and young adult patients with sickle cell disease after administering plerixafor. Studying these peripheral blood stem cells will help in optimizing the yield of peripheral CD34+ cells from pediatric and young adult patients with sickle cell disease, which in turn will help to develop better gene therapies for these patients. Primary Objectives * Determine the safety profile associated with administration of plerixafor in pediatric and young adult patients with sickle cell disease (SCD). * To estimate the number of CD34+ cells/kg of body weight that can be collected with peripheral apheresis after administration of plerixafor in pediatric and young adult patients with SCD. Exploratory Objectives * To describe the kinetics of CD34+ cell mobilization in peripheral blood after - + cells obtained from pediatric and young adult patients with SCD. * To study the effect of hydroxyurea therapy on senescence in plerixafor-mobilized CD34+ cells obtained from pediatric and young adult patients with SCD. | Sickle Cell Disease | ALL | CHILD, ADULT | |
| Added Value of Speckle Tracking in the Evaluation of Patients With Sickle Cell Disease | Sickle Cell Disease is a serious disease that is life-threatening for patients being homozygous for the SS form or heterozygous for the SC or βthal forms. The CHU Brugmann hospital currently regularly treats about 70 homozygous adult patients and this number is in constant augmentation. Sickle cell disease patients may develop a cardiomyopathy due to chronic anemia, the haemosiderosis risk or, less frequently, to coronary vaso-occlusive damages. The hypervolemia in patients with sickle cell disease causes an overestimation of the ejected left ventricular fraction measured by echocardiography, this parameter being very dependent of the blood volume.It has already been shown that the left ventricular ejection fraction was normal in most patients with sickle cell disease, but that its evaluation by parameters independent from the blood volume showed the existence of a dysfunction. Myocardial strain, as measured by speckle tracking, is a echographic evaluation method of the cardiac function, independent of the blood volume. This technique hasn't been used much in sickle cell disease patients. A study using 3D speckle tracking on a limited number of sickle cell disease patients failed to show a strain anomaly. Moreover, the study highlighted a higher global longitudinal strain in this patient population. The investigators find these data hard to explain and in contradiction with previous studies using other cardiac function evaluation techniques, independent from the blood volume. The primary goal of this study is thus * to study the longitudinal strain by 2D echography * to determine if anomalies of the longitudinal strain exist in sickle cell disease patients with a normal ejected left ventricular fraction, compared to a control group of healthy patients. The secondary goal of this study is to correlate, inside the sickle cell disease group, the possible strain anomalies with biological gravity parameters of the disease. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | CHU Brugmann, Brussels, 1020, Belgium |
| Sickle Cell Disease Treatment With Arginine Therapy (STArT) Trial | The trial of IV arginine therapy in children with Vaso-occlusive painful episodes (VOE) in sickle cell disease (SCD) is designed to further knowledge on efficacy and safety of the therapy. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's Hospital Los Angeles, Los Angeles, California, 90027, United States|UCSF Benioff Children's Hospital, San Francisco, California, 94158, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States|Children's Healthcare of Atlanta at Hughes Spalding, Atlanta, Georgia, 03322, United States|Children's Healthcare of Atlanta at Egleston, Atlanta, Georgia, 30322, United States|Washington University/St. Louis Children's Hospital, Saint Louis, Missouri, 63110, United States|Nationwide Children's Hospital, Columbus, Ohio, 43205, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Texas Children's Hospital/Baylor College of Medicine, Houston, Texas, 77030, United States|Medical College of Wisconsin/Wisconsin Children's Hospital, Wauwatosa, Wisconsin, 53226, United States |
| Establishing a Database of People With Sickle Cell Disease (Comprehensive Sickle Cell Centers Collaborative Data Project [C-Data]) | Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause intense pain episodes. The purpose of this study is to gather medical information from children and adults with SCD and establish a database so that researchers can contact people to participate in future SCD research studies. | Anemia, Sickle Cell | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital of Oakland, Oakland, California, 94609, United States|University of California, San Francisco, San Francisco, California, 94134, United States|University of Miami, Miami, Florida, 33136, United States|Kosair Children's Hospital, Louisville, Kentucky, 40202, United States|Brigham & Women's Hospital, Boston, Massachusetts, 02115, United States|Children's Hospital of Boston, Boston, Massachusetts, 02115, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|Montefiore Medical Center, Bronx, New York, 10463, United States|University of North Carolina-Chapel Hill, Chapel Hill, North Carolina, 27599, United States|Duke University, Durham, North Carolina, 27710, United States|Cincinnati Children's Hospital, Cincinnati, Ohio, 45229, United States|University of Oklahoma Health Sciences, Oklahoma City, Oklahoma, 73104, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Thomas Jefferson University Hospital, Philadelphia, Pennsylvania, 19107, United States|St. Christopher's Hospital for Children, Philadelphia, Pennsylvania, 19134, United States|Saint Jude Children's Hospital, Memphis, Tennessee, 38105, United States|Children's Medical Center of Dallas, Dallas, Texas, 75235, United States|University of Texas Southwestern and Parkland, Dallas, Texas, 75235, United States|University of Texas at Galveston, Galveston, Texas, 77555, United States |
| Arginine Therapy for Sickle Cell Disease Pain | The aim of this study is to determine whether giving extra arginine, a simple amino acid, to patients with sickle cell disease seeking treatment for a pain crisis (vaso-occlusive painful events (VOE) will decrease pain scores, decrease the need for pain medications or decrease length of hospital stay or emergency department visit. Funding Source - FDA OOPD. | Sickle Cell Disease|Vaso-occlusive Pain Episode | ALL | CHILD, ADULT | Children's Healthcare of Atlanta at Hugh Spalding, Atlanta, Georgia, 30303, United States|Children's Healthcare of Atlanta at Egleston, Atlanta, Georgia, 30322, United States|Children's Healthcare of Atlanta at Scottish Rite, Atlanta, Georgia, 30342, United States |
| Age of Blood in Sickle Cell Transfusion | The Investigators hypothesize that older red cell units trigger phagocytosis and activation of circulating macrophages with a downstream immunomodulatory cascade and release of excess Non Transferrin Bound Iron(NTBI) that leads to increased rates of infection in adults with Sickle Cell Disease(SCD). To test this hypothesis, the study staff will perform a randomized prospective clinical trial. In aim 1, the study staff will determine the biochemical differences between ≥30 day-old versus ≤10 day-old units. In aim 2, the study staff will determine the physiologic effects of the transfused blood in a patient with SCD. Lastly, in aim 3, the study staff will explore the clinical implications of receiving older red cells over a 3 month period. | Sickle Cell Disease | ALL | CHILD, ADULT | Emory University, Atlanta, Georgia, 30322, United States|University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, 27599, United States|Versiti Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| Screening Patients With Sickle Cell Disease for Kidney Damage | This study aims to study the temporal course of sickle nephropathy and assess novel biomarkers that can predict patients prone to nephropathy. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Emory University (Children's Healthcare of Atlanta Pediatric Hospital), Atlanta, Georgia, 30322, United States|Univeristy of Louisville (Kosair Children's Hospital), Louisville, Kentucky, 40202, United States|National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States|Akron Childen's Hospital, Akron, Ohio, 44308, United States |
| Pain Management Protocol for Pediatric Sickle Cell Disease | Sickle cell disease (SCD) is a devastating chronic health condition that primarily affects African-Americans. Painful episodes are the most frequent form of morbidity in SCD and the most frequent reason for hospitalization. Cognitive-behavioral interventions for pain management have shown efficacy for improving coping abilities, reducing the amount of medication needed to manage pain, and improving daily functioning during painful episodes. However, difficulties with disseminating and implementing cognitive-behavioral treatments have resulted in almost no use of these techniques in pediatric settings. In South Carolina these difficulties are compounded by social and geographical factors that pose particular challenges. A major issue with implementing quality pain management protocols is the difficulty with providing adequate practice and monitoring of the use of the techniques, particularly given the rural population in South Carolina and transportation difficulties for economically disadvantaged families. Due to a history of under-treating pain in SCD it is also critical that psychological and medical treatments are presented in an integrated manner so that these approaches are viewed as complimentary, not mutually exclusive, approaches to pain management. Finally, we believe the same implementation issues for improving the use of behavioral coping skills are also important for improving adherence to medication protocols for appropriate home-based pain management. The purpose of this proposal is to develop, implement, and evaluate a pain management protocol that uses portable electronic devices and other technologies to increase the practice of psychosocial pain management techniques, improve adherence to the overall biopsychosocial pain management protocol, and improve the clinician's ability to track progress with fewer office visits. In addition to addressing important dissemination issues, by embedding methods to assess for adherence into the technology it will be possible to continuously evaluate and modify protocol efficacy, resulting in a product that is effective, empirically sound, and flexible. Participants will be randomly assigned to the intervention or waitlist control condition. Those on the waitlist condition will receive the same study procedures after a 2 month wait periods. We anticipate that the intervention will result in better pain management and less impairment in the participants. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's Cancer and Blood Disorder, Columbia, South Carolina, 29203, United States |
| A Pilot Study of Fecal Microbiome and Neutrophil Cellular Adhesion Molecules in Patients With Sickle Cell Disease (SCD) | Patients with sickle cell disease often develop painful crisis without any obvious reasons. Some patients are more likely to develop this complication than others. It is now clear that painful crisis only occurs when sickled red blood cells stick to white blood cells that have been activated, usually by inflammation or infections. A recent study in mice with sickle cell disease showed that the use of long term antibiotics could reduce the number of activated white blood cells and reduce death of the mice during sickle cell crisis. The investigators believe that sickle cell patients who develop frequent painful crisis may have a different pattern of bacteria in their intestine when compared to those whose painful crisis occurs infrequently. In this study, the investigators propose to study sickle cell subject's blood to determine how many activated white blood cells he/she have. The investigators will also examine his/her stool to compare the bacteria in his/her stool to those other sickle cell patients. The investigators will then investigate whether or not the results from the blood and stool tests correlate with how frequently the patient develops painful crisis. The investigators will examine the patients' medical records to find out how many times they have been admitted to the hospital for sickle cell crisis in the last 12 months. The investigators will also obtain information on the following: their age, their sex, whether they are taking hydroxyurea or Penicillin, when they last had a transfusion or exchange transfusion therapy and painful crisis needing hospital admission, whether they have received any antibiotics (other than Penicillin) in the last 4 weeks, and whether they are experiencing a painful crisis at the time that they enter the study. The investigators will obtain, from their previous laboratory results, their levels of hemoglobin F and markers of inflammation. The investigators will check their hemoglobin F level if they have not already had this tested. The investigators expect to enroll 50 subjects into this study at Rhode Island Hospital/Hasbro Children's Hospital. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | |
| Macitentan in Pulmonary Hypertension of Sickle Cell Disease | This is a pilot study to assess the safety and efficacy of macitentan in patients with pulmonary hypertension of sickle cell disease. This study will enroll approximately 10 subjects. Study participation for each subject will last approximately 24 weeks from screening to end of treatment follow-up. | Pulmonary Hypertension|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Boston University School of Medicine, Boston, Massachusetts, 02118, United States |
| Web-MAP Intervention for Youth With Sickle Cell Disease | The proposed study will determine whether the efficacy of WebMAP cognitive behavioral therapy (CBT) treatment study generalizes to pediatric sickle cell disease (SCD), and explore whether the intervention is feasible and acceptable to this population. Feasibility of multi-institutional recruitment from sickle cell centers will also be determined. The short-term goal is to produce preliminary data to apply for an R01 proposal to carry out a multi-institutional randomized controlled trial (RCT) of internet-delivered behavioral intervention in a large population of youth with SCD. The long-term goal of this research is to develop effective, easily accessible, behavioral pain interventions for youth with SCD to reduce the long-term impact of pain on function, quality of life, and health service use in this population. The design of this study is an experimental 2 (group) x 3 (time of measurement) randomized controlled trial design to test the acceptability and efficacy of the Web-MAP intervention in reducing pain and functional impairment in youth with sickle cell disease. (Figure 1) Subjects will be randomized to either the behavioral intervention or the online patient education control group. The treatment protocol will be implemented over 8 weeks in Internet-based treatment modules. The primary study outcome is pain and functional impairment measured at baseline, immediately post-treatment, and at 3-month follow-up. | Sickle Cell Disease | ALL | CHILD, ADULT | CCMC, Hartford, Connecticut, 06106, United States |
| Secondary Pulmonary Hypertension in Adults With Sickle Cell Anemia | The purpose of this study is to determine how often people with sickle cell anemia develop pulmonary hypertension a serious disease in which blood pressure in the artery to the lungs is elevated. Men and women 18 years of age and older with sickle cell anemia may be eligible for this study. Participants will undergo an evaluation at Howard University s Comprehensive Sickle Cell Center in Washington, D.C. or at the National Institutes of Health in Bethesda, Maryland. It will include the following: * medical history * physical examination * blood collection (no more than 50 ml., or about 1/3 cup) to confirm the diagnosis of sickle cell anemia, sickle cell trait or beta-thalassemia (Some blood will be stored for future research testing on sickle cell anemia.) * echocardiogram (ultrasound test of the heart) to check the pumping action of the heart and the rate at which blood travels through the tricuspid valve. Following this evaluation, a study nurse will contact participants twice a month for 2 months and then once every 3 months for the next 3 years for a telephone interview. The interview will include questions about general health and recent health-related events, such as hospitalizations or emergency room visits. | Pulmonary Hypertension|Sickle Cell Anemia|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Howard University Hospital, Washington, District of Columbia, 20060, United States|National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Stroke Prevention in Young Adults With Sickle Cell Anemia | Sickle cell disease (SCD) is the most common genetic disease, affecting about 25 million people worldwide. Approximately 150,000 Nigerian children are born each year with sickle cell disease (SCD), making it the country with the largest burden of SCD in the world. Recent advancements in care for children with SCA have translated into improved survival of children in both high and low-resource settings. However, more complications of SCD are seen in those who survive to adulthood. Silent cerebral infarcts (SCI) and strokes are among the most devastating complications of SCD, affecting 40% and 10% of children, respectively. The overall goal of this study is to extend the Investigator's successful capacity-building effort in the assessment of neurological morbidity in children with SCD living in northern Nigeria (Kano) to young adults with SCD living in the same region. About 50% of all adults with SCD live in Nigeria. Despite the high prevalence of SCD in Africa, the neurological morbidity is not well characterized, limiting opportunities for primary and secondary stroke prevention strategies. At least 50% of young adults with sickle cell anemia (SCA), the most severe form of the disease, will have SCIs and an estimated 10% will have strokes, based on studies in high-resource settings. In high-resource settings, screening for abnormal transcranial Doppler (TCD) velocities in children with SCA, coupled with regular blood transfusion has resulted in a 92% reduction of relative risk for strokes. Despite this effective strategy, regular blood transfusion therapy does not seem sustainable in sub-Saharan Africa due to shortages and the risk of transfusion transmissible infections. Additionally, there is a lack of evidence-based stroke prevention strategies in young adults with SCA, either in the high-income or in low-resource settings. Based on the foregoing, the Investigators propose to determine the prevalence of neurological injury (overt stroke, transient ischemic attacks, and silent cerebral infarcts) in young adults at the transition age from 16-25 years. The Investigators will also, for the first time, assess conventional risk factors of stroke in the general population to determine whether a different prevention strategy is required to reduce the incidence of neurological injury in this high-risk population. | Sickle Cell Disease|Sickle Cell Anemia|Stroke, Ischemic|Silent Stroke|Silent Cerebral Infarct|Stroke | ALL | CHILD, ADULT | Vanderbilt University Medical Center, Nashville, Tennessee, 37232-9000, United States|Aminu Kano Teaching Hospital, Kano, Nigeria |
| Effects of HQK-1001 in Patients With Sickle Cell Disease | The purpose of this study is to evaluate the effects of HQK-1001 on Hb F in subjects with sickle cell disease. | Sickle Cell Disease|Sickle Cell Anemia|Sickle Cell Disorders|Hemoglobin S Disease|Sickling Disorder Due to Hemoglobin S | ALL | CHILD, ADULT | University of South Alabama, Mobile, Alabama, 36617-2238, United States|Children's Hospital and Research Center - Oakland, Oakland, California, 94609, United States|Children's National Hospital, Washington, District of Columbia, 20010, United States|Howard University Hospital, Washington, District of Columbia, 20060, United States|Georgia Health Sciences University, Augusta, Georgia, 30912, United States|University of Illinois at Chicago, Chicago, Illinois, 60612, United States|Tufts Medical Center, Boston, Massachusetts, 02111, United States|The Children's Hospital at Montefiore Medical Center, Bronx, New York, 10467, United States|New York Methodist Hospital, Brooklyn, New York, 11215, United States|University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, 27599, United States|Virginia Commonwealth Univeristy - Center on Health Disparities, Richmond, Virginia, 23298, United States|University Health Network Toronto General Hospital, Toronto, Ontario, M5G2C4, Canada|Abu El Reesh Pediatric University Hospital, Cairo, Egypt|Ain Sham University Hospital, Cairo, Egypt|University of the West Indies, Mona, Kingston 7, Jamaica|American University of Beirut Medical Center, Beirut, Lebanon|Chronic Care Center, Beirut, Lebanon|Rafik Hariri University Hospital, Beirut, Lebanon |
| Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease | This is a study to evaluate the safety and toxicity of a treatment regimen consisting of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic hematopoietic stem cell transplantation from a haploidentical donor in patients with sickle cell disease. The overall goal of this study is to expand the donor pool for hematopoietic stem cell transplantation in sickle cell disease using haploidentical donors, and to develop a non-toxic, myeloablative regimen, with the goal of achieving a consistent donor chimerism utilizing pre-transplant immunosuppressive therapy. | Sickle Cell Disease | ALL | CHILD, ADULT | City of Hope Medical Center, Duarte, California, 91010, United States |
| Neutrophil Extracellular Traps and Sickle Cell Disease | NETs formation in vaso-occlusive events in sickle cell disease and the role of hydroxyurea treatment.The study hypothesis is that NETs formation by neutrophils has a role in the induction of vaso occlusive disease in blood vessels. It is possible that high leukocyte count in children with sickle cell anemia is a bad prognostic sign due to NETs formation supporting occlusion of peripheral and central blood vessels.Hydroxyurea treatment might prevent vaso occlusive syndrome not only by increasing HbF but also by decreasing neutrophil count and inhibiting NETs formation. | Sickle Cell Anemia | ALL | CHILD, ADULT | |
| Pulmonary Hypertension, Hypoxia and Sickle Cell Disease | The study will look at the risk factors for pulmonary hypertension (high blood pressure in the lungs) in children and adolescents with sickle cell anemia (SCA) and examine the role of hypoxia (oxygen shortage) in the disease. In patients with SCA, red blood cells become sickle-shaped and tend to form clumps that get stuck in blood vessels, blocking blood flow to the limbs and organs. Blocked blood vessels can cause pain, serious infections, and organ damage. Many patients with SCA also develop pulmonary hypertension. Children and adolescents with SCA or Chuvash polycythemia (another blood disorder that carries an increased risk for pulmonary hypertension) may be eligible for this study. Participants undergo the following procedures at the beginning (baseline) and end of the study: * History, physical examination and blood tests . * Echocardiography (ultrasound study of heart function). * Transcranial doppler (brain ultrasound study to measure brain blood flow). * Lung function tests. * 6-minute walk (measure of the distance covered in 6 minutes of walking). In addition, patients are followed by telephone or by clinic visits every 6 months for a review of their medical history and medications. A physical examination is also done at 12 months. | Chuvash Polycythemia|Cerebrovascular Disease|Pulmonary Hypertension|Sickle Cell Anemia | ALL | CHILD, ADULT | Howard University Hospital, Washington, District of Columbia, 20060, United States|Childrens National Medical Center, Washington, District of Columbia, United States|University of Chicago, Chicago, Illinois, 60637, United States|National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States|University of Michigan, Ann Arbor, Michigan, 48109-0624, United States|Baylor College of Medicine, Houston, Texas, 77030, United States|Republic Cardiac Center in Cheboksary, Chuvashia, Russian Federation |
| Decision Aid for Therapeutic Options In Sickle Cell Disease | Sickle cell disease (SCD) is an inherited disorder with chronic multi-system manifestations affecting 100,000 individuals in the US, largely of minority origin and associated with substantial morbidity, premature mortality, individual suffering, healthcare costs and loss of productivity. Disease modifying treatments such as hydroxyurea, chronic blood transfusion and curative bone marrow transplantation are offered to patients based on physician preference and current practice informed by clinical trials. Decision aids are tools that could help translate evidence from these sources into practice by helping clinicians involve patients in making deliberate choices based on accessible information about the options available and their outcomes and to help them make decisions based on their values and preferences. The overarching goal of this project is to implement a web based decision aid individualized to patient characteristics to help patients with SCD achieve more accurate perception of risks and benefits of treatment options and make decisions in congruence with their values and preferences. Investigators will use a randomized controlled trial of the effectiveness of a web-based decision aid to give patients accurate information about risks and benefits of therapies that enable patients to make decisions based on their individual values and preferences. | Sickle Cell Disease|Sickle Cell Anemia|Hemoglobin SS|Hemoglobin SC|Hemoglobin Beta Thalassemia | ALL | CHILD, ADULT, OLDER_ADULT | Emory University, Atlanta, Georgia, 30322, United States |
| Pulmonary Ultrasound in the Diagnosis of Acute Thoracic Syndrome in Vaso-occlusive Sickle Cell Crisis | ATS (acute thoracic syndrome) refers to acute pulmonary involvement in a sickle cell patient. The diagnosis is based on the association of clinical signs (fever or respiratory symptoms) with a recent pulmonary infiltrate on the chest x-ray. The main objective of the study is to evaluate the place of the pulmonary ultrasound for the diagnosis of ATS, in comparison with frontal chest x-ray. | Sickle-Cell Disease Nos With Crisis | ALL | CHILD | Hôpital Antoine Béclère, Clamart, 92141, France|Centre Hospitalier Intercommunal Créteil, Créteil, 94000, France|Hôpital Bicêtre, Le Kremlin-Bicêtre, 94270, France|Hôpital Armand Trousseau, Paris, 75012, France |
| Plasma DNA and Vascular Remodelling in Patients With Sickle Cell Disease | The purpose of this study is to evaluate the relationship between plasma DNA levels and micro- and macro-circulatory vascular remodelling in patients with sickle cell disease | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Hôpital Avicenne, Bobigny, Ile De France, 93009, France |
| EXTEND EXpanding Treatment for Existing Neurological Disease | The primary goal of the Phase II EXTEND trial is to investigate the effects of open-label hydroxyurea treatment, escalated to maximum tolerated dose, for children with Sickle Cell Anemia and either conditional (170 - 199 cm/sec) or abnormal (≥200 cm/sec) Transcranial Doppler velocities. The primary endpoint will be measured after 18 months of hydroxyurea but treatment will continue until a common study termination date. | Sickle Cell Anemia | ALL | CHILD | Sickle Cell Unit, Kingston, Jamaica |
| Improving Emergency Department Management of Adults With Sickle Cell Disease | The objective of this study is to design, implement and test quality improvement measures to improve the care of adults with sickle cell disease in the emergency department. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Wake Forest Baptist Medical Center, Winston Salem, North Carolina, 27157, United States |
| Phase 2 Study of Montelukast for the Treatment of Sickle Cell Anemia | In this feasibility trial, the investigators will compare participants treated with montelukast and hydroxyurea to those treated with placebo and hydroxyurea for a total of 8 weeks. | Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0) | ALL | CHILD, ADULT, OLDER_ADULT | Vanderbilt University Medical Center, Nashville, Tennessee, 37232-9000, United States|Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| Nonpharmacological Approaches and Parental Education in Children With Sickle Cell Disease | Aim: Sickle cell disease (SCD) is a hereditary disease. Approximately 5% of the world's population carries trait genes for haemoglobin disorders, mainly, sickle-cell disease and thalassaemia. The incidence of sickle cell disease varies according to different geographical locations. Persons with SCD experience both acute and chronic pain. it is important that nonpharmacological therapies be investigated and used as complements to pharmacological therapies to address and treat both acute and chronic pain for those with SCD. An educational program involving parents with children with SCD can help parents use nonpharmacological methods for their children in reducing pain. The training program allows parents to develop their knowledge of the SCD and the importance of non-pharmacological methods, and may provide parents with the opportunity to develop preventive attitudes towards reducing pain crises. The research has two purposes. The first is to identify the non-pharmacological approaches that parents use to help their children with sickle cell disease in Turkey, Chad. Second, to determine the effectiveness of non-pharmacological approaches given to parents in Turkey, Chad. Method: This study is an experimental research method using a quasi-experimental design. This study used a pretest and posttest, with a design that is used is the two group pretest-posttest design. The study was conducted on the parents of children with sickle cell disease diagnosed and followed up in the Pediatric Hematology Oncology polyclinics of two hospitals in Chad in Central Africa between September 2015 and February 2016. The study found in April 2016 and July 2016 between Turkey's southern Antalya and Mersin two university hospital outpatient Children's pediatric hematology oncology has made clinic on sickle cell disease diagnosed and monitored the children's parents. The parents were selected using eligibility criteria and the study was performed in three steps (Pre-intervention testing session, education session, and post-intervention testing session). In the first step, "Information Form", "Parents' Experience of Nonpharmacological Methods Questionnaire", and "Nonpharmacological Approaches Used by Parents for Their Children and Knowledge of Parents about Nonpharmacological Approaches Questionnaire" questionnaires were applied to parents. In the second step an individual education was conducted by the researcher using the education book. In the third step, the questionnaire were reapplied after 3 weeks. | Nursing Caries | ALL | CHILD, ADULT, OLDER_ADULT | Emine EFE, Antalya, 07058, Turkey |
| Promoting Resilience Among Adolescents and Young Adults With Sickle Cell Disease | Adolescents and young adults with sickle cell disease (SCD) face challenges managing their illness and maintaining their well-being. This study proposes to test the feasibility and acceptability of a resilience-promoting intervention through a Collaborative Care Model. The primary goal is to determine with the resilience intervention (PRISM) is feasible and acceptable for adolescents and young adults with SCD. Exploratory outcomes include whether this intervention improves depression, anxiety, and pain interference. | Sickle Cell Disease | ALL | CHILD, ADULT | |
| Genetics and Pain Severity in Sickle Cell Disease | Background: - Pain is the most common symptom of sickle cell disease. Episodes of severe sickle cell pain are known as "crises." High rates of pain crises are associated with a higher risk of early death. Some people with sickle cell disease have many severe pain crises while others experience fewer crises. This difference in pain crisis may be caused by sensitivity to pain. People with high sensitivity to pain may have more pain crises. Many factors, including a person's genetic makeup, determine sensitivity to pain. Comparing genetic information from people with sickle cell disease and healthy volunteers may provide more information on pain and sickle cell disease. Objectives: - To study genetics and pain sensitivity in sickle cell disease. Eligibility: * African or African American individuals at least 18 years of age with sickle cell disease. * Healthy African or African American volunteers at least 18 years of age. Design: * Participants will be screened with a medical history and physical exam. They will also provide blood and urine samples. * Participants will have the following tests: * Quantitative sensory testing to measure sensitivity to pressure, heat, cold, and mechanical pain. * EndoPat test to measure blood vessel function and reaction. * Questionnaires about mood, evidence of depression, pain, quality of sleep, and sleep disturbances. * Measures of daily pain, whether or not related to sickle cell disease. * After the first visit, those in the study will have monthly study visits for 6 months. The above tests will be repeated at these visits. | Genotype|Pain|Genetic Variation|Quantitative Sensory Testing (QST)|GCH1 | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| Iron Overload and Growth Velocity in Thalassemia and Sickle Cell Anemia | Iron overload impaired growth in Thalassemia patients due to iron deposition in the endocrine glands, including the hypophysis and gonads. The issue of iron overload in Sickle Cell Anemia is recently studied more extensively and preliminary studies shows that endocrine damage is rarer in those patients. Growth velocity was not systematically studied in patients with Iron Overload, even in thalassemia patients in spite several studies that assess the endocrine function in those patients. In Sickle Cell Patients this issue was not studied. The purpose of this study is to assess the growth velocity in a cohort of Thalassemia Major and Intermedia patients and compare the results to another group of Sickle Cell patients, including Sickle cell thalassemia. | Thalassemia|Sickle Cell Disease | ALL | CHILD, ADULT | Pediatric Hematology Unit - Ha'Emek Medical Center, Afula, 18101, Israel |
| Autologous Bone Marrow Stem Cell Transplantation for Osteonecrosis in Sickle Cell Disease | The purpose of this study is to evaluate the safety and efficacy of autologous bone marrow stem cells in sickle cell disease patients with osteonecrosis | Avascular Necrosis of Bone|Sickle Cell Disease | ALL | ADULT | |
| Functional and Mechanistic Characterization of Limb Ulcers in Patients With Sickle Cell Disease | Sickle Cell Disease is the most frequent genetic disease in the world (representing one birth over 1900, in France). The polymerization of the abnormal hemoglobin (i.e., HbS) when deoxygenated is at the origin of a mechanical distortion of red blood cells (RBC) into a crescent-like shape. Sickled RBCs are very fragile and rigid, which lead patients to have severe anemia and to develop frequent and repeated painful vaso-occlusive crises. Furthermore, the repetition of sickling-unsickling cycles causes irreversible damages to the RBCs, which shorten their half-life. Accumulation of free hemoglobin and heme in the plasma is involved in blood vessels lesions in both the macro- and micro- circulation. The resulting vascular dysfunction could explain why limb ulcers are 10 fold more frequent in patients with sickle cell disease compared to the general population and may happen at a younger age. Limb ulcers induce significant morbidity (delay of healing between 9 and 26 weeks in the french cohort), and are associated to significant pain (needing opioid pain-killer) and increase the risk of infection. Cost of care is also increased. Moreover, ulcers induce missed school and work days. Data on cutaneous microcirculation and ulcers physiopathology in patients with sickle cell disease are scarce. We want to realise a microcirculatory and neurological functional study of patients with with and without ulcers and a characterization of biomarkers present in the blood or in the wound fluid which can participate to ulcers physiopathology. To ensure healing, adapted therapeutics are essential. Several strategies are proposed such as: lifestyle measures (venous compression, lower limb elevation, rest), dressings, hyperbaric oxygenotherapy (also used in diabetic ulcers). The project is devoted to study the mechanisms involved in leg ulcers and the effects of therapeutical/behavioral strategies. | Ulcer|Anemia|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Groupement Hospitalier Edouard Herriot, Lyon, 69437, France |
| Stigma, Self-management, & Quality of Life in SCD | Many individuals with sickle cell disease experience both a poor quality of life and stigma. Individuals with SCD often experience high levels of stigma which can be a barrier to good self-management and hinder quality of life. The purpose of this research is to improve understanding of the relationships between stigma, self-management, and quality of life in SCD in the United States and Jamaica. The findings from this project will contribute to the development of a tool to measure self-management strategies and will also guide the development of interventions to improve SCD self-management. | Sickle Cell Disease|Quality of Life|Stigma, Social|Disease Self-Management | ALL | ADULT, OLDER_ADULT | Dominique Bulgin, Durham, North Carolina, 27705, United States|Sickle Cell Unit, The University of the West Indies, Mona, Kingston 7, Jamaica |
| Pilot Study "AHSP as a Biomarker of Sickle Cell Disease in a Population of Adults and Children" | Evaluation of AHSP concentration in total blood as a biomarker in adult and pediatric sickle cell patients | in Relation to Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | |
| Apixaban in Patients With Sickle Cell Disease | In patients with SCD, the use of low dose anticoagulation as an outpatient may lead to a significant decrease in morbidity and as a result, decrease healthcare utilization and costs. This study attempts to critically avoid admissions by reducing daily pain scores and pain crisis as an outpatient by use of a novel oral anticoagulant. | Vaso-occlusive Crisis|Reduction in Hospitalizations|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Duke University Medical Center, Durham, North Carolina, 27710, United States |
| Physical Rehabilitation in Sickle Cell Anemia | Sickle cell anemia (SCA) is one of the most neglected diseases worldwide, according to the World Health Organization. In the adult population with SCA, the systemic effects of the disease, such as respiratory and peripheral muscle dysfunction, cause a decrease in quality of life. As a consequence, there is a concern about functional rehabilitation, since the aging of this population is already a reality in our environment. Thus, the objective of this project is to evaluate the effects of functional rehabilitation on quality of life in adult patients over 18 years of SCA. In this longitudinal intervention study, patients will be submitted to a three-month rehabilitation program. Before and after the intervention, patients will be submitted to the following assessments: spirometry; quality of life questionnaire - Medical Outcomes Study 36-Item Short-Form Health Survey (SF-36); functional scale of joint integrity - Lower Extremity Functional Scale (LEFS); fatigue assessment scale - Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F); physical activity assessment questionnaire - International Physical Activity Questionaire (IPAQ); peripheral muscle assessment (handgrip and isometric dynamometry of the quadriceps muscle); and 6-minute walk test (6MWT). The protocol will consist of warm-up and cool-down exercises, muscle strengthening and endurance exercises, aerobic training, balance training and proprioception. Thus, it is expected that patients with sickle cell anemia will benefit significantly, with a consequent improvement in musculoskeletal function, pain and health-related quality of life. | Sickle Cell Anemia|Muscle Dysfunction|Quality of Life|Physical Activity|Exercise | ALL | ADULT | Agnaldo José Lopes, Rio de Janeiro, 22745-271, Brazil |
| Examining Cognitive Function and Brain Abnormalities in Adults With Sickle Cell Disease | Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause intense pain episodes and may lead to organ failure. Preliminary studies have shown that adults with SCD may have brain abnormalities that contribute to problems with cognitive functioning, including attention and memory difficulties. This study will use brain magnetic resonance imaging (MRI) and neuropsychological testing to examine the differences in cognitive functioning in adults with SCD and adults without SCD. 212 subjects participated in this cross-sectional study consisting of screening questionnaires, a neuropsychological testing battery, and MRI testing. Enrollment into this study ended in May 2008. | Anemia, Sickle Cell | ALL | ADULT | University of Southern California, Los Angeles, California, 90033, United States|Children's Hospital & Research Center at Oakland, Oakland, California, 94609, United States|Memorial Cancer Institute, Hollywood, Florida, 33021, United States|University of Miami Miller School of Medicine, Miami, Florida, 33136, United States|Medical College of Georgia, Augusta, Georgia, 30912, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, 27599, United States|Duke University Medical Center, Durham, North Carolina, 27705, United States|Cincinnati Children's Hospital, Cincinnati, Ohio, 45229, United States|University of Cincinnati Medical Center, Cincinnati, Ohio, 45267, United States|Children's Medical Center at Dallas, Dallas, Texas, 75390, United States|University of Texas Medical Branch, Galveston, Texas, 77555, United States |
| Assessment of Opioid Analgesia in Sickle Cell | To develop and validate a non-invasive, in vivo, phenotyping method for CYP2D6 using the non-injurious neuroselective electrical stimulation technique: pain perception threshold/pain tolerance threshold (PPT/PTT) in children and adolescents with sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's National Medical Center, Washington, District of Columbia, 20010, United States |
| Integrative Medicine in Pain Management in Sickle Cell Disease | The proposed research is to determine the clinical efficacy and neurobiological mechanisms of acupuncture analgesia in patients with sickle cell disease. | Sickle Cell Disease|Pain|Acupuncture|Quantitative Sensory Testing|Magnetic Resonance Imaging|Circulating Biomarkers|Electroencephalography|Functional Near-infrared Spectroscopy | ALL | CHILD, ADULT, OLDER_ADULT | Indiana University School of Medicine, Indianapolis, Indiana, 46075, United States |
| Prevention of Cerebral Infarction in Sickle Cell Anemia - Comprehensive Sickle Cell Center | To conduct a prospective study aimed at the early detection and treatment of cerebral vascular disease prior to irreversible brain injury in young children with sickle cell anemia (SCA). | Anemia, Sickle Cell|Blood Disease|Cerebrovascular Disorders|Cerebrovascular Accident | MALE | CHILD, ADULT, OLDER_ADULT | |
| Study of Intravenous GMI-1070 in Adults With Sickle Cell Disease | This Phase 1/Phase 2 study will evaluate GMI-1070, a pan-selectin inhibitor, in adults with stable sickle cell disease. The study will assess safety, pharmacokinetics, and microvascular effects of intravenous GMI-1070 in the outpatient setting. | Sickle Cell Disease | ALL | ADULT | Children's Hospital & Research Center Oakland, Oakland, California, 94609, United States|University of California at Davis, CCRC, Sacramento, California, 95817, United States|Duke Comprehensive Sickle Cell Center, Durham, North Carolina, 27710, United States |
| Mesenchymal Stromal Cells for Haplo Hematopoietic Cell Transplantation for Sickle Cell Disease | This trial is being conducted as a step toward testing the long-term hypothesis that freshly cultured, autologous mesenchymal stromal cells (MSCs) grown in platelet lysate-containing medium will modulate recipient T-cell immune responses and promote engraftment in haploidentical hematopoietic cell transplant (HCT) recipients. As a phase I, dose escalation trial of autologous MSCs in patients with sickle cell disease (SCD) undergoing haploidentical HCT, the main aim is to evaluate the safety of this therapy with a secondary aim to evaluate its effects on engraftment and graft-versus-host disease (GVHD). | Sickle Cell Disease | ALL | CHILD, ADULT | |
| Pilot Study on the Value of Bedside Pleuropulmonary Ultrasound in Patients With Sickle Cell Disease Presenting With Vaso-occlusive Crisis | Describe the proportion of patients with major sickle cell syndrome in vaso-occlusive crisis presenting at least one pleuropulmonary ultrasound abnormality during one of the ultrasounds performed at D0, between D2 and D5 during hospitalization and at D-1 of discharge. We hypothesize that pleuropulmonary ultrasound will make it possible to detect the serious complications associated with vaso-occlusive crises in patients with major sickle cell syndrome earlier and more reliably, in departments where ultrasound tools are still underdeveloped, while avoiding the need for more conventional radiology examinations that cause radiation in multi-hospitalized patients. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | |
| Study of a Red Blood Cell Deformability Parameter in Patients With Sickle Cell Disease | Sickle cell disease is the most common genetic disease in the world. It results in the synthesis of an abnormal hemoglobin (HbS) which, in its deoxygenated form, polymerizes and causes structural changes in red blood cells (RBCs). They become more rigid and less deformable. The fragility of sickle-cell RBCs leads to their massive destruction, leading to chronic anemia (i.e. low hemoglobin in the blood) and to low tissue oxygenation. More rigid and less deformable, sickle-shaped RBCs tend to obstruct microvessels, leading to particularly painful vaso-occlusive crisis (VOC), which can cause organ failure (spleen, kidneys, brain, lungs, heart, liver, bone ...) and patient's lifethreatning. A preliminary work on red blood cells of sickle cell patients showed alteration of a parameter measuring the overall deformability of RBCs by assessing the nature of their movement in a shear flow. This parameter is altered sickle cell patients at basal state compared to a population of healthy individuals. This alteration is increased when sickle cell patients are in crisis. The main objective of this project is to study the evolution of this parameter in sickle cell patients according to their health status (basal state vs vaso-occlusive crisis). The investigators hypothesize that the alteration of the RBC deformability parameter is significant before symptoms of vaso-occlusive crisis (several hours to several days). The main objective is a weekly analysis of the evolution of the parameter in 30 sickle cell patients (SS or SB°) in the basal state and daily in at least 6 patients at the beginning, during and just after a vaso-occlusive crisis. The comparison between the parameter measured in a subject in the basal state and in the same subject in crisis will be performed. The criteria for the presence of a vaso-occlusive crisis were: the appearance of a attacks of pain affecting at least two territories +/- fever\> 38.3 +/- dyspnea and / or sputum. The investigators will differentiate the moderate VOC managed at home with low-level analgesics and VOC requiring hospitalization. The number of days of hospitalization, the occurrence of thrombotic complications, the degree of anemia and hemolysis will be noted. The measurement of the parameter will be performed on a capillary sampling of 40 microliters performed at the fingertip, weekly outside crises and daily when a crisis occurs. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Assistance Publique Hôpitaux de Marseille, Marseille, 13005, France |
| Mindfulness and Yoga Therapy for Acute Pain in Sickle Cell Disease | Patients with sickle cell disease suffer from acute and chronic pain that diminishes their quality of life. The purpose of this study is to assess the feasibility and acceptability of mindfulness meditation, breathing exercises, and gentle yoga therapy as supportive measures for the management of acute vaso-occlusive pain crises in the inpatient setting. | Sickle Cell Crisis|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of Texas Southwestern Medical Center, Dallas, Texas, 75390, United States |
| New Hemolysis Parameters in Sickle Cell Disease | The 6-minute walk (6MWT) test is used in adults and children affected by a wide range of chronic diseases to evaluate their sub-maximal exercise capacity. It reflects the global response of various physiological systems (respiratory, cardio-vascular, neurologic, metabolic and musculosquelettic) in a situation simulating a daily life activity. In children with sickle cell disease, the 6MWT is correlated with a low level of hemoglobin, a low level of fetal hemoglobin and low red cell deformability. Our team previously reported that in a population of children with sickle cell disease, highly treated with hydroxyurea, the sole factor which was independently linked to the 6MWT was the presence of silence infarct. As the cardio-vascular and cerebro-vascular injury in sickle cell disease are directly correlated with hemolysis, the investigators aim to evaluate a) the clinical relevance of endothelial and inflammation parameters and new hemolysis markers and b) if the presence of silent infarct and the 6MWT are correlated with this biological markers. This cross-sectional study will include sickle cell disease patients regularly followed for more than 5 years at Hôpital Universitaire des Enfants Reine Fabiola, Centre Hospitalier Universitaire (CHU)-Brugmann, Centre Hospitalier Etterbeek-Ixelles, CHU Saint-Pierre, Cliniques Universitaires Saint-Luc (Bruxelles, Belgium). Inclusion criteria are: sickle cell disease (SS, Sbeta°, SC, Sbeta+), age range : 6 to 25 years, signed informed consent. Exclusion criteria are: transplanted patients, inability to perform the 6MWT (severe cognitive disability, femoral osteonecrosis with functional impairment), hospitalization and/ or transfusion in the last 3 months for acute event. Demographic data and clinical data will be retrospectively recorded. Blood test and 6MWT will be performed in steady state. Studied analysis will be: coagulation factors, free hemoglobin, Pro-B type natriuretic peptide (Pro-BNP), High sensitivity C reactive protein (HS-CRP), Intercellular Adhesion Molecule (ICAM), Vascular Cell Adhesion Molecule (VCAM) and Selectins. With this study, the investigators expect to validate new predictive markers for cardio-vascular or cerebrovascular injury and to identify patients at high risk to develop these complications. | Sickle Cell Disease | ALL | CHILD, ADULT | |
| Evaluation of Knowledge Among Adolescents With Sickle Cell Disease. | Evaluation of knowledge about contraception in sickle cell adolescents. | Sickle Cell Disease | ALL | CHILD, ADULT | Centre Hospitalier Intercommunal de Créteil, Créteil, Creteil, 77500, France |
| Adherence to Hydroxyurea in Children With Sickle Cell Anemia | Medication non-adherence is a true public health problem. Despite advancements in the molecular understanding of disease and improvements in therapy, patient health outcomes will not improve unless patients take prescribed medications regularly. Decreasing the gap between efficacious and effective therapy for patients with SCD is an essential research agenda. Hydroxyurea has been shown to be safe and efficacious in children and infants. However, the effectiveness of the prophylaxis depends on adherence to the recommended regimen. Medication adherence in SCD has previously been found to be sub-optimal in patients taking penicillin, desferoxamine, and pain medication. Adherence to HU has been studied to some extent in children with SCD. Based on estimates of adherence in other chronic illness we expect approximately 50% of patients to be \>80% adherent with their HU administration. There is no gold standard for improving adherence to treatment. There have been a few attempts in the SCD population to improve adherence. These include a day camp to promote education about desferoxamine and peer support, a combination of a slide-show about SCD and it complications, weekly phone calls by the clinic social worker and a calendar, and a seven-phase educational program. Given the striking improvements in the peripheral blood smear findings of patients with SCD on HU therapy, with reduction in the numbers of sickled cells, we hypothesize that viewing the peripheral blood smear of patients with poor adherence to HU compared to a blood smear of someone on HU can be used to improve adherence in non-adherent patients. We will conduct a randomized trial between the intervention of regularly showing children and their parents the peripheral blood smear and standard care, including reminders of the importance of compliance and review of complete blood count parameters, including WBC, MCV, and Hgb concentration. The outcome measures will be increase in hemoglobin concentration and %HbF and increase in perceived QOL. QOL will be measured with age-appropriate and parent/proxy PedsQL™. Medication adherence will also be monitored throughout the study with pharmacy prescription refills, physician assessment, and self-report via a visual analogue scale. Adherence estimates, hemoglobin concentration, %HbF and QOL will be measured at baseline, 3 months and 6 months. | Sickle Cell Anemia | ALL | CHILD | Duke University Medical Center, Durham, North Carolina, 27710, United States |
| Acute Kidney Injury in Patients With Sickle Cell Disease | Patients with sickle cell disease may be at risk for acute kidney injury (AKI)during sickle cell crisis (pain or acute chest syndrome). This study will evaluate the role of hemolysis during SCD crisis on the development of AKI and the role for monitoring urine biomarkers during an admission for crisis and during well clinic follow-up. | Sickle Cell Disease|Kidney Injury|Kidney Diseases|Kidney Disease, Chronic | ALL | CHILD, ADULT | University of Alabama at Birmingham, Birmingham, Alabama, 35223, United States |
| Recruitment and Engagement in Care to Impact Practice Enhancement (RECIPE) for Sickle Cell Disease | The goal of this observational study is to help us understand more about the best ways to help individuals living with Sickle Cell Disease (SCD) get the best care. The main question it aims to answer is: How to find individuals unaffiliated from SCD specialist care use three distinct pathways? Once unaffiliated individuals are found using the pathways, Investigators will employ linkage coordinators (trained staff) to engage these patients in care. Participants will be asked to fill out an assessment survey which will cover areas such as previous and current treatment, clinic and hospital experience, pain, and quality of life. Participants will also be given the option of participation in a 1-hour long interview how they feel about treatment for sickle cell disease including clinic experience, pain, and quality of life? | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of Alabama, Birmingham (UAB), Birmingham, Alabama, 35233, United States|University of California, San Francisco (UCSF), Oakland, California, 94609, United States|Augusta University, Augusta, Georgia, 30901, United States|University of Illinois, Chicago, Illinois, 60612, United States |
| Safety Study of MP4CO in Adult Sickle Cell Patients | Sickle Cell Anemia is caused by an inherited hemoglobin disorder. Healthy red blood cells are discoid and can deform and move through small blood vessels to carry oxygen to all parts of the body. In sickle cell disease, as red blood cells circulate and oxygen is released in the circulatory system, the deoxygenated abnormal hemoglobin S can begin to polymerize. When this occurs, the red blood cells can become sticky and elongated. These sickled red blood cells are less flexible and will obstruct small blood vessels and block normal red blood cells from traveling through the circulatory system, which limits oxygen delivery to tissues and organs. This is known as a "sickle crisis". Patients suffering from a sickle crisis experience severe pain and are at risk of stroke, heart attack or even death. By lowering the level of oxygen pressure at which sickling occurs and opening the vasculature and rapidly delivering oxygen directly to ischemic tissues, the addition of MP4CO to existing treatment protocols may alleviate pain associated with a sickle cell crisis, abort a crisis and/or potentially reduce the duration of a crisis. This could mean less time in the hospital and an improved quality of life for patients with sickle cell anemia. | Anemia, Sickle Cell|Sickle Cell Anemia|Sickle Cell Disease|Sickle Cell Disorders|Hemoglobin SC Disease|Sickle Cell Hemoglobin C Disease | ALL | ADULT, OLDER_ADULT | Hôpital Henri Mondor, Creteil, France|Sickle Cell Unit, University of West Indies, Kingston, Jamaica|Rafic Hariri University Hospital, Beirut, Lebanon|Guy's Hospital, London, United Kingdom|King's College London, London, United Kingdom |
| Losartan to Reverse Sickle Nephropathy | Sickle cell disease causes kidney damage with increasing age, leading to chronic kidney disease and renal failure in nearly one third of patients with sickle cell disease. Currently, there is no treatment for sickle cell related kidney disease. | Nephropathy|Sickle Cell Anemia | ALL | CHILD, ADULT, OLDER_ADULT | University of Illinois at Chicago, Chicago, Illinois, 60612, United States|University of Louisville, Louisville, Kentucky, 40201, United States|NHLBI, Bethesda, Maryland, 20892, United States|Akron Children's Hospital, Akron, Ohio, 44308, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States|University of Cincinnati, Cincinnati, Ohio, 45229, United States|Nationwide Children's Hospital, Columbus, Ohio, 43205, United States|University of Texas Southwestern, Dallas, Texas, 75390, United States|Texas Children's Hospital, Houston, Texas, 77030, United States |
| Ph I/II Study of Allogeneic SCT for Clinically Aggressive Sickle Cell Disease (SCD) | The investigators propose to determine the engraftment and transplant related morbidity and mortality after a non-myeloablative allogeneic hematopoietic stem cell transplant protocol using immune- suppressive agents and low-dose total body irradiation (TBI) without standard chemotherapy in patients with aggressive sickle cell disease who are not candidates for or experienced complications from hydroxyurea therapy. Fully HLA matched siblings will be used as donors for hematopoietic stem cells to reduce the risk of morbidity and mortality in this cohort of patients. | Sickle Cell Disease | ALL | CHILD, ADULT | University of Illinois at Chicago, Chicago, Illinois, 60612, United States |
| Clinical Importance of Treating Iron Overload in Sickle Cell Disease | Hypothesis: The investigators suspect that significant degrees of iron overload in subjects with SCD result in decreased red cell survival, abnormal endothelial function and markedly dysregulated autonomic function. Furthermore, the investigators anticipate that the magnitude of these effects is proportional not only to the magnitude of total body iron stores but also to the duration of exposure to the high iron levels in tissues. Primary objective To determine if red cell survival as assessed by 51Cr red cell survival analysis, hemoglobin level, reticulocyte count, lactic acid dehydrogenase, and plasma hemoglobin in sickle cell patients is related to the degree of iron overload. Secondary objective(s) 1. Determine if the magnitude of endothelial-dependant vasodilation is related to The degree of iron overload. 2. Determine if the degree of change in cardiac beat to beat variability in response to hypoxic exposure or to cold exposure ("cold-face-test") is related the magnitude of iron overload. The primary measure of iron overload will be MRI determination of liver iron concentration. | Anemia, Sickle Cell|Transfusion Hemosiderosis | ALL | CHILD, ADULT, OLDER_ADULT | Childrens Hospital Los Angeles, Los Angeles, California, 90027, United States |
| Zinc for Infection Prevention in Sickle Cell Anemia-2 (ZIPS-2) | A randomized double-blinded placebo-controlled trial of zinc to reduce the incidence of severe or invasive infections in Ugandan children with sickle cell anemia (SCA) | Sickle Cell Disease | ALL | CHILD | |
| Spectralis HRA+OCT Imaging of the Retina With Autofluorescence in Sickle Cell Disease | To determine the retinal and choroidal thickness in patients with sickle cell disease compared to age, race matched population without sickle cell disease to allow a better understanding of the clinical manifestations of sickle cell retinopathy. The purpose of this research study is to evaluate the relationship between sickle cell disease and the eye. The research study is recruiting African American population with or without Sickle Cell Disease. The investigator in charge of this study is Dr E. Bowie. Approximately 60 subjects of both sexes will be enrolled at the Medical University of South Carolina. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Medical University of South Carolina, Storm Eye Institute, Charleston, South Carolina, 29425, United States|MUSC Storm Eye Institute, Charleston, South Carolina, 29425, United States |
| Trial of Oral Glutamine in Patients With Sickle Cell Anemia | Children with sickle cell anemia (SCA) seem to have higher energy needs than children who do not have the disease. This may be the reason why children and teenagers with sickle cell anemia tend to be smaller, weigh less, and have less fat and muscle than children and teens that do not have the disease. This study is being done to find out if giving a supplement called glutamine will help children with sickle cell anemia by lowering their energy needs and improving their growth and strength. Children will be randomly assigned (like a flip of a coin) to one of two groups. One group will take glutamine and one group will take a placebo (a protein mixture that looks like glutamine but may not have the same effect in the body). No one will know which group is taking which supplement until the study has been completed. Children will be in the study for 12 months. | Anemia, Sickle Cell | ALL | CHILD, ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Low Dose Iron Chelation as TReatment of Oxidative Damage in Sickle Cell Disease | Objective: To study the safety and efficacy of deferasirox as treatment of oxidative stress in adult subjects with sickle cell disease. Endpoints: The investigators will determine whether treatment with iron chelators results in decreased sickling of RBCs, oxidative stress, neutrophil activation, inflammation, endothelial activation and hypercoagulability and ultimately reduced disease severity. If the hypothesis is confirmed in this pilot dose-finding study, a larger randomized controlled clinical trial will be initiated. Study design: This will be an open-label pilot study, including 12 patients per dose group with a maximum of 3 dose groups. As the antioxidant capacity of deferasirox might be dose-dependent, the investigators will start with the highest dose of deferasirox (360 mg) deemed adequate for chronic use without causing iron depletion in adult SCD patients. Study population: Adult patients with sickle cell anemia (HbSS) or HbS-β0-thalassemia (HbSβ0-thal) visiting the outpatient-clinic of the Academic Medical Center, Amsterdam will be asked for inclusion in the study. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Amsterdan UMC location AMC, Amsterdam, Meibergdreef 9, 1105 AZ, Netherlands |
| Acupuncture for Adults With Sickle Cell Disease (SCD): A Feasibility Study | The investigators long-term goal is to demonstrate the effectiveness of acupuncture for the treatment of adults with chronic pain due to sickle cell disease (SCD), a debilitating pain syndrome characterized by acute and chronic pain. The objective of this study is to explore the feasibility and acceptability of acupuncture with adult patients with SCD. All participants will receive acupuncture treatments twice per week for 5 weeks. Subjects will complete measures at baseline and post-treatment, and a measure of study acceptability at post-treatment. The investigators will describe the procedures and potential challenges to implementing the acupuncture protocol, and expect to identify and rectify any procedural problems that subjects report regarding the 10-session study protocol. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of Illinois at Chicago College of Nursing, Chicago, Illinois, 60612, United States |
| Safety and Efficacy of Orally Administered NUV001 Nutraceutical Supplement in Sickle Cell Disease Patients | A total of 170 patients male or female who are carrying SS or Sbeta0 versions of the beta globin gene will be included in the study. The subjects will be assigned with 1:1:1 ratio of either NUV001 Immediate release IR or NUV001 Gastro resistant GR or Placebo. The treatment duration of the study will be 90 days which has in total 5 visits. The primary end point of this study is to check the safety and tolerance of the orally administered nutraceutical supplement. This endpoint will be checked by assessing the Adverse events, Vital signs of the subject and the Change in hematological parameters from Baseline to Final visit. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Aman Hospital and Research Center, Vadodara, Gujarat, Vadodara-390021, India|Kingsway Hospital, Nagpur, Maharashtra, Nagpur-440001, India|Sai Krupa Hospital & Research Centre, Ahmedabad, India|Thalassemia & Sickle Cell Society, Hyderabad, India|Index Medical College, Indore, India|NRSMC Hospital, Kolkata, India|Arihant Hospital, Nagpur, India|Shalinitai Meghe Hospital & Research Centre, Nagpur, India |
| CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease | This is a phase 1 pilot study of CSL200 in adult subjects with severe sickle cell disease. The primary objectives of this study are to evaluate the safety of the following: collection of CD34+ hematopoietic stem / progenitor cells by apheresis after mobilization with plerixafor, reduced intensity conditioning with melphalan, and administration of CSL200. | Anemia, Sickle Cell | ALL | ADULT | City of Hope Medical Center, Duarte, California, 91010, United States |
| Nasopharyngeal Bacterial Carriage and Antibiotic Resistance in Children With Sickle Cell Disease in Ile-De-France | The objective of this study is to to determine the rate of nasopharyngeal carriage of Streptococcus pneumoniae (Sp) in children with sickle cell disease over 6 months and under 15 years of age over a 9-month period in Ile-De-France. | Sickle Cell Disease | ALL | CHILD | |
| A Study of IMR-687 in Adult Participants With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia) | Study of IMR-687 in adult participants with sickle cell anemia (SCA) (homozygous HbSS or sickle-β0 thalassemia). | Sickle Cell Disease | ALL | ADULT | UCSF Benioff Children's Hospital Oakland, Oakland, California, 94609, United States|University of Connecticut Health Center, Farmington, Connecticut, 06030, United States|Foundation for Sickle Cell Disease Research, Hollywood, Florida, 33021, United States|University of Illinois, Chicago, Illinois, 60612, United States|Loretto Hospital, Chicago, Illinois, 60644, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|Baylor Scott & White Health, Temple, Texas, 76508, United States|Sandwell & West Birmingham Hospital, Birmingham, B18 7QH, United Kingdom|Bristol Haematology and Oncology Centre, Bristol, BS2 8ED, United Kingdom|Royal London Hospital, London, E1 1BB, United Kingdom|University College London Hospital, London, NW1 2PG, United Kingdom|Guy's Hospital, London, SE1 9RT, United Kingdom|Oxford Cancer & Haematology Centre, The Churchill Hospital, Oxford, OX3 7LE, United Kingdom |
| Vitamin D Supplementation in Children With Sickle Cell Disease | Sickle cell disease (SCD) is a genetic disease characterized by abnormal hemoglobin, the main constituent of red blood cells. People with SCD have nutritional deficiencies, and vitamin D deficiency is one of the most common. Symptoms of vitamin D deficiency are similar to those of SCD and include chronic pain and bone complications. Correcting vitamin D nutrition of children with SCD represents a treatment that will improve their health. A single oral high-dose of vitamin D3 will be given to SCD children during one of their follow-up visits at the SCD clinic of CHU Sainte-Justine, Montreal, Canada. This mode of administration was chosen to ensure a better adherence to the treatment. The investigators will determine whether this dose is safe and its administration feasible in clinic. The impact of this dose on blood vitamin D and calcium, urinary calcium, growth, inflammation, bone health, pain and quality of life will also be assessed. This study intends to propose a new intervention to improve the nutrition of children with this disease. | Sickle Cell Disease | ALL | CHILD | CHU Sainte-Justine, Montreal, Quebec, H3T 1C5, Canada |
| Exploration of the Parameters Influencing the Effort Limitation of Patients Suffering From Homozygous Sickle Cell Anemia | The cardiovascular complications of sickle cell disease are associated with major morbidity and mortality, as well as impaired quality of life. Stratifying the cardiovascular risk of these patients solely on resting parameters (blood pressure, cardiac ultrasound) is limited. The hypothesis that an exhaustive analysis of the physiological parameters of exercise is launched allows more relevant phenotyping and therefore much better stratification of the individual risk of these patients.. Recent studies have shown a paradigm shift in the use of physical activity. Hitherto inadvisable (acidosis, vaso-occlusive crises), if adapted, moderate and regular, it can lead to functional improvement and a reduction in crises. As a result, cardiovascular exercise rehabilitation is becoming increasingly popular in this population. It's easy to see how effort assessment, particularly through cardiorespiratory exchanges, will become more common in this pathology, making its analysis accessible and interesting. | Analyze the Results of Cardiorespiratory Stress Tests|Resting and Stress Echocardiography|Capillary Lactatemia Measurements at the Ear | ALL | ADULT, OLDER_ADULT | Assistance Publique Hôpitaux de Paris-Hôpital Henri Mondor, Créteil, Paris, 94010, France |
| Genetic and Haematological Modifiers of SCD Severity in Kaduna State, Northern Nigeria | This study is aimed to assess the genetic and haematological modifiers of disease severity among patients with Sickle Cell Disease (SCD) in Kaduna State, northern Nigeria. It is composed by two separate study designs: a cross-sectional study and a longitudinal study. The cross-sectional study will evaluate clinical and laboratory parameters in paediatric Sickle Cell Anaemia (SCA) patients (ages 2-18 years) in steady state and during Vaso-Occlusive Crisis (VOCs) to determine the parameters that can be used as a guide to monitor the course of the disease towards early recognition and management of sickle cell crises. In addition, the study will explore genotype-phenotype correlations in SCA patients by targeted Next-Generation Sequencing (NGS) of genetic modifiers for haemoglobinopathies. The longitudinal study will collect clinical and laboratory data over time for a paediatric cohort of SCD patients (9 months old; followed up to 2 years of age) and parental samples will be collected to determine the βS-globin haplotype in family trios. The aim is to determine the temporal relationships among foetal haemoglobin (HbF) levels, haematological parameters and frequency of sickle cell crises in SCD patients in relation to the type of the βS-globin haplotype and the sickle genotype. In addition, samples collected at 24 months of age will also be analysed by NGS to identify genetic modifiers of clinical manifestations and severity of SCA. Participants from the following centre will be involved: Ahmadu Bello University Teaching Hospital (ABUTH) Zaria. Consent from all the study parents/legally designated representatives as well as assent from minors will be sought. Consent for genetic analyses will be sought as well. Clinical and haematological analyses will be performed at ABUTH while genetic analyses will be performed at the Cyprus Institute of Neurology and Genetics (CING). | Sickle Cell Disease | ALL | CHILD, ADULT | |
| High Dose Vitamin D for Sickle Cell Disease | Vitamin D deficiency (VDD) is very common among African American adolescents and adults in the US, ten times higher than is seen in Caucasians. VDD is also quite common in sickle cell disease (SCD). Both VDD and SCD can cause chronic pain, compression fractures, and muscle weakness. The investigators believe VDD may contribute to poor musculoskeletal health and chronic pain seen in pediatric SCD. In this study, the investigators aim to show that children and adolescents with SCD and chronic pain have lower levels of vitamin D compared to those without chronic pain. The investigators also aim to determine the clinical characteristics in SCD patients related to their vitamin D status. About 60 subjects (7 to 21 years old) will be enrolled on this study, 30 with chronic pain and 30 without chronic pain. The investigators will assess baseline characteristics including vitamin D levels, bone turnover rates (measured by C telopeptide blood levels \[CTx\]), markers of inflammation and oxidative stress levels in blood, baseline hemoglobin and other laboratory parameters, presence of abnormal bones on chest x-ray, pulmonary function, opioid analgesic use, overall muscle strength, quality of life and depression. To evaluate the impact of vitamin D replacement on these baseline characteristics, the investigators will randomize subjects to receive either placebo or high dose vitamin D for 6 weeks after which time the investigators will evaluate overall vitamin D status, muscle and bone health, depression, quality of life, pain status and use of opioid pain medications, inflammation and oxidative status comparing before and after treatment with high dose vitamin D. The investigators will give-at no cost to subjects-a daily supplement that will provide the recommended daily allowance of calcium and vitamin D that contains 500mg Calcium and 200IU vitamin D to subjects throughout the study period. Subjects will be in the study for 7 months and have five to six study visits. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States |
| Sickle Cell Pro-Inflammatory Response to Interval Training Study | Recommendations for exercise prescription currently do not exist for individuals with sickle cell anemia (SCA) despite the known impact that SCA-related complications has on physical functioning and fitness. A major barrier to increasing physical activity in SCA is the concern that the well-described inflammatory effects of exercise could precipitate or exacerbate complications such as vaso-occlusive pain or airway bronchoconstriction (i.e. exercise-induced asthma). Although the investigator's preliminary data suggest that increasing physical activity may be beneficial rather than harmful in children with SCA, the pro-inflammatory effects associated with repeated bouts of moderate to vigorous exercise remain poorly understood in this population. The long term goal is to address the safety and health impact of regular exercise in children with SCA. This proposal would help establish the safety of moderate to vigorous intensity exercise in children with SCA and importantly, will inform the design of future clinical trials focused on exercise training as a transformative strategy to improve fitness and overall well-being in this population. | Sickle Cell Disease | ALL | CHILD, ADULT | University of Illinois at Chicago, Chicago, Illinois, 60608, United States|Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease | This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). The study will evaluate the hematopoietic stem cell transplantation (HSCT) using CRISPR/Cas9 edited red blood cells (known as CRISPR_SCD001 Drug Product). | Sickle Cell Disease | ALL | CHILD, ADULT | University of California, Los Angeles, Los Angeles, California, 90095, United States|UCSF Benioff Children's Hospital, Oakland, California, 94609, United States |
| Study of Blood Platelets in Sickle Cell Disease | We are studying if sickle cell disease blood platelets are larger than normal and how they may cause obstruction of blood vessels. We are also trying to study the reasons why large platelets are found in patients with sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Rockefeller University Hospital, New York, New York, 10021, United States |
| Folic Acid Supplementation in Children With Sickle Cell Disease | Folic acid supplementation (1mg/d) is the standard recommendation for Canadian children with Sickle cell disease (SCD), even though it can provide up to six times the recommended intake amount for healthy children. There is growing concern that too much folic acid can be detrimental to health as high folate levels and circulating unmetabolized folic acid (UMFA), which occurs in blood with doses of folic acid as low as 0.2mg/d, have been associated with accelerated growth of some pre-cancerous cells, and altered DNA methylation and gene expression. To inform the efficacy and potential harm of high-dose folic acid supplementation in Canadian children with SCD, a double-blind randomized controlled cross-over trial is proposed. Children with SCD (n=36, aged 2-19 y) will be recruited from BC Children's Hospital and randomized to initially receive 1 mg/d folic acid or a placebo for 12-weeks (wk). After a 12-wk washout period, treatments will be reversed. | Anemia, Sickle Cell | ALL | CHILD, ADULT | BC Children's Hospital, Vancouver, British Columbia, V6H 3N1, Canada |
| Interest of Famotidine in Children With Sickle Cell Disease | The purpose of this study is to determine whether oral famotidine, a histamine type 2 receptor antagonist already widely used with very few side effects in other indications in children, is effective in reducing endothelial expression of P-selectin in children with sickle cell disease (SCD). This pilot study will constitute the essential prerequisite for a randomized clinical trial comparing the efficacy of famotidine with that of placebo in the prevention of vaso-occlusive crises in SCD patients. | Sickle Cell Disease | ALL | CHILD | Necker - Enfants malades Hospital; Department of Pediatrics and Infectious Diseases, Paris, 75015, France |
| Pilot Study of Fructose for Sickle Cell Crisis | OBJECTIVES: I. Evaluate the efficacy and tolerability of fructose administered every 6 hours for up to 72 hours to patients in active sickle cell crisis. II. Obtain tolerability information in selected patients treated with fructose for more than 72 hours. | Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | |
| Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease | Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen; this in turn can injure organs including the heart, lungs, and kidneys. SCD can lead to serious illness and death. Treatments such as bone marrow transplants and gene therapies can cure SCD, but they are not widely available. Current drug treatments for SCD are not always effective. This natural history study will examine how a study drug (mitapivat) affects red blood cells in people with SCD. Objective: To learn how mitapivat affects red blood cells in people with SCD. Eligibility: People with SCD who are enrolled in the parent study, NIH protocol IRB001565-H. Design: Procedures for this study will be done during visits already scheduled for the parent study. Participants will have additional blood drawn during study visits. The additional amount will be about 3.5 teaspoons. Participants will undergo a test called near infrared spectroscopy (NIRS) up to 9 times. Probes will be placed on their skin. A blood pressure cuff will be placed on their arm. The cuff will be filled with air for up to 5 minutes and then released. Participants may be asked to breathe at a certain rate or to hold their breath during these measurements. NIRS measures oxygen levels, blood flow, and the makeup of skin and muscle. Researchers will draw additional information for this study from participants medical records. | Sickle Cell Anemia|Sickle Cell Thalassemia|Sickle Cell Pain|Hbss|Hbsc|Sickle Beta Thalassemia|Sickle Beta Zero Thalassemia|Sickle Cell Syndrome Variant | ALL | CHILD, ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Zinc for Infection Prevention in Sickle Cell Anemia (ZIPS) | A randomized double-blinded placebo-controlled trial of zinc to reduce the incidence of severe or invasive infections in Ugandan children with sickle cell anemia (SCA). | Sickle Cell Disease | ALL | CHILD | Jinja Reginal Referral Hospital, Jinja, Uganda |
| Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of FTX-6058 | This is a study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of FTX-6058 in participants with sickle cell disease. | Sickle Cell Disease|Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | University of Arkansas for Medical Sciences (UAMS), Little Rock, Arkansas, 72205, United States|University of California, Los Angeles, Los Angeles, California, 90095, United States|Foundation for Sickle Cell Disease Research, LLC, Hollywood, Florida, 33021, United States|University of Miami Health System, Miami, Florida, 33136, United States|Visionaries Clinical Research, Atlanta, Georgia, 30329, United States|Atlanta Center for Medical Research, Atlanta, Georgia, 30331, United States|Augusta University, Augusta, Georgia, 30912, United States|Our Lady of the Lake Hospital, Baton Rouge, Louisiana, 70808, United States|Axon Clinical Research Institute, Baltimore, Maryland, 21237, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|Mississippi Center for Advanced Medicine, Madison, Mississippi, 39110, United States|Jacobi Medical Center, Bronx, New York, 10461, United States|Queens Hospital Cancer Center, Jamaica, New York, 11432, United States|University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, 27599, United States|Lynn Health Science Institute, Oklahoma City, Oklahoma, 73112, United States|University of Texas Houston, Houston, Texas, 77030, United States|Inova Schar Cancer Institute, Fairfax, Virginia, 22031, United States|Virginia Commonwealth University, Richmond, Virginia, 23298, United States|Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, 2193, South Africa |
| Parent Educational Program for Children With Sickle Cell Disease | Children with sickle cell disease (SCD) are at risk for central nervous system (CNS) complications, which may affect academic achievement. This study will evaluate an educational support program for parents that aims to improve academic achievement in children with SCD. | Hemoglobin SC Disease|Anemia, Sickle Cell | ALL | CHILD | Mailman Center for Child Development, Miami, Florida, 33131, United States |
| Examining the Knowledge, Attitudes, and Beliefs of Sickle Cell Disease Patients, Parents of Patients With Sickle Cell Disease, and Providers Towards the Integration of CRISPR in Clinical Care | Background: Sickle cell disease (SCD) is caused by a genetic defect that affects how hemoglobin is made. Due to this, people with SCD have abnormally-shaped red blood cells, which can result in poor oxygen transport in the body and increase risk of blood clots. CRISPR Cas9 is a new tool which allows scientists to snip and edit genes in a way that is faster, cheaper, and more precise than other gene-editing tools. Recently, research has been done using CRISPR Cas9 to correct the sickle cell gene in animal models and human cells. Researchers want to understand the views of those with SCD, parents of people with SCD, and the providers of these patients regarding use of CRISPR Cas9 in clinical trials and treatment. Objectives: To study the attitudes, beliefs, and opinions of those with SCD, parents of those with SCD, and providers on the use of CRISPR Cas9 gene-editing. An additional purpose of this study is to assess the utility of an educational tool for improving understanding of CRISPR Cas9. Eligibility: People ages 18 and older who speak English and either have SCD, are a parent of someone with SCD, or are a physician for people with SCD. Design: Participants will be screened via phone. Those with SCD will be screened with data from their SCD genotype. Participation lasts about 2 hours. Participants will fill out three surveys. Participants will watch a video about CRISPR Cas9. Participants will engage in a focus group session. This will be audiotaped and analyzed. The data from the survey questions and focus groups may be used for future research. However, all personally identifiable information will be removed before data is shared. Participants data will be identified with a code number instead of their name. Participants may be invited to join future studies of SCD. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | National Human Genome Research Institute (NHGRI), Bethesda, Maryland, 20892, United States |
| Evaluation of Nocturnal Enuresis and Barriers to Treatment Among Pediatric Patients With Sickle Cell Disease | Pediatric patients with sickle cell disease are at greater risk for exhibiting nocturnal enuresis (bedwetting) compared to the general population. This increased risk has been attributed to a decreased ability to concentrate urine caused by sickling-induced nephropathy. The sociodemographic, psychosocial, and medical factors associated with nocturnal enuresis are not well defined. In addition, the impact of these behaviors on emotional and behavioral functioning, along with health-related quality of life are not clear. Despite the availability of evidence-based interventions for nocturnal enuresis, very few families with a child with sickle cell disease have utilized these methods. The reasons for this underutilization of interventions are not clear. | Sickle Cell Disease | ALL | CHILD | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Bone Marrow Transplantation in Treating Children With Sickle Cell Disease | RATIONALE: Sickle cell disease is an inherited disorder in which abnormal, crescent-shaped red blood cells interfere with the ability of the blood to carry oxygen through the body and can cause severe pain, stroke, and organ damage. Bone marrow transplantation, is a procedure in which the soft, sponge-like tissue in the center of bones producing white blood cells, red blood cells, and platelets is replaced by bone marrow from a another person. Bone marrow transplantation may be an effective treatment in relieving the symptoms of sickle cell disease. PURPOSE: Phase I/II trial to study the effectiveness of bone marrow transplantation in treating children who have sickle cell disease. | Sickle Cell Anemia | ALL | CHILD | Children's Hospital of Oakland, Oakland, California, 94609, United States|Fred Hutchinson Cancer Research Center, Seattle, Washington, 98109, United States |
| Pathophysiology of Acute Pain in Patients With Sickle Cell Disease | Background: Sickle Cell Disease (SCD) is a blood disorder that occurs mainly in people of African descent. Researchers want to learn more about the painful attacks and complications associated with SCD. They want to look for a relationship between SCD and specific changes in the blood. They want to study the role of genetics, inflammation, and blood clotting factors in SCD. They will do this with blood samples collected during an acute painful attack and in between attacks. Objective: To learn more about the painful attacks and complications associated with SCD. Eligibility: People ages 18-80 with SCD or who are healthy Africans or African Americans without SCD Design: * Participants will be screened with medical history and physical exam. * Healthy participants will have one visit. * Participants with SCD will have their first visit when they are not having a pain attack. They will have their next visit during a pain attack. About 3-4 months after this attack, they will have a final visit. * Visits will include a physical exam, and blood and urine tests. * Participants may have their blood samples used for genetic testing for research. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Exhaled Carbon Monoxide as a Marker of Hemolysis in Sickle Cell Disease- an Exploratory Study | The investigators propose to evaluate etCO in patients with HbSS, HbSC, and HbS-beta thalassemia during routine clinic visits, and longitudinally. Our goal is to know whether etCO differs amongst subjects with different sickle cell syndrome genotypes, and whether it is a stable marker of hemolytic rate, as reflected in routine labs obtained for clinical care (including total hemoglobin, reticulocyte count, lactate dehydrogenase, and, when sampled, total and direct bilirubin). We hope to establish whether this inexpensive and non-invasive test faithfully reflects hemolytic parameters in sickle cell syndromes. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Seidman Cancer Center, University Hospitals, Cleveland, Ohio, 44106, United States |
| A Study of SHP655 (rADAMTS13) in Sickle Cell Disease | TAK-755 (previously known as SHP655) is a medicine used to treat sickle cell disease (SCD). The main aim of the study is to measure the safety and tolerability of TAK-755 in SCD participants. Study participants will receive TAK-755 or placebo on Day 1. Their SCD will be treated by their doctor according to their doctor's usual clinical practice. During the study, participants will be asked to follow-up on 13 days following SHP655 or placebo administration for safety assessment. Maximum duration of participation is expected to be about 2 months. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of Alabama at Birmingham, Birmingham, Alabama, 35233, United States|Arkansas Children's Hospital, Little Rock, Arkansas, 77202, United States|University of Colorado Sickle Cell Treatment and Research Center, Aurora, Colorado, 80045, United States|Sickle Cell Center, Denver, Colorado, 80262, United States|University of Illinois, Chicago, Illinois, 60612-4325, United States|Ochsner Health System, New Orleans, Louisiana, 70121, United States|Johns Hopkins University School Of Medicine, Baltimore, Maryland, 21218, United States|Levine Cancer Institute, Charlotte, North Carolina, 28204, United States|Duke University Medical Center, Durham, North Carolina, 27705, United States|East Carolina University, Greenville, North Carolina, 27858, United States|Ohio State University Medical Center, Columbus, Ohio, 43210, United States|Medical University of South Carolina (MUSC), Charleston, South Carolina, 29425, United States|University of Tennessee -- Memphis, Memphis, Tennessee, 38163-2116, United States|VCU Health - Research Parent, Richmond, Virginia, 23298, United States |
| Arginine Therapy in Sickle Cell Disease-VOC Clinical Trial | Vaso-occlusion contributes significantly to morbidity in sickle cell disease (SCD). Vaso-occlusive painful episodes (VOE) are common and debilitating, causing the majority of emergency department visits. Currently efforts to treat painful episodes with use of non-steroidal pain relievers and intravenous narcotics offer symptomatic relief only, without targeting the underlying mechanisms of vaso-occlusion.Investigators have found that an arginine deficiency and low NO bioavailability occurs during painful events in SCD. Since arginine is the obligate substrate for NO production, and an acute deficiency is associated with VOE, investigators hypothesized that arginine supplementation may be a safe and beneficial treatment for sickle cell pain. | Vaso-occlusive Pain Episodes | ALL | CHILD, ADULT | Childrens Hospital Research Center Oakland, Oakland, California, 94609, United States |
| Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507) | This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD). Based on their age and entry criteria patients are stratified into two groups: (1) children with severe SCD; and (2) adults with severe SCD. | Sickle Cell Disease | ALL | CHILD, ADULT | University of Alabama at Birmingham, Birmingham, Alabama, 35294, United States|UCSF Benioff Children's Hospital Oakland, Oakland, California, 94609, United States|University of Colorado - Denver/Children's Hospital of Colorado, Aurora, Colorado, 80045, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States|University of Florida College of Medicine, Gainesville, Florida, 32610, United States|Nicklaus Children's Hospital/University of Miami Children's Hospital, Miami, Florida, 33155, United States|H. Lee Moffitt Cancer Center, Tampa, Florida, 33612, United States|Northside Hospital, Atlanta, Georgia, 30342, United States|Riley Children's Hospital at IU Health, Indianapolis, Indiana, 46020, United States|Indiana University Medical Center, Indianapolis, Indiana, 46202, United States|Johns Hopkins University, Baltimore, Maryland, 21287, United States|Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|Helen Devos Children's at Spectrum Health, Grand Rapids, Michigan, 49503, United States|Cardinal Glennon Children's Hospital, Saint Louis, Missouri, 63104, United States|Washington University, St. Louis, Saint Louis, Missouri, 63110, United States|Hackensack University Medical Center, Hackensack, New Jersey, 07601, United States|Roswell Park Cancer Center, Buffalo, New York, 14203, United States|Northwell Health/Monter Cancer Center, Lake Success, New York, 11042, United States|Levine Children's Hospital, Charlotte, North Carolina, 28203, United States|Duke University Medical Center, Durham, North Carolina, 27705, United States|University Hospitals of Cleveland/Case Western, Cleveland, Ohio, 44106, United States|Cleveland Clinic Foundation, Cleveland, Ohio, 44195, United States|Nationwide Children's Hospital, Columbus, Ohio, 43205-2696, United States|University of Pittsburgh, Pittsburgh, Pennsylvania, 15260, United States|Methodist Healthcare/West Cancer Center, Memphis, Tennessee, 38104, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|Vanderbilt University Medical Center, Nashville, Tennessee, 37232, United States|Texas Children's Hospital (Baylor), Houston, Texas, 77030, United States|University of Texas/MD Anderson Cancer Center, Houston, Texas, 77030, United States|Texas Transplant Institute, San Antonio, Texas, 78229, United States|Fred Hutchinson Cancer Research Center, Seattle, Washington, 98109, United States|The Medical College of Wisconsin, Wauwatosa, Wisconsin, 53226, United States |
| Evaluation of the Lung Capillary Blood Volume in Children With Sickle Cell Disease | Sickle cell disease (SCD) is the most common inherited disease of the world affecting African and Caribbean populations. SCD is caused by the homozygous inheritance of the gene for sickle hemoglobin (HbS). Most patients with SCD develop abnormal pulmonary function characterized by airway obstruction, restrictive lung disease, abnormal diffusing capacity, hypoxemia and pulmonary hypertension In healthy subjects, lung capillary blood volume (Qc) and membrane diffusing capacity (Dm) can be accurately measured by the nitric oxide-carbon monoxide (NO-CO) method. We propose to study, for the first time, lung capillary blood volume and alveolar membrane diffusing capacity, using the NO-CO method, in children with SCD aged of at least 6 years Early determination of lung function and pulmonary circulation in children with SCD is very important, not only for the understanding of physiopathologic mechanisms of the disease but also for a better therapeutic management of these children. | Sickle Cell Disease | ALL | CHILD, ADULT | Hopital Robert DEBRE, Paris, 75019, France |
| Home-based Assessment of PRO Measures in SCD Using A Smartphone App Platform: A Feasibility Study | The overarching goal of this proposal is to identify modifiable behavioral strategies based on patient-reported outcomes (PROs) and health-related quality of life (HRQOL) that will improve hydroxyurea (HU) adherence among adolescents and young adults with sickle cell disease (SCD). In this proposed study, we intend to test the functionality of a PROs-toolbox feature, which will be integrated into our existing smartphone application platform (SCD-app), over a 24-week period in a cohort of SCD patients and their caregivers. | Sickle Cell Disease|Sickle Cell Hemoglobin C|Sickle Beta Zero Thalassemia|Sickle B+ Thalassemia | ALL | CHILD, ADULT | Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States |
| MAP to Provide Access to Crizanlizumab, for Sickle Cell Disease Patients | The purpose of this Cohort Treatment Plan is to allow access to crizanlizumab (SEG101) for eligible patients diagnosed with sickle cell disease (SCD) to prevent or reduce the frequency of vaso-occlusive crises (VOC). The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | |
| Qualitative Survey of Potential Exercise Activity in Adults With Sickle Cell Anemia (SCA) | The purpose of this study is to assess the potential interventions of exercises in adults with sickle cell anemia (SCA) and cardiopulmonary disease; only including the more severe genotypes of sickle cell disease. | Cardiovascular Diseases|Sickle Cell Disease|Exercise | ALL | ADULT, OLDER_ADULT | Memorial Healthcare System, Hollywood, Florida, 33021, United States |
| Vascular Function Intervention Trial in Sickle Cell Disease | Sickle cell disease (SCD) is the most common inherited disorder worldwide affecting 300,000 births annually, most occuring in sub-Saharan Africa (SSA) where poor detection and care result in high childhood mortality, malnutrition, illness and disability in survivors. SCD is caused by abnormal haemoglobin, the compound in red blood cells(RBC) that carries oxygen. Much of the disability in SCD may be caused by vascular damage from the breakdown of damaged RBC. Research in high-income countries has led to some effective therapies but these are currently costly and complex. The investigators will test two different formulations of an affordable, ready-to-use supplementary food (RUSF) specifically tailored for children with SCD. As well as containing energy, protein, essential fats, vitamins and minerals, the vascular RUSF (RUSFv) will be fortified with the amino-acids arginine and citrulline and be delivered with a daily chloroquine dose to create a novel "nutraceutical" intervention. Arginine is converted to nitric oxide which is essential for vascular health. Arginine levels are low in SCD because the arginine-degrading enzyme, arginase, is released from RBCs. The investigators propose that by supplying additional arginine (and citrulline which converts to arginine) and suppressing arginase activity (an action of chloroquine) the investigators can improve vascular function. Our study will test this theory, and if provision of RUSF improves growth in children with SCD. | Sickle Cell Disease | ALL | CHILD | Muhimbili University of Heath and Allied Sciences (MUHAS), Dar es Salaam, Tanzania |
| Safety of Blood Stem Cell Mobilization With Plerixafor in Patients With Sickle Cell Disease | The objective of this study is to investigate if up to two injections of plerixafor represent a safe and effective strategy to mobilize adequate numbers of CD34+ hematopoietic stem progenitor cells (HSPC) for autologous hematopoietic cell transplantation (HCT) in sickle cell disease (SCD) patients | Sickle Cell Disease | ALL | ADULT | City of Hope Medical Center, Duarte, California, 91010, United States |
| Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe Β-Hemoglobinopathies | A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new genetic material in order to fix or replace a diseased gene, with the goal of curing the disease. The procedure is similar to a bone marrow transplant, in that the patient's malfunctioning blood stem cells are reduced or eliminated using chemotherapy, but it is different because instead of using a different person's (donor) blood stem cells for the transplant, the patient's own blood stem cells are given back after the new genetic material has been introduced into those cells. This approach has the advantage of eliminating any risk of Graft-Versus-Host Disease (GVHD), reducing the risk of graft rejection, and may also allow less chemotherapy to be utilized for the conditioning portion of the transplant procedure. The method used to fix or replace a diseased gene is called gene editing. A person's own cells are edited using a specialized biological medicine that has been formulated for use in human beings. Fetal hemoglobin (HbF) is a healthy, non-sickling kind of hemoglobin. Investigators have recently discovered a gene called BCL11A that is very important in the control of fetal hemoglobin expression. Increasing the expression of this gene in sickle cell patients could increase the amount of fetal hemoglobin while simultaneously reducing the amount of sickle hemoglobin in their blood, and therefore potentially cure the condition. | Sickle Cell Disease|Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)|Beta-Thalassemia|Transfusion Dependent Beta-Thalassaemia | ALL | CHILD, ADULT | Boston Children's Hospital, Boston, Massachusetts, 02115, United States |
| A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease | This clinical trial is a Phase 2 study that will evaluate the safety and clinical activity of etavopivat in patients with thalassemia or sickle cell disease and test how well etavopivat works to lower the number of red blood cell transfusions required and increase hemoglobin. | Sickle Cell Disease|Thalassemia | ALL | CHILD, ADULT, OLDER_ADULT | TOI Clinical Research, Cerritos, California, 90703, United States|[Legal] Children's Hospital Los Angeles, Los Angeles, California, 90027, United States|University of Californ LA-UCLA, Los Angeles, California, 90095, United States|UCSF Oakland Benioff ChildHosp, Oakland, California, 94609, United States|[Legal] Children's Hospital of Orange County on behalf of CHOC Children's Hospital of Orange County, Orange, California, 92868, United States|UCI Health, Orange, California, 92868, United States|Children's National Health Hospital, Washington, District of Columbia, 20010, United States|Children's Hospital of Atlanta, Atlanta, Georgia, 30342, United States|[Legal] Children's Hospital of Michigan, Detroit, Michigan, 48201-2018, United States|Weill Medical College of Cornell University, New York, New York, 10065, United States|Duke University, Durham, North Carolina, 27710, United States|East Carolina University, Greenville, North Carolina, 27858, United States|[Legal] Children's Hospital Medical Center dba Cincinnati Children's, Cincinnati, Ohio, 45229, United States|Children's Hosptl Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Master Centre for Canada, Mississauga, Ontario, L4W 4XI, Canada|The Hospital for Sick Children, Toronto, Ontario, M5G 1X8, Canada|University Health Network - Toronto General Hospital, Toronto, Ontario, M5G 2C4, Canada|CHU Sainte-Justine Mother and Child University Hospital, Montreal, Quebec, H3T 1C5, Canada|Abu El-Reesh El-Mounira Children University Hospital, Cairo, 11471, Egypt|Cairo University, Cairo, 12613, Egypt|Galliera Hospital Centro Anemie Congenite, Genova, 16128, Italy|Fondazione IRCSS Ca' Granda Ospedale Maggiore Policlinico, Milano, 20122, Italy|A.O.U. Università Studi della Campania "Luigi Vanvitelli", Naples, 80138, Italy|AORN A. Cardarelli, Napoli, 80131, Italy|Chronic Care Center, Baabda, RGWX 4CG, Lebanon|Hospital Nini, Tripoli, 1434, Lebanon|Sandwell and West Birmingham NHS Trust SCAT/ haematology, Birmingham, B18 7QH, United Kingdom|Barts Health NHS Trust - The Royal London Hospital, London, E1 1FR, United Kingdom|University College Hospital - University College London Hospitals NHS Foundation Trust, London, NW1 2PG, United Kingdom|Kings College London, London, SE5 9RS, United Kingdom|Imperial College Healthcare NHS Trust - Hammersmith Hospital, London, W12 0HS, United Kingdom|Manchester University NHS Foundation Trust, Manchester, M13 9WL, United Kingdom |
| Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial | Many youth with chronic disease have difficulty taking medication every day and therefore do not receive full benefit from treatment. Sickle Cell Disease (SCD) is an inherited blood disease that affects African Americans and other underserved communities. Hydroxyurea (HU) is the sole FDA-approved drug therapy for SCD and is highly effective and improves quality of life. The proposed study, a 5-site four-year randomized control trial (RCT), builds upon the investigators' recent feasibility study of the same title. Overall goals are reducing barriers to HU use and improving adherence for youth 10-18 years through creation of a daily medication habit. The goal of the proposed multi-site study is to test the efficacy of the HABIT intervention at 6 months and sustainability of the effect at 12 months. | Sickle Cell Disease | ALL | CHILD, ADULT | Albert Einstein College of Medicine, Bronx, New York, 10461, United States|Feinstein Institute for Medical Research, Manhasset, New York, 11030, United States|Columbia University Irving Medical Center, New York, New York, 10032, United States|The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States |
| Intravenous Gammaglobulin for Sickle Cell Pain Crises | The purpose of this study is to determine whether intravenous immune globulin is safe and effective in the acute treatment of pain crises in sickle cell disease. Funding Source: Food and Drug Administration (FDA), Office of Orphan Products Development (OOPD) | Sickle Cell Disease|Pain | ALL | CHILD, ADULT, OLDER_ADULT | Montefiore Medical Center, Bronx, New York, 10467, United States |
| Evaluation of a Training Program for Homozygous Sickle Cell Disease Patients | Sickle cell disease (SCD) is the most frequent inherited disease in the world. Literature reports that SCD patients display intolerance to exercise, important muscle weakness and profound remodeling of skeletal muscle including amyotrophy and rarefied microvascular network. Because strenuous exercise induces acidosis, hemorheological alterations, endothelial activation and oxidative stress, it constitutes a potential triggering factor of sickling and vaso-occlusive crisis. As a consequence, physical activity is usually discouraged in patients with SCD. However, moderate and regular physical activity seems to be not only safe but also beneficial for SCD patients. | Sickle Cell Hemoglobin C Disease|Hemoglobin S Disease | ALL | ADULT, OLDER_ADULT | Hopital Avicenne, Bobigny, 93000, France|Centre hospitalier sud francilien, Corbeil-essonnes, 91100, France|CHU Henri MONDOR, Creteil, 94010, France|CHU Kremlin-Bicêtre, Le Kremlin Bicetre, 93270, France|Hopital Europeen Georges POMPIDOU, Paris, 75015, France|Hopital Necker, Paris, 75015, France|Hopital Tenon, Paris, 75020, France|Centre hospitalier de Saint-Denis, Saint-denis, 93200, France|CHU de SAINT-ETIENNE, Saint-etienne, 42000, France |
| Study of Ventilatory Mechanics in Patients With Sickle Cell Anemia | The Sickle Cell Anemia (SCA) is a recessive genetic condition, monogenic, resulting in defects in the red cell structure. In the investigators' country, this disease affects about 3,000 children each year and is considered one of the most prevalent disorders among the group of existing hereditary diseases. The lungs are frequently affected in this disease by Acute Chest Syndrome (STA). Besides being the leading cause of death and the second leading cause of hospitalization in SCA, the STA is correlated with cognitive impairment frame these patients, resulting secondary Stroke vaso-occlusion of capillaries that supply the brain tissue. Traditional tests of pulmonary function allow assess whether the person has any commitment in the respiratory system, whether obstructive, restrictive or mixed. To run these tests it is necessary that the patient understands and performs a forced expiratory maneuver to obtain reliable results. In the particular case of SCA, performing these tests it is very difficult due to the presence of cognitive impairment of varying degrees. This results in underdiagnosis of early changes in the lung parenchyma during the therapeutic window, committing the proper monitoring and treatment offered to these patients. | Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | |
| Safety and Efficacy of Sodium Nitrite in Sickle Cell Disease | This study will determine if administration of sodium nitrite is safe and can improve small vessel blood flow and tissue oxygenation when given as an additional treatment in patients with acute vaso-occlusive crisis (pain crisis) associated with sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT | Childrens Hospital Los Angeles, Los Angeles, California, 90027, United States |
| Development and Evaluation of an Information Management System and Communication System for Population-wide Point-of-care Infant Sickle Cell Disease Screening | Although over 75% of children with sickle cell disease (SCD) are born in sub-Sahara where the disease highly contributes to under-5 mortality and causes life-long debilitation, evidence-based strategies to control SCD are not widely implemented in this region. Early detection of SCD by universal infant screening is a pillar of SCD control. Despite the affordability and move to adopt point-of-care (POC) SCD screening assays in sub-Sahara Africa, the absence of screening information management and communication systems (SIMCS) impedes standardized, systematic, coordinated, nationwide SCD screening programs. The long-term goal of the proposed research is to develop a SCD SIMCS that will enable universal SCD screening in the sub-Sahara African setting. The objective is to test and optimize a custom SCD SIMCS app and digital network to facilitate SCD screening and then evaluate its impact on access to SCD screening and care and on clinical outcomes of children with SCD in Uganda. The central hypothesis is that the SCD SIMCS will facilitate accurate and coordinated POC SCD screening that is accessible at health centers in urban and rural Uganda. The rationale is to build a custom SCD SIMCS on existing nationwide digital and health infrastructure in Uganda to standardize use of affordable POC assays at health centers nationwide. The central hypothesis will be tested by pursuing two specific aims: 1) Develop and evaluate a four-module ≥3G cell phone app for a novel SCD SIMCS (R21 Phase); 2) Evaluate the impact of the SCD SIMCS on access to screening and care and outcomes of children with SCD (R33 Phase). The investigators will pursue these aims using an innovative combination of software design and re-organization of SCD screening workflows. These include assembly of off-the-shelf software that is compatible with iOS and Android operating systems to reliably, accurately, and handily capture, interpret, transmit, and retrieve/playback information for patient's IDs, test results, salient clinical events, and education. The novel screening workflows are expected to dramatically reduce the cost and increase access to SCD screening and care. The proposed research is significant, because it will determine how to use POC SCD screening assays on a large nationwide scale. It will also enable coordination of evidence-based care and continuity of care between primary and specialist providers and longitudinally over the patient's lifetime - a critical aspect in controlling this life-long disease. The SCD SIMCS will also facilitate real time data management for research and policy for SCD control. The expected immediate outcome of this research is a SCD SIMCS that optimally functions on the digital and health infrastructure in Uganda and demonstration of its impact on access to SCD screening and care and on clinical outcomes of children with SCD. The expected long-term outcome is that the SCD SIMCS will be adopted, integrated, and scaled-up in the health systems of Uganda and other sub-Sahara Africa countries, particularly those where the POC assays have already been adopted as the national standard of SCD screening. If effective, the SCD SIMCS will have an important positive impact because it will reduce the cost of SCD screening, take screening services and evidence-based care closer to rural communities where the majority of children in sub-Sahara Africa live, and, ultimately, save millions of children from preventable and disability death. | Sickle Cell Disease | ALL | CHILD | Baylor College of Medicine, Houston, Texas, 77030, United States|College of Health Sciences, Makerere University, Kampala, 256, Uganda |
| Diabetic Retinopathy and Sickle Trait | To more clearly ascertain the relationship between ocular manifestations of sickle cell disease and diabetes, specifically; whether the presence of sickle cell trait exacerbates the disease progression of diabetic retinopathy. | Diabetic Retinopathy|Sickle Cell Trait | ALL | CHILD, ADULT, OLDER_ADULT | Medical University of South Carolina, Storm Eye Institute, Charleston, South Carolina, 29425, United States |
| Effect of Atorvastatin on Endothelial Dysfunction and Albuminuria in Sickle Cell Disease | The purpose of this research study is to learn about the effect of the drug, atorvastatin, on blood vessels in patients with sickle cell disease. The primary hypothesis is that endothelial dysfunction is an important contributor to the pathophysiology of albuminuria in SCD. The investigators propose that atorvastatin will improve endothelial dysfunction, decrease levels of soluble fms-like tyrosine kinase-1 (sFLT-1), and decrease albuminuria in SCD patients. Participants will be individuals with sickle cell disease, age 18 to 60, who have some degree of albuminuria. A total of 19 subjects, males and females, will be enrolled. The study is made up of Screening, Treatment, and Follow Up phases and has a cross-over design. After patients are screened for eligibility, they will be randomized to receive atorvastatin or placebo in the initial six-week treatment period. When that is complete, there will be a four-week washout period before they begin another six-week treatment period. In the second treatment period, they "cross-over" to the other treatment arm. Four weeks after the end of the second treatment period, follow-up safety assessments will be done. | Sickle Cell Disease|Sickle Cell Nephropathy | ALL | ADULT | UNC School of Medicine Clinical&Translational Research Ctr, Chapel Hill, North Carolina, 27599, United States |
| Sickle Cell Disease: Targeting Alloantibody Formation Reduction; Risk Factors, and Genetics | The focus of the study is the pathophysiological mechanism of allo-antibody formation after red blood cell transfusion in sickle cell disease patients. | Alloimmunization|Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Academic Medical Center Amsterdam, Amsterdam-Zuidoost, Netherlands|HagaZiekenhuis, Den Haag, Netherlands|Radboudumc, Nijmegen, Netherlands|Erasmus MC, Rotterdam, Netherlands |
| Establishing a Repository of Blood and DNA Samples From People With Sickle Cell Disease (Comprehensive Sickle Cell Centers Collaborative Genotype-Phenotype Database and Sample Repository) | Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause intense pain episodes. The purpose of this study is to collect, test, and archive blood and DNA samples from children and adults with SCD to study the role that genes play in SCD. Blood and DNA samples will be stored for use in future SCD studies. | Anemia, Sickle Cell | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital Oakland, Oakland, California, 94609, United States|University of California, San Francisco, San Francisco, California, 94134, United States|Brigham & Women's Hospital, Boston, Massachusetts, 02115, United States|Children's Hospital of Boston, Boston, Massachusetts, 02115, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|Montefiore Medical Center, Bronx, New York, 10463, United States|University of North Carolina-Chapel Hill, Chapel Hill, North Carolina, 27599, United States|Cincinnati Children's Hospital, Cincinnati, Ohio, 45229, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|St. Christopher's Hospital for Children, Philadelphia, Pennsylvania, 19134, United States|Children's Medical Hospital of Dallas, Dallas, Texas, 75235, United States|University of Texas Southwestern & Parkland, Dallas, Texas, 75235, United States|University of Texas at Galveston, Galveston, Texas, 77555, United States |
| Atorvastatin Therapy to Improve Endothelial Function in Sickle Cell Disease | This study will examine the effects of oral atorvastatin on the linings of blood vessels in patients with sickle cell disease, plus the agent's effect on blood markers of inflammation and blood vessel function. Sickle cell disease is a recessive genetic disorder and the most common genetic disease affecting African Americans. Inherited are abnormal genes that make hemoglobin, the substance within red blood cells that carries oxygen from the lungs to the body. In the disease, sickle hemoglobin leads to rigidity or hardness of the red cells, causing obstruction in small blood vessels, inflammation, and injury to organs when the flow of blood to them is blocked. Some medications already prescribed for other diseases, such as atorvastatin, which is used for lowering cholesterol levels, can improve blood flow. Patients 18 to 65 years of age who have sickle cell disease, who have not had an acute pain episode within the previous week, and who are not pregnant or lactating may be eligible for this study. They will undergo a complete medical history; physical examination; baseline blood tests; and echocardiogram, in which an ultrasound wand is placed against the chest wall to get images inside the heart and blood vessels. In addition, patients will have blood flow studies. During the procedure, they will lie in an adjustable reclining chair for 5 to 6 hours. There will be 20- to 30-minute rests between specific activities and blood samples will be drawn intermittently for testing. Small tubes will be placed in the artery of the forearm at the inside of the elbow. Normal saline will be infused into one tube. A small pressure cuff will be applied to the wrist and a larger cuff to the upper arm. Both cuffs will be attached to an inflation device. A device like a rubber band, a strain gauge, will be placed around the widest part of the forearm. When the pressure cuffs are inflated, blood will flow into the arm, stretching the gauge proportion to blood flow, and information will be recorded. Then light reflected from the patients' hand and the blood flow in the forearm will be measured. Activity of the genes in the white blood cells will be measured as well. Small amounts of sodium nitroprusside, widely used to reduce blood pressure in people with dangerously high blood pressure, will be injected and blood flow will be measured. Later, small amounts of acetylcholine will be injected. It usually causes blood vessels to expand. After that, small amounts of L-NMMA will be injected. It usually decreases local blood flow by blocking the production of nitric oxide in the cells lining the arm's blood vessels. Then acetylcholine combined with L-NMMA will be injected. After that, oxypurinol, an agent taken by many patients to prevent gout, will be injected. The procedures will be repeated, with oxypurinol given along with each of the agents, and the measurement of blood flow in the forearm will be measured after each drug combination. Afterward, patients will be treated for 4 weeks at home with oral atorvastatin. They will be asked to visit the Clinical Center every 2 weeks for collection of blood samples and an examination. After 4 weeks of taking atorvastatin orally, they will be asked to return to repeat the blood flow studies, but only the first half will be conducted. The part using oxypurinol will not be needed. Regarding some of the blood samples collected during the study, there will be an examination of the genes found in the white blood cells. Specific attention will go to those genes that make proteins for cell-to-cell interaction and inflammation, plus those that cause blood cells to stick to the lining of blood vessels. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| Improving Scientific Rigor of Renal Clinical Endpoints for Sickle Cell Anemia | The investigators will attempt to develop a more accurate equation to estimate eGFR in pediatric and adult sickle cell patients | Sickle Cell Disease|Renal Disease|Glomerular Disease | ALL | CHILD, ADULT | University of Alabama at Birmingham, Birmingham, Alabama, 35223, United States|University of Illinois Chicage, Chicago, Illinois, 60612, United States|St Jude Childrens Research Hospital, Memphis, Tennessee, 38106, United States|University of Tennessee Health Science Center, Memphis, Tennessee, 38163, United States |
| Characteristics and Treatment Patterns of Patients With Sickle Cell Disease in Globin Research Network for Data and Discovery Registry | This was a retrospective observational cohort study. This study was a secondary analysis of individuals with sickle cell disease (SCD) enrolled in the GRNDaD registry. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Novartis Pharmaceuticals, East Hanover, New Jersey, 07936, United States |
| Rifaximin to Modify the Disease Course in Sickle Cell Disease | In this single-arm, one-stage Phase II study, the investigators hypothesize that gut decontamination with rifaximin will reduce the frequency of hospital admission due to painful crisis in patients with SCD. The study will accrue 20 SCD patients who had at least two hospital admissions in the previous 12 months. These patients will receive rifaximin 550 mg twice a day for a total of 12 months. This following clinical parameters will be measured: 1. Changes in the annual rate of hospital admissions due to painful crisis; 2. Changes in the annual rate of days hospitalized; 3. Annual rates of uncomplicated crises; 4. Annual rate of acute chest syndrome; 5. Changes in the quality of life; and 6). Toxicities. The following laboratory parameters will be measured: 1. Changes in the number of circulating activated neutrophils; 2. Changes in the intestinal microbiome diversity; 3. Changes in the urinary 3-indoxyl sulfate levels; 4. Changes in the serum biomarkers of intestinal permeability (lipopolysaccharides; zonulin, citrulline, and fatty acid binding proteins). | Sickle Cell Disease|Antibiotics | ALL | ADULT, OLDER_ADULT | Westchester Medical Cancer Cancer Institute, Valhalla, New York, 10532, United States |
| Pulmonary Functions in Sickle Cell Disease: Response to Acu-TENS | pulmonary complications are common in sickle cell disease patients. Respiratory training using inspiratory muscle trainer (IMT) is usually a good choice to improve these complications. Recently, acupuncture like transcutaneous electrical nerve stimulation (AC-tens) may also improve these complications. | Sickle Cell Disease | ALL | ADULT | faculty of physical therapy Cairo university, Dokki, Giza, Egypt |
| A Study to Learn About Sickle Cell Disease In Adult Patients | The purpose of this clinical trial is to evaluate the performance of the sickle cell disease (SCD) electronic diary in people with SCD who are on treatment that will change SCD and those not on such a treatment. SCD is a type of condition when there are fewer red blood cells to carry oxygen around the body. This disease can be passed on from parent to child and may cause pain, infections and damage to organs. This study is seeking participants who: * are confirmed with SCD * are on a stable regimen of disease changing treatment or have not received any disease changing treatment before the start of the study and do not plan any changes in their treatment during the 6-month study observation period For 6 months, participants will be asked to complete a daily electronic diary to report on their experience in the past 24 hours with sickle cell pain crisis (if they got any treatment and what medications they took), worst pain, worst tiredness, and their ability to perform usual physical activities. We will compare the experiences of people who are taking SCD-modifying therapy to those that are not taking a SCD-modifying therapy. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Foundation for Sickle Cell Disease Research, Hollywood, Florida, 33024, United States|Mid-Atlantic Permanente Medical Group Largo Medical Center, Largo, Maryland, 20774, United States|Mid-Atlantic Permanente Medical Group Largo Medical Center, Upper Marlboro, Maryland, 20774, United States|Sanguine Biosciences, Inc., Waltham, Massachusetts, 02451, United States|Cohen Children's Medical Center, New Hyde Park, New York, 11040, United States |
| Avascular Bone Necrosis in Sickle Cell Disease: a Pediatric Study. | Avascular necrosis (AVN) is a serious complication of sickle cell disease, especially in pediatric patients where the prevalence is between 3% and 8% and are more frequent in patients with multiple vaso-occlusive crisis (VOC). The prevalence of AVN is usually made by a study of the hip through radiography, whereas other possible sites of ischemic infarcts are evaluated only in case of specific symptoms. In addition, bone infarcts may be the trigger for additional VOC. In this study, we want to investigate the presence of possible bone lesions even in asymptomatic or paucisymptomatic children. This is a prospective interventional and monocentric study whose objective is to describe the prevalence of osteonecrosis in children with sickle cell disease in Italy | Sickle Cell Disease | ALL | CHILD | AOU San Luigi Gonzaga, Orbassano, Torino, 10043, Italy |
| Quantifying the Presence of Lung Disease and Pulmonary Hypertension in Children With Sickle Cell Disease | The proposed research study is a cross-sectional study enrolling young children with sickle cell disease between 5 and 12 years of age. They will be screened as outpatients for consent to perform pulmonary function testing (PFT) and echocardiography. In addition, the degree of bronchodilator response will be assessed at each session. To estimate presence of pulmonary hypertension, echocardiography will be performed at the time of PFT measures. Study Design: 1. Enroll children aged 5 to 12 years of age with sickle cell disease (HbSS, HbSC, HbS beta plus thalassemia, HbS beta zero thalassemia, and HbS OArab) who are established patients within the Duke Pediatric Sickle Cell Clinic. 2. Perform a chart review of all enrolled subjects to obtain specific details regarding birth history, nutritional status (weight, height), family history, sickle cell genotype, parental smoking history, recent laboratory parameters, parental smoking history, any concurrent conditions (atopy, asthma, airway anomaly), history of sickle cell complications and prescribed medications. 3. Perform spirometry and plethysmography with the administration of albuterol. 4. Before or after completion the PFT session, the patient will have echocardiography in the PFT lab area 5. Using medical record information, determine number of hospitalizations for any pulmonary symptoms indicative of acute chest syndrome (ACS) (dyspnea, fever, wheezing, hypoxia, cough, chest pain). In addition, we will track any respiratory or cardiac symptoms or therapies for each subject 6 years after enrollment up to age 18 years using the registry. 6. As standard of care, refer any child identified as having lung disease or pulmonary hypertension to a pediatric pulmonologist and/or cardiologist for monitoring, treatment and ongoing care. | Sickle Cell Disease | ALL | CHILD | Duke University Medical Center, Durham, North Carolina, 27710, United States |
| Sickle Cell Disease Conditioning for Bone Marrow Transplant | Most bone marrow transplants for children with sickle cell disease are performed using high doses of two chemotherapy agents: busulfan and cyclophosphamide for the pre-transplant conditioning. This approach produces cure in most cases (approximately 95%). It, however, has serious side effects, including seizures and infertility. The primary goal of this study is to determine how much we can lower the dosages of busulfan and cyclophosphamide by incorporating fludarabine, a safer chemotherapy agent, into conditioning. The secondary goal is to develop a better understanding of how bone marrow transplants cause neurologic problems like seizures. | Bone Marrow Transplantation|Sickle Cell Disease | ALL | CHILD, ADULT | University of Alabama-Birmingham, Birmingham, Alabama, United States|Children's National Medical Center, Washington, D.C., District of Columbia, United States|All Children's Research Institute Inc., Saint Petersburg, Florida, 33701, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States|Tulane University, New Orleans, Louisiana, 70112, United States|Wayne State University, Detroit, Michigan, 48202, United States|University of Mississippi Medical Center, Jackson, Mississippi, United States|Montefiore Medical Center, The Bronx, New York, United States|University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, 27516, United States|University of Texas Southwestern, Dallas, Texas, United States |
| Pain in Sickle Cell Epidemiologic Study | To measure the variability in pain and response to pain in sickle cell disease, and to build multivariate models to explain both patients' pain and their response to pain, especially, utilization of health care. | Blood Disease|Anemia, Sickle Cell | ALL | CHILD, ADULT, OLDER_ADULT | |
| Study of Vitamin D in Children With Sickle Cell Disease | This pilot study aims to answer the question whether monthly oral vitamin D3 supplementation, 100,000 IU, will be safe and effective in raising serum 25-hydroxyvitamin D (form of vitamin D measured in the blood) to levels considered sufficient (30 ng/mL) but well below the threshold for toxicity (150 ng/mL) in children with sickle cell disease. Information from this study will be crucial before we perform a larger clinical trial to determine the effects of vitamin D in reducing respiratory complications in patients with sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT | Columbia University Medical Center, New York, New York, 10032, United States |
| Integrative Training Program for Pediatric Sickle Cell Pain | This research aims to answer the question: does a group training program specifically for teens with chronic sickle cell disease (SCD) pain that teaches skills to strengthen the mind and body help improve everyday functioning and reduce pain symptoms? The program will be tailored to address challenges related to frequent or chronic sickle cell pain and may improve participants' physical and emotional health. The program, called I-STRONG for SCD (Integrative Strong Body and Mind Training for Sickle Cell Disease), may help improve everyday functioning and pain symptoms in teens with chronic pain related to SCD. The research team aims to determine how participants (teens and parents) respond to this program. | Sickle Cell Disease | ALL | CHILD, ADULT | Connecticut Children's Medical Center, Hartford, Connecticut, 06106, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States|Arthur M. Blank Hospital | Children's Healthcare of Atlanta, Atlanta, Georgia, 30329, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States |
| Iron Overload Assesment in Sickle Cell Anemia and Sickle Cell Thalassemia | Iron overload is well study in Thalassemia patients and it's not only related to blood transfusions, since intestinal iron absorption is also increased in those patients. Sickle cell patients didn't develope significant clinical symptoms and signs of iron overload in spite frequent transfusions. The purpouse of this study is to assess the iron overload in Sickle cell anemia and Sickle cell Thalassemia patients using clinical parameters and cardiac T2\*MRI in order to determine the cardiac and liver iron. | Sickle Cell Anemia|Sickle Cell Thalassemia|Iron Overload|MRI | ALL | ADULT, OLDER_ADULT | Pediatric Hematology Unit and Pediatric Dpt B - HaEmek Medical Center, Afula, 18101, Israel |
| A Phase II Trial of Regadenoson in Sickle Cell Anemia | This research study is a Phase II clinical trial, which tests the safety and effectiveness of an investigational drug called Regadenoson (or Lexiscan) to learn whether the drug works in treating a specific disease, in this case Sickle Cell Disease (SCD). "Investigational" means that the drug is being studied. It also means that the FDA has not yet approved the drug for your type of disease. SCD is an inherited blood disorder that causes the red blood cells to change their shape from a round shape to a half-moon/crescent or sickled shape. People who have SCD have a different type of protein that carries oxygen in their blood (hemoglobin) than people without SCD. This different type of hemoglobin makes the red blood cells change into crescent shape under certain conditions. Sickle-shaped cells are a problem because they often get stuck in the blood vessels blocking the flow of blood, and cause inflammation and injury to important areas in the body. Regadenoson (trade name Lexiscan) is a drug that may prevent this inflammation and injury caused by the sickle shaped cells. This drug is approved by the FDA to be used as a fast infusion during a heart stress test in people who are unable to exercise enough to put stress on their heart by making the heart beat faster. Regadenoson has been studied as a long infusion at this dose in adults, and no safety issues have been identified (ClinicalTrials.gov Identifier: NCT01085201). This is the first study to look at patient benefit with the long infusion of the drug. This drug has been used in laboratory experiments and information from those other research studies suggests that this drug may help to protect the body from damage caused by sickle-shaped cells in this research study. In this research study, the investigators are specifically looking to see if Regadenoson is an effective treatment for pain crises and acute chest syndrome in SCD. | Sickle Cell Anemia | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital and Research Center at Oakland, Oakland, California, 94609, United States|University of Illinois at Chicago, Chicago, Illinois, 60612, United States|Johns Hopkins University, Baltimore, Maryland, 21205, United States|Boston Children's Hospital, Boston, Massachusetts, 02215, United States|Brigham and Women's Hospital, Boston, Massachusetts, 02215, United States|Dana-Farber Cancer Institute, Boston, Massachusetts, 02215, United States|Wayne State University/Karmanos Cancer Institute, Detroit, Michigan, 48201, United States|Washington University in St. Louis, Saint Louis, Missouri, 63110, United States|Duke University, Durham, North Carolina, 27705, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States|Baylor College of Medicine, Houston, Texas, 77030, United States|Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| Sildenafil Therapy for Pulmonary Hypertension and Sickle Cell Disease | This study will examine whether the drug sildenafil can lower blood pressure in the pulmonary artery (the blood vessel that leads from the heart to the lungs) in patients with sickle cell disease and pulmonary hypertension (high blood pressure in the lungs). It will see if this treatment can reduce disease complications, such as shortness of breath, pain crisis, pneumonia, and increase survival. Patients 12 years of age and older with sickle cell disease and pulmonary hypertension may be eligible for this study. Participants are randomly assigned to receive sildenafil or placebo (sugar pill) for 16 weeks. Before starting treatment, patients have baseline studies, including a pregnancy test for females of childbearing age; a chest x-ray; pulmonary function tests to measure how much air the patient can breathe in and out; an echocardiogram (heart ultrasound); a 6-minute walk test to measure exercise capacity; a quality-of-life assessment and a pain inventory. Patients with moderate to severe pulmonary hypertension undergo heart catheterization to evaluate the severity of hypertension before beginning sildenafil therapy. During treatment, patients are monitored with the following: * Blood tests: weeks 6, 10 and 16. * Echocardiogram: weeks 6 and 16. * 6-minute walk test: weeks 6, 10 and 16. * Measurements of weight, blood pressure and heart rate: weeks 6, 10 and 16. * Pregnancy test for women of childbearing age: weeks 6, 10 and 16. * Pain questionnaire once a day for a week: weeks 6 and 1.0 * Quality-of-life questionnaire: week 16. * Heart catheterization: week 16 for patients with moderate to severe hypertension. At the end of the 16-week period, patients may opt to continue to receive sildenafil and monitoring in an open-label phase of the study for up to 1 year. | Sickle Cell Disease|Pulmonary Hypertension | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital, Oakland, Oakland, California, 94609, United States|University of Colorado, Denver, Colorado, 80220-3706, United States|Howard University Hospital, Washington, District of Columbia, 20060, United States|University of Illinois at Chicago, Chicago, Illinois, 60612, United States|Johns Hopkins University, Baltimore, Maryland, 21205, United States|National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States|Albert Einstein College of Medicine, Bronx, New York, 10461, United States|Childrens Hospital, Pittsburgh, Pittsburgh, Pennsylvania, 15213-2583, United States|Imperial College London and Hammersmith Hospital, London, United Kingdom |
| Dose-Escalation Study of SCD-101 in Sickle Cell Disease | The purpose of this study is to determine the safety and clinical effects of SCD-101 when given to adults with sickle cell disease. | Sickle Cell Disease|Sickle-Beta Zero Thalassemia | ALL | ADULT | King's County Hospital, Brooklyn, New York, 11203, United States |
| Crizanlizumab Improves Tissue Oxygen Supply Demand Matching in Patients With Sickle Cell Anemia | Hypothesis Efficient unloading of oxygen to regions of high metabolic demand requires a healthy microvasculature to sense local oxygen tension and regulate flow, accordingly. In sickle cell disease patients, the investigators have demonstrated oxygen supply-demand mismatch, or SDM, in proportion to anemia severity. SDM occurs in both the peripheral circulation and the brain, and four characteristics: 1) Hyperemia beyond expected for the level of anemia, 2) Corresponding loss of vascular dilatory reserve, 3) Impaired oxygen unloading to the tissues, and 4) Tissue hypoxia. In sickle cell disease, red blood cell (RBC) and white blood cell (WBC) adhere to vascular endothelium triggering transient or irreversible microvascular damage as well as releasing vasoactive substances that contribute to microvascular dysregulation. The investigators postulate that ongoing microvascular damage/dysregulation in the setting of increased total blood flow contributes to SDM. The investigators believe SEG101, by lowering RBC and WBC adhesion to the microvasculature, will improve SDM and tissue oxygenation. Objectives * Primary - The investigators will test whether SEG101 improves SDM in patients with sickle cell anemia by measuring the change in tissue oxygenation measured by near infrared spectroscopy (NIRS). * Secondary/Exploratory - The investigators will identify end-organ disease and whether improvement of SDM by SEG101 occurs in patients with sickle cell anemia. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | |
| Efficacy of Vorinostat to Induce Fetal Hemoglobin in Sickle Cell Disease | Sickle Cell Disease (SCD) is a hereditary anemia that causes the red blood cells to change their shape from a round and doughnut-like shape to a half-moon/crescent, or sickled shape. People who have SCD have a different type of hemoglobin (protein that carries oxygen). This different type of hemoglobin makes the red blood cells change into a crescent shape under certain conditions. Sickle-shaped cells are a problem because they often get stuck in the blood vessels blocking the flow of blood and can cause inflammation and injury to important areas of the body. All babies are born with hemoglobin called fetal hemoglobin (HbF). Soon after birth, HbF production slows down and another hemoglobin called adult hemoglobin (HbA) is made. Clinical studies have shown that increasing the amount of HbF in the blood may prevent sickling of the red blood cells. Vorinostat has been used in the treatment of cancers and in other research studies and information from those suggests that it may help treat SCD by increasing the amount of HbF in the blood. The purpose of this research study is to determine the effectiveness and safety of vorinostat when used to treat SCD. | Sickle Cell Disease|Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | Brigham and Women's Hospital, Boston, Massachusetts, 02115, United States|Children's Hospital Boston, Boston, Massachusetts, 02115, United States|Dana-Farber Cancer Institute, Boston, Massachusetts, 02115, United States |
| Stem Cell Transplantation With Identical Donors for Patients With Sickle Cell Disease | This protocol studied the effect of administration of a myeloablative pretransplant preparative regimen followed by an infusion of donor stem cells in children with severe sickle cell disease. The donor graft consisted of bone marrow or cord blood derived from a genetically matched sibling. The primary aim of the study was to evaluate how well the donated cells migrated to the bone marrow and begin producing healthy red blood cells, white blood cells and platelets (engrafted), how well the recipients immune system recovered, and assess any regimen related toxicities including a potentially life-threatening transplant related complication called graft-versus-host-disease or GVHD. | Sickle Cell Disease | ALL | CHILD, ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Comparing Acute Pain Management Protocols for Patients With Sickle Cell Disease | The goal of this pilot study is to improve emergency department (ED) pain management for adults with sickle cell disease. Sickle cell disease (SCD) is the most common genetic disorder in the United States, and occurs primarily among African Americans. Management of painful episodes associated with SCD, referred to as vaso-occlusive crises (VOC), is the most common reason for SCD patients to visit the ED. Currently, there is no standard approach to managing VOC pain in the ED that is widely accepted and used, and pain management for vaso-occlusive crisis in persons with SCD is very different between providers and not based on research. Many times, patients who come to the ED with sickle cell pain feel that they do not receive adequate pain control. If EDs could provide efficient, effective, safe, patient-centered analgesic management, it may be possible to improve pain management for adults with SCD experiencing a VOC. Guidelines for treating vaso-occlusive crises caused by sickle cell disease will soon be published by the National Heart, Lung and Blood Institute of the National Institutes of Health. These guidelines recommend patient-specific pain treatment protocols or a standardized pain management protocol for SCD when a patient does not already have a pain treatment protocol designed for them. The purpose of this pilot study is to compare these two ways to treat vaso-occlusive pain in the ED for adults with sickle cell disease, and to determine if a large randomized controlled trial is feasible and required. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Mount Sinai Hospital, New York, New York, 10029, United States|University of Cincinnati Medical Center, Cincinnati, Ohio, 45219, United States |
| The Montreal Cognitive Assessment.Test in Adults With Sickle Cell Disease | This study will assess the performances of the Montreal Cognitive Assessment (MoCA) to screen for cognitive impairment in adults with sickle cell anemia. The results of the MoCA and its subscores will be compared to a standardized neuropsychological evaluation using validated tests. | Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | Internal Medicine Department - Tenon, Paris, 75020, France |
| Arterial Function Parameters and Transcranial Doppler Velocity in Paediatric Patients With Sickle Cell Disease | Structural and functional changes in arteries are increasingly being recognized as significant features of sickle cell disease. This study aims to determine whether there are differences in arterial function parameters between children with sickle cell disease with normal and abnormal transcranial Doppler velocity. After informed consent is obtained, participants will have vascular, Transcranial Doppler, haematological and biochemical parameters measured. Researchers will compare children with sickle cell disease who have normal Transcranial Doppler velocity and no history of stroke with children with those who have an abnormal Transcranial Doppler velocity with or without a history of stroke to see if there are significant differences in arterial function parameters. | Sickle Cell Disease | ALL | CHILD | Caribbean Institute for Health Research, The University of the West Indies, Kingston, Saint Andrew, KGN20, Jamaica |
| Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment: HABIT | The investigators propose that culturally aligned community-based interventions in our multi-ethnic sickle cell disease (SCD) population, augmented by task-focused communication technology, can improve self-managed adherence to hydroxyurea (HU) by decreasing barriers to use, supporting parent-youth partnerships for chronic disease self-management and reinforcing the behavior of daily medication use. Culturally aligned community health workers (CHW) are a well-established means to support chronic disease self-management by underserved families, in partnership with medical homes. CHWs can identify and address multiple barriers and reinforce developmentally appropriate self-management to help youth reach and maintain their best fetal hemoglobin (HbF) levels. However, this strategy alone may be insufficient to achieve daily HU adherence. The investigators therefore propose a feasibility trial to test the feasibility and acceptability of a structured intervention of CHW support to address existing barriers to improve HU use, augmented by daily cue-based parent and youth text message reminders, to efficiently extend CHW family support and reinforce family partnerships for self-management. | Sickle Cell Disease | ALL | CHILD, ADULT | Montefiore Medical Center - Albert Einstein College of Medicine, Bronx, New York, 10461, United States|Columbia University Medical Center, New York, New York, 10032, United States |
| FOCUS for Pediatric Sickle Cell Disease and Cancer | Aim. Pilot FOCUS. A pilot randomized controlled trial will compare FOCUS to standard care. Investigators will randomize a total of 60 12- to 18-year-old patients to either FOCUS intervention (n=15 with SCD; n=15 with cancer) or treatment as usual (n=15 with SCD; n=15 with cancer). Randomization will be stratified to match patients based on age, sex, and medical condition (SCD type, cancer type). FOCUS participants will engage in the intervention and complete measures for 10 days post hospital discharge. Control participants will complete similar measures but not receive the intervention. Mixed qualitative and quantitative measures of feasibility, acceptability, and preliminary outcomes will be conducted to evaluate both the intervention and study procedures. | Pediatric Cancer|Sickle Cell Disease|Quality of Life|Depressive Symptoms|Coping Skills | ALL | CHILD, ADULT | Georgia State University, Atlanta, Georgia, 30302, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30342, United States |
| Near Infrared Spectroscopy in Sickle Cell Pediatric Patients | Endothelial dysfunction contributes to vaso-occlusion and acute pain in sickle cell disease. Near infrared spectroscopy (NIRS) technology can measure tissue oxygenation and endothelial function. The main objective of this study is to study the natural history of tissue muscle oxygenation using NIRS in pediatric sickle cell subjects experiencing acute pain and pediatric sickle cell patients in steady-state. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's National Medical Center, Washington, District of Columbia, 20010, United States |
| Far Infrared Radiation for Sickle Cell Pain Management | Most patients with sickle-cell disease have periodic intensely painful episodes. To manage this pain, we are proposing the drinking of at least 500 mL of water followed by far infrared radiation. | Anemia, Sickle Cell | ALL | CHILD, ADULT, OLDER_ADULT | The Centre for Incurable Diseases, Mississauga, Ontario, L5R 3G9, Canada |
| Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies | The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with Hemoglobinopathies to halt disease progression. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | University of Louisville, Louisville, Kentucky, 40202, United States|Duke University Medical Center, Durham, North Carolina, 27705, United States|St. Christopher's Hospital for Children, Pittsburgh, Pennsylvania, 19134, United States |
| Anticoagulation For Pulmonary Hypertension in Sickle Cell Disease | Sickle cell disease (SCD) is often referred to as a hypercoagulable state. However, the contribution of coagulation activation to the pathogenesis of SCD remains uncertain. Pulmonary hypertension (PHT) is a common complication associated with significant morbidity and mortality. Autopsy studies of SCD patients with PHT show evidence of in situ thrombosis involving pulmonary vessels, similar to findings in non-sickle cell patients with PHT. Anticoagulation has been reported to be of benefit in non-sickle cell patients with PHT. With the evidence of increased coagulation activation in SCD, PHT represents a clinical endpoint that may be used to evaluate the contribution of coagulation activation to the pathophysiology of SCD. The investigators hypothesize that increased thrombin generation, as well as platelet activation are central to the pathophysiology of SCD and contribute to the occurrence of several SCD-related complications, including PHT. As a consequence, treatment modalities that down-regulate thrombin generation would be expected to delay the progression of PHT and result in improved survival in patients with SCD. | Pulmonary Hypertension | ALL | CHILD, ADULT, OLDER_ADULT | University of North Carolina, Chapel Hill, North Carolina, 27599, United States |
| Nonmyeloablative Conditioning for Mismatched Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Disease | The aim of this study is to evaluate the overall safety and feasibility of using haploidentical or one antigen mismatch unrelated hematopoietic stem cell transplant (HSCT) for adult patients with severe sickle cell disease (SCD) who undergo a non-myeloablative preparative regimen consisting of total body irradiation (TBI), cyclophosphamide and alemtuzumab (and fludarabine for haplo-SCT only) and graft vs. host disease (GvHD) prophylaxis consisting of post-transplant cyclophosphamide (PT-Cy), mycophenolate mofetil (MMF), and sirolimus. The investigators anticipate that this approach will expand the donor pool and offer a safe and less toxic curative intervention. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Washington University School of Medicine, Saint Louis, Missouri, 63110, United States |
| A Study of IMR-687 in Subjects With Sickle Cell Disease | A Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of Alabama at Birmingham School of Medicine - 1917 Clinic, Birmingham, Alabama, 35233, United States|Arkansas Primary Care Clinic, Little Rock, Arkansas, 72204, United States|University of California San Diego Moores Cancer Center, La Jolla, California, 92093, United States|Center For Inherited Blood Disorders, Santa Ana, California, 92705, United States|The Oncology Institute Long Beach, Whittier, California, 90603, United States|University of Connecticut Health Main Building, Farmington, Connecticut, 06030, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30342, United States|The University of Illinois at Chicago College of Medicine, Chicago, Illinois, 60612-4333, United States|Johns Hopkins Hospital, Baltimore, Maryland, 21287, United States|Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|Newark Beth Israel Medical Center, Newark, New Jersey, 07112, United States|Weill Cornell Medicine - Center for Blood Disorders, New York, New York, 10021, United States|Baylor Scott & White Medical Center-Temple, Temple, Texas, 76508, United States|Virginia Commonwealth University Health - Ambulatory Care Center, Richmond, Virginia, 23219, United States|Korle Bu Teaching Hospital, Accra, PO Box 77, Ghana|Kintampo Health Research Centre, Kintampo, Brong-Ahafo Region, Ghana|Laiko General Hospital of Athens, Athens, Attica, 11526, Greece|University General Hospital of Patras, Patra, 26504, Greece|Ippokrateio General Hospital of Thessaloniki, Thessaloníki, 54642, Greece|Azienda Ospedaliero - Universitaria San Luigi Gonzaga, Turin, Orbassano, 10043, Italy|Fondazione Policlinico Universitario Agostino Gemelli, Roma, Rome, 00168, Italy|Ente Ospedaliero Ospedali Galliera, Genoa, 16128, Italy|Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, 20122, Italy|Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello, Palermo, 90146, Italy|Kenya Medical Research Institute - Kisumu, Kisumu, Nyanza, 40100, Kenya|Gertrude's Children's Hospital, Nairobi, 00100, Kenya|The Centre for Respiratory Diseases Research - Kenya Medical Research Institute, Nairobi, 00100, Kenya|Hopital Nini, Tripoli, North Governorate, Lebanon|American University of Beirut Medical Center, Beirut, 01107 2020, Lebanon|Chronic Care Center, Hazmiyeh, Lebanon|Hôpital d'Enfants Rabat, Rabat, 10100, Morocco|Hagaziekenhuis Van Den Haag - Leyweg, Den Haag, South Holland, 2545 AA, Netherlands|Sultan Qaboos University Hospital, Muscat, 123, Oman|Centre National De Transfusion Sanguine - Du Senegal, Dakar, 5002, Senegal|Hedi Chaker Hospital, Sfax, 3089, Tunisia|Centre Hôpital Universitaire Farhat Hached, Sousse, 4000, Tunisia|Centre National de Greffe de la Moelle Osseuse, Tunis, 1006, Tunisia|Hospital Aziza Othmana, Tunis, 1008, Tunisia|Jinja Regional Referral Hospital, Jinja, PO Box 43, Uganda|Uganda Cancer Institute, Kampala, PO Box 3935, Uganda|Makerere University College of Health Sciences, Kampala, PO Box 7072, Uganda|Joint Clinical Research Center - Lubowa, Kampala, Wskiso District, Uganda|Infectious Diseases Research Collaboration - Tororo, Tororo, 256, Uganda|University Hospitals Bristol NHS Foundation Trust, Bristol, England, BS1 3NU, United Kingdom|University College London Hospitals NHS, London, England, NW1 2PG, United Kingdom|Guy's and Saint Thomas' NHS Foundation Trust, London, England, SE1 9RT, United Kingdom|King's College Hospital NHS Foundation Trust, London, England, SE5 9RS, United Kingdom|Manchester University NHS Foundation Trust, Manchester, England, M13 9WL, United Kingdom|Oxford University Hospitals NHS Foundation Trust, Oxford, England, OX3 7LE, United Kingdom |
| Analysis of T-Cell Immune Reconstitution Following Allogeneic Hematopoietic BMT for Severe SCD | In this study, patient blood samples from NMA transplants will be provided by Pittsburgh, and samples from myeloablative transplants will be provided by Atlanta (comparative controls). Samples would be obtained pre- and post-BMT from the recipient at a total of 7 timepoints, and from the donor at one timepoint. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States |
| Dronabinol for Pain and Inflammation in Adults Living With Sickle Cell Disease | This study is designed to address the feasibility of a randomized, double masked, cross-over study of dronabinol as a palliative agent in the treatment of pain, inflammation, and other complications of sickle cell disease (SCD). | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Yale New Haven Hospital Smilow Cancer Center, New Haven, Connecticut, 06510, United States |
| Hydroxyurea to Prevent Brain Injury in Sickle Cell Disease | This is a pilot study of hydroxyurea versus placebo to reduce central nervous system complications (abnormally fast blood flow to the brain, silent cerebral infarct or stroke) in young children with sickle cell disease. The investigators plan to identify children 12 to 48 months old without central nervous system complications and randomly assign 20 to treatment with hydroxyurea and 20 to treatment with placebo for 36 months. Neither the study doctors nor the participants will know which treatment they are receiving. | Sickle Cell Disease|Stroke | ALL | CHILD | University of Alabama, Birmingham, Alabama, 35233, United States|Johns Hopkins Hospital, Baltimore, Maryland, 21287, United States|Mercy Children's Hospital, Kansas City, Missouri, 64108, United States|St. Louis Children's Hospital, Saint Louis, Missouri, 63110, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States |
| Sickle Cell Anemia in an Arab Bedouin Village in the Northern Israel | Sickle cell anemia and sickle cell thalassemia are frequent diseases among the israeli arab population. The purpose of this study is to assess the clinical characteristics of the patients in one arab village and the laboratory characteristics in the carriers of this gene based in the screening for pregnant women that is carried out in the population of northern Israel. The results can be useful in order to institute universal screening for sickle cell anemia in northern Israel. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Pediatric Hematology Unit - HaEmek Medical Center, Afula, 18101, Israel |
| Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell AntibodY | This multicenter prospective study seeks to determine if daratumumab given, prior to HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus, can prevent pure red blood cell aplasia with an acceptable safety profile in patients with anti-donor red blood cell antibodies, achieving an event-free survival similar to transplanted patients without such antibodies. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's National Hospital, Washington, District of Columbia, 20010, United States |
| ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension Study | As safety information pertaining to the long-term use of HU remains incomplete in spite of the first safety study (ESCORT-HU), an extension of the latter is proposed. ESCORT-HU Extension study aims at evaluating the long-term safety of Siklos® focusing on some questions regarding its safety when used in current practice in adults and paediatric patients treated with Siklos® and followed for up to 5 years. The study will focus on the following concerns : occurrence and incidence of malignancies, leg ulcers, male fertility impairment and serious unexpected AEs causally related to Siklos®. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Amiens - Picardie Hospital Adults, Amiens, 80054, France|Amiens Picardie Hospital Children, Amiens, 80054, France|Clinique de l'Europe Amiens, Amiens, France|Angers Hospital Adults, Angers, 49933, France|Angers Hospital Center Children, Angers, 49933, France|Robert Ballanger Hospital Adults, Aulnay-sous-Bois, 93602, France|Robert Ballanger Hospital Children, Aulnay-sous-Bois, 93602, France|Avicenne Hospital Adults, Bobigny, 93009, France|Jean Verdier Hospital Children, Bondy, 93143, France|Bordeaux Hospital Adults, Bordeaux, 33000, France|Bordeaux Hospital Children, Bordeaux, 33000, France|Ambroise Paré Hospital Children, Boulogne-Billancourt, 92100, France|Tours Regional University Hospital Center Adults, Chambray-lès-Tours, 37170, France|Estaing Hospital Children, Clermont-Ferrand, 63003, France|Louis Mourier Hospital Adults, Colombes, 92700, France|Louis Mourier Hospital Children, Colombes, 92700, France|Sud Francilien Hospital Center Adults, Corbeil-Essonnes, 91100, France|Centre Hospitalier Sud Francilien, Corbeil-Essonnes, France|Sud Oise Public Hospital Group Adults, Creil, 60100, France|Henri Mondor Hospital Adults, Créteil, 94010, France|Intercommunal Hospital Center of Créteil Adults and Children, Créteil, 94010, France|Dreux Hospital Center Children, Dreux, 28102, France|Gonesse Hospital Children, Gonesse, 95500, France|Marne-La-Vallée Hospital Center Children, Jossigny, 77600, France|Grenoble Hospital Adults, La Tronche, 38700, France|Grenoble Hospital Children, La Tronche, 38700, France|Bicêtre Hospital Adults, Le Kremlin-Bicêtre, 94270, France|Hôpital Bicêtre, Le Kremlin-Bicêtre, France|CH du Mans, Le Mans, France|Jeanne De Flandre Hospital Children, Lille, 59037, France|Hôpital Saint-Vincent de Paul, Lille, France|Hôpital Mère-Enfant - CHU de Limoges, Limoges, France|Edouard Herriot Hospital Adults, Lyon, 69003, France|Hematology and oncology institute Children, Lyon, 69373, France|Timone Hospital Adults, Marseille, 13005, France|Timone Hospital Children, Marseille, France|Meaux Hospital Center Adults, Meaux, 77100, France|Meaux Hospital Center Children, Meaux, 77100, France|St Eloi Hospital Adults, Montpellier, 34070, France|Montpellier Hospital Children, Montpellier, 34295, France|Emile Muller Hospital Adults, Mulhouse, 68100, France|Nantes Hospital Children, Nantes, 44000, France|Nantes Hospital Adults, Nantes, 44093, France|Nice Hospital Children, Nice, 06200, France|Orléans Hospital Adults, Orléans, 45066, France|Armand Trousseau Hospital Children, Paris, 75012, France|Saint-Antoine Hospital Adults, Paris, 75012, France|Georges Pompidou European Hospital Adults, Paris, 75015, France|Necker Hospital Adults, Paris, 75015, France|Necker Hospital Children, Paris, 75015, France|Robert Debré Hospital Children, Paris, 75019, France|Centre hospitalier Perpignan, Perpignan, France|Poitiers Hospital Adults, Poitiers, 86020, France|American Hospital Children, Reims, 51100, France|Reims Hospital Adults, Reims, 51100, France|Pontchaillou Hospital Adults, Rennes, 35033, France|Charles Nicolle Hospital Adults, Rouen, 76000, France|Rouen Hospital Children, Rouen, 76000, France|Yves Le Foll. Hospital Center Children, Saint-Brieuc, 22000, France|Delafontaine Hospital Center Adults, Saint-Denis, 93200, France|Delafontaine Hospital Center Children, Saint-Denis, 93200, France|Saint-Quentin Hospital Center Adults, Saint-Quentin, 02321, France|Hautepierre Hospital Children, Strasbourg, 67098, France|Children Hospital, Toulouse, 31059, France|Toulouse University Institute of cancer Adults, Toulouse, 31059, France|Clocheville Regional University Hospital Center Children, Tours, 37000, France|Nancy Hospital Children, Vandoeuvre les nancy, 54500, France|Versailles Hospital Center Children, Versailles, 78157, France|CH de Cayenne, Cayenne, 97300, French Guiana|Centre hospitalier de Kourou Children, Kourou, 97387, French Guiana|Centre Hospitalier de l'Ouest Guyanais Franck Joly Adults, Saint-Laurent-du-Maroni, 97393, French Guiana|Centre Hospitalier de l'Ouest Guyanais Franck Joly Children, Saint-Laurent-du-Maroni, 97393, French Guiana|Charité Hospital Children, Berlin, Germany|Düsseldorf Hospital Children, Düsseldorf, Germany|Freiburg Hospital Children, Freiburg, Germany|Hamburg Hospital Children, Hamburg, Germany|Koblenz Hospital Children, Koblenz, Germany|Laiko General Hospital Adults, Athens, Greece|Hôpital RICOU - CHU Pointe-à-Pitre Abymes Children, Pointe-à-Pitre, 97159, Guadeloupe|Hôpital RICOU - CHU Pointe-à-Pitre Abymes, Pointe-à-Pitre, 97159, Guadeloupe|AUO Policlinico di Modena, Modena, Italy|Napoli Hospital Children, Napoli, Italy|Padova Hospital Children, Padova, Italy|Verona Hospital Children, Verona, Italy|CHU Fort de France Children, Fort-de-France, 97261, Martinique|CHU Martinique Adults, Le Lamentin, 97232, Martinique|CHU Sud Réunion, Saint-Pierre, Réunion |
| mAnaging siCkle CELl disEase Through incReased AdopTion of hydroxyurEa in Nigeria | Large knowledge gaps remain regarding strategies to promote the adoption of hydroxyurea (HU), particularly in sub-Saharan African countries including Nigeria, where more than 75% of annual sickle cell anemia births occur. The vast majority of people with SCD in Africa do not receive evidenced-based health care (e.g., newborn screening, health education, prophylaxis for infection, optimal nutrition and hydration, blood transfusion, transcranial Doppler screening, and HU therapy), despite its effectiveness in reducing SCD-related adverse outcomes and mortality. The use of HU in SSA is \<1% among SCD patients. The investigators' preliminary findings indicate that provider-level barriers are significant and must be addressed to improve HU adoption. To address HU adoption, the investigators will use the NIH-funded study (e.g., Realizing Effectiveness Across Continents with Hydroxyurea (REACH) Clinical Trial (NCT01966731)) that developed an evidence-informed, clinical, practical, and easy-to-follow algorithm to 1) Screen patients for sickle cell disease (SCD), 2) Initiate HU treatment, and 3) Maintain HU dosage over time (SIM) for the improved management of SCD as our intervention. The Nigerian government released guidelines supporting the SIM intervention for HU adoption for improved SCD management, and HU is on the list of essential medicines for Nigeria. The investigators' implementation strategy for improving SCD management in Nigeria uses a practical and replicable evidence-based task-sharing strategy, TAsk-Strengthening Strategy for Hemoglobinopathies (TASSH), adopted from the TAsk-Strengthening Strategy for Hypertension control (TASSH) trials in Ghana and Nigeria containing the essential components of i) Training healthcare workers/providers to be more patient-centered in clinical consultations, ii) Clinical reminders, and iii) Practice facilitation (TCP) known as (TASSH TCP) for SCD management. Using a sequential exploratory mixed-methods study design, the investigators will conduct this study using the Exploration, Preparation, Implementation, and Sustainment (EPIS) framework in four sequential phases to assess the effectiveness of SIM adoption by providers in the context of the TASSH TCP implementation strategy in Nigeria. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | |
| Intravenous Magnesium for Sickle Cell Vasoocclusive Crisis | The purpose of this study is to determine the safety and efficacy of intravenous magnesium in shortening the duration of a pain crisis and to determine the health-related quality of life and short term outcomes of children treated with intravenous magnesium during an acute pain crisis. | Sickle Cell Disease | ALL | CHILD, ADULT | Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States|Johns Hopkins Hospital, Baltimore, Maryland, 21287, United States|Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|Nationwide Children's Hospital, Columbus, Ohio, 43205, United States|Children's Hospital of Philadelphia Research Institute, Philadelphia, Pennsylvania, 19104, United States|University of Pittsburgh, Pittsburgh, Pennsylvania, 15213, United States|Baylor College of Medicine, Houston, Texas, 77030, United States|Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| Penicillin Prophylaxis in Sickle Cell Disease (PROPS) | To determine whether the regular daily administration of oral penicillin would reduce the incidence of documented infection due to Streptococcus pneumoniae in children with sickle cell anemia. | Anemia, Sickle Cell|Hematologic Diseases|Hemoglobinopathies|Infection (S. Pneumoniae)|Pneumonia | ALL | CHILD | |
| Study Exploring the Effect of Crizanlizumab on Kidney Function in Patients With Chronic Kidney Disease Caused by Sickle Cell Disease | The goal of the study was to evaluate descriptively the effect of crizanlizumab + standard of care and standard of care alone on renal function in sickle cell disease patients ≥ 16 years with chronic kidney disease due to sickle cell nephropathy. | Sickle Cell Disease (SCD) | ALL | ADULT, OLDER_ADULT | University of Alabama Birmingham, Birmingham, Alabama, 35233, United States|University of Illinois Hospital and Health Sciences System ., Chicago, Illinois, 60612, United States|Our Lady of the Lake Regional Medic ., Baton Rouge, Louisiana, 70809, United States|East Carolina University BrodySchool of Med 3, Greenville, North Carolina, 27858, United States|Univ of Tenn Health Sciences Ctr, Memphis, Tennessee, 38163, United States|University of Texas Health Science Center at Houston, Houston, Texas, 77030, United States|Novartis Investigative Site, Rio de Janeiro, RJ, 20.211-030, Brazil|Novartis Investigative Site, São Paulo, SP, 01232-010, Brazil|Novartis Investigative Site, Porto Alegre, 90035-003, Brazil|Novartis Investigative Site, Creteil, 94010, France|Novartis Investigative Site, Paris 15, 75015, France|Novartis Investigative Site, Athens, 115 27, Greece|Novartis Investigative Site, Larisa, 41221, Greece|Novartis Investigative Site, Dublin 8, DO8, Ireland|Novartis Investigative Site, Tripoli, 1434, Lebanon|Novartis Investigative Site, Amsterdam, 1105 AZ, Netherlands|Novartis Investigative Site, Panama, 0801, Panama|Novartis Investigative Site, Barcelona, Catalunya, 08035, Spain|Novartis Investigative Site, Adana, 01250, Turkey|Novartis Investigative Site, Adana, 01330, Turkey|Novartis Investigative Site, Antakya / Hatay, 31100, Turkey|Novartis Investigative Site, London, SE1 9RT, United Kingdom|Novartis Investigative Site, London, SE5 9RS, United Kingdom|Novartis Investigative Site, London, W12 0HS, United Kingdom |
| Best Noninvasive Predictor of Renal Function in Assessing Adult Sickle Nephropathy | Background: Sickle cell disease is a common inherited blood disorder. Kidney disease is a major cause of problems in people with sickle cell disease. In order to identify kidney problems early and stop the progression of kidney disease, doctors need the most accurate tests to check kidney function. Researchers hope to understand more about how to test for kidney disease in people with sickle cell disease. Objective: To determine which of two different lab tests is the best to measure kidney function in adults with sickle cell disease. Eligibility: People 18 years and older who have sickle cell disease Design: Participants will be screened with a medical history and blood tests. Participants will have up to 3 visits. Participants will collect their urine in a special container over 24 hours. At the first visit, participants will have blood tests. They will bring their container of urine to the visit. They will have an iothalamate test. For the test, they will get a catheter: a small tube will be inserted into a vein. A special contract agent will be injected into the vein. Blood will be collected over the next 4 hours to test kidney function. Participants will return the next day for a second visit. They will have blood tests. They will have an MRI. For the MRI, they will like on a table that slides into a machine that takes pictures of the kidneys. They may have the MRI in a third visit. ... | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Sickle Cell Pain: Intervention With Capsaicin Exposure | This study evaluates the safety and feasibility of using high dose topical capsaicin patches for the treatment of neuropathic pain in pediatric patients with sickle cell disease, as well as the feasibility of using a number of tests for the evaluation and monitoring of neuropathic pain. The hypothesis, based on evidence obtained from studies in adults with neuropathic pain related to other diseases as well as a single previously published study of capsaicin in pediatric patients, is that capsaicin will be well tolerated in this population. Additionally, it is hypothesized that it is feasible to monitor changes in neuropathic pain via the testing listed below. | Neuropathic Pain|Sickle Cell Disease | ALL | CHILD, ADULT | Children's Hospital of Michigan, Detroit, Michigan, 48201, United States |
| Epidemiology of Silent and Overt Strokes in Sickle Cell Disease | Sickle Cell Disease (SCD) is a rare disease occurring in an estimated 100,000 individuals, often poor and underserved, in the US. Silent and overt strokes contribute significantly to morbidity in adults with SCD, resulting in functional impairment, challenges with school and job performance, and premature death. Five NIH-funded randomized controlled trials have identified therapies to prevent silent and overt strokes in children with SCD, including monthly blood transfusion therapy (for preventing initial and recurrent strokes) and hydroxyurea (for preventing initial strokes). Despite the observation that at least 99% of children with SCD in high-income countries reach adulthood, and approximately 60% of adults will experience one or more strokes (\~50% with silent strokes and \~10% with overt strokes), no stroke trials have established therapeutic approaches for adults with SCD. For adults with SCD, inadequate evidence-based guidelines exist for secondary stroke prevention strategies. Applying stroke prevention strategies in children may not be effective for stroke prevention in adults with SCD, particularly given the high rate of co-morbidities. Identifying subgroups of adults with SCD and higher incidence coupled with the contribution of established stroke risk factors in the general population (smoking, diabetes, obesity, renal disease) will provide the requisite data required for the first-ever phase III clinical trials focused on secondary stroke prevention in adults. | Anemia, Sickle Cell|Sickle Cell Disease|Stroke|Sickle Cell Thalassemia|Sickle Cell-Beta0-Thalassemia | ALL | ADULT, OLDER_ADULT | University of Alabama at Birmingham, Birmingham, Alabama, 35233, United States|Washington University School of Medicine, Saint Louis, Missouri, 63110, United States|Vanderbilt University Medical Center, Nashville, Tennessee, 37232-9000, United States|Vanderbilt University Medical Center, Nashville, Tennessee, 37232, United States |
| Arginine Treatment of Acute Chest Syndrome (Pneumonia) in Sickle Cell Disease Patients | This is a study to determine if oral arginine will increase nitric oxide in sickle cell disease (SCD) patients with acute chest syndrome (ACS). It will also assess the effects of arginine in the body and how the body uses nitric oxide in ACS. | Anemia, Sickle Cell|Pneumonia | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital Oakland, Oakland, California, 94609, United States |
| Pilot Evaluation of a Motivational Interviewing Intervention Targeting Adherence Behaviors in Youth With Sickle Cell Disease | Sickle cell disease (SCD) is a group of inherited blood disorders affecting 100,000 individuals in the United States. SCD often leads to complications, including pain crises and organ damage. Many individuals with SCD require medications (e.g., Hydroxyurea or Endari) that research has demonstrated reduce risk of complications and improve quality of life. Despite the need for strong medication adherence, adolescents and young adults (AYAs; 13-25 years) have the lowest adherence rates compared to other age groups. Efforts to reduce AYA non-adherence risk should include youth in earlier childhood and persist throughout the AYA developmental period, with the goal of maintaining adherence throughout childhood and young adulthood. Motivational Interviewing (MI) has been effective in increasing pediatric and adult medication adherence via in-person or telehealth delivery; however, researchers have yet to empirically evaluate MI for feasibility, acceptability, and/or efficacy in improving pediatric/AYA SCD medication adherence. The proposed feasibility trial will provide preliminary feasibility data for a newly developed MI+education intervention targeting medication adherence for pediatric and adolescents and young adults (AYA) patients who have sickle cell disease. This trial will also evaluate study design feasibility to inform a future randomized controlled trial (RCT). The investigators are interested in delivering the intervention to AYA patients and to parents of younger children who have sickle cell disease because the investigators anticipate that establishing strong adherence in younger childhood could prevent future non-adherence during the AYA developmental period. Participants will include 13-22 year-old patients with sickle cell disease as well as parents of 0-22 year-old patients with sickle cell disease. The investigators will randomize ten families to a 4-session telehealth MI+education intervention and five families to a one-session education-only control condition. All participants will complete assessments at three times. Intervention participants will complete the T2 assessment at their last intervention session (week 4-8), and the T3 assessment 16-20 weeks after study enrollment. Education arm participants will complete T2 assessments 4-8 weeks after study enrollment and will complete T3 assessments 16-20 weeks after study enrollment. Primary outcomes include intervention feasibility and acceptability and study design feasibility. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Johns Hopkins All Children's Hospital, Saint Petersburg, Florida, 33701, United States |
| Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy | This is a multi-center, long-term safety and efficacy follow-up study for subjects with sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies. After completing the parent clinical study (approximately 2 years), eligible subjects will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in the study. | Sickle Cell Disease | ALL | CHILD, ADULT | University of Alabama, Birmingham, Alabama, 35294, United States|UCSF Benioff Children's Hospital Oakland, Oakland, California, 94609, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States|Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611-2991, United States|Warren Grant Magnuson Clinical Center, Bethesda, Maryland, 20892, United States|University of Minnesota Masonic Children's Hospital, Minneapolis, Minnesota, 55454, United States|Hackensack University Medical Center, Hackensack, New Jersey, 07601, United States|Cohen Children's Medical Center, New Hyde Park, New York, 11040, United States|Columbia University Medical Center, New York, New York, 10032, United States|The University of North Carolina, Chapel Hill, North Carolina, 27514, United States|Duke University Medical Center, Durham, North Carolina, 27705, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|Baylor College of Medicine, Houston, Texas, 77030, United States|Hospital Necker, Paris, 75015, France |
| Effectiveness of Arginine as a Treatment for Sickle Cell Anemia | Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited genetic disease that can cause intense pain episodes. This study will evaluate the effectiveness of the nutritional supplement arginine at improving blood cell function and disease symptoms in people with SCD. | Anemia, Sickle Cell | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital of Oakland and Research Institute, Oakland, California, 94609, United States|University of California - San Francisco, San Francisco, California, 94143, United States|University of Colorado at Denver and Health Sciences Center--Sickle Cell Treatment and Research Center, Denver, Colorado, 80262, United States|Kosair Children's Hospital, Louisville, Kentucky, 40202, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|University of Mississippi Medical Center (Adult), Jackson, Mississippi, 39215, United States|University of Mississippi Medical Center (Pediatric), Jackson, Mississippi, 39215, United States|Montefiore Medical Center, Bronx, New York, 10463, United States|Children's Hospital of Montefiore, Bronx, New York, 10467, United States|University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, 27599, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|Children's Hospital of Oklahoma, Oklahoma City, Oklahoma, 73104, United States|Thomas Jefferson University, Philadelphia, Pennsylvania, 19107, United States|St. Christopher's Children's Research Hospital, Philadelphia, Pennsylvania, 19134, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19444, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|Children's Medical Center of Dallas, Dallas, Texas, 75390, United States |
| RHD Genotype Matched Red Cells for Anti-D | This is a pilot study to evaluate the feasibility and safety of providing RH genotype matched D+ Red Blood Cells (RBCs) to chronically transfused patients with sickle cell disease (SCD) who type D+ but have formed anti-D and are currently transfused with D- RBC (Red Blood Cell) units. | Sickle Cell Disease|Anti-D Antibodies | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States |
| Adolescent, Caregiver, and Young Adult Perspectives of the Transition From Pediatric to Adult Care for Sickle Cell Disease: A Preliminary Evaluation of the Sickle Cell Disease Transition Program | Sickle cell disease (SCD) is a debilitating genetic disorder affecting 70,000-100,000 Americans. It is frequently associated with very serious medical complications. For children with SCD, successfully transitioning to adult care is a vital step in ensuring continuity of care, managing their disease, and improving their health outcomes. Transition programs have been created to facilitate the transition process. However, few studies have assessed transition readiness and whether transition program components meet the transition needs of patients and families. The purpose of this study is to explore transitioning from pediatric care to adult care and to assess components of the SJCRH SCD Transition Program from three perspectives: adolescents with SCD, their caregivers, and young adults with SCD who have transitioned to adult care. Data collection methods will include focus groups, questionnaires, and checklists. Qualitative data analysis procedures will be used to examine the data. | Sickle Cell Disease | ALL | CHILD, ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Medication Adherence in Youth With Sickle Cell Disease (SCD) | Youth diagnosed with sickle cell disease (SCD) may have difficulty taking medication as prescribed (adherence). Hydroxyurea (HU) is one medication that youth may take to help manage SCD. Electronic adherence monitoring is widely considered the gold standard in objective adherence measurement. These monitors provide continuous, real-time records of medication adherence and reveal problematic behavior patterns, including underdosing, overdosing, delayed dosing, "drug holidays" (i.e. where individuals do not take medications for a specified interval of time), and "white coat" adherence (i.e., a pattern of drug adherence as a function of time where individuals display good adherence immediately before and after clinic attendance with worsening adherence in the period between). Overall, electronic adherence measures are considered valid, reliable, and accurate, with clear advantages over pharmacy refill records, physician estimates and self-report measures. Currently, only one electronic measure capable of monitoring medications in both pill and liquid form is being manufactured: WisePill and WiseBag. While data are limited regarding its validity and reliability, preliminary data support the use of Wise technology to measure adherence to medication. The current study will determine the Wise device's ability to feasibly measure adherence to liquid and solid form HU medication in a pediatric SCD population. | Sickle Cell Disease | ALL | CHILD | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Sex Hormones and Inflammatory Biomarkers in Patients With Sickle Cell Disease | This study aims to characterize sex differences in the pathophysiology of vaso-occlusive crises (VOC) occurring among individuals with sickle cell disease (SCD). * The study will compare CRP and other biomarkers between females with SCD in the follicular phase of the menstrual cycle and males with SCD. * The study will explore potential sex differences in biomarker changes between females and males with SCD during and following resolution of VOC. * The study will compare neutrophil and platelet adhesion to the endothelium and real time fibrin deposition in the blood. | Sickle Cell Disease|Vaso-occlusive Crisis | ALL | ADULT | Penn Medicine University City, Philadelphia, Pennsylvania, 19104, United States|Pennsylvania Hospital, Philadelphia, Pennsylvania, 19107, United States|Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, 19143, United States |
| Desmopressin as a Therapy for Bedwetting in Children With Sickle Cell Disease | This study assesses if using the medication desmopressin will decrease nightime bedwetting in children with sickle cell disease. | Nocturnal Enuresis|Anemia, Sickle Cell | ALL | CHILD, ADULT | Children's Hospital at Montefiore, Bronx, New York, 10467, United States |
| Assessing the Safety of Buprenorphine in People With Sickle Cell Disease | This study will assess the safety of changing pain medications (opioids) adult sickle cell patients take to another type of medication therapy (buprenorphine). Patients will be asked questions about their quality of life. Other tools for assessment will also be administered. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Johns Hopkins Hospital, Baltimore, Maryland, 21205, United States |
| A Brief Laboratory-Based Hypnosis Session for Pain in Sickle Cell Disease | This pilot study will assess the effects of a brief laboratory-based guided imagery procedure on responses to pain in patients with sickle cell disease (SCD) and healthy controls. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | University of California, Los Angeles, Los Angeles, California, 90095, United States |
| A Stratified Sickle Event Randomized Trial (ASSERT) | The purpose of this study is to compare the effects of ICA-17043 to placebo with or without hydroxyurea (an oral drug used for treatment of sickle cell disease) in patients with sickle cell disease who have had 2 or more acute sickle-related painful crises requiring a visit to a medical facility within the past 12 months. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Mobile, Alabama, United States|Little Rock, Arkansas, United States|Davis, California, United States|Los Angeles, California, United States|Oakland, California, United States|Denver, Colorado, United States|Hartford, Connecticut, United States|Washington, District of Columbia, United States|Holly Hill, Florida, United States|Hollywood, Florida, United States|Jacksonville, Florida, United States|Augusta, Georgia, United States|Savannah, Georgia, United States|Chicago, Illinois, United States|Iowa City, Iowa, United States|New Orleans, Louisiana, United States|Shreveport, Louisiana, United States|Baltimore, Maryland, United States|Boston, Massachusetts, United States|Detroit, Michigan, United States|Jackson, Mississippi, United States|Kansas City, Missouri, United States|Las Vegas, Nevada, United States|Hackensack, New Jersey, United States|New Brunswick, New Jersey, United States|Newark, New Jersey, United States|Bronx, New York, United States|Brooklyn, New York, United States|Chapel Hill, North Carolina, United States|Durham, North Carolina, United States|Greenville, North Carolina, United States|Winston-Salem, North Carolina, United States|Cincinnati, Ohio, United States|Cleveland, Ohio, United States|Philadelphia, Pennsylvania, United States|Pittsburgh, Pennsylvania, United States|Charleston, South Carolina, United States|Memphis, Tennessee, United States|Nashville, Tennessee, United States|Houston, Texas, United States|San Antonio, Texas, United States|Richmond, Virginia, United States|Porto Alegre, Brazil|Rio de Janeiro, Brazil|Sao Paulo, Brazil|Creteil, France|Kingston, Jamaica|Port of Spain, Trinidad and Tobago|London, United Kingdom |
| A Study of Oral L-citrulline in Sickle Cell Disease | Sickle cell disease is a genetic red blood cell disorder that can result in blocking of the small blood vessels from sickle shaped red blood cells. This causes pain, the main feature of sickle cell disease. Also, low amounts of nitric oxide can occur in sickle cell disease, a substance important for widening the blood vessel wall and therefore preventing blockage of the small blood vessels. Citrulline is a drug that is known to increase nitric oxide. This is a phase I study of citrulline given by mouth to evaluate the safety, tolerability and appropriate dosing of this medication for individuals with sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT | University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States |
| Losartan for Diffuse Myocardial Fibrosis in Sickle Cell Disease | This study is a pilot, phase II, open-label study of the angiotensin II receptor blocker, losartan, in patients with Sickle Cell Disease (SCD) 6 years or older for 12 months. The investigators will enroll 24 patients with SCD over the course of 1 year with a goal to complete all study procedures in 2 years. The short-term goal is to obtain clinical pilot data regarding the safety and efficacy of losartan in stabilizing or decreasing extracellular volume fraction (ECV) after 12 months of therapy. | Sickle Cell Disease|Diffuse Myocardial Fibrosis | ALL | CHILD, ADULT, OLDER_ADULT | Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States |
| Prevalence and Pathophysiology of Systemic Arterial Pressure Abnormalities in Childhood Sickle Cell Disease | It is usually found that the blood pressure of adults with sickle cell disease is lower than in non-sickle cell patients. On the other hand, three recent prospective studies in children with sickle cell disease show prevalence of hypertension diagnosed by ambulatory blood pressure measurement (ABPM) ranging from 32 to 45% but on small numbers of patients (n = 54 at most). This hypertension appears to affect kidney function and has been previously associated with the risk of hemorrhagic stroke. It is therefore important to know the prevalence of hypertension in children with sickle cell disease and to determine its mechanisms. The factors which could explain this high prevalence are the increase in arterial stiffness and the increase in systemic vascular resistance linked to the alteration of the sympathovagal balance contributing to the regulation of vascular tone. Indeed, a disturbance of this balance with an increase in vasoconstrictor sympathetic tone has already been found. Hypothesis: In a subgroup of sickle cell children there is systemic hypertension (prevalence: main objective) linked to the alteration of the sympathovagal balance already established during sickle cell disease (increase in sympathetic tone and decrease in parasympathetic tone) affecting systemic vascular resistance (secondary pathophysiological objectives). | Sickle Cell Disease | ALL | CHILD, ADULT | Robert Debre Hospital, Paris, 75019, France |
| A Study Aimed to Assess the Needs of Subjects With Sickle Cell Disease and Healthcare Professionals Managing Sickle Cell Disease Patients in Selected Nigerian Centres | This pilot research is aimed to assess the needs of patients and health workers involved in Sickle Cell Disease (SCD) management in Nigeria. To achieve this, a questionnaire will be administered to SCD patients or parents of children affected by SCD. Another questionnaire will be administered to doctors and nurses working with SCD patients. A focus group discussion with patients/parents willing to participate will be also scheduled. Participants from the following centres will be involved: Barau Dikko Teaching Hospital Kaduna State University, Ahmadu Bello University Teaching Hospital Zaria, National Hospital Abuja, Federal Medical Centre Katsina. Data will be qualitatively and quantitatively analysed and presented as aggregated data. Consent from all the study participants will be sought. Questionnaires will be coded and no personal data will be disclosed to authorised third parties. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | National Hospital Abuja, Abuja, Nigeria|Barau Dikko Teaching Hospital, Kaduna State University, Kaduna, Nigeria|Federal Medical Centre Katsina, Katsina, Nigeria|Ahmadu Bello University Teaching Hospital Zaria, Zaria, Nigeria |
| Hypnosis to Manage Pain and Symptoms in Patients With Sickle Cell Disease | This study will examine whether hypnosis can reduce the frequency and intensity of pain in patients with sickle cell disease. Patients 18 years of age and older with sickle cell disease and a history of pain associated with their disease may be eligible for this study. Participants are interviewed to assess their frequency and intensity of pain, sleep quality, coping strategies, mood and anxiety and are then randomly assigned to study Group A or B (see below). All participants are given pain diaries to complete at home and turn in at each clinic visit. They undergo the following procedures: Group A Weeks 1-4: Receive weekly 60-minute hypnosis sessions, in which they are given suggestions for relieving pain, reducing anxiety, improving sleep and enhancing their health and well-being. The sessions are audio- and videotaped. Week 5: Are interviewed to assess pain, sleep, coping strategies, mood and anxiety. Week 6: Receive a DVD player and DVD with instruction on how to perform self-hypnosis. They practice hypnosis at home as often as needed, but at least once a day. They record in a pain diary in the morning and the evening their amount of pain, medication use, school or work attendance, quality and amount of sleep and number of times they use self-hypnosis. Weeks 8, 10 and 12: Turn in their pain diaries and have a pain assessment. Week 12: Are assessed for how they respond to the hypnosis. Group B Weeks 1-4: Receive weekly 60-minute sessions of education about sickle cell disease. Week 5: Are interviewed to assess pain, sleep, coping strategies, mood and anxiety. Week 6: Turn in their daily pain diaries and receive a DVD player and DVD that contains educational materials about sickle cell disease. Weeks 8, 10 and 12: Turn in their pain diaries and have a pain assessment. Weeks 13-24: Follow the procedures described in weeks 1-12 for Group A. | Sickle Cell Disease|Pain Management | ALL | ADULT, OLDER_ADULT | Howard University Hospital, Washington, District of Columbia, 20060, United States|National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease | The purpose of this study is to examine how abnormal blood flow in the small vessels (microvessels) of the heart, muscle and kidney in paroxysmal nocturnal hemoglobinuria (PNH) or sickle cell disease leads to poor functioning of the heart and kidney. To test this question, the investigators will perform imaging tests (contrast ultrasound perfusion imaging) to look at the flow and function of these microvessels and compare this information to heart and kidney function. To further look at this question, patients who have PNH will be studied before and after starting a new drug (Soliris) that decreases damage to blood cells. In patients with sickle cell disease, patients will be studied at baseline (not during a pain crisis) and also during a pain crisis if one develops. | Rheologic Disease|Sickle Cell Disease|Paroxysmal Nocturnal Hemoglobinuria | ALL | ADULT, OLDER_ADULT | Oregon Health & Science University, Portland, Oregon, 97239, United States |
| Effect of NUV001 Supplementation in Patients Suffering From Sickle Cell Disease (SCD) | This is a pilot study of daily dosing of NUV001 as a dietary supplement in 12 sickle cell disease patients with 3 months of follow-up plus 1 month after supplementation.The present study is designed to evaluate, first, the safety and tolerability parameters as well as to measure the plasma and urinary residues of daily oral doses of NUV001. Secondly, the study will evaluate the impact of NUV001 on biological parameters and quality of life of patients. | Sickle Cell Disease | ALL | ADULT | Aphm Hopital La Timone Adultes Sce Medecine Interne (Umap), Marseille, 13005, France |
| A Reduced Toxicity Allogeneic Unrelated Donor Stem Cell Transplantation (SCT) for Severe Sickle Cell Disease | Majority of patients who are eligible for allogeneic HSCT for cure of severe sickle cell disease lack a matched family donor. This study aims for cure of sickle cell disease by performing unrelated donor (outside family) allogeneic HSCT. Donors or unrelated cord blood units will be selected from the NMDP database. It is designed to estimate the safety of a novel reduced toxicity, yet an immunosuppressive and myeloablative preparative regimen. This is meant for patients \<21 years old who have severe complications from sickle cell and do not have matched sibling donors in the family to undergo stem cell transplant. Patients will undergo transplant using unrelated donor stem cells after receiving the protocol therapy. They will be followed for 1 year to monitor for engraftment of donor cells and complications like graft versus host disease (GVHD), infections and death. | Sickle Cell Disease | ALL | CHILD, ADULT | Nationwide Children's Hospital, Columbus, Ohio, 43205, United States |
| Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders | The primary objectives of this prospective mixed-method interview study are to use semi-structured interviews in parents of sickle cell disease (SCD) patients to describe parental attitudes of research involving genomic sequencing, including concerns about participation and expectations from researchers and to use surveys to quantitatively measure genetic/genomic knowledge, trust in health care provider, and literacy/numeracy ability in parents of children with SCD and adolescents with SCD. Investigators hope to use the results of the planned surveys and interviews to reduce the risk of misunderstanding about DNA and genetic research and build strong relationships between SCD families and researchers in the future, and to design educational information and study materials that will help parents with children with SCD understand important details about DNA and genetic research. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Motivations, Expectations, and Decision-making of Sickle Cell Patients in Clinical Research | Background: Sickle cell disease is an inherited blood disorder. People with this disease have a problem with their hemoglobin. That is a protein in red blood cells that carries oxygen in the body. Some people with this disease are enrolled in research at NIH. Researchers want to learn more about the thoughts and opinions of those people. This may improve the way researchers explain clinical studies, risks, and benefits to people with the disease. Objective: To learn about the motivations, decisions, and experiences in clinical research of people with sickle cell disease. Eligibility: Adults ages 18 and older who have sickle cell disease. They must be in an NIH study on this condition. They must have been invited to join either a gene therapy or peripheral blood stem cell transplantation study. Design: Participants will have 1 interview. It will be done in a quiet room in the NIH Clinical Center or by video call. It will take about 60 minutes. The interview will be audio-recorded if the participant agrees. Participants will be asked about: * Their experiences with and thoughts on sickle cell disease * Their decision to participate in clinical research * Factors that may have affected their decision to participate. These may include family, disease history, or faith. Participants may complete a few brief questionnaires. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Neurovascular Determinants of Cognitive Function in Adults With Sickle Cell Disease | Cognitive impairment is a poorly understood, serious, and emerging complication for adult patients with sickle cell disease. Because there is extensive microvascular damage from oxidative damage in sickle cell disease, the investigators hypothesize that this is also present in the cerebral microvasculature to cause cognitive impairment. The investigators plan to test this by correlating markers of inflammation and oxidative damage with cognitive performance and 7 Tesla brain MRI microvascular findings in these patients, with the long term goal of understanding the mechanisms and risk factors of cognitive impairment in sickle cell disease. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | |
| Links Between Cognitive Functions and Clinical, Biological and Neuroradiological Outcomes in Adults With Sickle Cell Disease. | Sickle cell disease (SCD) is an inherited blood disorder. Symptoms include acute and chronic complications. Due to progress in SCD care, patients with SCD are living longer than before and we focus more attention in chronic complications. Children with SCD experience worse cognitive functions than healthy children, and fewer is known about cognitive functions in adults. Studies suggest lower cognitive performance in SCD, mostly in executive functions and processing speed, but the biological and anatomical substrates of cognitive decline are not yet well established in SCD. Often times, cognitive impairments and cerebral disorders are not diagnosed and treated in adults with SCD. The main objective of this study is to propose a deep neuropsychological assessment in adults with SCD and cognitive complaints and to highlight links between cognitive functions and clinical, biological and neuroradiological markers. The hypothesis of this study is that cognitive functions are associated with severity of the SCD, with bood abnormalities, with MRI markers and Transcranial Doppler (TCD) markers of cerebrovascular disease. The secondary objective of this study is to validate a brief cognitive assessment tool (BEARNI tool) in adults with SCD. This study is an observational cross-sectional study that will enroll adults with SCD and cognitive complaint. | Sickle Cell Disease|Drepanocytosis | ALL | ADULT, OLDER_ADULT | Hôpital Edouard Herriot, Bron, 69437, France |
| Early Human Leukocyte Antigen (HLA) Matched Sibling Hematopoietic Stem Cell Transplantation | This study aims to enroll 58 pre-adolescent (\<13 years) pediatric participants with sickle cell disease (SCD) who have a pre-adolescent sibling bone marrow donor. All participants will go through a pre-transplant evaluation to find out if there are health problems that will keep them from being able to receive the transplant. It usually takes 2 to 3 months to complete the pre-transplant evaluation and make the arrangements for the transplant. Once they are found to be eligible for transplant, participants will be admitted to the hospital and will start transplant conditioning. Conditioning is the chemotherapy and other medicines given to prepare them to receive donor cells. It prevents the immune system from rejecting donor cells. Conditioning will start 21 days before transplant. Once they complete conditioning, participants will receive the bone marrow transplant. After the transplant, participants will stay in the hospital for 4-6 weeks. After they leave the hospital, participants will be followed closely in the clinic. Outpatient treatment and frequent clinic visits usually last 6 to 12 months. Routine medical care includes at least a yearly examination for many years after transplant by doctors and nurses familiar with sickle cell disease and transplant. The researchers will collect and study information about participants for 2 years after transplant. | Sickle Cell Disease | ALL | CHILD | Children's Hospital of Alabama, Birmingham, Alabama, 35233, United States|Phoenix Children's, Phoenix, Arizona, 85016, United States|Yale University, Yale Cancer Center, New Haven, Connecticut, 06520, United States|Children's National Hospital, Washington, District of Columbia, 20010, United States|Children's Healthcare of Altanta, Atlanta, Georgia, 30322, United States|University of Chicago, Chicago, Illinois, 60637, United States|Riley Children's Health/Indiana University, Indianapolis, Indiana, 46202, United States|Dana-Farber Cancer Institute/Boston Children's Hospital, Boston, Massachusetts, 02215, United States|Washington University School of Medicine, Saint Louis, Missouri, 63110, United States|Hackensack University Medical Center, Hackensack, New Jersey, 07601, United States|University of North Carolina Medical Center, Chapel Hill, North Carolina, 27514, United States|Atrium Health Levine Cancer Institute, Charlotte, North Carolina, 28204, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Cancercare Manitoba/Winnipeg Children's Hospital, Winnipeg, Manitoba, Canada|Centre Hospitalier Universitaire Sainte-Justine, Montréal, Quebec, Canada |
| Daily Vitamin D for Sickle-cell Respiratory Complications | This study aims to answer the question whether daily oral vitamin D supplementation can reduce the risk of respiratory or lung complications in children and adolescents with sickle cell disease. Respiratory problems are the leading causes of sickness and of death in sickle cell disease. The investigators hypothesize that daily oral vitamin D3, compared to monthly oral vitamin D, will rapidly increase circulating vitamin D3, and reduce the rate of respiratory complications by 50% or more within the first year of supplementation in children and adolescents with sickle cell disease. This study is funded by the FDA Office of Orphan Products Development (OOPD). | Sickle Cell Disease|Anemia, Sickle Cell|Anemia, Hemolytic, Congenital|Respiratory Tract Diseases|Respiration Disorders|Acute Chest Syndrome|Lung Diseases|Asthma|Respiratory Tract Infections|Nutrition Disorders|Deficiency Diseases Vitamin|Vitamin D Deficiency | ALL | CHILD, ADULT | Columbia University Medical Center, New York, New York, 10032, United States |
| An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease | The primary objective of this study is to evaluate a potential behavioral intervention (MED-Go app). To meet this objective, the researchers will conduct a pilot randomized controlled trial to test the feasibility and acceptability of MED-Go app in adolescents and young adults (AYA) with sickle cell disease (SCD). The long-term goal of this research is to promote medication adherence behavior and improve health outcomes in AYA with SCD. | Sickle Cell Disease|Sickle Beta Zero Thalassemia|Sickle B+ Thalassemia|Sickle Cell Hemoglobin C | ALL | CHILD, ADULT | Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States |
| SMYLS: A Self-management Program for Youth Living With Sickle Cell Disease | The purpose of this study is to find out whether a web-based intervention using a mobile device is helpful for teens learning to care for and manage symptoms of sickle cell disease. The intervention lasts 12 weeks with a 3-month follow up period, and uses a smartphone or a tablet. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Medical University of South Carolina, Charleston, South Carolina, 29425, United States |
| Multi-Center Study of Iron Overload: Survey Study (MCSIO) | The purpose of this study is to demonstrate that a sufficient number of iron-overloaded thalassemia (THAL), Sickle Cell Disease (SCD)and Diamond Blackfan Anemia (DBA) populations with similar duration of chronic transfusion, and age at start of transfusions would be available for a confirmatory study. The study will examine the hypothesis that a chronic inflammatory state in SCD leads to hepcidin- and cytokine-mediated iron withholding within the RES (reticuloendothelial system), lower plasma NTBI (non-transferrin bound iron) levels, less distribution of iron to the heart in SCD. | Sickle Cell Disease|Thalassemia|Diamond-Blackfan Anemia | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital & Research Center Oakland, Oakland, California, 94609, United States|Georgia Regents University, Augusta, Georgia, 30912, United States|Children's Memorial Hospital, Chicago, Illinois, 60614, United States|Adult Comprehensive Sickle Cell Center, Duke University Medical Center, Durham, North Carolina, 27710, United States|Thomas Jefferson SCD Program, Philadelphia, Pennsylvania, 19107, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105-3678, United States|Universitätsklinikum Hamburg-Eppendorf, Hamburg-Eppendorf, Germany|UCL Cancer Institute, London, WC1E 6BT, United Kingdom |
| Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome | This study evaluates the safety of defibrotide in subjects with sickle cell disease (SCD)-associated acute chest syndrome (ACS). | Sickle Cell Disease|Acute Chest Syndrome | ALL | CHILD, ADULT | New York Medical College, Valhalla, New York, 10595, United States |
| Intravenous L-Citrulline for Vaso-occlusive Pain Episode in Sickle Cell Disease | The goal of this clinical trial is to learn if intravenous citrulline works to treat acute pain in hospitalized patients with sickle cell disease. It will also learn about the safety of intravenous citrulline. The main questions it aims to answer are: * Does intravenous citrulline decrease the duration of sickle cell pain during hospitalization * What medical problems do participants have when taking intravenous citrulline? Researchers will compare intravenous citrulline to a placebo (a look-alike substance that contains no drug) to see if intravenous citrulline works to treat acute pain. Participants will: * Receive baseline tests and intravenous citrulline for 16 hours during the hospital stay * After hospital discharge, visit the clinic in about 30 days for checkup and tests | Sickle Cell Disease|Vaso-occlusive Pain Episode | ALL | CHILD, ADULT | Children's National Hospital, Washington, District of Columbia, 20010, United States |
| Feasibility of a Stress Reduction Intervention Study in Sickle Cell Disease | Stress is known to trigger acute pain crisis of sickle cell disease (SCD). SCD is an inherited blood disorder that afflicts about 100,000 people in the United States, and is among the most common lethal genetic diseases in the United States. Though worldwide in distribution, in the US it is most commonly found in African Americans. Its best known complication is severe, recurrent relentless pain, often known as pain crisis. Non-drug treatment for SCD pain such as cognitive coping interventions have been shown to be effective for reducing SCD pain intensity, but they are complicated, multifaceted, and time-consuming. A simple and cost-effective alternative such as guided imagery (GI) could reduce the effect of stress on SCD pain. GI is an intervention where patients listen to and view audio-visual recordings while being directed to visualize themselves being immersed in that scene or scenario. There are no published studies on the use of GI as a simple stress coping intervention or tracking stress in a systematic manner as a trigger for SCD pain. | Sickle Cell Disease|Stress | ALL | ADULT, OLDER_ADULT | |
| Hydroxyurea and Erythropoietin to Treat Sickle Cell Anemia | This study will examine the use of hydroxyurea and erythropoietin for treating sickle cell disease in patients who also have kidney disease or pulmonary hypertension (high blood pressure in the lungs). Hydroxyurea increases production of fetal hemoglobin in the red blood cells of patients with sickle cell disease, reducing the amount of sickle cells that cause pain and other complications requiring hospitalizations. However, hydroxyurea treatment has limitations: patients with sickle cell disease who have developed kidney disease may not be able to get the full benefit of the medicine, and hydroxyurea alone may not be able to treat life-threatening complications such as pulmonary hypertension or stroke. This study will determine which of two dosing schedules of hydroxyurea and erythropoietin is more effective for treating patients with sickle cell disease who also have kidney disease or pulmonary hypertension, and will examine whether the two drugs can lower blood pressure in the lungs. Patients 18 years of age and older with sickle cell anemia and kidney disease or pulmonary hypertension, or both, may be eligible for this study. Candidates are screened with a medical history, physical examination, blood tests, a 6-minute walk test (test to see how far the subject can walk in 6 minutes), and echocardiogram (ultrasound of the heart to measure blood pressure in the lungs). Participants undergo the following tests and procedures: Stabilization Phase: Patients take 2 hydroxyurea tablets a day until their fetal hemoglobin levels stabilize, usually over 2 to 4 months. They have blood tests every 2 weeks to monitor hemoglobin and fetal hemoglobin levels. At some time during this period, they undergo a test to measure kidney function, in which they are injected with an iodine-containing dye and wear a small pump for 1 day that injects a small amount of dye under the skin over 24 hours. They come to the clinic for 2 or 3 blood tests collected over 4 hours. Sequence I (Standard): When the fetal hemoglobin levels have been stable for 2 months, patients have a repeat echocardiogram and 6-minute walk test. Erythropoietin is then added to the hydroxyurea regimen. It is given 3 days a week, as an injection under the skin, along with iron supplements. Patients have blood tests and blood pressure measurements every week or every other week. Patients with pulmonary hypertension have another echocardiogram and 6-minute walk test once the hemoglobin level is stable. Sequence II (Cycled): When hemoglobin levels have stabilized with hydroxyurea once a day and erythropoietin 3 times a week, the hydroxyurea is adjusted so that the amount taken in 7 days is "cycled" over 4 days, and the erythropoietin is cycled over 3 days, with the dose increased twice, every 3 to 4 weeks. Blood pressure and hemoglobin are monitored once or twice a month. Patients with pulmonary hypertension have another echocardiogram and 6-minute walk test once the hemoglobin level is stable. Patients who develop complications while taking the drugs have their treatment regimens adjusted as needed. | Sickle Cell Disease|Chronic Kidney Disease|Pulmonary Hypertension | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease | The purpose of this study is to determine the correct prasugrel dosage to be given to children with sickle cell disease (SCD). | Sickle Cell Disease | ALL | CHILD | For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Oakland, California, 94609, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Washington, District of Columbia, 20060, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Chicago, Illinois, 60614, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., New Orleans, Louisiana, 70112, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Boston, Massachusetts, 02115, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., St Louis, Missouri, 63104, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Chapel Hill, North Carolina, 27599, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Cincinnati, Ohio, 45229, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Pittsburgh, Pennsylvania, 15224, United States |
| Transplantation for Patients With Sickle Cell Disease From Mismatched Family Donors of Bone Marrow | The purpose of this study is to learn if it is possible and safe to treat persons with severe sickle cell disease (SCD) by bone marrow transplant (BMT) from human leukocyte antigen (HLA) half-matched related donors. Preparation before transplant includes the chemotherapy drugs hydroxyurea, fludarabine, thiotepa, anti-thymocyte globulin, and cyclophosphamide. It also includes radiation. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's Healthcare of Atlanta, Atlanta, Georgia, 30033, United States |
| Blood Flow and Pain Crises in People With Sickle Cell Disease | Background: - Many people with sickle cell disease have repeated episodes of severe pain that lasts for days, requiring hospital care. These episodes, called pain crises, may be caused by changes in blood flow. Researchers want to study blood flow in people with sickle cell disease who are having a pain crisis and compare it with their blood flow after the pain crisis has resolved. They also want to compare these measurements against blood flow in healthy people who do not have sickle cell disease. Objectives: - To study whether changes in blood flow cause pain crises in people with sickle cell disease. Eligibility: * Individuals at least 18 years of age who have sickle cell disease and are being treated for a pain crisis. * Individuals at least 18 years of age who have sickle cell disease and are not experiencing a pain crisis. * Healthy volunteers matched by age and gender with the participants who have sickle cell disease. Design: * Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. * Participants having a sickle cell pain crisis will have two visits, one during the crisis and one about 4 weeks after the crisis has resolved. * Participants not having a sickle cell pain crisis will have one or two study visits. Blood samples will be collected during at least one of these visits. * Healthy volunteers will have one or two study visits. Blood samples will be collected during at least one of these visits. * During each visit for all participants, cameras and blood flow monitoring equipment will be used to measure blood flow in the forearm. sickle cell disease. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| Sickle Cell Anemia and Cerebral Microcirculation : Multimodal Exploration | The aim of this study is to evaluate determinants of cerebral oxygenation and perfusion at the microcirculatory level in children with sickle cell anemia (SCA) using combined novel investigational tools: Arterial Spin Labeling (ASL) perfusion MR (Magnetic Resonnance) imaging, brain Near Infra-Red Spectroscopy (NIRS) and red blood cell (RBC) rheological properties. | Sickle Cell Disease | ALL | CHILD | Centre Hospitalier Intercommunal, Créteil, France|Necker - Enfants Malades hospital, Paris, France |
| Vitamin D Status in Children With Sickle Cell Disease Living in Lyon, France | Vitamin D deficiency may be under-diagnosed in sickle cell disease French children. Therefore, the investigator need an epidemiologic study about the prevalence of vitamin D deficiency in this specific population. There are not specific guidelines neither testing nor treatment. The investigator propose to test vitamin D status in all children with sickle cell disease who are consulting their referring haematologist doctor or in the emergency ward. | Vitamin D Deficiency|Sickle Cell Disease | ALL | CHILD | Hôpital Femme Mère enfant - Urgences pédiatriques, Bron, 69500, France |
| HLA Haploidentical Bone Marrow Transplant in Patients With Severe Sickle Cell Disease | multicentric interventional biomedical research phase II, prospective, non-randomized evaluating a haploidentical marrow transplants after reduced-intensity conditioning and prevention of GvHD based on cyclophosphamide administration post transplantation in patients with severe sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT | CHU Henri-Mondor, Créteil, 94000, France|intercommunal hospital of Créteil, Créteil, 94000, France|CHU La Timone, Marseille, France|Hospital Necker, Paris, France|Hospital Robert-Debré, Paris, France|Saint-Louis hospital, Paris, France|CHU Strasbourg, Strasbourg, France |
| Hemostasis in Sickle Cell Disease--Infancy to Adulthood | To assess in older children and adults with sickle cell disease (SCD) whether intrinsic activation (relevant to the origin of pain and acute inflammation) occurs only during vasocclusive crisis (VOC). | Anemia, Sickle Cell|Blood Disease | MALE | CHILD, ADULT, OLDER_ADULT | |
| Long Term Effects of Hydroxyurea Therapy in Children With Sickle Cell Disease | The primary objectives of this prospective, observational study are (1) to describe the long-term cellular, molecular, and clinical effects of hydroxyurea therapy in sickle cell disease, and (2) to perform hydroxyurea pharmacokinetics studies. This study will follow sickle cell patients being treated with hydroxyurea for a long period of time to evaluate the long-term cellular and molecular effects of the drug on the patients' body. This study will consist of two patient groups. One group will be made up of patients who have received hydroxyurea therapy before entering the study. The second group will be made up of patients who have not received hydroxyurea before study entry. | Sickle Cell Disease | ALL | CHILD, ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Nitric Oxide to Improve Blood Flow in Sickle Cell Disease | Nitric oxide is important in regulating blood vessel dilation, and consequently, blood flow. This gas is continuously produced by cells that line the blood vessels. It is also transported from the lungs by hemoglobin in red blood cells. This study will examine how this gas regulates blood vessels and blood flow in people with sickle cell anemia. It will also look at a possible benefit of using certain genetic information to compare the white blood cells of people with sickle cell anemia to those without the disease. Patients with sickle cell anemia and healthy normal volunteers 18 to 65 years of age may be eligible for this study. Candidates will be screened with a medical history, cardiovascular physical examination, electrocardiogram and routine blood tests. Participation of volunteers without sickle cell anemia will be limited to a single blood draw for genetic study. Sickle cell disease patients will undergo the following procedures: Patients will lie in a reclining chair during the study. After administration of a local anesthetic, small tubes will be inserted through a needle into the artery and vein of the patient's forearm. These are used to measure blood pressure and draw blood samples during the study. Forearm blood flow will be measured using pressure cuffs placed on the wrist and upper arm, and a strain gauge (a rubber band device) placed around the forearm. When the cuffs are inflated, blood flows into the arm, stretching the strain gauge, and the flow measurement is recorded. A small lamp will be positioned over the hand. Light reflected back from the hand provides information about nitric oxide and hemoglobin in the blood of the skin. A squeezing device called a dynamometer will be used to measure handgrip strength. Baseline blood flow, nitric oxide, hemoglobin, and handgrip will be measured after an infusion of glucose (sugar) and water. These measurements will be repeated at various times before, during and after administration of small doses of the following drugs: * Sodium nitroprusside - causes blood vessels to dilate and increases blood flow to the heart * Acetylcholine - causes blood vessels to dilate and slows heart rate * LNMMA - decreases blood flow by blocking the production of nitric oxide There will be a 20- to 30-minute rest period between injections of the different drugs. When the above tests are completed, the patient will breathe a mixture of room air and nitric oxide for 1 hour through a facemask placed over the face, after which forearm blood flow and light reflected from the hand will be measured. Then the patient will do the handgrip exercise for 5 minutes, after which blood flow and hand lamp measurements will be taken. After a 20-minute rest period (with continued breathing of room air/nitric oxide), L-NMMA will be infused again. The handgrip exercise, blood flow and hand lamp measurements will be repeated. The face mask will then be removed, and the tubes will be removed 20 minutes later. Blood samples will be collected at various times during the 5- to 6-hour study through the tubes in the arm. Some of the blood will be used to look at genes that make proteins involved in cell-to-cell communication, inflammation, and in making red and white blood cells stick to the lining of blood vessels. | Sickle Cell Anemia | ALL | CHILD, ADULT, OLDER_ADULT | Warren G. Magnuson Clinical Center (CC), Bethesda, Maryland, 20892, United States |
| Observational Study to Deeply Phenotype Major Organs in Sickle Cell Disease After Curative Therapies | Background: People with sickle cell disease (SCD) have problems with their heart, brain, kidneys, liver, and lungs as they age. These problems may improve after transplant. Researchers want to learn how and why this happens. Objective: To study the benefits of treatments that are intended to cure SCD. Eligibility: People aged 18 and older with SCD who are either receiving curative therapy in the next 3 months or don t have any plans to receive a curative therapy in the next 2 years. Design: At their first visit, participants will be screened with their medical history and a physical exam. Participants will then have a baseline visit. This will take about a week to complete and will include: Blood and heart tests MRI of the brain, heart, and lungs. Participants will lie on a bed that will move into the MRI scanner. Special padding may be placed around their head to keep it still. Interactive games. Participants will complete computer games that test memory, attention, problem solving, language, spatial orientation, processing speed, and emotion. Questionnaire rating quality of life Iothalamate test. An IV catheter will be placed into a vein. A contrast agent will be injected through the IV. Blood will then be collected at different time points. Lung function tests and a 6-minute walk test Vibration controlled transient elastography. A probe placed on the abdomen will measure liver scarring. DOS test. A light attached to the finger or toe will measure blood oxygen. Participants will have an end-of-study visit about 2 years after their baseline visit. This will include repeats of the baseline visit tests. | Mortality in Sickle Cell|Sickle Cell Cardiopulmonary Complications|Sickle Cell Organ Damage|Sickle Cell Life Expectancy and Risk Factors for Early Death|Sickle Cell Lung Disease and Sudden Death | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Clinical Study of BRL-101 in the Treatment of Sickle Cell Disease | This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (BRL-101). | Sickle Cell Disease | ALL | CHILD, ADULT | |
| Transfusion Treatment in Patients With SCD | The "National Transfusion Treatment Survey in patients with sickle cell disease (SCD)" is a prospective longitudinal systemic study that was created in order to evaluate the therapeutic approach, mainly transfusional, in patients affected by SCD throughout Italy and to improve the quality of care and implement research. The survey will evaluate all patients affected by different forms of sickle cell disease (HbS homozygosis, Thalassoso-drepanocytosis, HbS / HbC compound heterozygosis, other possible genetic compounds). Patients will be selected according with a SCD diagnosis confirmed by standardized biochemical criteria or by DNA analysis. Patients will be excluded from the study who do not meet the these requirements, who are unable to understand the protocol or able to give informed consent in the absence of any legal representative. All data will be collected through a standard web-based application, which will be completed by the responsable investigator or by sub-investigators selected by each center, after registration on the site providing personal data and indicating the affiliation structure. All data will be subsequently encrypted by the Central Server. The operator will subsequently be able to access the patient's clinical data to perform the updates, in order to follow the patient's clinical evolution over time. The study will not involve any additional tests compared to the routine of patient control. | Anemia, Sickle Cell | ALL | CHILD, ADULT, OLDER_ADULT | Gian Luca Forni, Genova, Ge, 16121, Italy |
| Vitamin D Supplementation in Children With Sickle Cell Disease | Children aged 6 months to 12 years of age will be randomised to receive vitamin D 60,000IU once a month for 3 months or a placebo. The vitamin D will be in form of granules supplied in sachets. The primary study outcomes will be incidence of hospitalisation and change in vitamin D levels following supplementation. Secondary outcomes will include incidence of vaso-occlusive crisis (VOC), acute severe respiratory illness, Vitamin D related Severe adverse events and requirements for blood transfusion | Children With Sickle Cell Disease at Mulago Hospital | ALL | CHILD | |
| Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease | This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data. | Sickle Cell Disease | ALL | CHILD, ADULT | Lucile Packard Children's Hospital, Palo Alto, California, 94304, United States|Washington University, St. Louis, Missouri, 63110, United States|Nationwide Children's Hospital, Columbus, Ohio, 43205, United States |
| Psychometric Evaluation of the IPPAQ in Pediatric Patients With Sickle Cell Disease Hospitalized With Vasoocclusive Pain | Historically, sickle cell disease has not been viewed in the chronic pain paradigm because of its recurrent nature. Patients with sickle cell disease may be hospitalized for extended periods of time. As the hospital stay progresses, patients with SCD pain are often observed by clinicians to have improvements in function in areas such as self-care, mobility, and recreation despite continued self-report of high pain scores. This pattern of functional improvement with continued report of high pain intensity scores is common in patients with recurrent and chronic pain. A functional assessment tool that can assess function in the acute inpatient setting is needed. The purpose of this study is to evaluate the Inpatient Pediatric Physical Activity Questionnaire (IPPAQ), as a measure of daily function in children with sickle cell disease hospitalized with vasoocclusive pain. | Sickle Cell Disease|Pain | ALL | CHILD, ADULT | Connecticut Children's Medical Center, Hartford, Connecticut, 06106, United States |
| Vitamin D for Sickle-cell Respiratory Complications | This study aims to answer the question whether oral vitamin D supplementation can decrease lung complications in children and adolescents with sickle cell disease. Lung complications are the leading causes of morbidity and of death in sickle cell disease. Infections and increased inflammation play important roles in the development of the lung problems in sickle cell disease. Emerging evidence shows that vitamin D helps the immune system to fight infection and to control inflammation and could potentially help prevent respiratory complications in patients with sickle cell disease. The investigators hypothesize that oral vitamin D3, 100,000 IU (2.5 mg), given once a month to a group of children and adolescents with sickle cell disease, will reduce the rate of respiratory events (infection, asthma exacerbation and acute chest syndrome) compared to the rate in a group given standard dose oral vitamin D3, 12,000 IU (0.3 mg) given once a month. Funding Source - U.S. Food \& Drug Administration, Office of Orphan Products Development | Sickle Cell Disease|Vitamin D Deficiency|Acute Chest Syndrome|Asthma|Respiratory Infections | ALL | CHILD, ADULT | Columbia University Irving Medical Center, New York, New York, 10032, United States |
| Assessing Physical Function in Sickle Cell Patients Taking Voxelotor | Voxelotor is a novel hemoglobin polymerization inhibitor which has been demonstrated to reduce hemolysis and improve hemoglobin levels. There have been numerous studies examining the clinical impact of voxelotor in sickle cell disease (SCD) patients, but there are few published reports on the effects of treatment on physical function in patients with SCD. The hypothesis to be tested is that anemic SCD patients will have improvements in performance after 6 months of voxelotor treatment. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Inova Schar Cancer Institute, Falls Church, Virginia, 22042, United States |
| Alternative Dosing And Prevention of Transfusions (ADAPT) | ADAPT is a prospective cohort study at Jinja Regional Referral Hospital (JRRH) primarily to assess the effect of hydroxyurea on blood transfusion utilization and secondarily to determine the feasibility of PK-guided hydroxyurea dosing. | Sickle Cell Disease | ALL | CHILD | Jinja Regional Referral Hospital (JRRH), Department of Paediatrics, Sickle Cell Clinic, Jinja, Uganda |
| Pediatric -Adult Care Transition Program of Patients With Sickle Cell Disease | Background The pediatric-adult care transition is a risk-disrupting time for patients with chronic disease. This care transition takes place during adolescence; a period of psychological upheavals and adaptations of family roles. During this period, medication adherence is non-optimal and absenteeism at medical appointments is high. Sickle cell disease (SCD) is the first genetic disease detected in France. It is chronic disease characterized by frequent painful vaso-occlusive crises (VOC) requiring emergency hospitalization when they are severe. Other serious complications are acute chest syndromes (ACS) and stroke. In order to improve the health status of teenagers with sickle cell disease, it is necessary to anticipate this care transition and to involve the pediatric and adult sectors. The biopsychosocial health approach and the Social-Ecological Model of Adolescent and Young Adult Readiness to Transition (SMART) describe a care transition integrating bioclinical and psychosocial factors such as integration of the patient's family, education on disease and therapeutics, psychological management of pain and medico-social orientation. The pediatric-adult transition program proposed is based on this biopsychosocial approach. It aims to improve the health status of adolescents with SCD, their quality of life and the use of health care service. Objective of the study To assess the impact of a pediatric-adult transition program on the incidence of sickle-cell-related complications leading to hospitalization on 24-months after transfer to the adult sector. The evaluation focuses on severe complications leading to hospitalization, such as VOC, ACS, and stroke. Study design Multicenter Open-label individual Randomized Controlled Trial Population : Patients aged at least 16 years old with sickle cell disease, and their parents (or legal representatives Number of subject : 196 patients (98 patients by arm) The study will last 24 months Expected results For patients and families Better health and quality of life for patients is expected, including better use of medical care after the transition program. It is also expected a better experience of the pediatric-adult care transition and indirectly a better experience of intrafamilial relations. For health professionals This project is expected to provide solutions to improve the pediatric-adult care transition of patients with chronic disease. Indeed, the methodological quality of the study will make it possible to evaluate the efficiency of the proposed program, to possibly adapt it and test it to other chronic diseases presenting the same care transition problematic. In terms of public health SCD mainly affects populations of sub-Saharan origin, with low visibility and high social vulnerability. By focusing on this population, this project will reduce the social inequalities in health, experienced by patients with SCD and their families. By improving the health, quality of life and care of patients with SCD, this project is expected to decrease the cost of the pediatric-adult care transition period. | Sickle Cell Disease | ALL | CHILD | CHU de Fort de France, Fort-de-France-La Martinique, La Martinique, France|Centre Hospitalier Intercommunal de Creteil, Créteil, France|Hôpital Mondor, Créteil, France|Hôpital Bicêtre, Le Kremlin-Bicêtre, France|Hospices Civils de Lyon, Lyon, France|Hôpital Européen Georges Pompidou, Paris, France|Hôpital Necker, Paris, France|Centre Hospitalier de Pontoise, Pontoise, France |
| A Safety Study of Eptifibatide in Patients With Sickle Cell Disease | This study will evaluate the safety of eptifibatide in sickle cell patients and how well it works during the course of painful crises. The overall hypothesis that we seek to test is that increased platelet activation and the resultant inflammatory responses are important contributors to the problems of sickle cell disease. Sickle cell disease has been referred to both as a condition associated with increased risk of blood clots and increased inflammation. A painful crisis represents the most common cli nical problem in sickle cell disease, but the treatment of these crises remains inadequate. | Sickle Cell Disease | ALL | ADULT | University of North Carolina, Chapel Hill, North Carolina, 27599-7305, United States |
| Haploidentical Stem Cell Transplant for Patients With Sickle Cell Disease and Prior Stroke or Abnormal Transcranial Ultrasound | Sickle cell disease is a life-long blood condition that can cause damage to the brain and other organs of the body. Children may develop severe, debilitating clinical states, with stroke or abnormal blood flow to the brain. Treatment generally includes chronic blood transfusions which may cause iron overload, potentially leading to severe and sometimes fatal complications. Hematopoietic stem cell transplant using cells obtained from a sibling or an unrelated volunteer donor who is a perfect HLA "match" (same tissue type) for the recipient has shown to help, and possibly cure, sickle cell disease. Unfortunately, only about 10-20% of sickle cell patients have a HLA matched sibling donor, and the likelihood of locating an appropriate HLA matched unrelated donor through the various donor registries is limited. Stem cells from partially HLA matched family members (also called haploidentical transplant) is an option currently being explored for this patient population. This type of transplant has been used and found to be successful in some patients, mostly those with cancers of the blood. However, there can be significant complications with haploidentical transplant, primarily infection, failure of the graft to grow (graft failure), and a disorder called graft-versus-host disease. In addition, few patients with sickle cell disease have undergone this procedure. Therefore, the risks and benefits of haploidentical transplants for patients with sickle cell disorder are not as well established as those using an HLA identical sibling or unrelated donor. The primary objective of this study is to assess the safety of haploidentical stem cell transplantation for children and adolescents with severe sickle cell disease and stroke or abnormal transcranial Doppler ultrasound requiring chronic transfusion therapy. The treatment plan will be considered safe if there is not excessive toxicity. Toxicity for this protocol is defined as graft failure/graft rejection, severe acute GVHD, or regimen related death within 100 days after the last cellular product administered. Of note, the protocol was closed to accrual in September 2007 as we had met the stopping rules related to graft integrity (graft failure and graft rejection). Participants currently enrolled continue to be followed per protocol. | Sickle Cell Disease | ALL | CHILD | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Study on Use of Omega-3 Fatty Acids to Improve Outcomes in Individuals With Sickle Cell Disease | Sickle cell disease (SCD) is associated with significant morbidity and mortality. Pain and many adverse outcomes occurring in sickle cell disease are inflammatory driven. Recent data has shown that gut dysbiosis is present in individuals with sickle cell disease. Gut dysbiosis has been linked to inflammation in certain diseases. Omega -3-fatty acids (fish oil) has been shown to improve pain outcomes in individuals with sickle cell disease, but its acceptance is variable. The aim of this study is to determine if a plant-based omega-3-fatty acids will be more acceptable and also improve outcomes in individuals with sickle cell disease | Sickle Cell Disease | ALL | CHILD, ADULT | University of Alabama at Birmingham, Birmingham, Alabama, 35233, United States |
| Efficacy of a Decision Aid for Hydroxyurea in Sickle Cell Disease | The trial is a prospective, randomized study to determine the efficacy of training for use of a web based Decision Aid for Hydroxyurea(HU) usage among subjects with Sickle Cell Disease(SCD). Subjects are randomized primarily either to using a web based decision aid with training versus without training. Subjects in each group will be further randomized to 1) subjects receiving pretest surveys; and 2) subjects that do not receive pretest surveys. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Grady Health System, Atlanta, Georgia, 30303, United States|Hughes Spalding Children's Hospital, Atlanta, Georgia, 30303, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States |
| Sickle Cell Anemia Screening and Prevention in Northern Israel | Since 1987, a screening for β Thalassemia in pregnant women is carried on in northern Israel, and from 1999 all the samples were tested also for Hgb S, Hgb C, Hgb D, Hgb O Arab and others. In this study, the investigators intend to summarize the results of this preventive program aiming to detect couples at risk for having offspring with Thalassemia or SCA, the compliance regard to genetic counseling and prenatal diagnosis and the incidence of new affected babies born. | Thalassemia|Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | Pediatric Hematology Unit - Ha'Emek Medical Center, Afula, 18101, Israel |
| Evaluating Barriers to Stroke Screening and Prevention in Children With Sickle Cell Disease | DISPLACE is a three part, multi-center U.S. based study to evaluate the barriers to stroke screening and prevention in children with sickle cell anemia (SCA). In the United States, TCD (Transcranial Doppler ultrasound) is a proven method of screening children with SCA for stroke. However, many children are not getting the screening they need. This study will examine the issues that hinder and help children get the screening at 28 different hospitals and sickle cell centers to improve care for all children with sickle cell anemia. The investigators will then plan a study (part 3) aimed to improve stroke screening and prevention in sickle cell anemia. | Stroke Ischemic|Sickle Cell Disease | ALL | CHILD, ADULT | Medical University of South Carolina, Charleston, South Carolina, 29425, United States |
| A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS) | This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of etavopivat and test how well etavopivat works compared to placebo to improve the amount of hemoglobin in the blood and to reduce the number of vaso-occlusive crises (times when the blood vessels become blocked and cause pain). | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | University of Alabama at Birmingham (UAB), Birmingham, Alabama, 35249, United States|Phoenix Children's Hospital, Phoenix, Arizona, 85016, United States|Woodland International Research Group, Little Rock, Arkansas, 72211, United States|University of California, Irvine, Irvine, California, 92697, United States|Collaborative Neuroscience Research, LLC., Long Beach, California, 90806, United States|Pacific Research Partners, LLC, Oakland, California, 94607, United States|UCSF Benioff Children's Hospital Oakland, Oakland, California, 94609, United States|UC Davis Medical Center - UC Davis Comprehensive Cancer Center - Hemotology/Oncology Clinic, Sacramento, California, 95817, United States|University of Connecticut (UCONN) Health, Farmington, Connecticut, 06030, United States|Yale University, New Haven, Connecticut, 06519, United States|Children's National Health Center, Washington, District of Columbia, 20010, United States|Howard University, Washington, District of Columbia, 20060, United States|Cornerstone Research Institute, Altamonte Springs, Florida, 32701, United States|University of Florida Hematology, Gainesville, Florida, 32610, United States|Office of Gershwin T. Blyden, MD, Hollywood, Florida, 33021, United States|University of Miami - Miller School of Medicine, Miami, Florida, 033136, United States|Advanced Pharma CR LLC., Miami, Florida, 33147, United States|Arnold Palmer Hospital for Children - Haley Center for Children's Cancer and Blood Disorders, Orlando, Florida, 32806, United States|Emory University School of Medicine, Atlanta, Georgia, 30322, United States|Sonar Clinical Research, Atlanta, Georgia, 30331, United States|Children's Healthcare of Atlanta - Pediatric Research Center, Atlanta, Georgia, 30342, United States|Augusta University Center for Blood Disorders., Augusta, Georgia, 30912, United States|iResearch Atlanta, LLC, Decatur, Georgia, 30030, United States|University of Illinois at Chicago Sickle Cell Center, Chicago, Illinois, 60612, United States|Children's Hospital New Orleans, New Orleans, Louisiana, 70118, United States|University of Maryland School of Medicine, Baltimore, Maryland, 21201, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|Barbara Ann Karmanos Cancer Institute, Detroit, Michigan, 48201, United States|Detroit Medical Center (DMC) - Children's Hospital of Michigan (CHM), Detroit, Michigan, 48201, United States|Washington University School of Medicine Barnes - Jewish Hospital, Saint Louis, Missouri, 63110, United States|University of Nebraska Medical Center, Omaha, Nebraska, 68198, United States|Jacobi Medical Center, Bronx, New York, 10461, United States|Children's Hospital at Montefiore, Bronx, New York, 10467, United States|Kings County Hospital, Brooklyn, New York, 11203, United States|Queens Hospital Center, Jamaica, New York, 11432, United States|Long Island Jewish Medical Center, New Hyde Park, New York, 11042, United States|Columbia University Medical Center, New York, New York, 10032, United States|UNC School of Medicine, Chapel Hill, North Carolina, 27514, United States|Levine Cancer Institute, Charlotte, North Carolina, 28204, United States|Duke University - Sickle Cell Center, Durham, North Carolina, 27705, United States|East Carolina University (ECU) Physicians, Greenville, North Carolina, 27834, United States|Wake Forest Baptist Medical Center, Winston-Salem, North Carolina, 27157, United States|University of Cincinnati Cancer Center, Cincinnati, Ohio, 45219, United States|Cincinnati Childrens Hospital Medical Center, Cincinnati, Ohio, 45229, United States|Neuro-Behavioral Clinical Research, North Canton, Ohio, 44720, United States|Lynn Institute of Tulsa, Tulsa, Oklahoma, 74135, United States|The Children's Hospital of Philadelphia (CHOP), Philadelphia, Pennsylvania, 19104, United States|Thomas Jefferson University Hospital, Philadelphia, Pennsylvania, 19107, United States|Rhode Island Hospital, Providence, Rhode Island, 02903, United States|Medical University of South Carolina (MUSC), Charleston, South Carolina, 29425, United States|Prisma Health, Greenville, South Carolina, 29605, United States|St. Jude Children's Research Hospital (SJCRH), Memphis, Tennessee, 38105, United States|Methodist University Hospital, Nashville, Tennessee, 38104, United States|Baylor College of Medicine, Houston, Texas, 77030, United States|The University of Texas Health Science Center at Houston, Houston, Texas, 77030, United States|Virginia Commonwealth University, Richmond, Virginia, 23284, United States|Mary Bridge Children's Health Center, Tacoma, Washington, 98405, United States|Blood Center of Wisconsin (BCW), Milwaukee, Wisconsin, 53226, United States|Universite de Montreal - Centre Hospitalier Universitaire (CHU) Sainte-Justine, Montréal, H3T 1C5, Canada|The Hospital for Sick Children, Toronto, M5G 1X8, Canada|University Health Network Toronto General Hospital, Toronto, Canada|Providence Hematology, Vancouver, V6Z 2A5, Canada|Alexandria Clinical Research Center, Alexandria, Egypt|Alexandria Faculty of Medicine - Clinical Research Center, Alexandria, Egypt|Ain Shams University Hospital, Cairo, Egypt|Al Kasr AL Aini Hospital, Cairo, Egypt|Cairo University, Cairo, Egypt|Sydnawy University Hospital, Zagazig, Egypt|CHU Montpellier, Hôpital Saint-Eloi, Montpellier, Montpellier Cedex 5, 34295, France|Hôpital Henri Mondor, Créteil, 94010, France|Hôpital Edouard HERRIOT, Lyon, 69437, France|Hôpital Emile Muller, Mulhouse, 68100, France|CHU Paris - Hôpital Robert Debré, Paris, France|Charite Berlin - University Medicine, Berlin, Germany|Univertatsklinikum Frankfurt, Frankfurt, Germany|Universitätsklinikum Freiburg, Freiburg, 79106, Germany|UKE Hamburg, Hamburg, Germany|University Hospital of Heidelberg, Heidelberg, 69120, Germany|Universitatsklinikum Regensburg, Regensburg, Germany|General University Hospital of Patras, Rio, Patras, 26504, Greece|Hippocratio General Hospital, Athens, 115 27, Greece|General University Hospital of Larissa, Larissa, 41221, Greece|General Hospital of Thessaloniki "Hippokration", Thessaloniki, 54642, Greece|Azienda Ospedaliero Universitaria Policlinico "G. Rodolico - San Marco", Catania, 95123, Italy|Azienda Ospedaliera Universitaria San Luigi Gonzaga, Orbassano, 10043, Italy|Azienda Ospedale Università Padova, Padova, 35128, Italy|Fondazione IRCCS Policlinico San Matteo, Pavia, 27100, Italy|IRCCS Ospedale Pediatrico Bambino Gesu, Rome, Italy|American University of Beirut Medical Center, Beirut, Lebanon|Nini Hospital, Tripoli, Lebanon|Sultan Qaboos University Hospital, Muscat, 123, Oman|Sultan Qaboos University Hospital, Muscat, 123, Oman|Hospital De Cruces, Barakaldo, 48013, Spain|Hospital Universitari Vall d'Hebron de Barcelona, Barcelona, 08035, Spain|Hospital Clínico San Carlos, Madrid, 28040, Spain|Universidad Autonoma de Madrid (UAM) - Hospital Universitario La Paz, Madrid, 28046, Spain|Hospital Universitario Virgen del Rocio, Sevilla, 41013, Spain|Leicester Royal Infirmary, Leicester, LE1 5WW, United Kingdom|Guy's and St Thomas' NHS Foundation Trust, London, SE18 3RA, United Kingdom|King's College Hospital, London, SE5 9RS, United Kingdom|Hammersmith Hospital - Imperial College Healthcare NHS Trust, London, W2 1NY, United Kingdom|Imperial College Healthcare NHS Trust - St Mary's Hospital, London, W2 1NY, United Kingdom|Oxford University Hospitals NHS Trust - Churchill Hospital - Cancer and Haematology Centre, Oxford, OX3 7LE, United Kingdom|The Royal Hallamshire Hospital, Sheffield Teaching Hospital NHS Foundation Trust, Sheffield, S10 2JF, United Kingdom |
| Dietary Intake and Dietary Behaviors in Adults With Sickle Cell Disease | Background: Sickle Cell Disease (SCD) causes blood cells form a crescent shape. It is caused by a genetic mutation in the hemoglobin gene. People with SCD are at increased risk for illnesses like stroke, chronic pain, and heart problems, as well as decreased overall health and well-being. Researchers want to learn more about how nutrition and diet can help relieve or reduce the symptoms of SCD. Objective: To understand how diet, dietary patterns and behaviors, nutrition, and other related factors in adults with SCD affect their overall health. Eligibility: Adults aged 18 and older with SCD. Design: Participants will be screened with a review of their medical records. They will take a pregnancy test if needed. Participants will have a physical exam and medical history. Their height, weight, and waist and hip circumference will be measured. They can complete this exam (1) via telehealth along with a visit to an outpatient laboratory center or (2) by going to the NIH Clinical Center. Participants will complete 2 interviews about their diet. They will talk about the foods they ate in the past 24 hours. They will also complete 1 interview about diet-related behaviors such as food shopping and cooking. They can complete the interviews in person, by phone, or by telehealth visit. Participants will complete surveys about their demographics (such as age and gender), SCD pain, mood, stress, diet, and nutrition. It may take about 1 hour to complete all of the surveys. Participants will give blood and urine samples. They will need to fast for at least 8 hours overnight before giving blood samples. Participation will last for about 2 weeks. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| A Voxelotor for Sickle Cell Anemia Patients at Highest Risk for Progression of Chronic Kidney Disease | This study is a single center, prospective exploratory pilot study of Sickle Cell Anemia (SCA) participants. The study will enroll patients with early stages of sickle cell nephropathy (Chronic Kidney Disease (CKD) stage 1 or 2) who are at the highest risk of CKD progression (presence of both hemoglobinuria and urine albumin concentration ≥ 30 mg/g creatinin | Sickle Cell Disease|Sickle Cell Nephropathy | ALL | ADULT, OLDER_ADULT | University of Illinois, Chicago, Illinois, 60612, United States |
| Implication of the Oxydative Stress in the Pathophysiology of Sickle Cell Anemia: | Despite important advances in the current understanding of sickle cell vaso-occlusion, the basis of its control and prevention remain partially unknown. The primary purpose is to test the hypothesis of a control of the sickle cell vaso-cocclusive (VOC) process by the anti band 3 antibodies by assessing the level of these antibodies in the steady state and during the crises in SCA patients. To assess the relationship between the level of band 3 antibodies, the oxidation status, the expression of microparticles and the hemorheological alterations of the sickle red cells (SS RBs), the severity of VOC. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Hospital University Center of Pointe-à-Pitre, Pointe-à-Pitre, 97159, Guadeloupe |
| Longitudinal Changes in Exercise Capacity in Children and Young Adults With Sickle Cell Anemia | The purpose of this study is to use comprehensive exercise testing to examine longitudinal changes in exercise capacity over a 2 year period in children and young adults with sickle cell anemia. | Sickle Cell Anemia | ALL | CHILD, ADULT | Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States |
| Steroid Treatment for Sickle Cell Pain Crisis | The painful episode is the most common problem experienced by children with sickle cell disease. Although various treatments are available during painful episodes, the medication most commonly given for pain is a pain medication such as morphine. Fluids are also used. Even with these treatments, many children still have severe pain that is difficult to control. In addition to pain medications, there are other medications that may be useful. Methylprednisolone (solumedrol) and prednisone are a group of medications called steroids that may be helpful for painful episodes. These medications are known to lower the amount of inflammation (this means swelling, tenderness, and soreness) in the body. Because this medication may help with your pain, you are being asked to be a part of this study. These types of medications are used in other illnesses such as asthma, especially during times when the illness has gotten worse. The main purpose of this study is to see if the methylprednisolone and prednisone will lower the amount of pain and the length of hospital stay. In addition to the pain medication you will normally receive, you will be assigned to one of 2 groups: 1) the experimental group with the active form of the medicine, or 2) a comparison group without the active form of the medicine. In either group, you will still receive all of the treatments you would normally receive for a painful episode, including pain medicines and fluids. You and your doctors will not know what group you will be assigned. If you decide to be a part of the study the following will happen: For the first 5 days, you will be asked to: 1) describe your current pain (0=no pain to 10=a lot of pain), worst pain (0=no pain to 10=a lot of pain), least pain (0=no pain to 10=a lot of pain), and the amount of pain relief (0=no relief to 10=complete relief); 2) describe any signs or symptoms you feel, including filling out a pain scale form each day; 3) and take the medicines for 5 days, either at home or when in the hospital. Thirty days after the study, a study researcher will call and will ask questions about your pain, any painful episodes, and any medications you had. If you are discharged home sooner than 5 days after the start of the study, research staff will call you to ask you these questions, remind you to fill out your pain forms, and remind you to take your medicine. If you are discharged home, you will be given pain scales to fill out each day at home. | Sickle Cell Disease|Vaso-occlusive Crisis | ALL | CHILD, ADULT, OLDER_ADULT | Texas Childrens Hospital, Houston, Texas, 77030, United States |
| Methadone in Pediatric and Adult Sickle Cell Patients | To determine the pharmacokinetics of methadone in children and adults with SCD who are experiencing a painful episode. | Sickle Cell Disease | ALL | CHILD, ADULT | Barnes Jewish Hospital/St. Louis Children's Hospital, Saint Louis, Missouri, 63110, United States |
| Severe Acute Respiratory Syndrome CoV 2 COVID-19 Survey and Vaccination Coverage in the Sickle Cell Population in Ile-De-France | The objective of this study is to determine the seroprevalence of severe acute respiratory syndrome-CoV-2 in unvaccinated sickle cell patients living in an area with high viral circulation and at risk of high viral transmission, after the 4th epidemic wave of COVID-19 in Ile-de -France, over a period of 3 months (for example, last quarter of 2021). | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | |
| Hypoxic Red Blood Cells in Sickle Cell Anemia | The overall objective of this study is to evaluate the effectiveness and safety of transfusing hypoxic red blood cells manufactured with the Hemanext ONE system in patients with sickle cell anemia. The Hemanext ONE device was cleared through the De Novo process in September 2023. | Sickle Cell Anaemia|Sickle Cell Anemia Crisis|Sickle Cell Anemia in Children|Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0) | ALL | CHILD, ADULT, OLDER_ADULT | New England Sickle Cell Institute, University of Connecticut, Farmington, Connecticut, 06030, United States|Johns Hopkins All Children's Hospital, Saint Petersburg, Florida, 33701, United States|Emory University School of Medicine, Atlanta, Georgia, 30322, United States|John Hopkins University School of Medicine, Baltimore, Maryland, 21287, United States|University of Pittsburgh, Pittsburgh, Pennsylvania, 15213, United States|University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, 15260, United States |
| Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease | The study is a Phase II clinical trial. Patients will receive intensity modulated total body irradiation (TBI) at a dose of 3 Gy with standard fludarabine/ i.v. cyclophosphamide conditioning prior to human leukocyte antigen (HLA)-haploidentical hematopoietic stem cell transplant (HSCT). The primary objective of the study is to determine the engraftment at Day +60 following HLA-haploidentical hematopoietic stem cell transplant protocol using immunosuppressive agents and low-dose total body irradiation (TBI) for conditioning and post-transplant cyclophosphamide in patients with sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT | University of Illinois at Chicago, Chicago, Illinois, 60612, United States |
| Effects of the Contraceptive Implant in Women With Sickle Cell Disease | The objective of this study is to measure the acceptability and impact of the progestin implant on frequency of vaso-occlusive crises, quality of life, and hematologic parameters in women with SCD. | Sickle Cell Disease | FEMALE | ADULT | Penn Medicine University City, Philadelphia, Pennsylvania, 19104, United States |
| Laboratory-based Hypnosis Intervention on Pain Responsivity in Adolescents With Sickle Cell Disease | The purpose of this study is to test the effects of a laboratory-based hypnosis session compared to an attention control condition on peripheral blood flow, autonomic stress responses, and acute pain responses in adolescents (ages 12-21) with sickle cell disease, and examine how perceived disease-related stigma may affect these responses. | Sickle Cell Disease | ALL | CHILD, ADULT | University of California, Los Angeles, Los Angeles, California, 90095, United States |
| Do Alemtuzumab Levels Predict T Cell Chimerism After MSD SCT for SCD? | Rationale: Non-myeloablative allogeneic stem cell transplantation (SCT) has become a feasible curative treatment option for sickle cell disease (SCD) patients with an available matched sibling donor. Chemotherapy free conditioning with alemtuzumab and 3 Gy total body irradiation (TBI) is increasingly being used as preferred conditioning scheme for these patients. This regimen typically results in mixed donor chimerism and has only few toxic effects. However, the risk of graft failure (rejection) is still significant, with an occurrence of 13% in the latest series. Levels of T cell chimerism are crucial for the success of this kind of transplantation. A donor T cell level of at least 50% at 1-year post-transplantation seems to be sufficient to allow the discontinuation of immunosuppressive medication without risk of graft rejection. Low levels of alemtuzumab prior to or shortly after SCT are thought to facilitate rejection of the donor graft. Recently, a positive correlation between alemtuzumab levels on day+14 was found with levels of T cell chimerism +2 and +4 months post-transplantation in adult SCD patients receiving matched sibling donor SCT. However, in this study alemtuzumab levels prior to the infusion of hematopoietic stem cells and beyond day +28 post-transplantation were not measured. Furthermore, the alemtuzumab levels were measured in 2 patient groups undergoing two different conditioning regimens. Here, the investigators aim to thoroughly investigate the correlation of alemtuzumab levels and T cell chimerism. This will be the first study involving SCD patients receiving matched sibling donor SCT with alemtuzumab/TBI conditioning that includes alemtuzumab level measurements before the infusion of hematopoietic stem cells and beyond 1-month post-transplantation. Findings from this study will improve the insights into the etiology of graft failure in these patients and might ultimately lead to a more personalized approach in dosing alemtuzumab in order to achieve a more robust and stable engraftment of donor hematopoietic stem cells. Objectives: To investigate whether serum alemtuzumab concentrations are predictive of the robustness of engraftment in SCD patients undergoing a matched sibling donor transplantation with alemtuzumab/TBI conditioning resulting in mixed chimerism. Study design: Prospective observational laboratory study. Serum alemtuzumab concentration will be measured at various time points before and after stem cell infusion (days -3, 0, +7, +14, +28, +60). Study population: Adult SCD patients that are planned for a matched sibling donor transplantation with alemtuzumab/TBI conditioning at the Amsterdam UMC. Main study parameters/endpoints: The correlation between serum alemtuzumab concentration and levels of donor chimerism. Secondary endpoints: correlation between serum alemtuzumab levels and patients with and without successful engraftment. Correlation of serum alemtuzumab levels and the dosing of alemtuzumab in mg/kg, number of patient lymphocyte count and total number of infused enucleated donor-derived cells. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Amsterdam Medical Centre, Amsterdam, 1105AZ, Netherlands |
| Insights Into Microbiome and Environmental Contributions to Sickle Cell Disease and Leg Ulcers Study (INSIGHTS Study) | Background: - People with sickle cell disease and other blood disorders sometimes get chronic leg ulcers. These are wounds that develop on the skin and don t go away. Current treatments do not work very well, so researchers want to learn more about why the ulcers happen. They want to find out which bacteria may cause it, and if external factors play a role. Objective: - To study social and environmental factors of sickle cell disease and the causes of sickle cell disease leg ulcers. Eligibility: - People age 18 and older who have sickle cell disease or another red cell disorder, with or without an active leg ulcer. Design: * Participants will have a medical history and clinical evaluation. They will also have blood drawn. * Participants will complete questionnaires about their life, health, environment, stress, and other topics. * Participants may provide a small sample of hair. * Participants will be asked to collect a small amount of saliva. * Participants with leg ulcers will have their skin microbiome sampled. The microbiome is all of the microbes (bacteria and and/or fungi) and their genes in and on the body. Researchers will use swabs to collect skin samples. Photographs will be taken of the skin sample area. * Some participants without leg ulcers also will have their skin microbiome sampled. * Some participants who have their skin microbiome sampled will return for a second visit. At this visit, their microbiome will be resampled. It will take place more than 30 days after the first visit. | Genetic Disease|Genomics | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States|Montefiore Medical Center/Albert Einstein College of Medicine, Bronx, New York, 10467, United States|University of Sierra Leone, College of Medicine and Allied Health Services, Freetown, Sierra Leone |
| SANGUINATE™ in Sickle Cell Disease Associated Leg Ulcer | SANGUINATE™ Sickle Cell Disease associated Leg Ulcers. | Leg Ulcer | ALL | ADULT, OLDER_ADULT | General Hospital Plaza de la Salud, Santo Domingo, Dominican Republic|Centro Hemato-Oncologico, Marbella, Panama |
| Specifying Interventions From the Sickle Cell Disease Implementation Consortium (SCDIC) | The goal of this study is to specify the interventions, implementation strategies and control conditions from the Sickle Cell Disease Implementation Consortium (SCDIC) using a mixed-methods approach to study site materials and conduct semi-structured qualitative interviews with site representatives (N=3 per site). We focused on the Emergency Department Working Group from the SCDIC working groups. | Anemia, Sickle Cell | ALL | ADULT, OLDER_ADULT | Washington University in St. Louis, Saint Louis, Missouri, 63110, United States |
| An Extension Study of IMR-687 in Adult Patients With Sickle Cell Anemia | This is an open-label extension study of IMR-687 in adult patients who completed Imara's blinded Phase 2a study (IMR-SCD-102). The open-label extension study will evaluate long-term safety and tolerability. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of Connecticut Health Center, Farmington, Connecticut, 06030, United States|Foundation for Sickle Cell Disease Research, Hollywood, Florida, 33021, United States|Baylor Scott & White Medical Center - Temple, Temple, Texas, 76508, United States|Bristol Haematology and Oncology Centre, Bristol, United Kingdom|University College London Hospital NHS Foundation Trust, London, NW1 2PG, United Kingdom|Guy's and St Thomas Hospital CRF, London, United Kingdom|Royal London Hospital, London, United Kingdom |
| The Predictive Capacity of Machine Learning Models for Progressive Kidney Disease in Individuals With Sickle Cell Anemia | This is a multicenter prospective, longitudinal cohort study which will evaluate the predictive capacity of machine learning (ML) models for progression of CKD in eligible patients for a minimum of 12 months and potentially for up to 4 years. | Sickle Cell Disease|Kidney Diseases, Chronic | ALL | ADULT, OLDER_ADULT | University of Illinois at Chicago, Chicago, Illinois, 60612, United States|Wake Forest University, Winston-Salem, North Carolina, 27109, United States|The University of Tennessee Health Science Center, Memphis, Tennessee, 38104, United States |
| Use of Hydroxyurea and Magnesium Pidolate for Treatment of Sickle Cell Disease | The purpose of this study is to estimate the MTD of Mg pidolate in combination with HU in patients with sickle cell disease who have been on a therapeutic dose (15-30 mg/kg/day) of HU for at least 6 months. | Anemia, Sickle Cell | ALL | CHILD | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| AB1 in Adult Patients with Sickle Cell Disease (SCD) | This will be an open-label, dose escalating study with a starting dose of 2mg. Up to 6 additional cohorts will be enrolled at subsequently higher doses of 4mg, 8mg, 10mg, 12mg, 16mg, and 32mg. In each dose escalation cohort, each dose will be taken orally, once daily, for 8 weeks. | Sickle Cell Disease | ALL | ADULT | Augusta University Medical Center, Augusta, Georgia, 30912, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|East Carolina University, Greenville, North Carolina, 27834, United States |
| Home Based Massage and Relaxation for Sickle Cell Pain | The purpose of this study is to compare the effects of in-home, family-administered massage and in-home relaxation training on measures of physical status and health care utilization in a sample of African American adolescents age 15 years and older and adults with chronic pain associated with sickle cell disease who have been randomly assigned to six sessions of either family-administered massage or progressive muscle relaxation training. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Los Angeles Orthopaedic Hospital - Vascular Medicine Program, Los Angeles, California, 90007, United States|Moffitt Cancer Center and Research Institute, Tampa, Florida, 33612, United States |
| Safety Evaluation of DREPADOM - Home Care Services and Hospitalizations for Sickle Cell Disease Patients | The objective of this study is to evaluate the incidence of ACS within the DREPADOM system and compare it to expected incidences of ACS (historic cohorts of PRESEV1 and PRESEV2) | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Henri Mondor Hospital, Creteil, 94000, France |
| Assessing Function in Pediatric Patients With Sickle Cell Disease | The purpose of this study is to evaluate the FIM™ as a measure of daily function in children with sickle cell disease hospitalized with vasoocclusive pain. Currently, the standard for pain assessment is a rating of pain intensity, as determined by observation (for younger children) or self-report (for older children and adolescents). However, these measures of pain intensity are not effective in recurrent or chronic pain states, and in sickle cell disease in particular. Pediatric patients who are hospitalized with vasoocclusive pain often do not report a decrease in pain intensity; however, other indications of clinical status, such as ambulation, less use of opiates from the patient-controlled analgesia (PCA) pump, increased food intake, and transition to oral pain medication, signify that the patient may be improving. As a result of our inability to get an accurate picture of the patients' condition, we would like to have a summary of improvement that would reflect these changes in clinical status and reflect the reduced impact of sickle cell pain on the patient's life. In this study, we plan to evaluate a standardized functional assessment measure in pediatric patients with sickle cell disease. It is hypothesized that FIM™ scores will correlate with other indicators of clinical status, such as movement, quality of sleep, use of IV opiates from the patient-controlled analgesia (PCA) pump, and use of intravenous vs. oral pain medications. It is also hypothesized that the FIM™ will demonstrate adequate responsiveness to change in functional status within a 3-7 day hospitalization by a progressive increase in scores and associations with other indicators of clinical improvement. | Sickle Cell Disease | ALL | CHILD, ADULT | Connecticut Children's Medical Center, Hartford, Connecticut, 06106, United States|Johns Hopkins University School of Medicine, Baltimore, Maryland, 21205, United States |
| Evaluation of Different Dose Regimens of Aes-103 Given for 28 Days to Subjects With Stable Sickle Cell Disease | Sickle cell disease (SCD) is a genetic blood disorder characterized by the presence of sickle-shaped red blood cells. In the U.S. and the U.K. this occurs primarily in persons of African origin. There is only one drug (hydroxyurea) approved to manage SCD, but it is not fully efficacious and can produce medically significant side effects. Aes-103 is being evaluated as a novel agent for the long term management of SCD. By directly reducing the sickling process, Aes-103 has a different mechanism of action than hydoxyurea. The active ingredient in Aes-103 is 5-hydroxymethyl furfural, a naturally occurring small molecule that is chemically related to glucose. This study will evaluate the safety and pharmacokinetic profile of two dosing regimens of Aes-103 for up to 28 days in up to 50 adult subjects with stable SCD compared with subjects receiving placebo. | Sickle Cell Disease | ALL | ADULT | Quintiles Ltd. - Quintiles Drug Research Unit, 6 Newcomen Street, London, SE1 1YR, United Kingdom |
| Adenosine 2A Agonist Lexiscan in Children and Adults With Sickle Cell Disease | Sickle cell disease (SCD) is an inherited blood disorder that causes the red blood cells to change their shape from a round shape to a half-moon/crescent or sickled shape. People who have SCD have a different type of protein that carries oxygen in their blood (hemoglobin) then people without SCD. This different type of hemoglobin makes the red blood cells change into a crescent shape under certain conditions. Sickle-shaped cells are a problem because they often get stuck in blood vessels blocking the flow of blood, and cause inflammation and injury to the important areas in the body. Lexiscan is drug that may prevent this inflammation and injury caused by the sickle shaped cells. This drug is approved by the FDA to be used as a fast infusion during a heart stress test in people who are unable to exercise enough to put stress on their heart by making it beat faster. Lexiscan has never been studied in patients with SCD and has never been given as a long infusion. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Howard University Hospital, Washington, District of Columbia, United States|Johns Hopkins University, Baltimore, Maryland, United States|Brigham and Women's Hospital, Boston, Massachusetts, 02115, United States|Childrens Hospital Boston, Boston, Massachusetts, 02115, United States|Dana-Farber Cancer Institute, Boston, Massachusetts, 02115, United States|Washington University, St. Louis, Missouri, United States|Blood Center of Wisconsin, Milwaukee, Wisconsin, United States |
| Low Systemic/High Local Exercise Load in Peds SCD | This research study wants to learn about what kind of exercise is best for kids with sickle cell disease. Participating children will have a small amount of blood drawn one time at the beginning of the study. Children will then complete some questionnaires that measure pain, physical function, and emotions (depression, anxiety) and complete some tests that measure physical fitness at the beginning and end of the study. Children will be randomized to either a home-based telehealth (1) walking or (2) strengthening exercise program that lasts for 8-weeks, 3-x week, for 45 minutes each session. Children's participation will last up to 10 weeks. | Sickle Cell Anemia in Children | ALL | CHILD | University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States |
| Sub-dissociative Intranasal Ketamine for Pediatric Sickle Cell Pain Crises | The purpose of this study is to determine if the use of ketamine, sniffed in the nose, is a safe and effective way to help reduce pain in pediatric sickle cell patients with pain crises in resource-limited settings. | Sickle Cell Disease | ALL | CHILD | Mbingo Baptist Hospital, Bamenda, Northwest Province, Cameroon|Muhimbili National Hospital, Dar es Salam, Tanzania |
| Feasibility and Efficacy of Attentional-Control Training in Sickle Cell Disease | Children with sickle cell disease (SCD) exhibit significantly reduced cognitive functioning (often difficulties with attention) compared to peers and siblings without SCD. EndeavorRx (Akili Interactive Labs: Boston, MA) is an FDA-approved home-based, electronic attentional-control training program designed to treat attention problems in youth. Users access EndeavorRx on a tablet device for 25-30 minutes each day, 5 days per week, for 4 weeks. The program involves training in a game-like environment that repeatedly challenges attentional-control abilities and adapts to user performance, becoming more difficult over time as performance improves. This pilot study is examining the feasibility, acceptability, and preliminary efficacy of EndeavorRx in a sample of 20 children with SCD ages 8-16 who are being treated with chronic blood transfusion therapy. | Sickle Cell Disease|Attention Deficit|Cognitive Deficit in Attention | ALL | CHILD | Children's National Hospital, Washington, District of Columbia, 20010, United States |
| Hydoxycarbamide and L-Carnitine Therapy in Sickle Cell Anemia | The role of the combination therapy of hydroxyurea and L-Carnitine was studied in thalassemic patients. nevertheless its role in sickle cell anemia patients was not investigated | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Safaa A A Khaled, Assiut, 71515, Egypt |
| Future of Spermatogenesis in Men With Sickle Cell Disease Medically Treated | The project's background: Sickle cell disease is, at present in France, the most frequent genetic illness. Recent progress in its treatment, in particular the use of hydroxyurea, has considerably modified the prognosis of this disease. Many more patients now reach reproductive age and do consider fathering. Exceptional studies have reported the potential impact of this medical treatment on the sperm parameters and fertility of male patients. In a retrospective analysis, the investigators found that the observed alterations of semen parameters due to sickle cell disease seem to be exacerbated by hydroxyurea treatment. The study hypothesis: A large prospective study is essential to assess the potential adverse impact of the medical treatment of sickle cell disease on spermatogenesis and consider the advisability of proposing sperm cryopreservation before this treatment is started. Primary purpose of the protocol: evaluate the impact of a treatment by hydroxyurea (20-30 mg/kg/day), 6 months after its beginning, in 34 men with sickle cell disease (18-60 years old). The main trial criterion will be the average difference of the concentration of spermatozoa s (millions/ml) in the ejaculate, before and after 6 months of medical treatment. | Drepanocytic Men Treated by Hydroxyurea for the First Time | MALE | ADULT | Department of biology of reproduction (TENON Hospital- AP-HP), Paris, 75020, France |
| GDF 15 in Sickle Cell Disease and Hereditary Spherocytosis | Patients with thalassemia intermedia, congenital dyserythropoietic anemia type I , and sideroblastic anemia were found to express very high levels of serum GDF15, and this contributed to the inappropriate suppression of hepcidin with subsequent secondary iron overload.The aim of our present study is to asses the levels of GDF15 and hepcidin in patients with Sickle cell disease and hereditary spherocytosis | Patients With Thalassemia Intermedia,|Congenital Dyserythropoietic Anemia Type I | ALL | CHILD, ADULT, OLDER_ADULT | |
| Measurement of Cerebral Blood Flow Using Transcranial Doppler Ultrasound in Children With Sickle Cell Disease | Sickle cell disease (SCD) affects haemoglobin - the molecule in blood cells which carries oxygen. It causes red blood cells to become abnormal crescent (or sickle)- shaped. Sickled red blood cells cannot travel through small blood vessels as easily as normal red blood cells which can lead to blockages. This means that oxygen may be prevented from getting to where it is needed. Individuals with sickle cell disease also suffer form abnormality in the lining of their blood vessels, which contributes to the damage. Damage and blockage can occur in the blood vessels in the brain and means that children with sickle cell disease have a significant risk of suffering from strokes. Research has shown that transcranial Doppler ultrasonography can be used in this setting to identify children at most risk of getting strokes. Ultrasound is therefore used in children with sickle cell disease to measure the blood flow in the vessels in the brain. This research has formed the basis of the National Health Service (NHS) Standard of Care for Sickle Cell Disease in the United Kingdom (UK) which uses transcranial Doppler ultrasonography at once a year to screen children with sickle cell disease aged 2 to 16. Ultrasound is used because it is portable, does not uses ionising radiation such as x-rays, is non-invasive and gives good results. However, the results are dependent on the operator. This means that the screening service is provided by centres of excellence with experienced scanning staff visiting clinics in smaller hospitals with portable machines. There is a lack of research comparing the use of portable machines to laboratory-based machines. This is important because screening can identify children at high risk of stroke and may be used by clinical staff to make a decision about the care of the child. | Sickle Cell Disease | ALL | CHILD | Imperial College Healthcare NHS Trust, London, W2 1NY, United Kingdom |
| A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) | The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult and adolescent participants with severe sickle cell disease (SCD). | Sickle Cell Disease|Hemoglobinopathies | ALL | CHILD, ADULT | UCSF Benioff Children's Hospital, Oakland, California, 94609, United States|Children's Hospital Colorado, Aurora, Colorado, 80045, United States|Smilow Cancer Hospital, New Haven, Connecticut, 06511, United States|Johns Hopkins All Children's Hospital, Saint Petersburg, Florida, 33701, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States|Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States|University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States|Hackensack University Medical Center, Hackensack, New Jersey, 07601, United States|Columbia University Medical Center - Department of Pediatrics, New York, New York, 10032, United States|Columbia University Medical Center, New York, New York, 10032, United States|The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, 27599, United States|Atrium Health, Charlotte, North Carolina, 28204, United States|University Hospitals Rainbow Babies & Children's Hospital, Cleveland, Ohio, 44106, United States|Cleveland Clinic, Cleveland, Ohio, 44195, United States|Nationwide Children's Hospital, Columbus, Ohio, 43205, United States|The James Cancer Hospital, Columbus, Ohio, 43210, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers, Nashville, Tennessee, 37203, United States|Texas Oncology - Baylor Charles A. Sammons Cancer Center, Dallas, Texas, 75246, United States|Cook Children's, Fort Worth, Texas, 76104, United States|Ottawa Hospital Research Institute, Ottawa, Ontario, K1H 8L6, Canada|Princess Margaret Cancer Centre, Toronto, Ontario, M5G 2M9, Canada|Centre Hospitalier Universitaire Sainte-Justine, Montréal, Quebec, H3T 1C5, Canada |
| Treatment of Hemoglobin SC Disease With Hydroxyurea | Sickle cell disease (SCD), specifically hemoglobin SC disease (HbSC), is a subtype of sickle cell disease with typically higher hemoglobin and milder or later disease complications. Sickle cell disease is a disorder in which red blood cells (RBCs) are abnormally shaped. This can result in painful episodes, serious infections, and damage to body organs. One medication used to treat sickle cell disease is hydroxyurea. Hydroxyurea therapy offers significant benefits for infants, children, and adolescents with sickle cell anemia. These include a reduction in the frequency of pain crises and acute chest syndrome (inflammation of the lungs). Hydroxyurea has been given to many HbSC patients but HbSC patients were not included in the large clinical trials used to test hydroxyurea in SCD, so less is known about how HbSC patients respond to hydroxyurea. The purpose of this research study is to see if hydroxyurea, a medication given to many children with the most common type of sickle cell, those who are homozygous for the sickle mutation (HbSS), helps children who have HbSC. The investigators will see if it helps by giving a questionaire when the medication is started, and then every two months at a clinic visit. The questionaire, called the Pediatric Quality of Life Inventory (PedsQL™) Sickle Cell Disease Module version 3.0, measures quality of life. The investigators will also see how hydroxyurea changes laboratory test numbers, and blood thickness. | Hemoglobin SC Disease | ALL | CHILD, ADULT | Texas Children's Hospital, Houston, Texas, 77030, United States |
| Screening for Renal Complications in Children and Young Adults With Major Sickle Cell Disease | Sickle cell disease is the subject of targeted neonatal screening (carried out when one of the two parents is from an endemic country - sub-Saharan Africa, South-East Asia, Central America, the Caribbean) during the Guthrie test. Haemolysis, which results from the abnormality of the haemoglobin, and the vascular activation it causes, are responsible for multiple organ damage. Major sickle cell syndromes (MSC), by several mechanisms, are responsible for a wide range of renal damage, culminating in end-stage renal failure at an average age of 45 years and with an average survival of 3 years beyond ESRD. The various renal disorders are : glomerular hyperfiltration and then glomerulosclerosis; tubular dysfunction, especially proximal and distal hyposthenuria (a factor in enuresis); papillary necrosis, renal infarction, episodes of acute renal failure during vaso-occlusive crises; dysregulation of the renin-angiotensin system with early arterial hypertension and, more rarely, extra-membranous glomerulonephritis. In the early stages of these conditions, simple paraclinical tests can identify them before the appearance of specific clinical signs. In patients suffering from MDS, the HAS (High Authority of Health) recommends an annual check-up carried out in a Competence Centre. According to the HAS recommendations for annual surveillance, in addition to the search for other organic complications, for renal pathology, only microalbuminuria and renal ultrasound are recommended. However, as the literature shows, microalbuminuria and ultrasound only detect some of these renal disorders and at a very late stage. A large number of publications in adults and, to a lesser degree, in children, demonstrate the correlation between the frequency of acute complications of sickle cell disease (episodes of haemolysis, etc.) and the occurrence of kidney damage. | Anemia|Sickle Cell Disease | ALL | CHILD, ADULT | CHU de Nice, Nice, 06001, France |
| Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD) | This study is evaluating a genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) product - OTQ923 to reduce the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT | University of Chicago, Chicago, Illinois, 60637, United States|Memorial Sloan Kettering Cancer Ctr, New York, New York, 10065, United States|St Jude Children's Research Hospital, Memphis, Tennessee, 38105-3678, United States |
| A Phase 1 Study of Continuous Intravenous L-citrulline During Sickle Cell Pain Crisis or Acute Chest Syndrome | Sickle cell disease is a genetic red blood cell disorder characterized by vaso-occlusion from sickling of red blood cells, that can lead to pain or organ complications such as acute chest syndrome. Sickle cell disease is associated with low amounts of nitric oxide, a compound important for dilating the blood vessel wall. Citrulline is a substance that is known to increase nitric oxide. The goal of this Phase I study are to find the highest safe dose of continuous IV citrulline that can be given to individuals with sickle cell disease experiencing a sickle cell pain crisis or acute chest syndrome without causing severe side effects. | Sickle Cell Disease | ALL | CHILD, ADULT | University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States |
| Topical Sodium Nitrite in Sickle Cell Disease and Leg Ulcers | The investigators are conducting a Phase II prospective and placebo controlled study of a topical cream containing sodium nitrite compared to the current standard of care. Sodium nitrite is a local donor of nitric oxide, which is known to improve blood flow and decrease bacterial load in the ulcer bed. The primary objectives are to evaluate the safety of topical sodium nitrite cream treatment in patients with sickle cell disease and chronic leg ulcers and to determine its effectiveness in accelerating the healing process and decreasing the pain associated with ulceration. Potential benefit will be a durable resolution or improvement of the leg ulcer and its associated pain. Possible side effects include decreased blood pressure and methemoglobinemia, secondary to sodium nitrite absorption through the ulcerated skin. Funding source FDA OOPD. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Montefiore Medical Center - Albert Einstein College of Medicine, Bronx, New York, 10461, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|University of Pittsburgh, Pittsburgh, Pennsylvania, 15260, United States |
| Pharmacokinetics, Pharmacodynamics and Safety of Epeleuton in Patients with Sickle Cell Disease | To assess the pharmacokinetics, pharmacodynamics and safety of Epeleuton capsules in adult SCD patients who are aged ≥18 years. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of Alabama at Birmingham (UAB), Birmingham, Alabama, 35294, United States|New England Sickle Cell Institute, UConn Health, Farmington, Connecticut, 06030-1163, United States|Medstar Health, Washington, District of Columbia, 20010, United States|Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta at Hughes Spalding, Atlanta, Georgia, 30303, United States|Emory University - Georgia Comprehensive Sickle Cell Center, Atlanta, Georgia, 30303, United States|Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta at Arthur M. Blank Hospital, Atlanta, Georgia, 30329, United States|UI Health Sickle Cell Center, Chicago, Illinois, 60612, United States|The Johns Hopkins University School of Medicine, Baltimore, Maryland, 21205, United States|The Center for Cancer and Blood Disorders, A Division of American Oncology Partners, PA, Bethesda, Maryland, 20817, United States|Kaiser Permanente Mid-Atlantic States, Largo, Maryland, 20774, United States|Karmanos Cancer Institute, Detroit, Michigan, 48201, United States|Robert Wood Johnson Medical School Rutgers, New Brunswick, New Jersey, 08901, United States|Newark Beth Israel Medical Center, Newark, New Jersey, 07112, United States|Jacobi Medical Center, Bronx, New York, 10461, United States|Queens Hospital Center, Jamaica, New York, 11432, United States|UNC Health, Chapel Hill, North Carolina, 27517, United States|East Carolina University, Greenville, North Carolina, 27834, United States|Science 37, Morrisville, North Carolina, 27560, United States|Foothills Medical Center, Calgary, Alberta, T2N 2T9, Canada|St Paul's Hospital Hematology/Oncology Research, Vancouver, British Columbia, V6E 1M7, Canada|Toronto General Hospital, Toronto, Ontario, M5G 2C4, Canada |
| Risk Clinical Stratification of Sickle Cell Disease in Nigeria, Assessment of Efficacy/Safety of Hydroxyurea Treatment | The vast majority of births with sickle cell disease (SCD) occur in Africa and 90% are thought to die before the age of five. Hydroxyurea (HU) is the only drug approved by the FDA for the treatment of sickle cell anemia. Although HU is used to treat small numbers of patients in Africa, cost, fear of toxicity, and lack of awareness and availability limit its use. The leukopenia that may be seen with HU raises the possibility of increased susceptibility to infection. Risk stratification - i.e., identification of patients most likely to benefit- could focus therapy and provide confidence that the risk:benefit ratio is favorable. Several clinical measures of future risk are well defined and findings on modifier genes in the US, primarily related to fetal hemoglobin (HbF), have further improved risk prediction. Whether the genetic variants predict severity in Africa is not known. The investigators have established a SCD cohort in Ibadan, Nigeria. In the first phase of this research the investigators will implement clinical risk examinations and assess the relationship between clinical characteristics (including levels of HbF) and known genetic markers. As a proxy for a birth cohort, the investigators will compare the frequency of the genetic markers in adult patients (i.e., "survivors") to children. In the second phase the investigators will randomize 40 high risk adult patients to fixed low dose HU or no HU treatment in a crossover design and monitor hematologic and physiologic parameters to document hematologic effects and safety. This work will lay the basis for a large-scale trial to document safety and efficacy. | Sickle Cell Disease|Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | University of Ibadan College of Medicine, Ibadan, Oyo State, Nigeria |
| Voxelotor Sickle Cell Exercise Study | This study is a pilot, open-label, single-arm study to evaluate the effect of the sickle cell medication voxelotor on exercise capacity, as measured by cardiopulmonary exercise testing (CPET) in patients 12 years of age and older with sickle cell anemia (SCA). | Sickle Cell Anemia | ALL | CHILD, ADULT, OLDER_ADULT | Pediatric Specialist of Virginia, Fairfax, Virginia, 22031, United States |
| "Association of Proteinuria and Progression of Kidney Dysfunction in Sickle Cell Disease"Disease | To describe change in ACR and eGFR during study follow-up, and assesss the association of baseline and change in ACR and eGFR, with progression of kidney failure and/or all-cause mortality. | Change in Albumin to Creatinine Ratio and Glomerular Filtration Rate|Progression of Kidney Failure and or All-cause Mortality | ALL | ADULT, OLDER_ADULT | Henri Mondor Hospital, Creteil, 94000, France |
| Resolution of Sickle Cell Leg Ulcers With Voxelotor | This study is a Phase 3, multicenter, randomized, placebo-controlled study to evaluate the efficacy of voxelotor and standard of care for the treatment of leg ulcers in participants with sickle cell disease. The study is divided into a 5 study periods: Screening, Run-in, Randomized Treatment, Open-label Treatment, and Follow-up/End of Study (EOS). The study will be conducted in approximately 80 eligible participants at approximately 20 global clinical trial sites. | Sickle Cell Disease|Leg Ulcers | ALL | CHILD, ADULT, OLDER_ADULT | Complexo Hospitalar Universitário Professor Edgard Santos- Universidade Federal Da Bahia, Salvador, Bahia, 40110-060, Brazil|Universidade Federal Da Bahia Hospital Universitário Professor Edgard Santos, Salvador, Bahia, 40110-060, Brazil|Hospital São Rafael, Salvador, Bahia, 41253-190, Brazil|Hospital das Clinicas da Universidade Federal de Minas Gerais, Belo Horizonte, MG, 30130-100, Brazil|Hospital das Clinicas da Universidade Federal de Minas Gerais, Belo Horizonte, Minas Gerais, 30130-100, Brazil|Multihemo Servicos Medicos S/A, Recife, Pernambuco, 50070-460, Brazil|Hospital das Clínicas da Faculdade de Medicina da Universidade de Sao Paulo, Cerqueira Cesar - Sao Paulo, SP, 05403-000, Brazil|Hospital das Clinicas da Faculdade de Medicina de Ribeirão Preto da Universidade de Sao Paulo, Ribeirão Preto, SÃO Paulo, 14051-140, Brazil|Esho Empresa De Servicos Hospitalares S.A/ Hospital Samaritano Higienopolis, Sao Paulo, 01232-010, Brazil|Hospital das Clínicas da Faculdade de Medicina da Universidade de Sao Paulo, Sao Paulo, 05403-000, Brazil|Hospital das Clínicas da Faculdade de Medicina da Universidade de Sao Paulo, São Paulo, 01246-000, Brazil|KEMRI Centre for Clinical Research Butere County Hospital, Butere, Kakamega County, 50100, Kenya|KEMRI Kondele Children's Hospital within Jaramogi Oginga Odinga Teaching and Referral Hospital., Kisumu, 40100, Kenya|Gertrude's Children's Hospital, Nairobi, 00100, Kenya|KEMRI-Centre for Respiratory Diseases Research-Nairobi, Nairobi, 00100, Kenya|Strathmore University Medical Centre, Nairobi, 00200, Kenya|KEMRI-CRDR, KEMRI Clinical Research Annex, Siaya, 40600, Kenya|SYNLAB, Gwagwalada, Abuja, 902101, Nigeria|University of Calabar Teaching Hospital, Calabar, Cross River, 540281, Nigeria|SYNLAB, Abuja, FCT, 902101, Nigeria|University of Abuja Teaching Hospital, Gwagwalada, FCT, 902101, Nigeria|Barau Dikko Teaching Hospital/Kaduna State University, Kaduna, 800212, Nigeria|Aminu kano Teaching Hospital, Kano, 700233, Nigeria|Lagos University Teaching Hospital, Lagos, 100254, Nigeria |
| Skin and Blood Research Samples From Healthy Volunteers and Patients With Hematologic Diseases | The investigators plan to obtain skin and blood samples from healthy volunteers and patients with a benign, inherited hematologic disease to use for research to use homologous recombination to correct β-globin gene mutations in therapeutically useful cells, like autologous induced pluripotent stem cells from sickle cell anemia patients. | Anemia, Sickle Cell | ALL | ADULT, OLDER_ADULT | Washington University School of Medicine, St. Louis, Missouri, 63110, United States |
| Improving Parental Psychosocial Functioning and Early Developmental Outcomes in Children With Sickle Cell Disease | There are all significant risk factors for poor early cognitive development and, as such, neurocognitive deficits have been demonstrated in pre-school children with sickle cell disease (SCD). This project assesses the efficacy of using an evidence-based early stimulation program, combined with components to help parents cope with stress, delivered during six routine monthly clinic visits to parents of children with sickle cell disease. It is hoped that this innovation will improve parental psychological outcomes, as well as child developmental outcomes. | Sickle Cell Disease|Cognitive Ability, General|Psychological Stress|Parenting | ALL | CHILD | Sickle Cell Unit, University of West Indies, Mona Campus, Kingston 7, Jamaica |
| Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN 1503) | This is a clinical trial that will compare survival and sickle related outcomes in adolescents and young adults with severe sickle cell disease after bone marrow transplantation and standard of care. The primary outcome is 2-year overall survival. | Sickle Cell Disease | ALL | CHILD, ADULT | Benioff Children's Hospital at Oakland, Oakland, California, 94609, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States|University of Florida Gainsville, Gainesville, Florida, 32611, United States|Foundation for Sickle Cell Research/Florida Sickle Inc., Hollywood, Florida, 33021, United States|University of Miami, Miami, Florida, 33136, United States|Grady Memorial Hospital, Atlanta, Georgia, 30303, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States|Emory University, Atlanta, Georgia, 30322, United States|Augusta University Medical Center, Augusta, Georgia, 30912, United States|University of Iowa, Iowa City, Iowa, 52242, United States|Children's Hospital of New Orleans, New Orleans, Louisiana, 70118, United States|Dana Farber Cancer Institute/Brigham & Women's Hospital, Boston, Massachusetts, 02114, United States|Dana Farber Cancer Institute/Massachusetts General Hospital, Boston, Massachusetts, 02115, United States|Boston University, Boston, Massachusetts, 02215, United States|Barbara Ann Karmanos Cancer Institute, Detroit, Michigan, 48201, United States|Washington University/St. Louis Children's Hospital, Saint Louis, Missouri, 63110, United States|Hackensack University Medical Center, Hackensack, New Jersey, 07601, United States|Newark Beth Israel Medical Center, Newark, New Jersey, 07112, United States|Montefiore Medical Center/Albert Einstein School of Medicine, Bronx, New York, 10467, United States|New York Presbyterian Brooklyn Methodist Hospital, Brooklyn, New York, 11215, United States|Cohen Children's Medical Center, New Hyde Park, New York, 11040, United States|Icahn School of Medicine at Mount Sinai, New York, New York, 10029, United States|Weill Cornell Medical College, New York, New York, 10065, United States|University of North Carolina Hospital at Chapel Hill, Chapel Hill, North Carolina, 27516, United States|Duke University Medical Center, Durham, North Carolina, 27705, United States|Ohio State University, Columbus, Ohio, 43210, United States|University of Oklahoma, Oklahoma City, Oklahoma, 73104, United States|Oregon Health Sciences University, Portland, Oregon, 97239, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|University of Texas Health Sciences Center, Houston, Texas, 77004, United States|Baylor College of Medicine/The Methodist Hospital, Houston, Texas, 77030, United States|University of Texas/MD Anderson CRC, Houston, Texas, 77030, United States|University of Virginia, Charlottesville, Virginia, 22908, United States|Virginia Commonwealth University, Richmond, Virginia, 23298, United States |
| Rejuvesol® Washed RBC in Sickle Cell Patients Requiring Frequent Transfusions | The objective of this proposal is to test the feasibility of red blood cell (RBC) rejuvenation to chronic transfusion in sickle cell disease (SCD) and the potential benefit of RBC rejuvenation in this population to determine if a larger clinical trial powered to definitively characterize the benefits of rejuvenation is warranted. This is a small pilot study is to see if restoring important energy molecules (ATP and 2,3,DPG) in stored red blood cells before they are transfused, with a rejuvenating solution (Rejuvesol), offers any advantages to individuals over standard blood transfusion. Subjects will receive either rejuvenated (R) or standard (S) RBCs with each transfusion for 6 transfusions (over approximately a 6-month period) in a pre-defined order to maximize detection of any signal. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States|Duke Univeristy Medical Center, Durham, North Carolina, 27710, United States |
| Ibuprofen and Opioid (Morphine or Diamorphine) for Acute Pain in Sickle Cell Disease - Sickle With Ibuprofen & Morphine | The use of oral ibuprofen combined with Opioid (Morphine or Diamorphine) administered through patient controlled analgesia (PCA) will be clinically effective for acute pain crisis in adults with sickle cell disease (SCD). | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | North West London Hospitals NHS Trust, London, NW10 7NS, United Kingdom|Imperial College Healthcare NHS Trust, London, W12 0HS, United Kingdom |
| MAST - Magnesium for Sickle Cell Acute Crisis in Children | The purpose of this study is to determine if intravenous magnesium sulfate treatment is effective in reducing the length of stay and pain in children with sickle cell disease suffering an acute vaso-occlusive episode. | Anemia, Sickle Cell | ALL | CHILD, ADULT | The Hospital for Sick Children, Toronto, Ontario, M5G 1X8, Canada |
| Tocilizumab for Acute Chest Syndrome | The investigators are evaluating the role of a low dose of tocilizumab in treating acute chest syndrome in patients with sickle cell disease. Tocilizumab inhibits interleukin-6 (IL-6) receptors and is used to treat rheumatoid arthritis and severe cytokine release syndrome, which can be seen with chimeric antigen receptor T-cell (CAR-T) therapy, and it is also authorized for treatment of COVID-19. Since IL-6 levels are elevated in the sputum of patients with acute chest syndrome, the investigators are hopeful that this will be an effective strategy. The investigators will be looking at how a low dose of tocilizumab affects oxygen status, clinical outcomes, and laboratory markers in patients admitted to the hospital with acute chest syndrome. | Sickle Cell Disease|Acute Chest Syndrome | ALL | CHILD, ADULT, OLDER_ADULT | University of Chicago, Chicago, Illinois, 60637, United States |
| Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease | This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical allogeneic stem cell transplantation with CD34 enrichment and T-cell addback. This patient population historically has a risk of developing sinusoidal obstructive syndrome (SOS) and Defibrotide has demonstrated efficacy in treatment of SOS. The Funding Source is FDA OOPD. | Sickle Cell Disease | ALL | CHILD, ADULT | University of California Los Angeles, Los Angeles, California, 90095, United States|University of Florida, Gainesville, Florida, 32610-0278, United States|New York Medical College, Valhalla, New York, 10595, United States|Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| A Study to Assess the Effect of Ticagrelor in Reducing the Number of Days With Pain in Patients With Sickle Cell Disease | The purpose of this study is to determine whether ticagrelor is effective in reducing the number of days of pain, intensity of pain, and reducing the use of analgesics due to sickle cell disease | Sickle Cell Disease | ALL | ADULT | Research Site, Miami, Florida, 33136, United States|Research Site, Bethesda, Maryland, 20817, United States|Research Site, Charleston, South Carolina, 29425, United States|Research Site, Alexandria, 21131, Egypt|Research Site, Cairo, 11562, Egypt|Research Site, Cairo, 11566, Egypt|Research Site, Bordeaux Cedex, 33076, France|Research Site, Strasbourg, 67091, France|Research Site, Verona, 37134, Italy|Research Site, Kikuyu, 00100, Kenya|Research Site, Kisian, 40100, Kenya|Research Site, Nairobi, 40100, Kenya|Research Site, Beirut, 1107 2020, Lebanon|Research Site, Beirut, 113-6044, Lebanon|Research Site, Adana, 01130, Turkey|Research Site, Mersin, 33079, Turkey|Research Site, Van, 65080, Turkey|Research Site, Harrow, HA1 3UJ, United Kingdom|Research Site, London, E1 1BB, United Kingdom|Research Site, London, E9 6SR, United Kingdom |
| Improving Sickle Cell Disease (SCD) Care Using Web-based Guidelines | The overall goal of this proposed project is to 1) increase co-management between sickle cell specialists and primary care providers (PCP's); 2) increase the use of hydroxyurea (HU) which prevents Vaso-Occlusive Episode (VOE), EDs and subsequent hospitalizations, and death; 3) identify and link patients not receiving primary or SCD specialty care to care, and 4) shift healthcare use from EDs and hospitalizations to primary and specialty co-management. Many persons with SCD experience a poor quality of life, serious medical complications and frequent painful events that require treatment from SCD specialty care, primary care and emergency department (ED) providers. There are two dominating models of care in the United States; neither are ideal. Many people with SCD have all of their healthcare needs addressed by sickle cell specialists who do not typically provide primary care and are often geographically distant from the patients' home. Other sickle cell patients receive all of their care in EDs. Both models are inadequate and result in an alarmingly high number of ED visits for many patients. Current care models are neither cost efficient nor promoting optimal patient outcomes. To improve outcomes, the investigators will implement a new model of care for SCD using nurse care managers, web based-interactive algorithms, and test if additional patient provided coaching can improve outcomes. | Sickle Cell Disease | ALL | CHILD, ADULT | Duke University, Durham, North Carolina, 27710, United States |
| Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females with Sickle Cell Disease | This research is being conducted to see if using an injectable contraception, Depot Medroxyprogesterone Acetate (Depo-Provera), can reduce the pain experienced by women with sickle cell disease. Participants in this study will be adult women with sickle cell disease who regularly experience sickle cell pain. They will complete a 3-month "baseline "with no use of hormonal contraception, and then a 3-month follow-up after receiving an injection of Depo-Provera. Participants will complete 6 to 7 in-person visits with a urine pregnancy test, blood draw, and surveys, as well as complete remote weekly surveys and monthly home pregnancy tests. | Sickle Cell Disease (SCD)|Vaso-Occlusive Pain Episode in Sickle Cell Disease | FEMALE | ADULT | Johns Hopkins Medical Center, Baltimore, Maryland, 21287, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|University of Pennsylvania, Philadelphia, Pennsylvania, 19104, United States |
| Safety and Efficacy of Gene Therapy of the Sickle Cell Disease by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains CD34+ Cells Transduced ex Vivo With the GLOBE1 Lentiviral Vector Expressing the βAS3 Globin Gene in Patients With Sickle Cell Disease (DREPAGLOBE) | The purpose of this study is to evaluate the Safety and Efficacy of Gene Therapy of the Sickle Cell disease by Transplantation of an Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced ex vivo with the GLOBE1 lentiviral vector expressing the βAS3 globin gene (GLOBE1 βAS3 Modified Autologous CD34+ Cells) in Patients with Sickle Cell Disease (SCD) | Sickle Cell Disease | ALL | CHILD, ADULT | Department of Biotherapy, Necker-Enfants Malades Hospital, Paris, 75015, France |
| Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease | The purpose of the study is to Evaluate the Effect of Ticagrelor versus Placebo in Reducing the Rate of Vaso-Occlusive Crises in Paediatric Patients with Sickle Cell Disease | Sickle Cell Disease | ALL | CHILD | Research Site, Fort Lauderdale, Florida, 33316, United States|Research Site, Miami, Florida, 33155, United States|Research Site, Chicago, Illinois, 60612, United States|Research Site, Oak Lawn, Illinois, 60453, United States|Research Site, Las Vegas, Nevada, 89135, United States|Research Site, Brooklyn, New York, 11212, United States|Research Site, Charleston, South Carolina, 29425, United States|Research Site, Brussel, 1200, Belgium|Research Site, Edegem, 2650, Belgium|Research Site, Porto Alegre, 90035-903, Brazil|Research Site, Rio de Janeiro, 20211-080, Brazil|Research Site, Salvador, 41253-190, Brazil|Research Site, São Paulo, 01221010, Brazil|Research Site, São Paulo, 08270-070, Brazil|Research Site, Al Sharkeya, 44519, Egypt|Research Site, Alexandria, 21131, Egypt|Research Site, Cairo, 11521, Egypt|Research Site, Cairo, 11566, Egypt|Research Site, Cairo, 12655, Egypt|Research Site, Dakahlia, 35516, Egypt|Research Site, Accra, 233, Ghana|Research Site, Ho, 0, Ghana|Research Site, Kumasi, 1934, Ghana|Research Site, Athens, 11521, Greece|Research Site, Mumbai, 400053, India|Research Site, Nagpur, 440003, India|Research Site, Nagpur, 440012, India|Research Site, Pune, 411001, India|Research Site, Pune, 411004, India|Research Site, Wardha, 442004, India|Research Site, Catania, 95123, Italy|Research Site, Naples, 80138, Italy|Research Site, Padova, 35128, Italy|Research Site, Verona, 37126, Italy|Research Site, Kisumu, 40100, Kenya|Research Site, Nairobi, 00200, Kenya|Research Site, Siaya, 40600, Kenya|Research Site, Beirut, 11-0236, Lebanon|Research Site, Tripoli, 961, Lebanon|Research Site, Cape Town, 7700, South Africa|Research Site, Soweto, 2013, South Africa|Research Site, Barakaldo, 48903, Spain|Research Site, Barcelona, 08035, Spain|Research Site, Madrid, 28007, Spain|Research Site, Madrid, 28009, Spain|Research Site, Mbeya, 2410, Tanzania|Research Site, Antalya, 7070, Turkey|Research Site, Mersin, 33343, Turkey|Research Site, Seyhan, 01130, Turkey|Research Site, Kampala, 10005, Uganda|Research Site, Masaka, 0000, Uganda|Research Site, Cardiff, CF14 4XW, United Kingdom|Research Site, Glasgow, G51 4TF, United Kingdom|Research Site, London, E1 1BB, United Kingdom|Research Site, London, SE1 7EH, United Kingdom |
| Rollover Study for Patients With Sickle Cell Disease Who Have Completed a Prior Novartis-Sponsored Crizanlizumab Study | This is a multi-center multi-national rollover study to allow continued access to crizanlizumab for patients with sickle cell disease (SCD) who are on crizanlizumab treatment in a Novartis-sponsored study (parent study) and are benefiting from the treatment as judged by the investigator. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | University Of Alabama, Birmingham, Alabama, 35233, United States|Childrens National Hospital, Washington, District of Columbia, 20010, United States|Augusta University Georgia, Augusta, Georgia, 30912, United States|East Carolina University, Greenville, North Carolina, 27834, United States|East Carolina University, Greenville, North Carolina, 27858, United States|Childrens Hospital Of Philadelphia, Philadelphia, Pennsylvania, 19104-4399, United States|Cook Childrens Medical Center, Fort Worth, Texas, 76104, United States|Novartis Investigative Site, Brussel, 1000, Belgium|Novartis Investigative Site, Laeken, 1020, Belgium|Novartis Investigative Site, Liege, 4000, Belgium|Novartis Investigative Site, Salvador, BA, 41253-190, Brazil|Novartis Investigative Site, Ribeirao Preto, SP, 14051-140, Brazil|Novartis Investigative Site, Sao Paulo, SP, 01232-010, Brazil|Novartis Investigative Site, Cali, Valle Del Cauca, 760012, Colombia|Novartis Investigative Site, Monteria, 230004, Colombia|Novartis Investigative Site, Creteil, 94010, France|Novartis Investigative Site, Paris 15, 75015, France|Novartis Investigative Site, Paris 15, 75015, France|Novartis Investigative Site, Heidelberg, 69120, Germany|Novartis Investigative Site, Padova, PD, 35128, Italy|Novartis Investigative Site, Orbassano, TO, 10043, Italy|Novartis Investigative Site, Beirut, 1107 2020, Lebanon|Novartis Investigative Site, Tripoli, 1434, Lebanon|Novartis Investigative Site, Muscat, 123, Oman|Novartis Investigative Site, Barcelona, Catalunya, 08035, Spain|Novartis Investigative Site, Madrid, 28009, Spain|Novartis Investigative Site, Adana, 01250, Turkey|Novartis Investigative Site, Adana, 01330, Turkey|Novartis Investigative Site, Antakya Hatay, 31100, Turkey |
| Dose Escalation Study of PF-07209326 in Healthy Participants and Participants With Sickle Cell Disease | This Phase 1 first-in-human, first-in-patient, single ascending dose and multiple dose study will be a randomized, double-blind, placebo-controlled investigation of the safety, tolerability, and pharmacokinetics of PF-07209326 in healthy participants and participants with sickle cell disease. | Healthy|Sickle Cell Anemia | ALL | CHILD, ADULT, OLDER_ADULT | New Haven Clinical Research Unit, New Haven, Connecticut, 06511, United States|Howard University College of Medicine, Washington, District of Columbia, 20060, United States|Golisano Children's Hospital of Southwest Florida, Fort Myers, Florida, 33908, United States|Lee Health - Golisano Children's Hospital of Southwest Florida, Fort Myers, Florida, 33908, United States|Foundation for Sickle Cell Disease Research, Hollywood, Florida, 33023, United States|Foundation for Sickle Cell Disease Research, Hollywood, Florida, 33024, United States|Children's Healthcare of Atlanta - Egleston Hospital-Aflac Cancer and Blood Disorders Center, Atlanta, Georgia, 30322, United States|University of Illinois at Chicago Clinical Research Center, Chicago, Illinois, 60612, United States|University of Illinois at Chicago, Chicago, Illinois, 60612, United States|Prism Research LLC dba Nucleus Network, Saint Paul, Minnesota, 55114, United States|Columbia University Medical Center - Herbert Irving Pavilion, New York, New York, 10032, United States|CUIMC Research Pharmacy, New York, New York, 10032, United States|CUMC Research Pharmacy, New York, New York, 10032, United States|UT Physicians Comprehensive Sickle Cell Center Houston, Houston, Texas, 77004, United States|Memorial Hermann clinical research unit, Houston, Texas, 77030, United States|UT Physicians Comprehensive Sickle Cell Center Houston, Houston, Texas, 77030, United States |
| Study of MGTA-145 and Plerixafor in Patients With Sickle Cell Disease | This research study is designed to investigate a new potential medicine for mobilizing stem cells and apheresis collection in patients with Sickle Cell Disease. MGTA-145, the new potential medicine, will be given with plerixafor. | Sickle Cell Disease | ALL | ADULT | National Institutes of Health, Bethesda, Maryland, 20892, United States|Boston Children's Hospital, Boston, Massachusetts, 02115, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| A Safety, Efficacy, and Pharmacokinetic (PK) Study of HBI-002, an Oral Carbon Monoxide (CO) Therapeutic, in Subjects With Sickle Cell Disease (SCD) | This is a multi-center, open label Phase 2a clinical trial in subjects with sickle cell disease to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO), with doses daily for 14 days. | Anemia, Sickle Cell | ALL | CHILD, ADULT | Contact Company, San Diego, California, 92121, United States |
| Pediatric Hydroxyurea in Sickle Cell Anemia (PED HUG) | To determine whether hydroxyurea prevents the onset of chronic end organ damage in young children with sickle cell anemia. | Anemia, Sickle Cell|Hematologic Diseases|Hemoglobinopathies | ALL | CHILD, ADULT | |
| Steroid Injection for Sickle Cell Arthropathy Pain Therapy | Sickle cell disease (SCD) is associated with arthropathy. Arthropathy may require periarticular corticosteroid injection therapy. This observational study examines efficacy, and safety of steroid injections in SCD patients. Data collection includes patient's gender, age, race, smoking history, alcohol intake, analgesic use, pain score, sleep quality, limb joint injections, post-injection analgesia, and post-injection complication. Pain is measured using numeric pain scale. Sleep quality is measured using Likert scale. | Sickle Cell Disease|Sickle Cell Abnormality | ALL | ADULT, OLDER_ADULT | Salem Anaesthesia Pain Clinic, Surrey, British Columbia, V3S 7J1, Canada |
| The Effect of Voxelotor on Cerebral Hemodynamic Response in Children with Sickle Cell Anemia | Voxelotor is a new drug for adolescents and adults with sickle cell disease that improves hemoglobin levels and reduces the incidence of worsening anemia. However, it is unclear whether this increase in hemoglobin is associated with a reduction in cerebral metabolic stress. This study will measure the effects of voxelotor on cerebral hemodynamics. | Sickle Cell Anemia in Children | ALL | CHILD, ADULT | Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States|Aflac Sickle Cell Comprehensive Clinics at Children's Healthcare of Atlanta, Scottish Rite, Atlanta, Georgia, 30342, United States |
| A Randomized Trial of LOVAZA in Pediatric Sickle Cell Disease (SCD) | The purpose of the study is to determine the effectiveness of LOVAZA (fish oil capsules) to decrease inflammation in children and adolescents with Sickle Cell Disease (SCD). It has been found that besides the damage caused by sickle red blood cells themselves, the inflammatory response that occurs in SCD patients could potentially play a significant role in the occurrence of painful episodes or pain crises. The investigators will also study whether the subject/caregiver feels that there is an improvement in the child's quality of life by taking the medication. Besides the effect of LOVAZA on inflammation,the investigators are also testing whether the drug will have a beneficial effect on blood clotting ability (which is known to be increased in SCD) and on the anemia (low red blood cells) that is part of the disease entity. | Sickle Cell Disease|HEMOGLOBIN SS|Hemoglobin S Beta-0 Thalassemia|Inflammation|Quality of Life | ALL | CHILD, ADULT | Thomas Jefferson University Hospital, Philadelphia, Pennsylvania, 19107, United States|St. Christopher's Hospital for Children, Drexel University, Philadelphia, Pennsylvania, 19134-1095, United States |
| Effect of Simvastatin Treatment on Vaso-occlusive Pain in Sickle Cell Disease | The purpose of this study is to determine whether simvastatin is effective in reducing the frequency and intensity of vaso-occlusive pain episodes in patients with sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital and Research Center Oakland, Oakland, California, 94609, United States |
| Study to Evaluate the Effect of Ticagrelor Versus Placebo in Reducing Vaso-Occlusive Crises Rate in Pediatric Patients With Sickle Cell Disease. | The purpose of this study is to compare the effect of ticagrelor vs placebo for the reduction of Vaso-Occlusive crises in paediatric patients with Sickle Cell Disease | Sickle Cell Disease | ALL | CHILD | |
| Hemolysis Related Complications in SCD. A Phase II Study With Voxelotor | Intro: Sickle cell disease is a genetic disorder caused by a mutation of the β hemoglobin called HbS, which causes red blood cell (RBC) abnormalities responsible for hemolysis, mainly intravascular, leading to chronic anemia. Intravascular hemolysis is responsible for severe inflammation and endothelial dysfunction. Maintaining hemoglobin in its oxygenated R-conformation is one of the strategies for inhibiting the polymerization of HbS. Previous experimental therapeutic approaches having this effect have been discontinued due to poor pharmaceutical properties or toxicity. Nevertheless, they proved the validity of the concept by demonstrating an increase in oxyhemoglobin and a decrease in biomarkers of hemolysis. Voxelotor binds to the α chain of globin and maintains Hb in its R conformation, thereby inhibiting the polymerization of HbS while increasing the affinity of Hb for oxygen. Because of its mechanism of action affecting anemia and hemolysis, Voxelotor is a promising treatment for the prevention and treatment of renal and cerebral arterial disease. Hypothesis/Objective : Investigator hypothesis is that the treatment by Voxelotor (GBT440) will improve intra vascular hemolysis and will increase the total mass of hemoglobin with beneficial effects on organ function. The primary objective of the study is to evaluate the biological activity of Voxelotor on the reduction of intra vascular hemolysis measured by plasma hemoglobin. The secondary objectives of the study will aim at characterizing the effects of GBT 440 Voxelotor on: * Intra vascular hemolysis measured by plasma Heme * Total hemoglobin mass (MHb) * RBCs lifespan * Blood volumes (plasma volume (PV), red blood cell mass (RBCM), total blood volume (BV)) * Blood viscosity * Cerebral perfusion * Cerebrovascular vaso-reactivity * Cognitive function (MoCA) * Six minute walk test * Renal perfusion and iron deposits in renal cortex * Measurement of Glomerular filtration rate Estimation of glomerular filtration rate (CKD/EPI equation) * Urine albumin/creatinine ratio * Ability to decrease or stop erythropoietin in patients under EPO treatment * Safety (VOC, ACS, Priapism) and tolerability of voxelotor * RBC properties Method: This is an open-label, single-arm, single-stage phase II trial in patients treated with Voxelotor 1500 mg daily for 48 weeks. Assessments will be done during the study at week 0, week 6, week 12, week 24, week 36 and week 48. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Hospital Henri Mondor, Créteil, 94010, France |
| Single-center Prospective Evaluation of Sickle Cell Patient Care in the CHU Brugmann Emergency Department | Sickle Cell Disease is a serious disease that is life-threatening for patients being homozygous for the SS form or heterozygous for the SC or bthal forms. The CHU Brugmann hospital currently regularly treats about 70 homozygous adult patients and this number is in constant augmentation. The age average of the patients is below 30. The hospital developed a close collaboration with the Queen Fabiola Kids University Hospital to optimize the transition of young sickle cell patients from the pediatric to the adult network. The emergency care of sickle cell patients remains a source of worry. Even with a correct treatment (Hydroxy-urea or exsanguineous transfusions), patients suffer from frequent sickle cell disease crisis when stress or infection cause hemolysis. The pain level is intolerable and causes emergency hospital admission (2 to 3 crisis per patient per year on average). The crisis are more frequent with poor compliance to the treatments. There are several obstacles to the rapid and optimal management of these patients: * fear of causing addiction to heavy pain releaf products (high dosis of morphine) * lack of biological parameters for the determination of the crisis severity. The prognostic value of the lactate dehydrogenase (LDH) level in a vaso-occlusive crisis was recently stressed while activation of the coagulation, translated by the elevation of various parameters including the rate of DD dimers, seemed associated with clinical complications. The deleterious role of increased oxidative stress has also been recently demonstrated in patients with sickle cell disease, opening new therapeutic avenues. This study aims to prospectively evaluate the management of sickle cell patients being admitted in the emergency department for a vaso-occlusive crisis. The level of satisfaction of the patients will be measured. The investigators will also evaluate the predictive value of several routine biological parameters regarding the severity of the crisis, including the values of nitrous albumin (PNA) as marker of oxydative stress. This last dosage will be made in collaboration with the team of Dr Wayenberg and Pr Bottari in Grenoble. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | CHU Brugmann, Brussels, 1020, Belgium |
| Vaporized Cannabis for Chronic Pain Associated With Sickle Cell Disease | Our primary objective is to assess whether inhaling vaporized cannabis ameliorates chronic pain in patients with sickle cell disease (SCD). As these patients will all be on chronic opioid analgesics, the investigators will also assess the possible synergistic affect between inhaled cannabis and opioids. The investigators will also assess the clinical safety of the concomitant use of cannabinoids and these opioids in patients with SCD by monitoring the short-term side effects associated with combined therapy. Finally, the investigators will evaluate the short-term effects of inhaled cannabis on markers of inflammation and disease progression in patients with SCD. Hypotheses are as follows: 1. Inhaled cannabis will significantly reduce chronic pain in patients with SCD. 2. Inhaled cannabis will significantly alter the short-term side effects experienced by patients who take opioids for SCD. 3. Inhaled cannabis will significantly alter markers of inflammation and disease progression in patients with SCD compared to placebo. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | San Francisco General Hospital, San Francisco, California, 94110, United States |
| A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD) | The main purpose of the study is to evaluate the efficacy and safety of the study drug known as prasugrel for the reduction of Vaso-Occlusive Crisis events in pediatric participants with sickle cell disease. The study will also investigate reduction in daily pain in children who have sickle cell disease. | Sickle Cell Disease | ALL | CHILD | Children's Hospital of Oakland, Oakland, California, 94609, United States|Stanford Univ Medical Center, Palo Alto, California, 94304, United States|Connecticut Children's Medical Center, Hartford, Connecticut, 06106, United States|Howard University Hospital, Washington, District of Columbia, 20060, United States|Emory University, Atlanta, Georgia, 30322, United States|Memorial Health University Medical Center, Savannah, Georgia, 31404, United States|Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States|Boston Children's Hospital, Boston, Massachusetts, 02115, United States|Childrens Hospital of Michigan, Detroit, Michigan, 48201, United States|Children's Mercy Hospital, Kansas City, Missouri, 64108, United States|Albert Einstein College of Medicine, Bronx, New York, 10467, United States|University of NC at Chapel Hill School of Medicine, Chapel Hill, North Carolina, 27599, United States|Childrens Hospital Medical Center, Cincinnati, Ohio, 45229, United States|Rainbow Babies and Children's Hospital, Cleveland, Ohio, 44106, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19134, United States|St Christophers Hospital For Children, Philadelphia, Pennsylvania, 19134, United States|Childrens Hospital of Pittsburgh, Pittsburgh, Pennsylvania, 15224, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|St Jude Childrens Research Hospital, Memphis, Tennessee, 38105, United States|Mary Bridge Children's Hospital and Health Center, Tacoma, Washington, 98405, United States|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Brussel, 1200, Belgium|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Montegnee, 4420, Belgium|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Rio De Janeiro, 20211-030, Brazil|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Montreal, Quebec, H3T 1C5, Canada|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Alexandria, 21131, Egypt|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Cairo, 11566, Egypt|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Fayoum, 63514, Egypt|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Ismailia, Egypt|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Mansoura, 35516, Egypt|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Zagazig, 44519, Egypt|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Agogo, Ghana|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Korle Bu, Ghana|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Genova, 16128, Italy|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Modena, 40124, Italy|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Monza, 20900, Italy|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Padova, 35138, Italy|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Verona, 37126, Italy|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Busia, 40100, Kenya|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Kisumu, Kenya|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Kombewa, Kenya|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Nairobi, Kenya|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Beirut, 5244, Lebanon|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Muscat, 123, Oman|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Jeddah, 21859, Saudi Arabia|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Balcali Adana, 01330, Turkey|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Mersin, 33079, Turkey|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Abu Dhabi, United Arab Emirates|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Tooting, London, SW17 0QT, United Kingdom|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., London, SE1 7EH, United Kingdom|For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Manchester, M13 9WL, United Kingdom |
| Study of Beet Juice for Patients With Sickle Cell Anemia | The investigators hypothesize that increasing plasma nitrite using dietary nitrate will improve platelet function and red cell deformability and decrease MCHC in patients with sickle cell disease. The investigators will test this hypothesis through administration of daily intake of beetroot juice (Unbeetable - Performance Drink) to patients with sickle cell disease for 28 days. The investigators will evaluate the safety of daily beet root juice intake in patients with sickle cell disease. In addition, the investigators will measure MCHC, red cell deformability, and platelet function (activation and aggregation) in response to daily intake of beet root juice in this patient population. | Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | Wake Forest University School of Medicine, Winston-Salem, North Carolina, 27157, United States |
| Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises | The purpose of this study was to determine whether the investigational drug SelG1 when given to sickle cell disease patients either taking or not taking hydroxyurea was effective in preventing or reducing the occurrence of pain crises. SelG1 prevents various cells in the bloodstream from sticking together. By stopping these cell-cell interactions, SelG1 may prevent small blood vessels from becoming blocked and therefore reduce the occurrence and severity of pain crises. Other effects of SelG1 was evaluated, as well as the safety of the drug and how long it stayed in the blood stream. Funding Source - FDA Office of Orphan Products Development (OOPD) | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Birmingham, Alabama, United States|Mobile, Alabama, United States|Little Rock, Arkansas, United States|Los Angeles, California, United States|Oakland, California, United States|Orange, California, United States|Sacramento, California, United States|Aurora, Colorado, United States|New Haven, Connecticut, United States|Washington, District of Columbia, United States|Daytona Beach, Florida, United States|Jacksonville, Florida, United States|Miami, Florida, United States|Orlando, Florida, United States|Tampa, Florida, United States|Atlanta, Georgia, United States|Augusta, Georgia, United States|Chicago, Illinois, United States|Peoria, Illinois, United States|Indianapolis, Indiana, United States|Louisville, Kentucky, United States|Baton Rouge, Louisiana, United States|New Orleans, Louisiana, United States|Baltimore, Maryland, United States|Bethesda, Maryland, United States|Boston, Massachusetts, United States|Detroit, Michigan, United States|Jackson, Mississippi, United States|Kansas City, Missouri, United States|Las Vegas, Nevada, United States|New Brunswick, New Jersey, United States|Newark, New Jersey, United States|Bronx, New York, United States|Brooklyn, New York, United States|New Hyde Park, New York, United States|Chapel Hill, North Carolina, United States|Durham, North Carolina, United States|Greenville, North Carolina, United States|Cleveland, Ohio, United States|Columbus, Ohio, United States|Oklahoma City, Oklahoma, United States|Philadelphia, Pennsylvania, United States|Pittsburgh, Pennsylvania, United States|Charleston, South Carolina, United States|Sumter, South Carolina, United States|Fort Worth, Texas, United States|Houston, Texas, United States|Norfolk, Virginia, United States|Richmond, Virginia, United States|Salvador, Bahia, Brazil|Porto Alegre, Rio Grande Do Sul, Brazil|Campinas, São Paulo, Brazil|São José do Rio Preto, São Paulo, Brazil|Rio de Janeiro, Brazil|São Paulo, Brazil|Kingston, Jamaica |
| Cannabinoids for the Reduction of Inflammation and Sickle Cell Related Pain | A randomized, double blind, study of dronabinol as a palliative agent in the treatment of pain, inflammation, and other complications of sickle cell disease (SCD). | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Mount Sinai Hospital, New York, New York, 10029, United States |
| Monocytic Expression of Heme Oxidase-1 (HO-1) in Sickle Cell Patients and Correlation With the Humoral Immune Response to Vaccine and With Allo-immunization. | Sickle cell disease (SCD) is an autosomal recessive disorder resulting from a substitution in the β chain of hemoglobin (Hb) which causes hemoglobin S to polymerize when deoxygenated. SCD patients present immune abnormalities that have always been attributed to functional asplenia. It it is now being recognized that patients with SCD have a pro-inflammatory condition with altered immune system activation contributing to the pathology of SCD. Increased levels of neutrophils, monocytes or cytokines have been reported in SCD patients. SCD is associated with many acute and chronic complications requiring immediate support. Actual strongly recommended therapies include chronic blood transfusions (CT) and hydroxyurea (HU). In addition, episodic transfusions are recommended and commonly used to manage many acute SCD complications.There is strong evidence to support the use of HU in adults with 3 or more severe vaso-occlusive crises during any 12-month period, with SCD pain or chronic anemia, or with severe or recurrent episodes of acute chest syndrome. HU use is now also common in children with SCD. Some patients receive chronic monthly RBC transfusion with the objective to reduce the proportion of HbS to \< 30 %. Long-term RBC transfusions prevent and treat complications of SCD decreasing the risk of stroke and the incidence of acute chest syndrome (ACS). Therapeutic complications, such as alloimmunization against RBC in 20-50% of patients or hematopoietic stem cell transplantation (HSCT) graft rejection, constitute an immune-based clinical issue in SCD. Poorly understood RBC alloimmunization is responsible for serious hemolytic transfusion reaction associated with severe mortality and morbidity underlying the need for a better understanding of the immunology of SCD to improve SCD transfusion support/outcome. Little evidence exists about HU effects on immune functions in SCD. HU treatment doesn't appear to have deleterious effects on immune function and appears to decrease the abnormally elevated number of total WBC and lymphocytes, while CT does not. Patients with SCD are at higher risk of infections and prophylactic vaccination is strongly recommended. Recent data suggest that vaccinal response to pneumococcal antigens in SCD patients is identical to healthy control while controversy concern the stability of the immune protection after vaccination of SCD patient. Antibody levels declined over the year and the need for more frequent vaccination in SCD patient should be investigated. Currently, there is no evidence whether HU may interfere with pneumococcal immune response. Purohit showed that immune response to inactivated influenza A (H1N1) virus vaccine was altered in patient with SCD receiving CT but little is known on immune response to vaccination in patients with SCD receiving HU. Recent data suggest that not only inflammatory status but also humoral immune response to antigens in SCD patients may differ according to treatment. Yazdanbakhsh reported an imbalance between regulatory T cell (Treg) and effector T cell (Teff) in alloimmunized SCD patients with as consequence an increase in antibody production. In a model proposed by the authors, the balance between Treg and Teff is dictated by the monocyte control of cytokines expression. Altered activity of monocyte heme oxidase-1 (HO-1) would be responsible of a decrease in IL-12 and an increase in IL-10 cytokines secretion impacting the Treg/Teff cells ratio and promoting antibody production by B cells. The objectives of the project are to assess whether different humoral immune responses to vaccines or to erythrocyte alloantigens are related to the type of treatment administered to patients with SCD. We also aim to study if these differences might be related to different expressions of HO-1 by monocytes. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | CHU Brugmann, Brussels, 1020, Belgium|HUDERF, Brussels, 1020, Belgium |
| A Study Evaluating the Long-Term Safety of ICA-17043 in Sickle Cell Disease Patients With or Without Hydroxyurea Therapy | This trial is a follow-up companion study to Protocol ICA-17043-10, a Phase III, multi-center, efficacy and safety study of ICA-17043. This is an open-label extension study collecting safety data on the use of ICA-17043 in subjects with sickle cell disease (SCD) (e.g., HbSS, HbSC, HbSb0-thalassemia, HbSb+-thalassemia subjects). All subjects who have successfully completed ICA-17043-10 will, if deemed appropriate by their study Investigator and appropriate consent by subject is given, enroll in the ICA-17043-12 study (Study 12). Only patients who participated in ICA-17043-10 are eligible for this open label study | Sickle Cell Disease|Sickle Cell Anemia | ALL | CHILD, ADULT, OLDER_ADULT | University of South Alabama, Mobile, Alabama, 36617, United States|Arkansas Children's Hospital, Little Rock, Arkansas, 72202, United States|Children's Hospital Oakland, Oakland, California, 94609, United States|University of California Davis Medical Center, Sacramento, California, 95817, United States|University of Colorado Health Sciences Center, Denver, Colorado, 80262, United States|Century Clinical Research, Inc., Holly Hill, Florida, 32117, United States|University of Florida Health Science Center, Jacksonville, Florida, 32209, United States|Medical College of Georgia, Augusta, Georgia, 30912, United States|Memorial Health University Medical Center, Savannah, Georgia, 31404, United States|University of Illinois Medical Center, Chicago, Illinois, 60612, United States|Sickle Cell Center of Northern Louisiana, Shreveport, Louisiana, 71103, United States|Johns Hopkins University School of Medicine, Baltimore, Maryland, 21205, United States|Brigham and Women's Hospital, Boston, Massachusetts, 02115, United States|Wayne State School of Medicine, Detroit, Michigan, 48201, United States|University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States|Hackensack University Medical Center, Hackensack, New Jersey, 07601, United States|Robert Wood Johnson Medical Center, New Brunswick, New Jersey, 08903, United States|Newark Beth Israel Medical Center, Newark, New Jersey, 07112-2027, United States|SUNY Downstate Medical Center, Brooklyn, New York, 11203, United States|New York Methodist Hospital, Brooklyn, New York, 11215, United States|University of North Carolina Hospitals, Chapel Hill, North Carolina, 27514, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|East Carolina University School of Medicine, Greenville, North Carolina, 27858, United States|Wake Forest University School of Medicine, Winston-Salem, North Carolina, 27157, United States|University of Cincinnati, Cincinnati, Ohio, 45219, United States|Thomas Jefferson University Hospital, Philadelphia, Pennsylvania, 19107, United States|St. Christopher's Hospital for Children, Philadelphia, Pennsylvania, 19134-1095, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|Baylor College of Medicine, Houston, Texas, 77030, United States|Medical College of Virginia, Richmond, Virginia, 23298-0157, United States|Virginia Commonwealth University, Richmond, Virginia, 23298, United States |
| Concurrent Single Gene and 24 Chromosome Aneuploidy Preimplantation Genetic Diagnosis (PGD) | Gene Security Network has developed a novel technology called Parental SupportTM (PS) which is used for Preimplantation Genetic Screening/Diagnosis (PGS/D) during in vitro fertilization (IVF). This technology allows IVF physicians to identify embryos, prior to transfer to the uterus, which have the best chance of developing into healthy children. The purpose of this study is to validate clinical use of PS to detect specific genetic mutation(s) known to cause severe inheritable diseases in embryos produced by at-risk couples. This may be done while simultaneously testing these embryos for aneuploidy. This study will allow for first of its kind commercial PGS/D testing to detect disease-associated genetic mutations together with aneuploidy screening. | Any Single Gene Disorder (Cystic Fibrosis, Tay-Sachs) | ALL | CHILD, ADULT | Gene Security Network, Redwood City, California, 94063, United States |
| Effect of Exercise With and Without HMB on Body Composition and Muscle Strength in Sickle Cell Anaemia | Wasting is a common and significant problem in sickle cell anaemia (SCA) that correlates with poorer clinical outcome such as frequent painful crises, acute chest syndrome and sub normal resistance to infection. Thus, improvement of nutritional status in SCA holds the potential of ameliorating the course of the disease. Elevated haemolysis and its effects are associated with hypermetabolism and have resulted in higher rates of protein breakdown and synthesis, and energy expenditure. Offering more food has not optimized nutritional status and metabolic performance in free-living patients with SCA. Moreover, appetite might be suppressed. Supplementation with β-hydroxy-β-methylbutyrate (HMB), which is produced in the body from leucine, has been shown to have inhibitory effect on protein breakdown and to promote lean tissue synthesis in humans with sarcopenia. Also, HMB has been implicated as an ergogenic tool to promote exercise performance and skeletal muscle hypertrophy. Therefore, the investigators hypothesize that in individuals with SCA, an intervention of resistance exercise with HMB supplement will have a greater enhancing effect on muscle mass and strength compared to receiving resistance exercise without HMB. | Sickle Cell Anemia | ALL | ADULT | |
| Achieving Understanding of the Natural History of Sickle Cell Trait (AUNT) | The main purpose of this study is to create a longitudinal cohort of those with Sickle Cell Trait (SCT) to better understand the hematologic phenotype for those that carry HbS, assess for differences in those with varying quantities of HbS and assess for potential clinical complications of SCT. | Sickle Cell Trait | ALL | ADULT, OLDER_ADULT | University of Alabama, Birmingham, Alabama, 35294, United States|Loma Linda University Health Care, Loma Linda, California, 92354, United States|Nemours Children's Hospital, Wilmington, Delaware, 19803, United States|Indiana University, Indianapolis, Indiana, 46202, United States|Johns Hopkins University, Baltimore, Maryland, 21224, United States|University of North Carolina, Chapel Hill, North Carolina, 27599, United States|Duke University, Durham, North Carolina, 27710, United States|East Carolina University, Greenville, North Carolina, 27834, United States|Nationwide Children's Hospital, Columbus, Ohio, 43205, United States |
| Indices of Severity and Prognosis for Sickle Cell Disease | To develop a clinical severity index that could prospectively identify sickle cell disease patients who were at high risk for a turbulent clinical course and a poor prognosis. | Anemia, Sickle Cell|Blood Disease | MALE | CHILD, ADULT, OLDER_ADULT | |
| Haploidentical PBMC Transplant for Severe Congenital Anemias | Background: Bone marrow transplantation (BMT), which involves transplanting a donor's marrow stem cells, is capable of curing some congenital anemias. BMT usually involves high-intensity treatment with chemotherapy and radiation to kill abnormal cells, which affects all systems of the body. People with anemias often have damage to other organs such as the kidneys, which can be further damaged by the chemotherapy. Only approximately 20 percent of patients have a full-matched donor, making treatment for many people with anemias unavailable. However, 90 percent of patients may have a half-matched donor, but using a half-matched donor increases the toxicity of BMT. Objectives: To determine if a research BMT with half-matched donor cells, low-intensity radiation, immunosuppressant drugs, and no chemotherapy will be effective in patients with sickle cell disease and Beta-thalassemia. To determine the effectiveness of cyclophosphamide, an immunosuppressant drug, in preventing rejection of the donor cells. Eligibility: Recipients are individuals at least 18 years of age who have been diagnosed with sickle cell disease and Beta-thalassemia, and who have a family member who is a haploidentical (i.e., half match) tissue match. Donors are healthy individuals between the ages of 2 and 80 who are found to be suitable donors. Design: Donors will undergo apheresis, which involves withdrawing blood from one arm vein, passing it through a machine that removes bone marrow stem cells, and returning the remaining blood through the vein in the other arm. Donors will receive a drug that causes the stem cells to be released into the bloodstream prior to the apheresis procedure. Recipients will undergo routine physical and laboratory examinations, including bone marrow sampling at the beginning of the study. After transplantation, physical and laboratory examinations will occur on a weekly or twice weekly basis at the outpatient clinic. Recipients will be examined every 6 months starting 100 days posttransplant for 5 years. Recipients will receive low-dose radiation in two treatments 1 and 2 days before the transplant. They will also be given immunosuppressant therapy with alemtuzumab and sirolimus. Another immunosuppressant drug, cyclophosphamide, will be given in the future as needed to subsets of the recipients to prevent rejection of donor cells. Recipients will receive the donor stem cells through a previously inserted central line. The process takes up to 8 hours. Recipients will receive blood transfusions as necessary to prevent anemia and bleeding during the posttransplant period. They may also receive intravenous antibiotics to prevent infection. | Sickle Cell Anemia | ALL | CHILD, ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| Music Therapy in Sickle Cell Disease Vaso-occlusive Crisis | Treatment of painful vaso-occlusive crises, the most common manifestation of sickle cell disease, is notoriously limited. vaso-occlusive crises pain is multifactorial with a psychological component. The hypothesis is that the music therapy program MUSIC CARE® can help alleviate severe vaso-occlusive crises pain in synergy with traditional treatment in sickle cell disease patients. The main objective of this prospective, randomized, open label study is to test the effect of the music therapy program MUSIC CARE® on daily mean morphine consumption during the 3 first days of hospitalisation for severe vaso-occlusive crises. | Vaso-occlusive Crisis | ALL | ADULT, OLDER_ADULT | Avicenne University Hospital, Bobigny, 93000, France |
| HRV-B for Symptom Management in Sickle Cell Patients | This study will test the hypothesis that Heart Rate Variability Biofeedback (HRV-B) restores autonomic balance and reduces pain and other symptoms among patients with sickle cell disease (SCD).The specific aims of this study are to: (1) conduct a randomized, wait list controlled, pilot intervention trial to determine whether HRV-B increases HRV coherence among SCD participants (minimum N of 30, up to 50 total); (2) determine whether HRV-B reduces pain, stress, fatigue, depression or insomnia among SCD participants; and (3) determine whether increases in HRV coherence are associated improvements in pain, stress, fatigue, depression, or sleep among study participants. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Center for Integrative Oncology and Survivorship, Greenville, South Carolina, 29605, United States |
| Study of SANGUINATE™ Versus Hydroxyurea in Sickle Cell Disease (SCD) Patients | The purpose of this study is to compare the safety of SANGUINATE™ versus Hydroxyurea in patients suffering from Sickle Cell Disease. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Fundacion BIOS, Barranquilla, Colombia|Fundacion Reina Isabel, Cali, Colombia|Hospital Pablo TobinUribe, Medellin, Colombia|PAMRI, Panama City, Panama |
| A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease | This is an open label, multicenter, Phase 1/2 study in approximately eight adults with severe Sickle Cell Disease (SCD). The study will evaluate the safety, tolerability, and efficacy of autologous hematopoietic stem cell transplantation using BIVV003. | Sickle Cell Disease | ALL | ADULT | UCSF Benioff Children's Hospital, Oakland, California, 94609, United States|University of California Davis Comprehensive Cancer Center, Sacramento, California, 95817, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States|Investigational Site Number 101, Bethesda, Maryland, 20892, United States|Karmanos Cancer Institute, Detroit, Michigan, 48201, United States |
| DREPAMASSE Study - Evaluation of a Newborn Screening for Sickle Cell Disease by Tandem Mass Spectrometry | Three methods are actually used in newborn screening for sickle cell disease (SCD) in France: isoelectric focusing, high performance liquid chromatography and capillary electrophoresis. New technologies are currently under development such as Matrix Assisted Laser Desorption Ionisation - Time of Flight (MALDI-TOF) and tandem mass spectrometry (MS/MS) using the SpOtOn Diagnostics Reagent Kit available in United Kingdom only. Zentech company (Liège, Belgium) is developing a package for SCD newborn screening using MS/MS technology. The main objective of the present study will be to compare this new technique with the technique actually used in the hospital center of Lille (sub-contractor for SCD newborn screening of Lyon) and the haemoglobin analysis to test its accuracy (sensitivity and specificity). | Sickle Cell Disease|Infant, Newborn, Disease|Drepanocytosis | ALL | CHILD | Groupement Hospitalier Est - Hospices Civils de Lyon, Bron, 69677, France|Hopital de la Croix Rousse, Lyon, 69004,, France|Centre Hospitalier Lyon Sud, Pierre-Bénite, 69495, France |
| Desmopressin for Bedwetting in Children With SCD | This study assesses if using the medication desmopressin will decrease nightime bedwetting in children with sickle cell disease. | Nocturnal Enuresis|Anemia, Sickle Cell | ALL | CHILD, ADULT | Children's Hospital at Montefiore, Bronx, New York, 10467, United States |
| Escalation of Plerixafor for Mobilization of CD34+ Hematopoietic Progenitor Cells and Evaluation of Globin Gene Transfer in Patients With Sickle Cell Disease | The purpose of this research study is to test the safety and efficacy of a drug called Plerixafor. Plerixafor is approved by the US FDA for use in increasing blood stem cell counts before collection in cancer patients. It is not yet approved for patients with sickle cell disease. The investigators want to find out if Plerixafor can be used to increase cell counts in patients with sickle cell disease. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Memorial Sloan Kettering Cancer Center, New York, New York, 10065, United States|Weill Cornell Medical College, New York, New York, 10065, United States |
| Assessment of Tolerance of Mobilizing Peripheral Hematopoietic Stem Cells by Plerixafor in Sickle Cell Patients | The purpose of this study is to assess the tolerance and efficacy of mobilizing hematopoietic stem cells after a single injection of plerixafor (0.24mg/kg) in 3 adult patients (or 5, if results of the first 3 patients are not reproducible) affected by sickle cell disease. | Major Sickle Cell Syndrome of Type SS or Sβ Thalassemia | ALL | ADULT, OLDER_ADULT | Hôpital Necker - Enfants Malades, Paris, 75015, France |
| Patient Centered Comprehensive Medication Adherence Management System in Patients With Sickle Cell Disease | The purpose of this research study is to learn about ways to help children and adults with sickle cell disease who are taking the medication, hydroxyurea. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Children's National Medical Center, Washington, District of Columbia, 20010, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States|University of Illinois at Chicago, Chicago, Illinois, 60607, United States|Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, 15224, United States |
| L-Glutamine Therapy for Sickle Cell Anemia | The primary purpose is to evaluate the effect of L-glutamine therapy on exercise endurance and breath by breath exercise response of sickle cell anemia patients The secondary purpose is to assess the effect of L-glutamine on incidence of painful crises; level of chronic pain, and amount of daily requirement for narcotics. | Sickle Cell Anemia|Thalassemia | ALL | ADULT, OLDER_ADULT | LA Biomed at Harbor-UCLA Medical Center, Torrance, California, 90502, United States |
| Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease | This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD). | Sickle Cell Disease | ALL | CHILD, ADULT | Children's National Health System, Washington, District of Columbia, 20010, United States|Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States|Columbia University, New York, New York, 10032, United States|Levine Children's Hospital, Charlotte, North Carolina, 28203, United States|Nationwide Children's Hospital, Columbus, Ohio, 43205, United States|Alberta Children's Hospital, Calgary, Alberta, T3B 6A8, Canada|The Hospital for Sick Children, Toronto, Ontario, M5G 1X8, Canada |
| Safety and Pharmacokinetics of SANGUINATE™ in Sickle Cell Disease (SCD) Patients | Prolong proposes to test safety, tolerability and pharmacokinetics of SANGUINATE™ in sickle cell disease (SCD) patients. Prolong's preclinical studies showed that SANGUINATE™ was safe in a number of different animal models and toxicology studies. In this Phase I trial, Prolong will test whether it is also safe and tolerable in sickle cell patients. The study will be conducted in 15 adult (\>18 years) patients. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Rambam Health Care Campus, Haifa, Israel |
| MBSR for Pain Catastrophizing in SCD | Significance: The purpose of this exploratory study is to test the feasibility, accessibility, and effects of a mindfulness-based stress reduction program (MBSR) on reducing pain catastrophizing in persons with sickle cell disease (SCD) and chronic pain. One of the most difficult symptoms for SCD patients to manage is chronic pain. Approximately one-third of SCD patients experience chronic pain, which is associated with pain catastrophizing. Pain catastrophizing is a negative mental state toward pain stimuli and pain experience, and is associated with increased pain severity, pain interference, and lower social functioning, physical functioning, and mental health. There have been no psychobehavioral intervention studies that have attempted to alter the experience of pain catastrophizing in persons with SCD. MBSR is a complementary group-based therapy that emphasizes nonjudgmental awareness of thoughts, feelings, and bodily sensations. With no pharmacological or non-pharmacological treatment for catastrophizing in persons with SCD, MBSR offers a potential solution to this highly significant problem for both SCD patients and providers. This project will be the first randomized controlled trial (RCT) of MBSR to reduce pain catastrophizing, and improve quality of life for SCD patients with chronic pain. Methods: This study will enroll 60 adult patients with SCD and chronic pain from the Duke Adult Sickle Cell Clinic. Patients will be randomized to a MBSR or wait-listed control group. The MBSR group will complete a 6- week, group-based telephonic MBSR program that is administered by a certified MBSR clinician once a week for 90 minutes. MBSR feasibility, acceptability, and effects on pain catastrophizing will be assessed by questionnaires at baseline, week 1, 3, and 6 in both groups. At the end of week 6, 10 randomly selected MBSR participants will complete semi-scripted telephone interviews to help assess intervention acceptability, and the wait-listed control condition will be offered the same MBSR intervention. | Anemia, Sickle Cell | ALL | ADULT, OLDER_ADULT | Duke University School of Nursing, Durham, North Carolina, 27701, United States |
| GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD) | An Open-label Extension Study of GBT021601 in Participants with Sickle Cell Disease | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Edward Jenner Research Group LLC., Miami, Florida, 33317, United States|Our Lady of the Lake Hospital, Inc., Baton Rouge, Louisiana, 70808, United States|University Medical Center Inpatient Pharmacy, New Orleans, Louisiana, 70112, United States|University Medical Center New Orleans, New Orleans, Louisiana, 70112, United States|Mississippi Center for Advanced Medicine, Madison, Mississippi, 39110, United States|University of Texas Health Science Center, Houston, Texas, 77030, United States|Inova Schar Cancer Institute, Fairfax, Virginia, 22031, United States|College of Medicine, University of Ibadan, Ibadan, OYO State, 200212, Nigeria|University College Hospital Ibadan, Ibadan, Oyo/ibadan North, 200212, Nigeria|Aminu kano Teaching Hospital, Kano, 700233, Nigeria|Lagos University Teaching Hospital, Lagos, 100254, Nigeria |
| Effect of MitoQ on Platelet Function and Reactive Oxygen Species Generation in Patients With Sickle Cell Anemia | MitoQ is commercially available as a dietary supplement and it has been tested as a potential drug in other diseases, but it has never been tested in patients with sickle cell disease. The goal of this research is to study if MitoQ, a molecule that works as an antioxidant by removing potentially damaging agents in a living organism, improves platelet function in patients with sickle cell disease (SCD). | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Magee Women's Hospital, Pittsburgh, Pennsylvania, 15213, United States|UPMC Montefiore, Pittsburgh, Pennsylvania, 15213, United States|UPMC Presbyterian, Pittsburgh, Pennsylvania, 15213, United States|Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, 15224, United States|Hillman Cancer Center, Pittsburgh, Pennsylvania, 15232, United States |
| Curative Versus Disease-Modifying Therapies in Children With Severe Sickle Cell Disease | The research proposed is a pilot study of pediatric and adolescent/young adult patients who have received the curative intervention (MSD-SCT), disease-modifying interventions (HU or CT) or SCC (control), with respect to three clinically important outcomes: quality-of-life (QOL), neurocognitive function, and reproductive potential. Comparable cohorts will be identified for each of the groups, drawing from patients treated by the SCD program of Children's Healthcare of Atlanta (CHOA). QOL measures and neuropsychiatric testing and will be administered. Reproductive endocrine function markers (laboratory studies and pubertal staging), will be collected and analyzed. A tracking system of such patients will also be developed, gathering available retrospective data and setting up a mechanism for collection of new data. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's Healthcare of Atlanta/Emory University, Atlanta, Georgia, 30322, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States |
| Sickle Cell Anemia - A Comparative Study Between Three Ethnical Communities, a Multicenter Study | The purpose of this study is to take advance of the presence of two different cohorts of SCA patients in one country, the first group included SCA patients from Bedouin Arab origin that lives in Israel for more than one century and originally comes from African countries or Saudi Arabia, those patients lives in north east Israel and are treated at the Hematology Unit of the Emek Medical Center, the second group are SCA patients from African origin that come to Israel in the last decades and belong to original African population, this group receive treatment at the Pediatric Hematology Unit, Dana Children's Hospital, Ichilov Medical Center. A third group is a cohort of SCA patients treated at Schneider Children's Hospital Hematology Unit. Those patients belong also to the Israel Arab population and patients from a village that African Muslims live for many years. The characteristics of the three groups will be compared to the characteristics of a fourth group, a cohort of Afro-American SCA patients that are followed up and treated at the Pediatric Hematology Unit, Detroit Children's Medical Center, Detroit, Michigan, USA. | Sickle Cell Anemia|Sickle Cell β+ or β0 Thalassemia | ALL | CHILD, ADULT | Children's Hospital of Michigan, Detroit Medical Center - Wayne State University, Detroit, Michigan, United States|Pediatric Hematology Unit HaEmek Medical Center, Afula, 18101, Israel|Pediatric Hematology Unit - Schneider Children's Hospital - Beilinson Medical Center, Petah Tikva, Israel|Pediatric Hematology Unit - Dana Children's Hospital - Ichilov Medical Center, Tel Aviv, Israel |
| MUSic Therapy to Improve Quality Of Life in Sickle Cell Disease (MUSIQOLS) | The purpose of this pilot study is to investigate the effects of a 6-session music therapy protocol on the pain, mood, quality of life, coping skills, and self-efficacy of adult patients with sickle cell disease (SCD) as compared to adult patients with SCD who receive standard care alone. The investigators will also determine the feasibility (delivery, acceptability, and usefulness) of the music therapy intervention for pain management and quality of life. | Sickle Cell Disease|Chronic Pain | ALL | ADULT, OLDER_ADULT | University Hospitals Seidman Cancer Center, Cleveland, Ohio, 44106, United States |
| A Study of the Efficacy and Safety of ICA-17043 (With or Without Hydroxyurea) in Patients With Sickle Cell Anemia. | ICA-17043 is being developed for the chronic treatment of patients with sickle cell disease (SCD) in both adults and children. ICA-17043 is a potent and specific inhibitor of a channel in human red blood cells (RBCs) that blocks RBC dehydration. ICA-17043 is expected to inhibit RBC dehydration and thus should prevent or delay the sickling process. By reducing sickled cells, an improvement in anemia, a reduction in painful crises, and ultimately, less end-organ disease is anticipated. | Sickle Cell Disease|Sickle Cell Anemia | ALL | ADULT | Study Site, Birmingham, Alabama, United States|Study Site, Oakland, California, United States|Study Site, San Francisco, California, United States|Study Site, Washington, District of Columbia, United States|Study Site, Augusta, Georgia, United States|Study Site, Chicago, Illinois, United States|Study Site, Baltimore, Maryland, United States|Study Site, Boston, Massachusetts, United States|Study Site, Detroit, Michigan, United States|Study Site, Jackson, Mississippi, United States|Study Site, Brooklyn, New York, United States|Study Site, New York, New York, United States|Study Site, Chapel Hill, North Carolina, United States|Study Site, Durham, North Carolina, United States|Study Site, Philadelphia, Pennsylvania, United States|Study Site, Pittsburgh, Pennsylvania, United States|Study Site, Nashville, Tennessee, United States|Study Site, Houston, Texas, United States|Study Site, Richmond, Virginia, United States |
| Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia | Background: Sickle cell disease (SCD) is an inherited disorder of the blood. It can damage a person s organs and cause serious illness and death. A blood stem cell transplant is the only potential cure for SCD. Treatments that improve survival rates are needed. Objective: To find out if a new antibody drug (briquilimab, JSP191) improves the success of a blood stem cell transplant Eligibility: People aged 13 or older who are eligible for a blood stem cell transplant to treat SCD. Healthy family members over age 13 who are matched to transplant recipients are also needed to donate blood. Design: Participants receiving transplants will undergo screening. They will have blood drawn. They will have tests of their breathing and heart function. They may have chest x-rays. A sample of marrow will be collected from a pelvic bone. Participants will remain in the hospital about 30 days for the transplant and recovery. They will have a large intravenous line inserted into the upper arm or chest. The line will remain in place for the entire transplant and recovery period. The line will be used to draw blood as needed. It will also be used to administer the transplant stem cells as well as various drugs and blood transfusions. Participants will also receive some drugs by mouth. Participants must remain within 1 hour of the NIH for 3 months after transplant. During that time, they will visit the clinic up to 2 times a week. Follow-up visits will include tests to evaluate participants mental functions. They will have MRI scans of their brain and heart. | Sickle Cell Anemia|Beta Thalassemia | ALL | CHILD, ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Optimizing Hydroxyurea Dosage With Pharmakokinetic in Patients Suffering of Moderate to Severe Sickle Cell Anemia | The goal of this study is to evaluate if patients with sickle cell disease can achieve a maximum tolerate dose of hydroxuyrea (HU) over a period of 12 months faster with pharmacokinetic testing than the standard of care bloodwork follow-up. Pharmacokinetic test is used to evaluate the process by which drugs are absorbed, distributed in the body, localized in the tissues, and is excreted. Patient will be a randomized (coin toss method) into 2 groups. Group A will have an increase of their HU dosage with pharmacokinetic results and Group B will have an increase of their HU dosage following the standard of care bloodwork follow-up. Group C will include patient with sickle cell disease that has been taking HU for at least 12 months and will undergo a pharmacokinetic dosage to check the level of HU only one time. | Sickle Cell Disease (SCD) | ALL | CHILD, ADULT | CHU Sainte-Justine, Montreal, Quebec, H3T 1C5, Canada |
| Multicenter Study of Hydroxyurea in Patients With Sickle Cell Anemia (MSH) | To assess the efficacy and safety of orally administered hydroxyurea in the treatment of painful crises in patients with sickle cell anemia. | Anemia, Sickle Cell|Hematologic Diseases|Hemoglobinopathies | ALL | ADULT | |
| A Phase Ib Study of NVX-508 in Sickle Cell Disease | This Phase 1b study in adults with sickle cell disease (SCD) in steady-state (non-acutely ill) aims to evaluate safety and toxicity of NVX-508 in a multi-dosing paradigm as well as to determine the maximum tolerated dose (MTD) in this population. The information gained from this study will be used in making decisions about the appropriate dose(s) and dosing schedule in future multicenter studies of the efficacy of NVX-508 in the treatment of vaso-occlusive episodes (VOE). | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | |
| Advancing Feasibility and Acceptability of Digital Cognitive Rehabilitation in Sickle Cell Disease | This is a single site nonrandomized pilot clinical trial of the feasibility, acceptability, and efficacy of a combination treatment involving digital Cogmed working memory skills training, social support components, and memory strategy training to improve adherence to Cogmed in youth with sickle cell disease ages 7-16 years. | Sickle Cell Disease | ALL | CHILD | Children's National Hospital, Washington, District of Columbia, 20010, United States |
| Non-myeloablative Haploidentical HCT Study for Patients With Sickle Cell Disease, Including Compromised Organ Function | Background: Sickle cell disease (SCD) is a genetic disorder where red blood cells, that carry oxygen, are stiff and become stuck in small blood vessels. As a result, affected patients can experience severe pain and serious organ damage. SCD can be cured with a hematopoietic cell transplant (HCT), that is, when they receive blood stem cells from a family donor. But HCT can also have serious side effects, especially in people with organ damage. Researchers want to find ways to make HCT safer for everyone. Objective: To test a new combination of drugs (briquilimab, abatacept, and alemtuzumab), used along with radiation, in people undergoing HCT for SCD. Eligibility: People aged 16 and older with SCD. They must be eligible for HCT and have a family member who is a good donor match. Donors must be aged 4 and older. Design: Participants with SCD will be screened. They will have blood tests and tests of organs including their heart and lung function. Donors will have blood drawn. Participants with SCD will have a tube inserted into a blood vessel in their chest (intravenously). This line will remain in place up to 2 months; it will be used to draw blood and administer the donor cells and other medications. Briquilimab will be administered intravenously 1 time, along with other drugs used to prepare for HCT. Participants will receive abatacept 6 times, from just before they receive their donor cells until 6 months after. Participants will undergo radiation therapy and take other drugs that are standard for HCT. Most HCT recipients remain in the hospital for about 30 days after HCT. Follow-up visits will continue for 5 years.... | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Tadalafil for Treatment of Priapism in Men With Sickle Cell Anemia | This research is being done to compare the effect of tadalafil with placebo (an inactive substance that looks like the study drug, but should have no effect) on the frequency of recurrent priapism (prolonged erection, unassociated with sexual interest or desire) and the nature of sexual experiences in male patients with sickle cell disease. | Sickle Cell Anemia|Priapism | MALE | ADULT | Johns Hopkins Outpatient Center; Johns Hopkins Medical Institutions, Baltimore, Maryland, 21287, United States |
| SCD-Haplo: Phase II Study of HLA-Haploidentical SCT for Aggressive SCD | Related donor stem cell transplantation using the alemtuzumab/ TBI platform has been shown to be a safe strategy to cure severe sickle cell disease. However, due to a lack of suitable donors, many patients cannot benefit from this strategy. Alternative donor sources are desperately needed to fill this gap. Nearly all patients will have a haploidentical family member who would be able to donate. The use of post transplantation cyclophosphamide has greatly improved the outcome of haploidentical stem cell transplantation. The investigators propose to combine this with alemtuzumab/TBI conditioning. | Sickle Cell Disease | ALL | CHILD, ADULT | University of Illinois Cancer Center, Chicago, Illinois, 60612, United States |
| Sildenafil for Treatment of Priapism in Men With Sickle Cell Anemia | This research is being done to evaluate if the phosphodiesterase type 5 (PDE5) inhibitor sildenafil has an effect on the frequency of recurrent priapism and the quality of life of males with sickle cell disease (SCD). | Sickle Cell Disease|Priapism | MALE | CHILD, ADULT | Johns Hopkins Hospital, Baltimore, Maryland, 21287, United States |
| Establishment of Functional MRI Imaging Parameters for Use in the Evaluation of Sickle Cell Disease | Patients with sickle cell anemia (SCA) are at an increased risk for damage to brain tissue due to their disease. The investigators are interested in how blood flow and cerebral inflammation are different in SCA patients and how that affects brain tissue- the investigators will use a relatively new set of dynamic MRI techniques to evaluate these parameters. The investigators will image participants with both SCA and matched controls with non-invasive MRI. | Sickle Cell Anemia | ALL | ADULT | Albert Einstein College of Medicine, Bronx, New York, 10461, United States|Montefiore Medical Center (Einstein), Bronx, New York, 10461, United States |
| Disseminating NIH Evidence Based Sickle Cell Recommendations in North Carolina | This project will improve the efficiency and quality of healthcare for persons with sickle cell disease, an under-served and at risk population by implementing a co-management model of care. Many patients with sickle cell disease (SCD) receive care primarily from specialty physicians and emergency departments (ED), thus resulting in a lack of primary care and a high number of ED visits and hospitalizations. The goal is to improve PCP and SCD specialist co-management. The overall purpose of this dissemination project is to evaluate utilization data, as well as patient and provider reported outcomes associated with the dissemination of a toolbox of decision support tools to PCP's and ED providers across NC and SC. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Community Care of North Carolina, Raleigh, North Carolina, 27607, United States |
| Inhaled Mometasone to Reduce Painful Episodes in Patients With Sickle Cell Disease | The proposed research is designed to test the global hypothesis that inhaled corticosteroids (ICS), a therapy developed to treat asthma, will prevent vasoocclusive painful episodes in adults with Sickle Cell Disease (SCD) who wheeze, but do not meet criteria for a diagnosis of asthma. The specific aims of this proposal are 1) Conduct a feasibility study - a randomized controlled trial of ICS for adults with SCD who do not meet criteria for a diagnosis of asthma but report recurrent cough or wheezing, 2) Measure the effects of ICS on biological correlates of pulmonary inflammation (as determined by exhaled nitric oxide) and vascular injury (as determined by sVCAM) in SCD, and 3) Compare properties of traditional and Bayesian adaptive clinical trial design for therapeutic trials in SCD in preparation for designing a definitive trial of ICS. These aims have the potential to 1) change the standard of care for individuals with SCD and recurrent cough or wheeze, 2) provide insight into the pathogenesis of non-asthmatic wheezing in SCD and its response to treatment, 3) explore the suitability of innovative clinical trial designs to overcome the challenges that have hindered therapeutic innovation for SCD. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Icahn School of Medicine at Mount Sinai, New York, New York, 10029, United States |
| Phase III Randomized Study of Poloxamer 188 for Vaso-Occlusive Crisis of Sickle Cell Disease | OBJECTIVES: I. Assess the efficacy of poloxamer 188 in reducing the duration of painful vaso-occlusive crisis in patients with sickle cell disease. II. Assess the effect of poloxamer 188 on duration and intensity of pain, total analgesic use, and length of hospitalization of these patients. | Sickle Cell Anemia | ALL | CHILD, ADULT, OLDER_ADULT | |
| PINPOINT: Gaming Technology for SCD Pain | Sickle cell disease (SCD) is a common genetic disorder characterized by episodes of pain, yet programs to assist SCD adolescents with better identification and communication about pain are lacking. Research shows that interactive gaming technology can enhance adolescents' learning, and can be especially effective in delivering health-related messages and tools to improve self-care. Pinpoint is an interactive gaming app that will be tested in a Phase II project to determine whether the app assists SCD teens with improving their communication and identification skills for pain self-report. | Sickle Cell Disease|Sickle Cell Anemia in Children|Sickle Cell Thalassemia|Sickle Cell SC Disease | ALL | CHILD | Klein Buendel, Inc., Golden, Colorado, 80401, United States|Hilton Publishing Company, Munster, Indiana, 463213963, United States |
| Evaluation of Non-invasive Endothelial Function in Children Sickle by Vascular Ultrasound | Sickle cell disease (SCD) is an inherited disorder characterized by recurrent painful crises with ischemia resulting from vascular occlusion. Adults with SCD have increased arterial stiffness and reduced flow-mediated dilation (FMD), due to impaired release of substances such as nitric oxide. The present study assess the vascular properties of carotid and brachial arteries in children with SCD compared with a control group without cardiovascular risk factors. | Sickle Cell Disease | ALL | CHILD, ADULT | University Hospital of Toulouse, Toulouse, 31000, France |
| Pulmonary Hypertension in Patients With Sickle Cell Disease in Nigeria | This study will explore how people with sickle cell disease (SCD) develop a complication called pulmonary hypertension (PHTN), a serious disease in which blood pressure in the lungs is higher than normal. PHTN is also caused by HIV, hepatitis C and schistosomiasis. Patients who have both SCD and one of these other infections may develop more severe PHTN. The number of Nigerians with SCD who also have PHTN is not known, nor is the cause of PHTN in this population. This study will examine genetic material in people with and without SCD to determine whether certain genes will allow doctors to predict which patients with SCD are likely to develop PHTN. Nigerian males and females 10 years of age and older with or without SCD may be eligible for this study. Patients must have SS, SC, or SB thalassemia or other genotype; control subjects must have hemoglobin A or AS genotype. Participants undergo a complete medical history and physical examination, blood tests, electrocardiogram (EKG), ultrasound tests of the heart and abdomen, and a 6-minute walk (distance test) to determine exercise capacity. Blood tests include screening for HIV, hepatitis B and C, schistosomiasis, hookworm and malaria. Patients who test positive for HIV, hepatitis B or C, schistosomiasis, hookworm or malaria are referred for treatment at Ahmadu Bello University Teaching Hospital in Zaria, Nigeria, and those who test negative for hepatitis B are referred for vaccination. Genetic tests focus on genes involved in SCD, PHTN, inflammation, blood vessel function and red blood cell function. | Secondary Pulmonary Arterial Hypertension|Sickle Cell Disease|Pulmonary Hypertension | ALL | CHILD, ADULT, OLDER_ADULT | Ahmadu Bello University Teaching Hospital, Kaduna, Nigeria |
| Non-Myeloablative Bone Marrow Transplant for Patients With Sickle Cell Anemia and Other Blood Disorders | RATIONALE: Giving low doses of chemotherapy, such as fludarabine and cyclophosphamide, and total-body irradiation before a donor bone marrow transplant helps stop the growth of abnormal cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving sirolimus and mycophenolate mofetil after transplant may stop this from happening. PURPOSE: This phase II trial is studying how well giving fludarabine and cyclophosphamide together with total-body irradiation followed by a donor bone marrow transplant works in treating patients with sickle cell anemia and other blood disorders. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, 21231-2410, United States |
| Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease | This is a clinical study evaluating the efficacy and safety of rivipansel (GMI-1070) in treating subjects with sickle cell disease (SCD) who are 6 years of age or older experiencing a pain crisis necessitating hospitalization. | Anemia, Sickle Cell | ALL | CHILD, ADULT, OLDER_ADULT | University of South Alabama Children's and Women's Hospital, Mobile, Alabama, 36604, United States|Arkansas Children's Hospital Research Pharmacy, Little Rock, Arkansas, 72202, United States|Arkansas Children's Hospital, Little Rock, Arkansas, 72202, United States|UCSF Benioff Children's Hospital Oakland, Oakland, California, 94609, United States|UC Davis Medical Center Main Hospital, Sacramento, California, 95817, United States|UC Davis Medical Center, Sacramento, California, 95817, United States|University of California Davis Medical Center, Sacramento, California, 95817, United States|University of Colorado CTRC, Aurora, Colorado, 80045, United States|University of Colorado Hospital, Aurora, Colorado, 80045, United States|Howard University Center for Sickle Cell Disease, Washington, District of Columbia, 20001, United States|Medstar Health Research Institute, Washington, District of Columbia, 20010, United States|Howard University Hospital, Washington, District of Columbia, 20060, United States|Golisano Children's Hospital of Southwest Florida, Fort Myers, Florida, 33908, United States|UF Health Davis Cancer Pavillion and Shands Med Plaza, Gainesville, Florida, 32608, United States|UF Health Shands Cancer Hospital, Gainesville, Florida, 32608, United States|UF Health Shands Hospital, Gainesville, Florida, 32610, United States|Jackson Memorial Hospital, Miami, Florida, 33136, United States|University Of Miami, Miami, Florida, 33136, United States|Investigational Drug Service Tampa General Hospital, Tampa, Florida, 33606, United States|Tampa General Hospital Center of Research Excellence, Tampa, Florida, 33606, United States|Tampa General Hospital, Tampa, Florida, 33606, United States|University of South Florida, Tampa, Florida, 33606, United States|St. Joseph's Children's Hospital, Tampa, Florida, 33607, United States|Children's Hematology and Oncology Associates, West Palm Beach, Florida, 33407, United States|St. Mary's Medical Center, West Palm Beach, Florida, 33407, United States|Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center, Atlanta, Georgia, 30303, United States|Grady Health System, Atlanta, Georgia, 30303, United States|Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center, Atlanta, Georgia, 30322, United States|Emory Children's Center, Atlanta, Georgia, 30322, United States|Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center, Atlanta, Georgia, 30342, United States|Children's Healthcare of Atlanta: Scottish Rite Campus, Atlanta, Georgia, 30342, United States|Augusta University Clinical Research Pharmacy, Augusta, Georgia, 30912, United States|Augusta University Medical Center, Augusta, Georgia, 30912, United States|Augusta University, Augusta, Georgia, 30912, United States|Memorial Family Medicine Ctr. @Memorial Health Univ Med Ct, Savannah, Georgia, 31404, United States|Memorial Health University Medical Center, Savannah, Georgia, 31404, United States|University of Illinois at Chicago Clinical Research Center, Chicago, Illinois, 60612, United States|University of Illinois Hospital and Health Sciences System, Chicago, Illinois, 60612, United States|The University of Chicago/Comer Children's Hospital, Chicago, Illinois, 60637, United States|University of Chicago, Investigational Drug Service Pharmacy, Chicago, Illinois, 60637, United States|University of Iowa Hospitals and Clinics, Iowa City, Iowa, 52242, United States|Kosair Charities Pediatric Clinical Research Unit, Louisville, Kentucky, 40202, United States|Norton Children´s Hospital, Louisville, Kentucky, 40202, United States|The Novak Center for Children's Health, Louisville, Kentucky, 40202, United States|University of Louisville Health Sciences Center, Louisville, Kentucky, 40202, United States|University of Louisville Physicians Pediatric Hematology Oncology, Louisville, Kentucky, 40202, United States|Our Lady of the Lake Regional Medical Center, Baton Rouge, Louisiana, 70808, United States|St. Jude Affiliate Clinic, Baton Rouge, Louisiana, 70808, United States|Our Lady of the Lake Physician Group-Medical Oncology, Baton Rouge, Louisiana, 70809, United States|University of Maryland Medical System Investigational Pharmacy, Baltimore, Maryland, 21201, United States|University of Maryland Medical System, Baltimore, Maryland, 21201, United States|Johns Hopkins Medicine, Baltimore, Maryland, 21205, United States|The Johns Hopkins University School of Medicine, Baltimore, Maryland, 21205, United States|The Johns Hopkins Hospital, Baltimore, Maryland, 21287-6180, United States|Johns Hopkins Medicine, Baltimore, Maryland, 21287, United States|Boston Children's Hospital, Boston, Massachusetts, 02115, United States|Brigham and Womens Hospital, Boston, Massachusetts, 02115, United States|Center for Clinical Investigation, Brigham & Women's Hospital, Boston, Massachusetts, 02115, United States|Investigational Drug Services, Boston, Massachusetts, 02115, United States|Boston Medical Center E7E, Boston, Massachusetts, 02118, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|Boston University Medical Center, Boston, Massachusetts, 02118, United States|Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|Detroit Medical Center Pharmacy, Detroit, Michigan, 48201, United States|Wayne State University / Detroit Receiving Hospital, Detroit, Michigan, 48201, United States|University of Mississippi Medical Center-Outpatient Clinical Research Unit, Jackson, Mississippi, 39216, United States|University Of Mississippi Medical Center, Jackson, Mississippi, 39216, United States|Children's Mercy Hospital, Kansas City, Missouri, 64108, United States|Center for Outpatient Health, Saint Louis, Missouri, 63108, United States|Barnes-Jewish Hospital Department of Pharmacy, Saint Louis, Missouri, 63110, United States|Barnes-Jewish Hospital, Saint Louis, Missouri, 63110, United States|Center for Advanced Medicine, Saint Louis, Missouri, 63110, United States|Washington University School of Medicine, Saint Louis, Missouri, 63110, United States|Bristol Myers Squibb Children's Hospital at Robert Wood Johnson University Hospital, New Brunswick, New Jersey, 08901, United States|Rutgers-Robert Wood Johnson Medical School, New Brunswick, New Jersey, 08901, United States|Rutgers Cancer Institute Of New Jersey, New Brunswick, New Jersey, 08903, United States|Jacobi Medical Center, Bronx, New York, 10461, United States|Kings County Hospital Center - Pharmacy Department, Brooklyn, New York, 11203, United States|Kings County Hospital Center, Brooklyn, New York, 11203, United States|State University of New York (SUNY) - Downstate Medical Center, Brooklyn, New York, 11203, United States|SUNY Downstate Medical Center University Hospital of Brooklyn, Brooklyn, New York, 11203, United States|Icahn School of Medicine at Mount Sinai, New York, New York, 10029, United States|Columbia University Medical Center Research Pharmacy, New York, New York, 10032, United States|MS CHONY Pediatric Emergency Department, New York, New York, 10032, United States|MS CHONY Pediatric Hematology/Oncology Unit, New York, New York, 10032, United States|Staten Island University Hospital, Staten Island, New York, 10305, United States|Duke University Hospital, Durham, North Carolina, 27710, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|East Carolina University Brody School of Medicine, Greenville, North Carolina, 27834, United States|Leo W. Jenkins Cancer Center, Greenville, North Carolina, 27834, United States|Vidant Medical Center, Greenville, North Carolina, 27834, United States|University of Cincinnati Medical Center/ Investigational Pharmacy, Cincinnati, Ohio, 45219, United States|University of Cincinnati Medical Center/Research Office, Cincinnati, Ohio, 45219, United States|University of Cincinnati Medical Center, Cincinnati, Ohio, 45219, United States|University of Cincinnati Physicians Company LLC, Cincinnati, Ohio, 45219, United States|University of Cincinnati- Hoxworth Building, Cincinnati, Ohio, 45219, United States|UC Health Ridgeway Hospital, Cincinnati, Ohio, 45229, United States|The Ohio State University Wexner Medical Center East, Columbus, Ohio, 43203, United States|Nationwide Childrens Hospital, Columbus, Ohio, 43205, United States|The Ohio State University Investigational Drug Services, Columbus, Ohio, 43210, United States|The Ohio State University James Comprehensive Cancer Hospital & Solove Research Institute, Columbus, Ohio, 43210, United States|The Ohio State University Wexner Medical Center, Columbus, Ohio, 43210, United States|Five Rivers Medical Surgical Health Center, Dayton, Ohio, 45402, United States|Miami Valley Hospital, Dayton, Ohio, 45409, United States|Einstein Medical Center Philadelphia, Philadelphia, Pennsylvania, 19141, United States|UPMC Presbyterian, Pittsburgh, Pennsylvania, 15213, United States|Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, 15224, United States|UPMC Hillman Cancer Center, Pittsburgh, Pennsylvania, 15232, United States|Hasbro Children's Hospital, Providence, Rhode Island, 02903, United States|Rhode Island Hospital-Pharmacy Service, Providence, Rhode Island, 02903, United States|Rhode Island Hospital, Providence, Rhode Island, 02903, United States|The Miriam Hospital, Providence, Rhode Island, 02906, United States|Medical University of South Carolina - Hospital, Charleston, South Carolina, 29425, United States|Medical University of South Carolina Lifespan Comprehensive Sickle Cell Center, Charleston, South Carolina, 29425, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|MUSC Investigational Drug Services, Charleston, South Carolina, 29425, United States|Cook Children's Hematology and Oncology Center, Fort Worth, Texas, 76104, United States|Cook Children's Medical Center, Fort Worth, Texas, 76104, United States|Cook Children's Hematology and Oncology Center-Grapevine, Grapevine, Texas, 76051, United States|Texas Children's Hospital - Clinical Research Center, Houston, Texas, 77030, United States|Texas Children´s Hospital, Houston, Texas, 77030, United States|University of Texas Medical School, Houston, Texas, 77030, United States|Primary Children's Hospital, Salt Lake City, Utah, 84113, United States|Main Hospital - Virginia Commonwealth University, Richmond, Virginia, 23298, United States|Virginia Commonwealth University-Investigational Drug Services, Richmond, Virginia, 23298, United States|Virginia Commonwealth University, Richmond, Virginia, 23298, United States|Royal Alexandra Hospital, Edmonton, Alberta, T5H 3V9, Canada|Miseracordia Community Hospital, Edmonton, Alberta, T5R 4H5, Canada|Kaye Edmonton Clinic 3 C, Edmonton, Alberta, T6G 1Z1, Canada|University of Alberta Hospital, Pharmacy Services, Edmonton, Alberta, T6G 1Z1, Canada|Stollery Children's Hospital, Edmonton, Alberta, T6G 2B7, Canada|University of Alberta Hospital, Edmonton, Alberta, T6G 2B7, Canada|Research transition Facility, Edmonton, Alberta, T6G 2V2, Canada|Grey Nuns Community Hospital, Edmonton, Alberta, T6L 5X8, Canada|St. Paul's Hospital, Vancouver, British Columbia, V6E 1M7, Canada|St. Paul's Hospital, Vancouver, British Columbia, V6Z 1Y6, Canada|Children's Hospital Of Eastern Ontario, Ottawa, Ontario, K1H 8L1, Canada|University Health Network, Toronto General Hospital, Toronto, Ontario, M5G 2C4, Canada|The Hospital for Sick Children, Toronto, Ontario, M5G1X8, Canada|Centre Hospitalier Universitaire Sainte-Justine, Montreal, Quebec, H3T 1C5, Canada|McGill University Health Centre, Royal Victoria Hospital, Montreal, Quebec, H4A 3J1, Canada|The Montreal Children's Hospital/ McGill University Health Centre, Montreal, Quebec, H4A 3J1, Canada |
| A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT) | Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD. In this study, depending on how participants' body responds to the fludarabine and dexamethasone, the study doctor may decide participants should receive another drug, called cyclophosphamide, instead of fludarabine. In addition, depending on the results of participants' routine blood tests, participants may receive the drugs bortezomib and rituximab, which also help with immune suppression. | Sickle Cell Disease|Thalassemia, Beta|Thalassemia | ALL | CHILD, ADULT | Memorial Sloan Kettering at Basking Ridge (Consent only), Basking Ridge, New Jersey, 07920, United States|Memorial Sloan Kettering Monmouth (Consent only), Middletown, New Jersey, 07748, United States|Memorial Sloan Kettering Bergen (Consent only), Montvale, New Jersey, 07645, United States|Memorial Sloan Kettering Suffolk - Commack (Consent only), Commack, New York, 11725, United States|Memorial Sloan Kettering Westchester (Consent only), Harrison, New York, 10604, United States|Memorial Sloan Kettering Nassau (All protocol activities), Rockville Centre, New York, 11553, United States |
| Early-goal Directed Automated Red Blood Cell Exchange for Acute Chest Syndrome in Sickle Cell Disease | Sickle cell disease (SCD) is characterized by recurrent vaso-occlusive pain crisis (VOC), which may evolve to acute chest syndrome (ACS), the most common cause of death among adult patients with SCD. Currently, there is no etiologic treatment to abort ACS. Therefore, management of ACS mostly involve a symptomatic approach including in routine, and as per recommendations, hydration, analgesics, supplemental oxygen, and transfusion. The polymerisation of sickle haemoglobin (HbS) is one major feature in the pathogenesis of vaso-occlusion. Current guidelines recommend red blood cell exchange transfusion (REX) in patients with severe ACS in order to improve oxygenation and reduce HbS concentration to blunt sickling. REX is often preferred over simple transfusion in this setting because it rapidly reduces HbS without raising final haematocrit. There are currently two methods for REX: manual (with sequential phlebotomies and transfusions) or automated (erythrocytapheresis). The former allows a sober use of red blood cell packs, while the latter achieves haematological targets (HbS and haematocrit) quickly and more consistently, but requires a special equipment and trained staff. As a result of inflammation and intravascular hemolysis, the plasma of patients with ACS may also contain several components that promote vaso-occlusion, lung injury and organ failure, including cytokines (e.g., IL-6), free haemoglobin and free haem. Conversely, it is depleted in haptoglobin and hemopexin, which normally bind to and clear cell-free haemoglobin. The addition of therapeutic plasma exchange to erythrocytapheresis during automated REX may therefore have a dual beneficial effect in patients with overt intravascular hemolysis: i) deplete the inflammatory mediators and products of hemolysis; ii) replete haptoglobin and hemopexin. REX modalities (automated vs manual) have not been tested during ACS. The hypothesis is that early-goal directed automated REX may accelerate the resolution of severe ACS as compared to manual REX. | Acute Chest Syndrome | ALL | ADULT, OLDER_ADULT | Armand MEKONTSO DESSAP, CRETEIL Cedex, Val De Marne, 94010, France |
| Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX) | This prospective mixed-method interview study aims to qualitatively describe the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among patients and parents of children with a rare disease targeted for treatment using gene therapy techniques. Using learned insights, the team will develop an online platform providing educational content and patient decision aids for patients and their families. | Sickle Cell Disease | ALL | ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Substudy of CADRE: for People With Extreme Phenotype: BIOCADRE | BIOCADRE is a CADRE substudy and aims to characterize more precisely the sickle cell patients with extreme phenotype. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Centre de Recherches er de Lutte contre la Drépanocytose, Bamako, Mali|Centre National de Transfusion Sanguine, Dakar, Senegal |
| Study of a Deformability Parameter of Red Blood Cell | Sickle-cell disease is one of the most common severe monogenic disorders in the world, it results in the synthesis of abnormal hemoglobin (HbS) instead of hemoglobin A. When deoxygenated, the sickle haemoglobin (HbS) polymerizes inducing the sickling of red blood cells (RBCs) and leading to decreased deformability and increased fragility. Therefore, sickle RBCs exhibit a reduced lifespan associated with intravascular hemolysis, hemolytic anemia and low tissue oxygenation. Sickle RBCs, which exhibit abnormal adhesive properties to endothelial cells, can block the microcirculation, causing the occurrence of painful vaso-occlusive crisis (VOC), acute chest syndrome (ACS), acute and chronic organ damage (heart, lung, liver, spleen, kidney, bone...) and shortened life span. A preliminary study performed on RBC from sickle cell patients (Hb SS) has shown an alteration of a parameter measuring the overall deformability of RBCs by evaluating the nature of their movement in a shear flow. This parameter is significantly lower in sickle cell patients in steady state compared to a population of healthy individuals. The parameter is also significantly lower in sickle cell patients during VOC when compared to patient in steady state. The main objective of this study is to evaluate the performance of the method for measuring the deformability of RBCs on an experimental prototype. Measurements will be performed on blood samples from subjects with a normal hemoglobin electrophoretic profile, from heterozygous carriers of sickle cell disease and from patients with sickle cell disease. Samples from paediatric patients will also be tested to study any specificity in comparison to adult subjects. | Drepanocytosis | ALL | CHILD, ADULT, OLDER_ADULT | |
| Research Study Investigating How Well NDec Works in People With Sickle Cell Disease | This study examines how well a new, potential medicine called NDec works and is tolerated in people with sickle cell disease. NDec is a combination of two medicines (decitabine-tetrahydrouridine). Both medicines are new for the treatment of sickle cell disease. Participants who are not taking Hydroxyurea (HU) will get NDec, NDec and placebo, or placebo. Participants who are on HU treatment before joining the study will get NDec, NDec and placebo, or continue on HU. Which treatment participants get is decided by chance. Participants getting NDec and/or Placebo will get capsules to take twice weekly. The study will last for about a year. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University Of South Alabama, Mobile, Alabama, 36617, United States|UCSF Oakland Benioff Children's Hospital, Oakland, California, 94609, United States|Center for Inherited Blood Dis, Orange, California, 92868, United States|Harbor-UCLA Medical Center, Torrance, California, 90502-2004, United States|Clinical and Transl Res Center, Aurora, Colorado, 80045, United States|Howard University, Washington, District of Columbia, 20060, United States|Foundation for Sickle Cell Disease Research, Hollywood, Florida, 33023, United States|University of Miami Hospital & Clinics, Miami, Florida, 33136, United States|University Of Illinois at Chicago, Chicago, Illinois, 60612, United States|Tulane Sickle Cell Ctr- So LA, Metairie, Louisiana, 70002, United States|Mississippi Center Advanced Medicine, Madison, Mississippi, 39110, United States|Cure 4 the Kids Foundation, Las Vegas, Nevada, 89135, United States|Jacobi Medical Center, Bronx, New York, 10461, United States|Mount Sinai School of Medicine, New York, New York, 10029, United States|East Carolina University_Greenville_0, Greenville, North Carolina, 27834, United States|Univ Oklahoma HSC_Okla City, Oklahoma City, Oklahoma, 73104, United States|University of Oklahoma Health Sciences Center_Oklahoma City, Oklahoma City, Oklahoma, 73104, United States|St Christopher Hosp for Child, Philadelphia, Pennsylvania, 19134, United States|Medical Univ of SC Charleston, Charleston, South Carolina, 29425, United States|Univ Texas HSC-Houston, Houston, Texas, 77030, United States|LHSC - Victoria Hospital, London, Ontario, ON N6A 5W9, Canada|Toronto General Hospital, Liver Clinic, Toronto, Ontario, M5G 2C4, Canada|Toronto General Hospital, Liver Clinic, Toronto, Ontario, M5G2C4, Canada|McGill University Health Centre, Montreal, Quebec, H4A 3J1, Canada|Centre Hospitalier Universitaire Grenoble Alpes-Site Nord Michallon-3, Grenoble Cedex 9, 38043, France|Hospices Civils de Lyon-Hopital Edouard Herriot, Lyon Cedex 03, 69437, France|Aghia Sophia Childrens' Hospital, Goudi, 11527, Greece|General Hospital Of Larissa Koutlibaneio And Triantafylleio, Larissa, 41221, Greece|Gen Univ Hospital of Patras, Thalassemia/Hemoglobinopathies, Patra, GR-26504, Greece|All India Institute of Medical Sciences (AIIMS), Raipur, Raipur, Chhattisgarh, 492099, India|Nirmal Hospital Pvt. Ltd., Surat, Gujarat, 395002, India|BAPS Pramukh Swami Hospital, Surat, Gujarat, 395009, India|SSG Hospital, Baroda, Vadodara, Gujarat, 390001, India|SSG Hospital, Baroda, Vadodara, Gujarat, 390006, India|Victoria Hospital (Bangalore Medical College and Research Institute), Bangalore, Karnataka, 560002, India|Victoria Hospital (Bangalore Medical College and Research Institute), Bangalore, Karnataka, 560002, India|J.S.S.Hospital, Mysore, Karnataka, 570004, India|Government Medical College, Kozhikode, Kozhikode, Kerala, 673008, India|K.J Somaiya Hospital and Research Centre, Mumbai, Maharashtra, 400022, India|Government Medical College and Hospital, Nagpur, Maharashtra, 440003, India|Government Medical College and Super Speciality Hospital, Nagpur, Nagpur, Maharashtra, 440003, India|Government Medical College and Super Speciality Hospital, Nagpur, Nagpur, Maharashtra, 440003, India|IMS and SUM Hospital, Bhubaneswar, Orissa, 751003, India|S.C.B. Medical College, Cuttack, Orissa, 753007, India|Christian Medical College Hospital, Vellore, Ranipet, Tamil Nadu, 632517, India|Christian Medical College Hospital, Vellore, Vellore, Tamil Nadu, 632004, India|Yashoda hospital, Hyderabad, Telengana, 500082, India|Sanjay Gandhi Postgraduate Institute of Medical Sciences, Lucknow, Uttar Pradesh, 226014, India|NRS Medical College & Hospital, Kolkata, West Bengal, 700014, India|NRS Medical College & Hospital, Kolkatta, West Bengal, 70014, India|KIMS - Kingsway Hospital, Nagpur, 440001, India|KIMS - Kingsway Hospital, Nagpur, 440001, India|Ospedali Galliera, Genova, 16128, Italy|Ospedali Galliera, Genova, 16128, Italy|Fondazione IRCSS Ca' Granda Ospedale Maggiore Policlinico, Milan, 20122, Italy|Azienda Ospedale Universita Padova, Padova, 35128, Italy|Policlinico GB Rossi, Verona, 37134, Italy|Hospital Nini, Tripoli, 1434, Lebanon|Sultan Qaboos University Hospital, Muscat, 123, Oman|Charlotte Maxeke Johannesburg Academic Hospital, Parktown, Johannesburg, Gauteng, 2193, South Africa|Hospital Universitario La Paz, Madrid, 28046, Spain|Hospital Universitario Regional de Málaga, Málaga, 29010, Spain|Acibadem Adana Hastanesi, Adana, 01130, Turkey|Cukurova Universitesi, Adana, 01130, Turkey|Hacettepe University Hematology, Ankara, 06230, Turkey|Mersin University Medical Faculty Hospital, Hematology, Mersin, 33110, Turkey|University Hospital of Wales, Cardiff, CF14 4XW, United Kingdom|Central Middlesex Hospital, London, NW10 7NS, United Kingdom|Guy's Hosptial, London, SE1 9RT, United Kingdom|Kings College Hospital, London, SE5 9RS, United Kingdom|Manchester Royal Infirmary_Manchester_0, Manchester, M13 9WL, United Kingdom |
| Efficacy, Safety Study and Benefit of Alkali Therapy in Sickle Cell Disease | The objective of this study is to assess the effect of alkali administration on bicarbonate and potassium levels in patients with Sickle Cell Disease (SCD) and depressed serum bicarbonate levels. The study is a prospective non-blinded evaluation of tolerability and efficacy of alkali repletion with 4 weeks of observation and two sequential 4 week courses of escalating oral sodium bicarbonate treatment. | Sickle Cell Anemia|Chronic Kidney Disease|Metabolic Acidosis | ALL | ADULT, OLDER_ADULT | University hospitals Case Medical Center, Cleveland, Ohio, 44106, United States |
| Autologous Bone Marrow Stem Cells for Chronic Leg Ulcer Treatment in Sickle Cell Disease | The purpose of this study is to evaluate the safety and efficacy of autologous bone marrow stem cell implantation for the treatment of leg ulcer in adult patients with sickle cell disease. | Chronic Leg Ulcer|Sickle Cell Disease | ALL | ADULT | |
| Fixed Dose Flavonoid Isoquercetin on Thrombo-Inflammatory Biomarkers in Subjects With Stable Sickle Cell Disease | Background: Sickle cell disease (SCD) is an inherited hemoglobin disorder. People with SCD have an increased chance for getting blood clots. Researchers want to see if a dietary supplement called isoquercetin can decrease levels of inflammation and blood clotting in people with SCD. Objective: To see how isoquercetin works in people with SCD. Eligibility: Adults age 18-70 years old who have SCD and are in a steady-state (have not experienced a pain crisis in the last 60 days and, if taking hydroxyurea, have not had a dose change in the past 90 days). Design: Participants will be screened with a physical exam, medical history, medicine review, and blood tests. Participants will be put in 1 of 2 treatment groups. They will take 4 capsules of isoquercetin or placebo all at once, by mouth, every day for 4 weeks. They will get a pill dispenser and keep a medicine diary. Participants may have an optional near infrared spectroscopy (NIRS) test to measure how treatment affects blood flow. In this test, probes will be placed on the skin to measure tissue oxygen level and blood flow. A blood pressure cuff placed on the arm will be filled with air briefly to restrict the blood flow in the arm (for up to 5 minutes) and then released. Participants may also be asked to breathe at a certain rate or hold their breath for as long as they can during measurements. Participants will take folic acid once a day. Participants will have an end-of-study drug visit. They will discuss any side effects and repeat some of the screening tests. They may have an additional optional NIRS test. About a month after the end of study drug visit, participants will be contacted by phone to see if they have any side effects. Those who do may have a follow-up visit. At this visit, they may have additional blood tests performed. Participation will last from 8 to 12 weeks. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Zinc Supplementation in Children With Sickle Cell Disease in Western Kenya | Zinc is a nutritionally essential trace element found in previous studies to reduce growth retardation and improve immune function, which may also result in decreased incidence of infectious diseases including malaria, pneumonia and diarrhea. Sickle Cell Disease (SCD) patients are known to be susceptible to zinc deficiency and appear to benefit from zinc supplementation. The proposed pilot research project aims to investigate the influence of zinc supplementation on incidence of malaria infections, incidence of bacterial infections and investigate the influence of zinc supplementation on morbidity in children with SCD in western Kenya. The differences in incidence of morbidity and other secondary endpoints will be compared between the zinc group and the control group. | Sickle Cell Disease|Zinc Deficiency|Infection | ALL | CHILD | |
| A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001) | A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine in which genetic material (mostly DNA) in the patient is changed to treat his or her own disease. In gene therapy, we introduce new genetic material in order to fix or replace the patient's disease gene, with the goal of curing the disease. The procedure is similar to a bone marrow transplant, in that the patient's malfunctioning blood stem cells are reduced or eliminated using chemotherapy, but it is different because instead of using a different person's (donor) blood stem cells for the transplant, the patient's own blood stem cells are given back after the new genetic material has been introduced into those cells. This approach has the advantage of eliminating any risk of graft versus host disease (GVHD), reducing the risk of graft rejection, and may also allow less chemotherapy to be utilized for the conditioning portion of the transplant procedure. To introduce new genetic material into the patient's own blood stem cells we use a modified version of a virus (called a 'vector') that efficiently inserts the "correcting" genetic material into the cells. The vector is a specialized biological medicine that has been formulated for use in human beings. Fetal hemoglobin (HbF) is a healthy, non-sickling kind of hemoglobin. The investigators have discovered a gene that is very important in controlling the amount of HbF. Decreasing the expression of this gene in sickle cell patients could increase the amount of fetal hemoglobin while simultaneously reducing the amount of sickle hemoglobin in their blood, specifically the amount in red blood cells where sickle hemoglobin causes damage to the cell, and therefore potentially cure or significantly improve the condition. The gene we are targeting for change in this study that controls the level of fetal hemoglobin is called BCL11A. In summary, the advantages of a gene therapy approach include: 1) it can be used even if the patient does not have a matched donor available; 2) it may allow a reduction in the amount of chemotherapy required to prepare the patient for the transplant; and 3) it will avoid certain strong medicines often required to prevent and treat GVHD and rejection. Our lab studies with normal mice, mice that have a form of SCD, and with cells from the bone marrow of SCD patients who have donated bone marrow for research purposes show this approach is very effective in reducing the amount of sickle hemoglobin in red cells. Our pilot trial testing this approach in 10 patients with SCD has shown that the treatment has not caused any unexpected safety problems, and that it increases HbF within the red blood cells. Our goal is to continue to test whether this approach is safe, and whether using gene therapy to change the expression of BCL11A will lead to decreased episodes of vaso-occlusive crisis pain in people with SCD. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's Hospital of Los Angeles, Los Angeles, California, 90027, United States|UCLA Medical Center, Los Angeles, California, 90095, United States|UCSF Benioff Children's Hospital Oakland, Oakland, California, 94609, United States|UC Davis Medical Center, Sacramento, California, 95817, United States|Children's Healthcare of Atlanta/Emory University, Atlanta, Georgia, 30322, United States|Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States|Boston Children's Hospital, Boston, Massachusetts, 02115, United States|Dana-Farber Cancer Institute/Brigham and Women's Hospital, Boston, Massachusetts, 02115, United States|Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| Ketamine for Acute Painful Crisis in Sickle Cell Disease Patients | Investigators hypothesize that administration of ketamine for pain relief in sickle cell patients with vaso-occlusive crisis early on will lead to a more rapid improvement in pain score and less narcotic requirement. | Sickle Cell Crisis | ALL | ADULT | Imam Abdulrahman Bin Faisal University, Dammam, Eastern, 31952, Saudi Arabia |
| Study of Propranolol as Anti-Adhesive Therapy in Sickle Cell Disease (SCD) | An open label, prospective, randomized cross-over phase II study in up to 60 sickle cell patients who are either homozygous for Hb S or have HbSB0 thalassemia. Initially, each patient will be treated for 6 weeks with placebo or a standard dose of propranolol (40 mg) every 12 hrs. This will be followed by a 2-week washout period after which, patients will receive the other treatment modality (placebo or propranolol). We Hypothesize that propranolol administered in vivo on a daily basis for 6 weeks (1) will decrease baseline adhesion to endothelial cells and will substantially abrogate epinephrine-stimulated adhesion to endothelial cells, as measured in vitro; (2) will improve biomarkers of endothelial activation and dysfunction; and (3) can be safely used in patients with SCD. Thus, the use of propranolol in SCD may represent a safe and effective means of anti-adhesive therapy in SCD. Study Objectives: Primary Objective: • To establish the safety and efficacy of long-term therapy with propranolol as an anti-adhesive therapy for SCD. Secondary Objective: • To evaluate changes in soluble markers of endothelial activation and dysfunction. Correlative Science Objective: • To determine whether response to propranolol therapy is associated with polymorphisms in genes encoding the proteins involved in the upregulation of Sickle Red Blood Cell (SS RBC) adhesion by epinephrine. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Duke University Medical Center, Durham, North Carolina, 27710, United States |
| Music Therapy in Sickle Cell Pain Mixed Methods Study | The purpose of this research study is to: 1. Investigate the effects of a single 20-minute music therapy intervention with a music therapist on the pain intensity, pain relief, and mood of adult patients with sickle cell disease as compared to: 1. Adult patients with SCD who listen to their preferred music for 20 minutes without the presence of a music therapist (music listening group) 2. Adult patients with SCD who receive standard care alone (control group) 2. Determine the feasibility (delivery, acceptability, and usefulness) of the music therapy intervention for pain management | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University Hospitals Seidman Cancer Center, Cleveland, Ohio, 44106, United States |
| Hydroxyurea in Young Children With Sickle Cell Anemia | The purpose of this study is to asses prospectively the safety and efficacy of hydroxyurea therapy in children with Sickle cell Anemia between ages 18 months and 5 years, with special emphasis on the ability of hydroxyurea to prevent or reverse chronic organ damage. | Sickle Cell Anemia | ALL | CHILD | Duke University Medican Center, Durham, North Carolina, 27710, United States |
| Relationship Between Abnormal Myocardial Perfusion and Diastolic Dysfunction in Sickle Cell Disease Using PET | There is limited information on what causes injury to the heart in individuals with Sickle Cell Disease (SCD). Researchers in this study want to see if decreased blood flow to the heart during stress could be causing the heart damage seen in SCD patients. They also want to test people who don't have SCD to see if their hearts react the same way under stress. Primary Objective * To estimate the coronary flow reserve (CFR) (also referred to as myocardial perfusion reserve), as measured by PET stress-rest myocardial perfusion imaging, in SCD patients with and without diastolic dysfunction, and healthy controls. Secondary Objectives * To investigate the relationship between decreased CFR (quantified with PET stress- rest myocardial perfusion imaging) and presence of abnormal diastolic parameters | Sickle Cell Disease | ALL | ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease | The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512. This will be the first evaluation of the potential therapeutic effect of ITU512 in healthy participants and patients with sickle cell disease (SCD). | Sickle Cell Disease | ALL | CHILD, ADULT | Quotient Sciences Sea View, Miami, Florida, 33126, United States |
| Comparative Health Status and Quality of Life of Patients With Sickle Cell Disease (SCD) Who Underwent Matched-sibling Hematopoietic Stem Cell Transplantation Versus Non Transplanted SCD Case-control Patients | The long term burden of morbidity and mortality in the natural history of sickle cell disease has not been compared up to date to the risks and mortality of a curative option like bone marrow transplantation in severe sickle-cell disease patients. Given this lack of data, primary-care Sickle Cell Disease (SCD) physicians and transplant physicians are prevented from a factual debate over the benefit/risk ratio for each patient and refining indications of transplant in patients. Therefore, the present study seeks to describe and compare the very long-term outcomes after either Human Leukocyte Antigen (HLA) -matched sibling transplantation (study arm) and "non-transplant care" for severe sickle cell disease SCA patients in order to yield robust comparative data regarding both arms. The main objective is to assess the benefit of Hematopoietic stem cell transplantation (HSCT) regarding quality of life compared to standard care after 10 years, in patients with severe Sickle Cell Disease (SCD). | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | |
| Osteopathic Manipulation in the Management of Pain Associated With Sickel Cell Disease | Background: Sickle cell disease is the most common monogenic disease in the world caused by a mutation in the β-globin gene which creates abnormal hemoglobin called HbS. This polymer deforms the erythrocyte, making it more fragile and less flexible, thus leading to the occlusion of small blood vessels. This obstruction is the cause of painful vaso-occlusive crises and ischemia-reperfusion phenomena. Patients with sickle cell disease undergo major acute and chronic pain responsible for a significant deterioration in their quality of life and a significant consumption of analgesics, often daily, sometimes with the development of addictive behavior. Improved analgesic management was associated with improved disease prognosis. Several studies have shown the effectiveness of the osteopathic approach in the management of chronic pain. Our hypothesis is that the association with the standard treatment of osteopathy sessions could improve but also prevent the chronic pain frequent in patients with sickle cell disease. Objectives: Our main objective is to study the effectiveness of an osteopathic treatment in adult sickle cell patients with chronic pain on the reduction of the consumption of level I and II analgesics at 3 months (D90 +/- 15 days). Methods/Experimental design: This is a single-blind prospective randomized controlled monocentric study. The study population will be composed of 37 sickle cell patients aged over 18 years. The patients included will be allocated into two groups: one group will receive the osteopathic treatment and the 2nd group will receive the "placebo" treatment. Analgesic consumption will be assessed by weekly self- questionnaire. The evaluation of the pain will be carried out by the visual analogue scale (VAS). The degree of stress will be measured using the Perceived Stress Scale (PSS). Patients will receive an osteopathic treatment or a "placebo" treatment, one session every 4 weeks for 12 weeks with a total of 3 sessions per patient. The duration of each session is 45 minutes. Pain and stress assessments will be done before each session. A final evaluation will be carried out 3 months after the end of the osteopathic or "placebo" treatment. Data analysis will be performed using SPSS version 17.0 software. The significance threshold will be set at 0.05. This is the first protocol that aims to evaluate, with scientific rigor, the impact of the osteopathic approach in the management of pain in patients with sickle cell disease. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Collège Ostéopathique de Provence Aix-Marseille, Marseille, Paca, 13001, France |
| Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure | Background: - Some sickle cell disease or beta-thalassemia can be cured with transplant. Researchers want to test a variation of transplant that uses low dose radiation and a combination of immunosuppressive drugs. They want to know if it helps a body to better accept donor stem cells. Objectives: - To see if low dose radiation (300 rads), oral cyclophosphamide, pentostatin, and sirolimus help a body to better accept donor stem cells. Eligibility: - People 4 and older with beta-thalassemia or sickle cell disease that can be cured with transplant, and their donors. Design: * Participants and donors will be screened with medical history, physical exam, blood test, tissue and blood typing, and bone marrow sampling. They will visit a social worker. * Donors: * may receive an intravenous (IV) tube in their groin vein. * will receive a drug injection daily for 5 or 6 days to move the blood stem cells from the bone marrow into general blood circulation. * will undergo apheresis: an IV is put into a vein in each arm. Blood is taken from one arm, a machine removes the white blood cells that contain blood stem cells, and the rest is returned through the other arm. * Participants: * may undergo red cell exchange procedure. * will remain in the hospital for about 30 days. * will receive a large IV line that can stay in their body from transplant through recovery. * will receive a dose of radiation, and transplant related drugs by mouth or IV. * will receive blood stem cells over 8 hours by IV. * will take neuropsychological tests and may complete questionnaires throughout the transplant process. * must stay near NIH for 4 months. They will visit the outpatient clinic weekly. | Sickle Cell Disease|Thalassemia|Stem Cell Transplantation|Graft vs Host Disease | ALL | CHILD, ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Safety, Pharmacokinetic, and Pharmacodynamic Study of NKTT120 in Adult Patients With Stable Sickle Cell Disease (SCD) | The purpose of this study is to determine the safety, pharmacokinetics, and pharmacodynamics of NKTT120 in adult patients with stable sickle cell disease. | Sickle Cell Disease | ALL | ADULT | Children's Hospital & Research Center at Oakland, Oakland, California, 94609, United States|Johns Hopkins School of Medicine, Baltimore, Maryland, 21205, United States|Brigham and Women's Hospital, Boston, Massachusetts, 02115, United States|Washington University School of Medicine, St. Louis, Missouri, 63110, United States|UNC Sickle Cell Program, Chapel Hill, North Carolina, 27514, United States|Blood Center of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| Re-Aiming at Hydroxyurea Adherence for Sickle Cell With mHealth | National Institutes of Health (NIH)/National Heart, Lung, and Blood Institute (NHLBI) guidelines recommend that hydroxyurea be offered to symptomatic adults and all children with sickle cell disease (SCD) (HbSS and HbSβ0-thal genotypes) age ≥9 months. Research has shown that hydroxyurea reduces hospitalizations and mortality, supporting its effectiveness outside of clinical trials. Hydroxyurea is given as a once-daily oral dose that costs \<$1 per day. Despite overwhelming evidence for positive effects, hydroxyurea is vastly underutilized. Given the relative ease of its administration, low cost, and safety profile, barriers to hydroxyurea utilization are primarily constrained to the health system and patient determinants. System-level barriers include insufficient access to SCD-specific care, limited access to medication (due to lack of health coverage), and providers' reluctance in prescribing it; while patient-level barriers include low acceptance (due to insufficient knowledge or misconceptions regarding risks and benefits), and forgetfulness leading to poor adherence. Mobile health (mHealth) refers to the practice of medicine and public health supported by mobile devices. Short message service (SMS) text messaging (through cell phones) is a widespread means of communication, particularly among adolescents and young adults and is an emerging intervention modality to improve medication adherence. Its low cost, simplicity, and prevalence allow for relatively easy adoption and dissemination in medical practices. This protocol seeks to examine barriers to hydroxyurea adherence among SCD patients between 15 and 45 years of age who are living in the Memphis region by conducting a needs assessment. In addition to examining these barriers, the needs assessment will provide data that will inform the development of an mHealth application (e.g. mobile phone application) for assisting patients in increasing their medication adherence. The developed mHealth intervention will then undergo a pilot trial to test its acceptability, satisfaction, and feasibility among 56 patients living with SCD. * To conduct multi-level needs assessment of hydroxyurea utilization barriers and facilitators, in Memphis, Tennessee (Phase I). * To test the feasibility and acceptance of a patient-informed smart phone application aimed at improving hydroxyurea adherence in the Memphis, Tennessee region, and to estimate the efficacy parameters needed to design a definitive large phase III trial (Phase II). | Sickle Cell Disease | ALL | CHILD, ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|Baptist Memorial Hospital, Baptist Clinical Research Institute, Memphis, Tennessee, 38120, United States |
| Dose-Finding Study of SC411 in Children With Sickle Cell Disease | This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, dose-finding study of SC411 in children with sickle cell disease (SCD). The primary objective of the study is to evaluate the safety and tolerability of three different doses of SC411 compared to a placebo. All patients will undergo eight weeks of oral study treatment and a four-week safety follow-up period. Patients will be randomized to one of three dose levels of SC411 or placebo. | Sickle Cell Disease | ALL | CHILD | Children's of Alabama - University of Alabama, Birmingham, Alabama, 35233, United States|UCSF Benioff Children's Hospital Oakland, Oakland, California, 94609, United States|University of Florida Health at Shands, Gainesville, Florida, 32610, United States|Batchelor Children's Research Institute - University of Miami, Miami, Florida, 33136, United States|Children's Healthcare of Atlanta - Emory University, Atlanta, Georgia, 30322, United States|Boston Children's Hospital, Boston, Massachusetts, 02115, United States|Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States|East Carolina University, Greenville, North Carolina, 27834, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|Texas Children's Hospital - Baylor College of Medicine, Houston, Texas, 77030, United States |
| SMYLS Multi-site Trial | The purpose of this study is to find out whether a web-based intervention using a mobile app is helpful for teens and young adults with sickle cell disease (SCD) in learning how to care for and manage their symptoms. | Sickle Cell Disease | ALL | CHILD, ADULT | Medical University of South Carolina, Charleston, South Carolina, 29425, United States |
| Self-Management for Youth and Families Living With SCD - SMYLS | The purpose of this proposal is to integrate family-centered self-management strategies with mobile health (mHealth) technology to improve reach, self-management behaviors, and child and caregiver physical and psychosocial symptoms and quality of life. Specifically, the investigators propose to conduct feasibility testing of SMYLS, which has been adapted based on user feedback in the first phase of this study. First the investigators will work with the Medical University of South Carolina (MUSC) Pediatric Sickle Cell Clinic to identify and recruit families with children with sickle cell disease (SCD) in the community, statewide. Next, the investigators will test the feasibility of the intervention with 30 dyads of children ages 8 - 17 with sickle cell disease and their parent or primary caregiver, (N=60) | Sickle Cell Disease | ALL | CHILD | Medical University of South Carolina, Charleston, South Carolina, 29425, United States |
| Inhaled Nitric Oxide for Pediatric Painful Sickle Crisis | Randomized, double blind placebo controlled clinical trial to evaluate effectiveness and safety of inhaled nitric oxide for the treatment of sickle cell painful crisis in pediatric patients with sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's Hospital Boston, Boston, Massachusetts, 02115, United States |
| Clinical Study on the Safety and Efficacy of BRL-101 in the Treatment of Sickle Cell Disease | This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (BRL-101) | Sickle Cell Disease | ALL | CHILD, ADULT | |
| Empowering Adolescents and Young Adults With Sickle Cell Disease as Partners in Treatment Decision Making (EMPOWER-AYA) | This study will evaluate the acceptability, feasibility, and preliminary efficacy of a shared decision making intervention for adolescents and young adults (AYAs) with sickle cell disease (SCD). 60 AYAs with SCD ages 15-25 and their caregivers and 8 SCD providers will participate in the pilot pragmatic trial. AYAs, caregivers, and providers will be recruited from Nemours Children's Hospital, Delaware (NCH-DE), Nemours Children's Hospital in Orlando, FL (NCH-ORL), and Nemours Children's Health at Wolfson Children's Hospital in Jacksonville, FL (NCH-JAX). NCH-DE participants (n=30) will receive the SDM intervention including a virtual reality patient health education component, whereas NCH-ORL and NCH-JAX participants (n=30) will receive the SDM intervention with standard patient education materials (print, video). SCD providers will be trained to use the toolkit components and will introduce decision aids during an outpatient clinic visit for AYAs who are candidates for one or more disease-modifying therapies. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Nemours Children's Hospital, Delaware, Wilmington, Delaware, 19803, United States |
| Study to Assess Efficacy and Safety of VIT-2763 (Vamifeport) in Subjects With Sickle Cell Disease | The purpose of this study is to investigate the effect of VIT-2763 on markers of hemolysis (breakdown in red blood cells) in sickle cell disease (SCD). The safety, tolerability and clinical beneficial effects of VIT-2763 for the treatment of SCD are also explored. | Sickle Cell Disease | ALL | ADULT | Investigator Site 709, Birmingham, Alabama, 35233-2110, United States|Investigator Site 708, Los Angeles, California, 90027, United States|Investigator Site 713, Aurora, Colorado, 80045, United States|Investigator Site 706, Hollywood, Florida, 33023, United States|Investigator Site 703, Chicago, Illinois, 60612, United States|Investigator Site 701, Greenville, North Carolina, 27834, United States|Investigator Site 711, Charleston, South Carolina, 29425, United States|Investigator Site 702, Milwaukee, Wisconsin, 53226, United States|Investigator Site 801, Colombes, 92700, France|Investigator Site 802, Lyon, 690003, France|Investigator Site 305, Athens, 11527, Greece|Investigator site 301, Athens, GR-11527, Greece|Investigator Site 302, Patra, Greece|Investigator Site 101, Baabda, Lebanon|Investigator Site 102, Beirut, Lebanon|Investigator Site 103, Tripoli, Lebanon|Investigator Site 606, Liverpool, L9 7AL, United Kingdom|Investigator Site 603, London, SE59RS, United Kingdom|Investigator Site 608, London, W12 0HS, United Kingdom|Investigator Site 601, London, United Kingdom|Investigator Site 605, London, United Kingdom|Investigator Site 607, Manchester, M13 9WL, United Kingdom |
| Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease | This study is an open-label, non-randomized, single-dose Phase 1/2 trial involving around 85 adult and pediatric participants aged between 2 and 50 years with sickle cell disease (SCD). It aims to assess the effectiveness of hematopoietic stem cell transplantation (HSCT) using BAH243 for SCD. | Sickle Cell Disease|Sickle-Cell Disease With Crisis | ALL | CHILD, ADULT, OLDER_ADULT | District One Hospital, Beijing, Beijing, 086-373, China |
| Gaming Technology to Engage Adolescent Sickle Cell Patients in Pain Management | Sickle cell disease (SCD) is a common genetic disorder characterized by episodes of pain, yet assessments to identify type, intensity, frequency, and phase of pain among SCD adolescents is lacking. Research shows that interactive gaming technology can enhance adolescents' learning, and can be especially effective in delivering health-related messages and tools to improve their self-care. Pinpoint is an interactive gaming tablet app that will be developed with the significant input of clinical experts to assist SCD teens with better identification and self-report of their pain. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Klein Buendel, Inc., Golden, Colorado, 80401, United States|Hilton Publishing Company, Munster, Indiana, 463213963, United States |
| Effect of Empowerment-Based Interventions on Self-Efficacy and Self-Care Capacity Among Adults With Sickle Cell Disease | This study aimed to To investigate the effect of empowerment-based interventions (EBI) on self-efficacy and self-care capacity among adults with Sickle Cell Disease (SCD). The hypotheses of this study were: Adults with SCD who receive EBI exhibit higher self-efficacy, self- management capacity and HRQoL than those who do not. | Self-Efficacy|Self-Care | ALL | ADULT | College of Health and Sport Sciences, University of Bahrain, Manama, 11511, Bahrain |
| Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease | Background: - Sickle cell disease (SCD) is a blood disease. The drug hydroxyurea (HU) is approved to prevent pain crises in people with SCD. Researchers want to see how higher doses of HU affect the blood. This will help them learn about the right dosage of HU to give to people with SCD. Objective: - To improve hydroxyurea dosing in people with SCD. Eligibility: - People age 15 or older with homozygous SCD (HbSS). Design: * Participants will be screened with medical history, physical exam, medication review, and blood and urine tests. * Participants will be in the study for about 15 months. * First 3 months: monthly study visits with blood and urine tests. * After 3 months: participants will take HU as a capsule by mouth. If you are already taking HU, your dose will be increased. * Within a month of starting or increasing HU: participants will keep a daily pain diary for 2 weeks. They will have an echocardiogram (ultrasound) of the heart, a 6-minute walk test. They will complete a quality-of-life questionnaire. * Participants will visit every month until they reach their highest tolerated dose of HU. They may need to come as often as every week sometimes to closely monitor their blood counts. Then they will alternate a phone call one month and a visit the next. At the visits, participants will bring their pill bottle, answer questions about side effects, and have blood tests. * Every 2 months, participants will have a medical history, physical exam, and blood tests. * Every 4 months, participants will have blood and urine tests. They will also complete another 2-week pain diary and quality-of-life questionnaire. * About 12 months after starting or increasing HU, participants will have blood tests, an echocardiogram, and a 6-minute walk test. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| Exercise-induced Pulmonary Hypertension in Patients With Sickle-cell Anemia | Pulmonary hypertension (PH) at rest is a risk factor for death in patients with sickle-cell anemia (SCA). Exercise echocardiography (EE) can detect latent PH. We sought to investigate the occurrence of exercise-induced PH in patients with SCA and normal pulmonary pressure (PP) at rest, and its relationship with clinical and echocardiographic variables.Forty-four patients with SCA and normal PP at rest were studied and divided into two groups: exhibiting normal PP after treadmill EE (TRV≤2.7m/s) (G1), and exhibiting exercise-induced PH (TRV\>2.7m/s) (G2). TRV cutoff points at rest and during exercise were based on data from healthy control subjects, matched for age, sex, and body surface area. Data obtained from EE were correlated with clinical, echocardiographic and ergometric variables.Exercise-induced PH occurred in 57% of the sample (G2), significantly higher than those of G1. Exercise-induced PH was related to higher levels of creatinine (p\<0.05), increased left atrial volume (p\<0.05) and right ventricular diastolic area (p\<0.05), larger E/Em waves ratio derived from spectral and tissue Doppler (p\<0.05), and higher TRV at rest (p\<0.005).We concluded that patients with SCA and normal PP at rest may exhibit exercise-induced PH, which was related to renal function, increased cardiac chambers, abnormal indices of diastolic function and baseline TRV levels. | Sickle Cell Anemia|Pulmonary Hypertension | ALL | CHILD, ADULT, OLDER_ADULT | |
| Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs | The main purpose of this project is to cure patients with high risk Sickle cell disease and other red cell disorders including thalassemia and diamond-blackfan anemia by bone marrow transplantation. The patients enrolled in this study will be those who lack matched sibling donors and therefore have no other option but to undergo bone marrow transplantation using matched but unrelated bone marrow or umbilical cord blood from the national marrow donor program registry. Since bone marrow transplantation for these disorders using matched unrelated donors has two major problems i.e. engraftment, or , the process of new marrow being accepted and allowed to grow in the the patient; and graft-versus-host disease, or the process where the new marrow "rejects" the host or the patient, this study has been devised with methods to overcome these two problems and thus make transplantation from unrelated donors both successful in terms of engraftment and safe in terms of side effects, both acute and long term. In order to accomplish these two goals, two important things will be done. Firstly, patients will get three medicines which are considered reduced intensity because they are not known to cause the serious organ damage seen with conventional chemotherapy. These medicines, however, do cause intense immune suppression so these can cause increased infections. Secondly, in addition to transplantation of bone marrow from unrelated donors, patients will also transplanted with mesenchymal stromal cells derived from the bone marrow of their parents. Mesenchymal stromal cells are adult stem cells that are normally found in the bone marrow and are thought to create the right background for the blood cells to grow. They have been shown in many animal and human studies to improve engraftment. In addition, they have a special property by which they prevent and are now even considered to treat graft versus host disease. Therefore, by using a reduced intensity chemotherapy regimen before transplant and transplanting mesenchymal stromal cells, we hope to improve engraftment while at the same time decrease the potential for severe side effects associated with a conventional transplant which uses extremely high doses of chemotherapy. | Sickle Cell Disease|Thalassemia|Diamond-Blackfan Anemia | ALL | CHILD, ADULT | Children's Hospital of Alabama, Birmingham, Alabama, 35233, United States|University of Minnesota, Minneapolis, Minnesota, 55455, United States |
| Patient-Provider Tools to Improve the Transition to Adult Care in Sickle Cell Disease | The purpose of the study is to develop patient-provider clinical support tools to improve clinical practice, patient self-management, and disease outcomes in sickle cell disease during transition to adult care. The investigators hypothesize that these clinical support tools (patient tool, provider tool, and patient/parent web-based portal) will be feasible, user friendly, and beneficial. The investigators hypothesize that participants will demonstrate better disease self-efficacy at the end of the 6 week intervention and maintain these gains during the follow-up period (up to 1 year post-intervention). | Sickle Cell Disease | ALL | CHILD, ADULT | Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States |
| Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease. | This is a prospective, interventional, phase II, open-label, multicentre, national, non-comparative study of a single administration of the new dispersible form of hydroxycarbamide at the usual dose in children with sickle cell disease who are already treated with the current form of hydroxycarbamide (Siklos® 100 mg and/or 1000 mg film-coated tablets). | Sickle Cell Disease | ALL | CHILD | InterCommunal Hospital Centre of Creteil, Créteil, 94000, France|Necker University Hospital, Paris, 75015, France|Robert Debré Hospital, Paris, 75019, France |
| Validation of the Sickle Cell Disease Pain Burden Interview | The purpose of this study is to validate a brief survey tool, the SCD Pain Burden Interview (SCPBI), which can be used in the clinical and/or research settings to assess the impact of pain on children with sickle cell disease. | Sickle Cell Disease|Pain | ALL | CHILD, ADULT | Connecticut Children's Medical Center, Hartford, Connecticut, 06106, United States |
| Nitric Oxide Inhalation to Treat Sickle Cell Pain Crises | This study will examine whether nitric oxide (NO) gas can reduce the time it takes for pain to go away in patients who are in sickle cell crisis. NO is important in regulating blood vessel dilation, and consequently, blood flow. The gas is continuously produced by cells that line the blood vessels. It is also transported from the lungs by hemoglobin in red blood cells. Patients 10 years of age or older with sickle cell disease (known SS, S-beta-thalassemia or other blood problems causing sickle cell disease) may be eligible for this study. Patients whose disease is due to hemoglobin (Hgb) SC are excluded. Candidates are screened with blood tests and a chest x-ray to look at the lungs and heart. Participants are admitted to the hospital in a pain crisis. They are evaluated and then randomly assigned to receive one of two treatments: 1) standard treatment plus NO, or 2) standard treatment plus placebo. The placebo used in this study is nitrogen, a gas that makes up most of the air we breathe and is not known to help in sickle cell disease. For the first 8 hours of the study, patients receive placebo or NO through a facemask. The mask may be taken off for 5 minutes every hour and for not more than 20 minutes to eat a meal. After the first 8 hours, the gas is delivered through a nasal cannula (small plastic tubing that rests under the nose) that may be taken off only while showering or using the restroom. Patients are questioned about the severity of their pain when they start the study and then every few hours while they are in the hospital. Their vital signs (temperature, breathing rate, and blood pressure) and medicines are checked. Patients will breathe the gas for a maximum of 3 days, but will stay hospitalized until the patient feels well enough to go home. Patients are followed up about 1 month after starting the study by a return visit to the hospital or by a phone call. | Anemia, Sickle Cell | ALL | CHILD, ADULT, OLDER_ADULT | University of Alabama, Birmingham, Alabama, 35294, United States|Children's Hospital Oakland, Oakland, California, 94609-1809, United States|Colorado Sickle Cell Treatement and Research Center, Aurora, Colorado, 80045, United States|Howard University Hospital, Washington, District of Columbia, 20060, United States|Johns Hopkins University, Baltimore, Maryland, 21205, United States|National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States|Brigham and Women's Hospital, Boston, Massachusetts, 02115, United States|Childrens Hospital, Boston, Boston, Massachusetts, 02115, United States|Case Western Reserve University Hospital, Cleveland, Ohio, 44106-2602, United States|St. Christopher's Hospital for Children, Philadelphia, Pennsylvania, 19134, United States|Childrens Hospital, Pittsburgh, Pittsburgh, Pennsylvania, 15213-2583, United States |
| An Indian Multi-centric Phase IV Study to Assess the Safety of Crizanlizumab in Sickle Cell Disease Patients | Sickle cell disease (SCD) is a genetic blood disorder. Crizanlizumab is indicated to reduce the frequency of vaso-occlusive crises (VOCs) in patients with SCD aged 16 years and older. The purpose of this local Phase IV study was to evaluate the safety of crizanlizumab specifically in Indian patients with SCD aged 16 years or older with a history of VOC leading to healthcare visit. | Sickle Cell Disease (SCD) | ALL | CHILD, ADULT, OLDER_ADULT | Novartis Investigative Site, Guwahati, Assam, 781003, India|Novartis Investigative Site, Raipur, Chhattisgarh, 492099, India|Novartis Investigative Site, Kozhikode, Kerala, 673008, India|Novartis Investigative Site, Bhubaneswar, Odisha, 751003, India|Novartis Investigative Site, Hyderabad, Telangana, 500082, India|Novartis Investigative Site, Lucknow, Uttar Pradesh, 226014, India|Novartis Investigative Site, Kolkata, West Bengal, 700014, India |
| Phase 2 Study of MP4CO to Treat Vaso-occlusive Sickle Crisis | Sickle Cell disease is caused by an inherited hemoglobin disorder. Healthy red blood cells are discoid and can deform and move through small blood vessels to carry oxygen to all parts of the body. In Sickle Cell disease, as red blood cells circulate and oxygen is released, the deoxygenated abnormal Hemoglobin S can begin to polymerize and cause red cells to become sticky and elongated. These "sickled" red cells are less flexible and will obstruct small blood vessels and prevent normal red cells from circulating freely, which limits oxygen delivery to tissues and organs. This is known as a "sickling crisis" or "vaso-occlusive crisis" and is the leading cause of hospitalization in patients with Sickle Cell disease. Patients suffering from a sickle crisis experience severe pain and are at risk of stroke, heart attack or even death. Current therapy is limited to hydration and symptomatic pain relief. The administration of MP4CO as an adjunct treatment to standard therapy may alleviate pain associated with a sickling crisis and potentially reduce the severity and duration of a crisis. This may shorten the time in hospital and potentially improve the quality of life for patients with sickle cell anemia. | Anemia, Sickle Cell|Sickle Cell Anemia|Sickle Cell Disease|Sickle Cell Disorders|Hemoglobin SC Disease|Sickle Cell Hemoglobin C Disease | ALL | CHILD, ADULT, OLDER_ADULT | Salmaniya Medical Complex, Manama, Bahrain|University Hospital Brugmann, Brussels, Belgium|Rio de Janerio Instituto Estadual de Hematologie, Rio de Janerio, Brazil|Hôpital Henri Mondor, Créteil, France|Georges Pompidou European University Hospital, Paris, France|American Univ. of Beirut Medical Center, Beirut, Lebanon|Univ. Medical Center Rizk Hospital, Beirut, Lebanon|Academic Medical Center, Amsterdam, Netherlands|Cornell Medical City, Doha, Qatar|Cukurova University Medical Facilty, Adana, Turkey|Mersin University Medical Faculty, Mersin, Turkey|Guys Hospital, London, United Kingdom|King's College Hospital, London, United Kingdom|Queen Mary Hospital, London, United Kingdom |
| Induction of Stable Chimerism for Sickle Cell Anemia | To investigate a modified hematopoeitic cell transplantation (HCT) procedure for sickle cell disease that significantly reduces the toxicity of HCT, yet retains its therapeutic benefit. | Blood Disease|Hematopoietic Stem Cell Transplantation|Anemia, Sickle Cell | ALL | CHILD, ADULT, OLDER_ADULT | |
| Ketorolac for Acute Vaso-Occlusive Crisis in Pediatric Sickle Cell Disease | Pediatric patients who present with acute vaso-occlusive pain crisis may have equivalent pain reduction scores at lower dosing of intravenous Ketorolac compared to standard dosing of 0.5 mg/kg/dose IV (\<16yo max 15mg, \>16yo max 30mg) x 1 dose. | Sickle Cell Disease|Sickle Cell Crisis|Veno-occlusive Disease | ALL | CHILD, ADULT | |
| Motixafortide and Natalizumab to Mobilize CD34+ Hematopoietic Stem Cells for Gene Therapies in Sickle Cell Disease (SCD) | Hematopoietic stem cell (HSC)-based gene therapies now offer curative potential for patients with sickle cell disease (SCD), with decreased toxicity compared to allogeneic hematopoietic cell transplantation. However, effective HSC-based gene therapy depends on collecting sufficient HSCs to generate the therapeutic product, and currently available mobilization regimens carry unacceptable risk for patients with SCD or do not reliably yield optimal numbers of HSCs for gene therapy. The investigators hypothesize that HSC mobilization with motixafortide (CXCR4i) alone and the combination of motixafortide plus natalizumab (VLA-4i) will be safe and tolerable in SCD patients. In addition, the investigators hypothesize that combined CXCR4 and VLA-4 blockade with motixafortide plus natalizumab will result in a rapid, robust, and synergistic increase in HSC mobilization to peripheral blood (PB) in patients with SCD, when compared to motixafortide alone. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Washington University School of Medicine, Saint Louis, Missouri, 63110, United States |
| Interest of Nutritional Care of Children With Sickle Cell Disease on Bone Mineral Density and Body Composition | This study is design to assess the effects of an increase in nutritional intake on the bone mineral density of children with sickle cell disease, for 12 months. | Sickle Cell Disease|Osteoporosis|Osteopenia | ALL | CHILD | CHR Orléans, Orléans, France |
| Evaluation of HemoTypeSC as a Novel Rapid Test for Point-of-Care Screening for Sickle-Cell Disease, Hemoglobin C Disease, and Carrier Status in Low-Resource Settings | Sickle cell disease is a life-threatening genetic disorder that can be effectively treated following early diagnosis via newborn screening. However, sickle cell disease is most prevalent in low-resource regions of the world, where newborn screening is rare due to the cost and logistical burden of laboratory-based methods. In many such regions, \>80% of affected children die, undiagnosed, before the age of five years. A convenient and inexpensive point-of-care test for sickle cell disease is thus crucially needed. In this study we will conduct a blinded, multicenter, prospective diagnostic accuracy study of HemoTypeSC(TM), an inexpensive 15-minute point-of-care immunoassay for detecting sickle cell disease, hemoglobin C disease, and trait phenotypes in newborns, children, and adults. | Diagnoses Disease | ALL | CHILD, ADULT, OLDER_ADULT | Silver Lake Research Corporation, Azusa, California, 91702, United States |
| Working Memory and School Readiness in Preschool-Aged Children With Sickle Cell Disease | Children with sickle cell disease (SCD) are at risk for neurobehavioral problems because of the impact the disease can have on the central nervous system. Specific impairments in working memory are particularly prevalent in school-aged children with SCD. Working memory is more strongly associated with school readiness and academic success than intellectual ability in the general population. The adverse effects of low socioeconomic status (SES) and poverty on cognition and neurodevelopment emerge early, before children have entered formal education. In addition, they affect language and executive function skills (e.g., working memory) more than other skills. SES is a proxy variable for other risk factors. Higher SES is associated with less parental stress, more supportive parenting practices, and better cognitive stimulation based on the availability of books, computers, and outings. PRIMARY OBJECTIVE: * To examine working memory and school readiness in young children with sickle cell disease in comparison to demographically matched control children without sickle cell disease. SECONDARY OBJECTIVE: * To examine the relationships of family/environmental factors (caregiver stress, parental responsiveness, and cognitive stimulation in the home) and disease severity to working memory and school readiness skills in preschool-aged children with SCD. | Sickle Cell Disease | ALL | CHILD | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Hydroxyurea for the Treatment of Patients With Sickle Cell Anemia | A total of fifty severely affected patients with homozygous sickle cell disease or other sickling disorders (e.g. B negative or B positive Thalassemia/Sickle) who are greater than 18 years of age will be eligible for treatment. Such patients must be able to tolerate an extensive period without blood transfusion and have relatively well preserved renal and hepatic function (creatinine less than 1.5 mg/dl and normal liver function test with exception of a mild elevation in transaminase). Evidence of severe sickle cell anemia will include recurrent pain crisis, chronic bone oain, evidence of aseptic necrosis with symptoms, and intractable leg ulcer, etc. On admission to the study, each patient will receive a complete history and physical examination. These data and standard laboratory evaluation, including a test for pregnancy if appropriate, will be adequate to ascertain whether any of the criteria for exclusion are present. Each patient must accept responsibility for for using an effective means of contraception. Patients who are found to be HIV positive will be excluded from the study.... | Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| Brain Structure and Neurocognitive Development in Sickle Cell Disease; a Longitudinal Cohort Study (BRICK Study) | Sickle cell disease (SCD) is an autosomal recessive red blood cell blood disorder. One especially vital organ affected in SCD is the brain. Individuals with SCD have an increased risk of both overt cerebral infarctions and silent infarctions. The latter are brain lesions without apparent neurological sequelae. Since cortical neurons in the brain lack the ability to regenerate, tissue damage accumulates throughout the already shortened lifespan of individuals with SCD, resulting in far-reaching consequences such as significant cognitive impairment. Currently, only hematological stem cell transplantation can halt the multiorgan tissue damage. However, the criteria to determine the timing of curative therapy do not center the brain, despite that subtle anomalies of this critical organ can have long-lasting consequences. Since it is not yet known whether brain tissue damage precedes, parallels, or lags behind non-brain tissue damage, it is critical to map these effects in youth with SCD. While importantly comparing images with a healthy reference population. Understanding how the brain is affected is critical for clinical decision making, such as timing of potentially curative interventions but also, to prevent long term irreversible brain damage in youth with SCD. In this study, a cohort of 84 SCD patients between the ages of 6 and 18 at baseline, will undergo MR imaging, neurological examination, neuropsychological assessment and blood sampling three times in total, with intervals of two years; results will be innovatively compared with children included in the Generation R population study (±8000 MRIs children and (young)adults) 6-20 years of age). Our hypothesis, based on the inability of the brain to generate new cortical neurons following cell death, is that brain function is impaired earlier than other organ systems and that there is an age-dependent limit in the brain's ability to remodel itself based on neuroplasticity. | Brain Development Abnormality|Sickle Cell Disease | ALL | CHILD, ADULT | Erasmus MC, Rotterdam, South Holland, 3000 CA, Netherlands|Amsterdam University Medical Center, Amsterdam, Netherlands |
| Effect of Virtual Reality Technology for Pain Management of Vaso-Occlusive Crisis in Patients With Sickle Cell Disease | Acute vaso-occlusive crisis (VOC) is the most common complication in patients with sickle cell disease (SCD) and pain related to VOC is often inadequately treated. This is a phase II randomized controlled clinical trial evaluating the efficacy of virtual reality technology when added to standard pain management for patients with sickle cell disease who are experiencing acute pain crisis in the ambulatory care setting. Patients will be randomized to receive either standard management only or standard management in addition to virtual reality therapy. The remainder of care for the painful event will continue per institutional standards according to clinical indication, including reassessment and documentation of pain and additional doses of pain medicines by intravenous (IV) or oral route. Pain scores and opioid requirement will be measured and compared across treatment arms, along with the outcomes of discharge from clinic versus admission to the inpatient unit. PRIMARY OBJECTIVE: To assess the efficacy of virtual reality (VR) technology in reducing pain at 30 minutes after intervention during an acute vaso-occlusive crisis in patients with sickle cell disease. Primary endpoint will be change in pain scores in Standard versus VR arms, between the first pain assessment at the time of presentation and the subsequent pain assessments up to 30 minutes after intervention. Secondary Objectives: * To compare total opioid consumption from the time of presentation to the time of discharge from acute care setting in Standard versus VR arms. * To assess the efficacy of virtual reality (VR) technology in reducing pain at 60 minutes after the first IV medication administered or 60 minutes after completion VR during an acute vaso-occlusive crisis in patients with sickle cell disease. | Sickle Cell Disease|Vaso-occlusive Crisis | ALL | CHILD, ADULT | Methodist Comprehensive Sickle Cell Center, Memphis, Tennessee, 38104, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Non Invasive Positive Pressure Ventilation for Prevention of Acute Chest Syndrome in Sickle Cell Disease | Acute chest syndrome is a severe respiratory complication of sickle cell disease. The standard prevention of this dangerous complication is spirometry in wich patient is required to take deep breaths trough a little device several times a day. This treatment is compromised when pain in important or when the patient is asleep and cannot participate. The investigators hypothesised that non invasive ventilation in wich patient have no effort to take might be a better prevention than spirometry and may improve pain and quality of sleep. Children with vaso-occlusive crisis necessitating morphinic treatment will be randomly assigned with either spirometry or ventilation and the investigators will monitor for occurrence of acute chest syndrome, pain and quality of sleep. | Sickle Cell-hemoglobin SS Disease|Vaso-occlusive Crisis | ALL | CHILD, ADULT | Hôpital Necker - Enfants Malades Hospital, Paris, 75015, France |
| Role of Transcranial Doppler and Magnetic Resonance Angiography for Future Management of Sickle Cell Anemia CNS. | This study aimed to determine the Predictive Value of Transcranial Doppler and Magnetic Resonance Angiography for Future Management of Sickle Cell Anemia. Specific aims are: Demonstrate silent parenchymal and vascular brain changes that are incidentally observed in neurologically free SCD children using screening TCD and MRA in Pediatric Hematology unit at Assiut University Hospital Detect any abnormality with vasculopathy, arterial occlusion and old SCI. Strokes in children with SCD can be prevented by checking a transcranial Doppler (TCD) ultrasound,MRA and providing blood transfusions to children with abnormal blood flow on the TCD and detect Silent cerebral and cerebrovascular changes in SCD. | Blood Disease|Brain Injuries | ALL | CHILD, ADULT | |
| Intermittent Preventive Treatment for Malaria in Patient With Sickle Cell Disease | Malaria prophylaxis is recommended for sickle cell disease patients. In Nigeria, daily proguanil or weekly pyrimethamine are the most commonly prescribed regimens, but the current policy is not effective due to poor compliance and drug resistance. Intermittent treatment with a long acting drug regimen administered under supervision at clinic visits may be more effective. The aim of this trial is to compare the tolerability and acceptability of supervised bimonthly treatment with either sulfadoxine-pyrimethamine plus amodiaquine (SP+AQ) or mefloquine plus artesunate (MQ+AS), with the daily proguanil. Two hundred and seventy patients with sickle cell disease attending the paediatric sickle cell disease clinic in Ilorin hospital who meet the eligibility criteria and have parental consent, will be randomized to one of three prophylactic regimens: daily proguanil, bimonthly sulfadoxine-pyrimethamine plus amodiaquine, or bimonthly mefloquine plus artesunate. Patients will be asked to return to clinic every two months and whenever they are sick. At enrollment, the study paediatrician will conduct a physical examination of the child, and collect a venous blood sample for a complete blood cell count and biochemical screen, determination of G6PD genotype, preparation of blood smears for malaria microscopy and a blood spot for determination of molecular markers of resistance. Four days after each clinic visit, patients will be interviewed (by phone and, for a subset, at home or in the clinic) to ask about compliance and adverse events. Participants will be followed for one year. The parents or carer will be encouraged to bring their child to the Outpatient Department clinic if the child becomes unwell. The primary outcome of the trial is tolerability, secondary outcomes are adherence to the regimen, and incidence of malaria and the number of hospitalizations over 12 months. If the bimonthly regimens are well tolerated and the preliminary data from this study are promising, a larger multicentre trial will be required to determine efficacy. | Malaria|Sickle Cell Crisis | ALL | CHILD, ADULT | Department of Paediatrics and Child Health, University of Ilorin Teaching Hospital, Ilorin, Kwara, Nigeria |
| THromboprophylaxis In Sickle Cell Disease With Central Venous Catheters (THIS) | Research Question: In adult SCD patients with CVC, is it feasible and safe to conduct an adequately powered RCT to evaluate the use of rivaroxaban as thromboprophylaxis in this population? Study Design: The study is a vanguard pilot double blind multi-centre randomized controlled trial. Participants with SCD and CVC will be randomized to either rivaroxaban 10mg PO daily or placebo for the duration of CVC in situ or for up to one year, whichever is less. After screening (day -7 to day -1), patients will be followed at day 1, months 3 (+/- 15 days), 6 (+/- 15 days), 9 (+/- 15 days), and 12 (+/- 15 days). Study Objectives: The primary objective is to estimate the proportion of eligible patients who will enroll into a trial of thromboprophylaxis. Secondary objectives include (a) document indications for central venous catheter (CVC), (b) summarize duration of CVC insertion prior to enrollment, (c) estimate adherence to the study drug, (d) estimate proportions of participants being compliant with study procedures, and lost to follow up. Exploratory objectives will assess thrombotic, bleeding, and quality of life outcomes. | Sickle Cell Disease|Central Venous Catheter Thrombosis|Venous Thromboembolism | ALL | ADULT, OLDER_ADULT | University of Alberta Hospital, Edmonton, Alberta, T6G 2B7, Canada|London Health Sciences Center, London, Ontario, N6A 5W9, Canada|Toronto General Hospital, Toronto, Ontario, M5G 2C4, Canada |
| A Study of Varespladib Infusion in Subjects With Sickle Cell Disease. | The purpose of this study is to determine the effectiveness of A 001 infusion in preventing acute chest syndrome in sickle cell disease (SCD) subjects with vaso-occlusive crisis, fever, and elevated serum C-reactive protein (CRP). | Sickle Cell Disease|Vaso-occlusive Crisis | ALL | CHILD, ADULT, OLDER_ADULT | Investigator Site 101, Atlanta, Georgia, 30342, United States |
| Cooperative Assessment of Late Effects for SCD Curative Therapies | Sickle Cell Disease is one of the most common genetic diseases in the United States, occurring in approximately 1 in 400 births. Approximately 100,000 individuals are diagnosed with SCD in the United States. Mortality for children with SCD has decreased substantially over the past 4 decades, with \>99% of those born in high resource settings, including the United States, France, and England, now surviving to 18 years of age. However, the life expectancy of adults with SCD is severely shortened. Dysfunction of the heart, lung, and kidney is directly associated with decreased life expectancy. With the variety of curative therapies that are now available for SCD, long-term health outcomes studies are time-sensitive. As of now, efforts to determine long-term health outcomes following curative therapies for SCD have been limited. Though curative therapies initially should provide a cure for symptoms of SCD, there is the risk of late health outcomes to consider. Defining health outcomes following curative therapy is essential to improve personalized decision-making when considering curative versus disease-modifying therapeutic options. The primary goal of this study is to determine whether curative therapies for individuals with SCD will result in improved or worsening heart, lung, and kidney damage when compared to individuals with SCD receiving standard therapy. The investigators will also explore whether certain genes are associated with a good or bad outcome after curative therapy for SCD. | Sickle Cell Disease|Pulmonary Disease|Renal Disease|Heart Disease | ALL | CHILD, ADULT, OLDER_ADULT | Children's National Medical Center, Washington, District of Columbia, 20010, United States|Emory University School of Medicine, Atlanta, Georgia, 30322, United States|Johns Hopkins Hospital, Baltimore, Maryland, 21287, United States|National Institutes of Health Clinical Center, Bethesda, Maryland, 20814, United States|Vanderbilt University Medical Center, Nashville, Tennessee, 37232-9000, United States |
| A Phase 2/3 Study in Adult and Pediatric Participants With SCD | The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Lynn Institute of the Ozarks, Little Rock, Arkansas, 72204, United States|Lundquist Institute for Biomedical Innovation at Harbor- UCLA Medical Center, Torrance, California, 90502, United States|Smilow Cancer Center at Yale New Haven Hospital, New Haven, Connecticut, 06511, United States|Smilow Cancer Center at Yale New Haven Hospital, New Haven, Connecticut, 06520, United States|Children's National Health System, Washington, District of Columbia, 20010, United States|Children's National Hospital, Washington, District of Columbia, 20010, United States|Edward Jenner Research Group LLC., Miami, Florida, 33317, United States|Kidz Hematology / Oncology a division of Kidz Medical, West Palm Beach, Florida, 33407, United States|Palm Beach Children's Hospital at St. Mary's Medical Center, West Palm Beach, Florida, 33407, United States|Sonar Clinical Research, Atlanta, Georgia, 30315, United States|Sonar Clinical Research, Atlanta, Georgia, 30331, United States|Alpha Clinical Research Georgia, Dunwoody, Georgia, 30350, United States|University of Illinois Hospital and Health Sciences System - Hospital Pharmacy Services, Chicago, Illinois, 60612-4333, United States|University of Illinois Hospital and Health Sciences System, Chicago, Illinois, 60612-7232, United States|University of Illinois at Chicago Clinical Research Center, Chicago, Illinois, 60612, United States|University of Illinois Hospital and Health Sciences System - Investigational Drug Services (IDS), Chicago, Illinois, 60612, United States|LSU Health Baton Rouge-North Clinic, Baton Rouge, Louisiana, 70805, United States|Our lady of the Lake Hospital, Baton Rouge, Louisiana, 70805, United States|Our Lady of the Lake Hopistal, Inc, Baton Rouge, Louisiana, 70808, United States|Our Lady of the Lake Hospital, Inc In-Patient Pharmacy, Baton Rouge, Louisiana, 70808, United States|Our Lady of the Lake Physicians Group, Medical Oncology, Baton Rouge, Louisiana, 70808, United States|University Medical Center New Orleans, New Orleans, Louisiana, 70112, United States|Mississippi Center for Advanced Medicine, Madison, Mississippi, 39110, United States|Truman Medical Center, Kansas City, Missouri, 64108, United States|UNC Hospitals, The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, 27599-7600, United States|UT Physicians Comprehensive Sickle Cell Clinic, Houston, Texas, 77004, United States|McGovern Medical School/Health Science Center Houston, Houston, Texas, 77030, United States|Memorial Hermann - TMC Investigational Drugs, IDS Pharmacy, Houston, Texas, 77030, United States|Memorial Hermann Hospital, Texas Medical Center - Clinical Research Unit (CRU), Houston, Texas, 77030, United States|UT Physicians Comprehensive Sickle Cell Clinic, Houston, Texas, 77030, United States|Inova Schar Cancer Institute Infusion Pharmacy, Fairfax, Virginia, 22031, United States|Inova Schar Cancer Institute, Fairfax, Virginia, 22031, United States|Clinical Research Services Unit - Virginia Commonwealth University Medical Center, Richmond, Virginia, 23298, United States|Virginia Commonwealth University, Richmond, Virginia, 23298, United States|Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo, Sao Paulo, SP, 05403-010, Brazil|Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP, Ribeirão Preto, SÃO Paulo, 14051-140, Brazil|HEMORIO - Unidade de Pesquisa Clínica, Rio De Janeiro, 20211-030, Brazil|Instituto Estadual de Hematologia Arthur Siqueira Cavalcanti - HEMORIO, Rio de Janeiro, 20211-030, Brazil|Real e Benemerita Associacao Portuguesa de Sao Paulo, Sao Paulo, 01321-000, Brazil|Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo, São Paulo, 05403-010, Brazil|Nirmal Hospital Pvt Ltd, Surat, Gujarat, 395002, India|Chopda Medicare & Research Centre Pvt. Ltd: Magnum Heart Institute, Nashik, Maharashtra, 422005, India|KEMRI/CRDR, Siaya, KEMRI Clinical Research Annex, Kisumu, Siaya, 40600, Kenya|Gertrude's Children's Hospital, Nairobi, 00100, Kenya|Kenya Medical Research Institute - Centre for Respiratory Disease Research, Nairobi, 00100, Kenya|Center for Research In Therapeutic Sciences (CREATES), Strathmore University Medical Centre, Nairobi, 00200, Kenya|Center for Research In Therapeutic Sciences (CREATES), Strathmore University Medical Centre, Nairobi, 200, Kenya|University College Hospital Ibadan, Ibadan, Oyo/ibadan North, 200212, Nigeria|Aminu Kano Teaching Hospital, Kano, 700233, Nigeria|Lagos University Teaching Hospital, Lagos, 100254, Nigeria|University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, BS2 8ED, United Kingdom|Bristol Royal Infirmary, Bristol, BS2 8EX, United Kingdom |
| A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease | This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects ≥2 and ≤50 years of age with sickle cell disease (SCD). The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using bb1111 (also known as LentiGlobin BB305 Drug Product for SCD). | Sickle Cell Disease | ALL | CHILD, ADULT | University of Alabama, Birmingham, Alabama, 35233, United States|Children's National Hospital, Washington, District of Columbia, 20010, United States|Tufts Medical Center, Boston, Massachusetts, 02111, United States|University of Minnesota, Minneapolis, Minnesota, 55455, United States|Hackensack University Medical Center, Hackensack, New Jersey, 07601, United States|Montefiore Medical Center, Bronx, New York, 10467, United States|Duke University, Durham, North Carolina, 27705, United States|Baylor College of Medicine/Texas Children's Hospital, Houston, Texas, 77030, United States|Virginia Commonwealth University (VCU), Richmond, Virginia, 23219, United States |
| Novel Cardiac Magnetic Resonance Imaging to Define a Unique Restrictive Cardiomyopathy in Sickle Cell Disease | The purpose of this study is to use cardiac magnetic resonance imaging (CMR) and echocardiographic tissue Doppler imaging to demonstrate a unique restrictive cardiomyopathy of sickle cell disease. The investigators will characterize its frequency and how it might change (e.g., presence/absence and severity) over a 2-year period. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States |
| Fitness Trial in Adults With Sickle Cell Disease (SCD Fit): A Feasibility Study | The purpose of this project is to develop novel approaches to promote health and longevity while enhancing quality of life among persons with Sickle cell disease (SCD). Therefore, investigators are aiming to adapt an evidence-based exercise intervention for adults with SCD informed by culturally- relevant and biologic factors and pre-test the adapted exercise program in a small sample of adults with SCD. | Cardiovascular Diseases|Sickle Cell Disease|Exercise | ALL | ADULT, OLDER_ADULT | Memorial Healthcare System, Hollywood, Florida, 33021, United States |
| PET Imaging of Vaso-Occlusive Crisis (VOC) in SCD | The purpose of this study is to find objective biomarkers of vaso-occlusion (blood vessel blockage) in people with SCD. Using information from earlier studies and work being done, researchers have developed a strategy to image VOC, using positron emission tomography (PET). The ability to see and measure VOC in SCD patients can help patient care, by showing when and how a VOC is occurring or going to occur. Studying this method will also help in future drug research, as it will allow researchers to deliver promising new medications that target hyper-adhesion and sickling in people with SCD. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | UPMC, Pittsburgh, Pennsylvania, 15213, United States |
| Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease | This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to \< 15 years old, with Sickle Cell Disease. The primary objective is to evaluate the effect of voxelotor on the TCD (Transcranial Doppler Ultrasound) measurements in SCD participants in this age range. | Sickle Cell Disease | ALL | CHILD | Children's of Alabama, Birmingham, Alabama, 35233, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States|University of Miami, Miami, Florida, 33136, United States|Children's Healthcare of Atlanta: Hughes Spalding, Atlanta, Georgia, 30303, United States|Children's Healthcare of Atlanta AFLAC Center, Atlanta, Georgia, 30342, United States|Children's Healthcare of Atlanta: Scottish Rite, Atlanta, Georgia, 30342, United States|Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States|Boston Childien's Hospital - Clinical Research Pharmacy, Boston, Massachusetts, 02115, United States|Boston Children's Hospital - Clinical Research Pharmacy, Boston, Massachusetts, 02115, United States|Boston Children's Hospital, Boston, Massachusetts, 02115, United States|Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States|Washington University School of Medicine, Saint Louis, Missouri, 63110, United States|The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, 15224, United States|Medical University of South Carolina: Investigational Drug Services, Charleston, South Carolina, 29425, United States|Medical University of South Carolina: Shawn Jenkins Women's and Children's Hospital, Charleston, South Carolina, 29425, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|St. Jude Children's Research Hospital - Pharmaceutical Services, Memphis, Tennessee, 38105, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|Texas Children's Hospital - Investigational Pharmacy, Houston, Texas, 77030, United States|Texas Children's Hospital- Wallace Tower, Houston, Texas, 77030, United States|Texas Children's Hospital, Houston, Texas, 77030, United States|Ain Shams University Hospital- Clinical Research Center (MASRI), Cairo, Abassia, 11566, Egypt|Zagazig University Hospital, Zagazig, Alsharkia, Egypt|Alexandria Clinical Research Center, Faculty of Medicine, Alexandria University, Alexandria, 21131, Egypt|Abu El Rich Hospital, Cairo University Hospital, Cairo, 11562, Egypt|Cairo University Hospital, Abu El Rish Hospital, Cairo, 11562, Egypt|Abu El Rich Hospital, Cairo University Hospital, Cairo, Egypt|Abu Elrish Children Hospital, Cairo, Egypt|Egyptian Thalassemia Association (satellite site), Cairo, Egypt|AP-HP Hopital Robert Debré, Paris, 75019, France|Komfo Anokye Teaching Hospital, Kumasi, Ashanti, 00233, Ghana|Department of Child Health, University of Ghana Medical School, College of Health Sciences, Korle-Bu, Accra, Greater Accra, GA-221-1570, Ghana|Azienda Ospedaliera Universitaria Meyer "A.O.U. Meyer" - SOC "Oncologia, Ematologia e TCSE", Firenze, 50139, Italy|Azienda Ospedaliera Universitaria (A.O.U.) "Luigi Vanvitelli", Napoli, 80138, Italy|Azienda Ospedaliera Universita' (AOU ) Padova, Padova, 35128, Italy|Azienda Ospedaliera Universita' (AOU) Padova, Padova, 35128, Italy|Kemri/Crdr,Siaya,Kemri Clinical Research Annex, Kisumu, Siaya, 40600, Kenya|KEMRI CRDR Clinical Research Annex, Nairobi, 00100, Kenya|Strathmore University Medical Centre, Nairobi, 00200, Kenya|Gertrude's Children Hospital, Nairobi, 100, Kenya|Lagos University Teaching Hospital, Surulere, Lagos, 100254, Nigeria|College of Medicine, University of Ibadan, Ibadan, OYO State, 200212, Nigeria|University of Nigeria Teaching Hospital, Enugu, 460000, Nigeria|Barau Dikko Teaching/Kaduna State University, Kaduna, 800212, Nigeria|Barau Dikko Teaching/Kaduna State University, Kaduna, 800242, Nigeria|Aminu Kano Teaching Hospital, Kano, 70001, Nigeria|Aminu Kano Teaching Hospital, Kano, 700233, Nigeria|Sultan Qaboos University Hospital, Muscat, 123, Oman|Sultan َQaboos University Hospital, Muscat, Oman|King Saud University Medical City, Riyadh, 11472, Saudi Arabia|King Abdullah International Medical Research Center (KAIMRC), Ministry of National Guard - Health, Riyadh, 11481, Saudi Arabia|Barts Health NHS Trust, London, E1 1BB, United Kingdom|King's College Hospital NHS Foundation Trust, London, SE5 9RS, United Kingdom |
| Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias | People with severe congenital anemias, such as sickle cell anemia and beta-thalassemia, have been cured with bone marrow transplantation (BMT). The procedure, however, is limited to children younger than the age of 16 because the risks are lower for children than for adults. The purpose of this study is to explore the use of a BMT regimen that, instead of chemotherapy, uses a low dose of radiation, combined with two immunosuppressive drugs. This type BMT procedure is described as nonmyeloablative, meaning that it does not destroy the patient s bone marrow. It is hoped that this type of BMT will be safe for patients normally excluded from the procedure because of their age and other reasons. To participate in this study, patients must be between the ages of 18 and 65 and have a sibling who is a well-matched stem-cell donor. Beyond the standard BMT protocol, study participants will undergo additional procedures. The donor will receive G-CSF by injection for five days; then his or her stem cells will be collected and frozen one month prior to BMT. Approximately one month later, the patient will be given two immune-suppressing drugs, Campath 1-H and Sirolimus, as well as a single low dose of total body irradiation and then the cells from the donor will be infused. Prior to their participation in this study, patients will undergo the following evaluations: a physical exam, blood work, breathing tests, heart-function tests, chest and sinus x-rays, and bone-marrow sampling. ... | Congenital Hemolytic Anemia|Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Real World Patient Characteristics and Treatment Patterns From Crizanlizumab Use: Preliminary Analysis From Select Sickle Cell Centers | This was a retrospective cohort study using secondary data from member sites of the National Alliance of Sickle Cell Centers (NASCC) with patients who had initiated crizanlizumab between 2019 and 2022. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Novartis Pharmaceuticals, East Hanover, New Jersey, 07936, United States |
| Macrolide Therapy to Improve Forced Expiratory Volume in 1 Second in Adults With Sickle Cell Disease | Sickle cell anemia (SCA) is a life-threatening, monogenic disorder associated with early death when compared to individuals without SCA. Pulmonary complications, namely acute chest syndrome, obstructive lung disease and pulmonary hypertension, are the most common causes of death in patients with SCA. Recent studies suggest that lung specific inflammation is a hallmark of SCA and underlies pulmonary pathology. To date, no therapy has been shown to improve the pulmonary complications of SCA. Macrolides have pleomorphic effects in the lung with improvement in pulmonary function, symptoms and inflammatory markers demonstrated in several inflammatory pulmonary conditions such as cystic fibrosis, asthma, COPD and post-transplant bronchiolitis obliterans. Investigators hypothesize that low dose macrolide therapy is well tolerated and can improve pulmonary function and symptoms in patients with SCA. The objective of this project is to assess the feasibility of macrolides to attenuate or reverse the decrease in %predicted FEV1 in adults with SCA in a single-site, randomized, placebo-controlled feasibility trial. | Sickle Cell Disease | ALL | ADULT | Vanderbilt University Medical Center, Nashville, Tennessee, 37203, United States |
| A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE) | A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult Sickle Cell Disease patients with frequent vaso-occlusive crises. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | WCG Sonar Clinical Research, Riverdale, Georgia, 30274, United States |
| Angiotensin-converting Enzyme Inhibitors and Early Sickle Cell Renal Disease in Children | Patients with sickle cell anaemia may develop renal disease. In fact, renal disease occurred in 40% of adults patients (macroalbuminuria) with evolution to end-stage renal disease for half of them. Microalbuminuria is an early and sensitive marker of glomerular damage. It appears during the first decade and occurred in 20 to 25% of infants (2 to 18 years). Physiopathology of renal scarring is not well understood actually. Renal scarring might be due to glomerular hyperfiltration and vascular and endothelial damage. Angiotensin-converting enzyme inhibitors (ACE) were studied and used in diabetic nephropathy. In a study on 26 sickle cell adults, albuminuria was reduced about 50% by ACE compared to placebo after six months treatment. It might be interesting studying ACE efficacy in sickle cell children with microalbuminuria because renal disease is directly related to sickle cell and is not influenced by other cardiovascular risk factors like in adult patients. We hypothesized to have a successful ACE treatment in more than 40% of cases after a nine months treatment period. A success is defined as a 50% reduction of the albuminuria/creatinuria ratio. | Sickle Cell Disease | ALL | CHILD, ADULT | Trousseau Hospital, Nephro-pediatric unit, Paris, 75012, France |
| Adding Azathioprine/Hydroxyurea Preconditioning to Alemtuzumab/TBI to Reduce Risk of Graft Failure in MSD HSCT in Adult SCD Patients | In this study the investigators will prospectively investigate whether the addition of a 3-months long preconditioning with azathioprine to the alemtuzumab/TBI non-myeloablative conditioning results in improved disease-free survival and donor chimerism after allo-SCT in SCD patients. Furthermore, the investigators will evaluate whether azathioprine/hydroxyurea preconditioning leads to more patients being able to taper and discontinue sirolimus at 12 months post-transplantation. | Sickle Cell Disease | ALL | CHILD, ADULT | Amsterdam Medical Centre, Amsterdam, Netherlands |
| Pharmacokinetics and Pharmacodynamics of Rifaximin Novel Formulations in Patients With Sickle Cell Disease | This is a randomized, double-blind, placebo-controlled study in sickle cell disease participants with a history of Vaso-occlusive Crises (VOCs). Approximately 60 participants with sickle cell disease will be enrolled and randomized: 12 participants in each of four active novel formulation rifaximin groups and 6 participants in each of 2 placebo groups. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Bausch Site 105, Orange, California, 92868, United States|Bausch Site 103, Denver, Colorado, 80220, United States|Bausch Site 104, Atlanta, Georgia, 30329, United States|Bausch Site 101, Syracuse, New York, 13210, United States|Bausch Site 102, Greenville, North Carolina, 27834, United States|Bausch Site 201, Montréal, Quebec, H2X 3E4, Canada|Bausch Site 501, Eldoret, 30100, Kenya|Bausch Site 502, Kisumu, 40100, Kenya |
| Early Diagnosis of Sickle Acute Chest Syndrome Using a Combination of Plasma Bimarkers and Chest Imaging | Background: Painful vasoocclusive crisis (VOC) occurs in people with sickle cell disease (SCD). People with VOC have many visits to the hospital. About 10 30 percent of these people will go on to develop acute chest syndrome (ACS). ACS can cause further ill health. It can also cause death. Researchers want to find ways to diagnose ACS more quickly. To do this, they want to use stored blood samples and scans from a study (the DeNOVO trial) that was closed in 2015. They want to see if scans and samples taken of people with VOC who later developed ACS could help diagnose ACS faster. The data of people in the DeNOVO study who did not develop ACS will serve as controls. Objectives: To look at data from the DeNOVO trial to find a way to diagnose ACS more quickly. Eligibility: People 10 85 years old who took part in NHLBI Protocol number 05-H-0019 (the DeNOVO trial). The trial lasted from 2004 to 2008. The study was closed in November 2015. Design: Scans and intact, frozen samples from a study that was closed in 2015 will be studied. No new participants will be enrolled. ... | Sickle Cell Disease (SCD) | ALL | CHILD, ADULT, OLDER_ADULT | National Heart, Lung and Blood Institute (NHLBI), Bethesda, Maryland, 20892, United States |
| A Research Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease | This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can reduce the damage to different organs, improve your exercise tolerance and reduce fatigue in people with sickle cell disease.The participants will either get etavopivat or placebo. Which treatment the participants will get is decided by chance. Etavopivat is a new medicine and is currently being tested in other studies in addition to this one. The study will last for about 2 years. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Uni of Alabama at Birmingham, Birmingham, Alabama, 35233, United States|Univer South Alabama Ped/Onc, Mobile, Alabama, 36604, United States|Phoenix Children's Hsptl, Phoenix, Arizona, 85016, United States|Arkansas Children's Hospital, Little Rock, Arkansas, 72202, United States|Children's Hospital Los Angeles - Endocrinology, Los Angeles, California, 90027, United States|UCLA Health, Los Angeles, California, 90095, United States|Valley Children's Hospital, Madera, California, 93636, United States|University Of California Irvine, Orange, California, 92868, United States|Stanford University_Palo Alto, Palo Alto, California, 94304, United States|Harbor-UCLA Medical Center, Torrance, California, 90502, United States|Clinical and Transl Res Center, Aurora, Colorado, 80045, United States|Nemours/AI duPont Hosp-Chld, Wilmington, Delaware, 19803, United States|Childrens National Medical Ctr, Washington, District of Columbia, 20010, United States|MedStar Hlth Res Institute, Washington, District of Columbia, 20010, United States|Memorial Healthcare, Hollywood, Florida, 33021, United States|Children's Healthcare Atlanta, Atlanta, Georgia, 30329, United States|Childrens Hospital of Chicago, Chicago, Illinois, 60611, United States|Univer Of Illinois at Chicago, Chicago, Illinois, 60612, United States|Children's Hosp-New Orleans, New Orleans, Louisiana, 70118, United States|Boston Children's Hospital, Boston, Massachusetts, 02115, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|Henry Ford Hospital_Detroit, Detroit, Michigan, 48202, United States|University of Minnesota, Minneapolis, Minnesota, 55455, United States|Washington University-St.Louis, Saint Louis, Missouri, 63110, United States|Children's Nebraska, Omaha, Nebraska, 68114, United States|Cure 4 the Kids Foundation, Las Vegas, Nevada, 89135, United States|Newark Beth Israel Medical Center, Newark, New Jersey, 07112-2027, United States|Jacobi Medical Center, Bronx, New York, 10461, United States|Montefiore Medical Ctr, Bronx, New York, 10467, United States|NYC Health+Hospitals, Brooklyn, New York, 11203, United States|Interfaith Medical Center, Brooklyn, New York, 11238, United States|Northwell Health, Mount Kisco, New York, 10549, United States|Cohen Children's Medical Ctr, Queens, New York, 11040, United States|Duke University Medical Center, Durham, North Carolina, 27705, United States|Duke Comprehen Sickle Cell, Durham, North Carolina, 27710, United States|East Carolina University_Greenville_0, Greenville, North Carolina, 27834, United States|Atrium Health-Wake Forest Bapt, Winston-Salem, North Carolina, 27157, United States|Univ Hosp Cleveland Med Ctr, Cleveland, Ohio, 44106, United States|Ohio State Univ Wexner Med Ctr, Columbus, Ohio, 43210, United States|Univ of OK Health Sciences Ctr, Oklahoma City, Oklahoma, 73104, United States|Children's Hosptl Philadelphia, Philadelphia, Pennsylvania, 19104, United States|St Christopher Hosp for Child, Philadelphia, Pennsylvania, 19134, United States|Methodist University Hospital, Memphis, Tennessee, 38104, United States|St. Jude Children's Res Hosp, Memphis, Tennessee, 38105, United States|UT Health University of Texas, Houston, Texas, 77030, United States|Inova Health System, Fairfax, Virginia, 22031, United States|Children's Hsptl Of The Kings, Norfolk, Virginia, 23507, United States|Virginia Comm Univ Medical Ctr, Richmond, Virginia, 23298, United States|Mary Bridge Children's Health, Tacoma, Washington, 98405, United States|Royal Prince Alfred Hospital, Camperdown, New South Wales, 2050, Australia|Prince of Wales Hospital, Randwick, New South Wales, 2031, Australia|Monash Health, Clayton, Victoria, 3168, Australia|Fiona Stanley Hospital - Haemophilia and Haemostasis Centre, Murdoch, Western Australia, 6150, Australia|CHU Saint-Pierre - UMC Sint-Pieter, Bruxelles, 1000, Belgium|CHU - UVC Brugmann, Bruxelles, 1020, Belgium|HUB - Hôpital Erasme, Bruxelles, 1070, Belgium|UZ Antwerpen - UZA - Haematology, Edegem, 2650, Belgium|UZ Antwerpen - UZA - Kinderhemato-Oncologie, Edegem, 2650, Belgium|UZ Leuven - Kindergeneeskunde, Leuven, 3000, Belgium|MultiHemo - Grupo Oncoclínicas, Recife, Pernambuco, 52070-460, Brazil|HC da FMUSP Hospital das Clínicas São Paulo, São Paulo, SP, 05403-010, Brazil|Hemocentro UNICAMP, Distrito De Barão Geraldo - Campinas, São Paulo, 13083-878, Brazil|Fundação Hemocentro de Ribeirão Preto, Ribeirão Preto, São, 14051-140, Brazil|Hemorio-Fundarj, Rio de Janeiro, 20211-030, Brazil|Hospital Samaritano Higienópolis - Instituto de Conhecimento Ensino e Pesquisa (ICEP), São Paulo, 01232 -010, Brazil|Foothills Med Ctr-Univ Calgary, Calgary, Alberta, T2N 2T9, Canada|Stollery Children's Hospital, Edmonton, Alberta, T6G 2B7, Canada|University of Alberta_Edmonton, Edmonton, Alberta, T6G 2B7, Canada|BC Children's Hospital, Vancouver, British Columbia, V6H 3V4, Canada|LHSC - Victoria Hospital, London, Ontario, N6A5W9, Canada|UHN-Toronto General Hospital, Toronto, Ontario, M5G 2C4, Canada|University of Toronto, Toronto, Ontario, M5G1X8, Canada|CHUM-Hosp de Univ Montreal, Montreal, Quebec, H2X 0A9, Canada|Montreal Children's Hospital, Montreal, Quebec, H4A 3J1, Canada|Sociedad de Oncología y Hematología del Cesar SAS (SOHEC), Antioquia, 4006, Colombia|Clinica de la Costa, Barranquilla, 080020, Colombia|Centro de investigaciones clínicas, fudación valle del lili, Cali, 760032, Colombia|Hospital Pablo Tobon Uribe, Medellin, 050034, Colombia|Clínica IMAT Oncomedica Auna S.A.S., Monteria, 230002, Colombia|Ap-Hp-Hopital Avicenne, Bobigny, 93000, France|Centre Hospitalier Universitaire de Bordeaux-Hopital Pellegrin, Bordeaux, 33000, France|Ap-Hp-Hopital Henri Mondor, Créteil, 94000, France|Centre Hospitalier Universitaire Grenoble Alpes-Site Nord Michallon-1, Grenoble cedex 9, 38043, France|Hospices Civils de Lyon-Hopital Edouard Herriot, Lyon Cedex 03, 69437, France|Ap-Hp-Hopital Necker-1, Paris, 75015, France|Ap-Hp-Hopital Robert Debre, Paris, 75019, France|Centre Hospitalier Universitaire de Rouen-Hopital Charles Nicolle, Rouen cedex, 76031, France|Agogo Presbyterian Hospital, Ghana, Agogo, 0000, Ghana|Kintampo Health Research Centre (KHRC), Kintampo, Ghana, Kintampo, 200, Ghana|Navrongo Health Research Centre, Ghana, Navrongo, 00000, Ghana|"Laiko" General Hospital of Athens, Athens, 115 26, Greece|Childrens' Hospital of Athens "Agia Sofia", Athens, 115 27, Greece|Hippokration Hospital, Athens, 11527, Greece|University General Hospital Of Ioannina, Ioannina, 45500, Greece|"AHEPA" University General Hospital of Thessaloniki, Thessaloniki, 546 36, Greece|Gauhati Medical College and Hospital, Guwahati, Assam, 781032, India|All India Institute of Medical Sciences (AIIMS), Raipur, Raipur, Chhattisgarh, 492099, India|Zydus Medical College & Hospital, Dahod, Gujarat, 389151, India|Nirmal Hospital Pvt. Ltd., Surat, Gujarat, 395002, India|SSG Hospital, Baroda, Vadodara, Gujarat, 390001, India|HOCC Haemato Oncology Care Centre, Vadodara, Gujarat, 390020., India|JSS Hospital, Mysore, Mysuru, Karnataka, 570004, India|Government Medical College, Kozhikode, Kozhikode, Kerala, 673008, India|Arihant Multispeciality Hospital, Nagpur, Maharashtra, 440026, India|IMS and SUM Hospital, Bhubaneswar, Orissa, 751003, India|RNT Medical College, Udaipur, Rajasthan, 313001, India|NIZAM'S Institute of Medical Sciences, Hyderabad, Telangana, 500082, India|Post Graduate Institute of Child Health, Noida, Uttar Pradesh, 201303, India|All India Institute of Medical Sciences_New Dehli, New Delhi, 110029, India|Azienda Ospedaliera Universitaria San Luigi Gonzaga, Orbassano, Torino, 10043, Italy|ASST Civili di Brescia, Piazzale Spedali Civili 1, 25123 Brescia, Brescia, 25123, Italy|ARNAS Ospedale Garibaldi, Catania, 95123, Italy|A.O. Universitaria di Modena, Modena, 41100, Italy|Fondazione IRCCS San Gerardo dei Tintori, Monza, 20900, Italy|A.O.U. Università Studi della Campania "Luigi Vanvitelli", Naples, 80138, Italy|Azienda Ospedale Universita Padova, Padova, 35128, Italy|Azienda Ospedaliera Ospedali Riuniti Villa Sofia - Cervello, Palermo, 90146, Italy|Policlinico GB Rossi, Verona, 37134, Italy|Ahero Clinical Trials Unit, Kisumu, Kenya, Kisumu, 40100, Kenya|Gertrude's Children's Hospital, Nairobi, Nairobi, 00000, Kenya|KEMRI Siaya Clinical Research Annex Siaya County Referral Hospital, Siaya, Siaya, 0000, Kenya|International Cancer Institute (ICI), Uasin Gishu County, 0000, Kenya|Hospital Nini, Tripoli, 1434, Lebanon|Amsterdam UMC Lokatie VUMC, Amsterdam, 1081 HV, Netherlands|Haga Ziekenhuis, Den Haag, 2545AA, Netherlands|Erasmus MC, Rotterdam, 3015 GD, Netherlands|UMC Utrecht, Van Creveldkliniek, Utrecht, 3584 CX, Netherlands|University College Hospital Paediatric Haematology and Oncology Unit, Ibadan, Ibadan, Oyo State, 0000, Nigeria|Centre for Sickle Cell Disease Research and Training University of Abuja (CESRTA), Abuja, Abuja, 0000, Nigeria|University of Nigeria Teaching Hospital, Enugu, Enugu, 00000, Nigeria|Lagos University Teaching Hospital, Lagos, Lagos, 102215, Nigeria|The Royal Hospital, Muscat, 111, Oman|Sultan Qaboos University Hospital, Muscat, 123, Oman|King Abdulaziz Hospital-Al Ahsa-National Guard, Al Ahsa, 36428, Saudi Arabia|National Guard Hospital Dammam, Dhahran, 34232, Saudi Arabia|King Faisal Specialist Hospital & Research Centre, Riyadh, Riyadh, 12713, Saudi Arabia|Hospital Vall d'Hebron, Barcelona, 08035, Spain|Hospital Gregorio Marañón, Madrid, 28009, Spain|Hospital Universitario La Paz, Madrid, 28046, Spain|Hospital Universitario Virgen del Rocío, Sevilla, 41013, Spain|Acibadem Adana Hastanesi, Adana, 01130, Turkey|Baskent Universitesi Adana, Adana, 01250, Turkey|Cukurova University Medical Faculty, Adana, 01250, Turkey|Hacettepe University Hematology, Ankara, 06230, Turkey|Mersin University Medical Faculty Pediatric Hematology, Mersin, 33110, Turkey|VM Medical Park Mersin Hospital, Mersin, 33200, Turkey|Jinja Regional Referral Hospital Children's Ward, Jinja, 0000, Uganda|Makerere University College of Health Sciences, Kampala, 0000, Uganda|Joint Clinical Research Centre (JCRC), Kampala, 10005, Uganda|Addenbrooke's Hospital, Cambridge, CB2 0QQ, United Kingdom|Whittington Hospital, London, N19 5NF, United Kingdom|Guy's Hospital, London, SE1 9RT, United Kingdom|Kings College Hospital, London, SE5 9RS, United Kingdom|Imperial College London, London, W12 0NN, United Kingdom |
| Patient Characteristics and Treatment Patterns From Early Crizanlizumab Use in Real-world Setting: Preliminary Analysis From Select Sickle Cell Centers | This was a retrospective cohort study using secondary data from member sites of the National Alliance of Sickle Cell Centers (NASCC) with at least five patients who initiated crizanlizumab. Patients who were prescribed crizanlizumab were included in the cohort. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | University of Alabama Birmingham, Birmingham, Alabama, 35233, United States|Cristiana Care, Wilmington, Delaware, 19801, United States|All Children's Hospital, Saint Petersburg, Florida, 33701, United States|Johns Hopkins University, Baltimore, Maryland, 21205, United States|Washington University of St. Louis, Saint Louis, Missouri, 63110, United States|Beth Israel Hospital, East Hanover, New Jersey, 07936, United States|Montefiore, New York, New York, 10461, United States|University of North Carolina, Chapel Hill, North Carolina, 27514, United States|University Hospital at Cleveland Medical Center, Cleveland, Ohio, 44106, United States|Ohio State University, Columbus, Ohio, 43203, United States|University of Texas (Houston), Houston, Texas, 77030, United States |
| Pressure Pain Tolerance in Relation to Balance and Strength in Children | Sickle cell disease (SCD) is the most frequent life-threatening genetic hemoglobinopathy in the world and occurs due to the synthesis of abnormal hemoglobin S (HbS).Cells with sickle cell hemoglobin are stiff and sticky. When they lose their oxygen, they form into the shape of a sickle or crescent. This can cause pain and tissue damage. Significant decrease in exercise capacity was seen in sickle cell anemic children. In the absence of a guidelines that can guide the prescription of exercise in SCD children. This study is a step for determine the forms of prescription of pain on the balance and muscle strength to build up in future studies a safety of physical exercises for children with sickle cell anemia and improve their functional abilities. | Sickle Cell Anemia | ALL | CHILD | El-Tahrir st.- in front of Ben El-Sarayat, Ad Doqi Al Giza, Giza Governate, Giza, 002, Egypt |
| Pain Management in Children and Young Adults With Sickle Cell Disease | This is a phase II double-blind placebo-controlled clinical trial evaluating the effect of gabapentin when added to standard pain management for patients with sickle cell disease experiencing acute pain crisis in the ambulatory care setting. Sickle cell pain is different for every patient. Some patients get complete relief from routine pain medicines, and others need more time or more doses of pain medicines before the pain goes away completely. It is known that humans have many types of pain, including something called neuropathic pain. Neuropathic pain in other conditions (such as diabetes) has been treated successfully with a medicine called gabapentin. The investigators in this study suspect that some sickle cell pain is a combination of pain types. They would like to see if adding gabapentin to the usual pain medicines makes pain go away faster or more completely. Primary Objective: * To assess the analgesic efficacy of gabapentin vs. placebo for pain during vaso-occlusive crisis (VOC) in participants with sickle cell disease (SCD). A response to study drug will be defined by a decrease in pain score of ≥ 33% between presentation to the acute care setting and assessment at 3 hours post administration of study drug. Secondary Objective: * To compare the total morphine equivalent dose (mg/kg) used to control pain during VOC between presentation to the acute care setting and assessment at 3 hours post administration of study drug in the gabapentin vs. placebo groups. | Sickle Cell Disease | ALL | CHILD, ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Cooperative Study of The Clinical Course of Sickle Cell Disease | To determine the natural history of sickle cell disease from birth to death in order to identify those factors contributing to the morbidity and mortality of the disease. | Anemia, Sickle Cell|Blood Disease | ALL | CHILD, ADULT | |
| SALT: Alternative Donor Bone Marrow and Cord Blood Transplantation for High Risk Sickle Cell Disease | We hope to gain valuable information about the safety, success of engraftment, and rates of complications using alternate donor transplantation for children with severe SCD. Crucial information will be also collected about late effects from alternate donor BMT sickle cell, providing valuable information to clinicians and families making decisions among interventions for children with severe sickle cell disease. If successful, alternate donor transplantation in this setting could pave the way to offering curative treatment to many more patients with severe SCD. | Sickle Cell Disease | ALL | CHILD | Children's Healthcare of Altanta, Atlanta, Georgia, 30322, United States |
| A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy | The primary purpose of this study is to evaluate the effect of mitapivat on albumin creatinine ratio (ACR) response in participants with sickle cell disease (SCD) and nephropathy. | Sickle Cell Disease|Nephropathy | ALL | CHILD, ADULT, OLDER_ADULT | |
| Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis | The purpose of this study is to determine and confirm the role of bone marrow transplantation in the treatment of disorders of the red cell and hemoglobin including sickle cell anemia, thalassemia and diamond blackfan anemia. | Genetic Disorders|Sickle Cell Anemia | ALL | CHILD, ADULT, OLDER_ADULT | Memorial Sloan-Kettering Cancer Center, New York, New York, 10065, United States |
| Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease | This study consists of four parts, Parts A, B, C, and D. * Part A is a single dose pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease ages 6 to 17 years. * Part B is a multiple dose, safety, exploratory, efficacy, and PK study in adolescent participants with Sickle Cell Disease ages 12 to 17 years. * Part C is a multiple dose, safety, tolerability, and PK study, which includes the assessment of hematological effects and the effect on TCD flow velocity of voxelotor in pediatric participants with Sickle Cell Disease ages 4 to 17 years. * Part D is a multiple dose, safety, tolerability, and PK study, which examines the hematological effects of voxelotor in pediatric participants with Sickle Cell Disease ages 6 months to \< 4 years. | Sickle Cell Disease | ALL | CHILD | Brentwood Clinic UCSF Benioff Children's Hospital Oakland, Brentwood, California, 94513, United States|UCSF Benioff Children's Hospital Oakland, Oakland, California, 94609, United States|UCSF Benioff Children's Hospital Walnut Creek, Walnut Creek, California, 94598, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States|Emory Children's Center, Atlanta, Georgia, 30322, United States|Children's Healthcare of Atlanta Scottish Rite, Atlanta, Georgia, 30342, United States|Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States|University of Illinois at Chicago Clinical Research Center, Chicago, Illinois, 60612, United States|University of Illinois Hospital and Health Sciences System, Chicago, Illinois, 60612, United States|Our Lady of the Lake Children's Hospital (IP Address), Baton Rouge, Louisiana, 70809, United States|St. Jude Affiliate Clinic at Our Lady of the Lake Children's Health, Baton Rouge, Louisiana, 70809, United States|Children's Mercy Hospital, Kansas City, Missouri, 64108, United States|Robert Wood Johnson University Hospital, New Brunswick, New Jersey, 08901, United States|Rutgers-Robert Wood Johnson Medical School, New Brunswick, New Jersey, 08901, United States|Rutgers-Robert Wood Johnson Medical School, New Brunswick, New Jersey, 08903, United States|Brody School of Medicine at East Carolina University, Greenville, North Carolina, 27834, United States|ECU Physicians, Greenville, North Carolina, 27834, United States|University Hospitals Cleveland Medical Center, Rainbow Babies & Children's Hospital, Cleveland, Ohio, 44106, United States|The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, 15224, United States|St. Jude Children's Research Hospital - Pharmaceutical Services, Memphis, Tennessee, 38105, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|American University of Beirut - Medical Center, Beirut, Lebanon|Rafik Hariri University Hospital, Beirut, Lebanon|Nini Hospital, Tripoli, Lebanon|University College London Hospital, NHS Foundation Trust, London, Greater London, NW1 2PG, United Kingdom|Barts Health NHS Trust, The Royal London Hospital, London, E1 1BB, United Kingdom|Guy's and St Thoma's NHS Foundation Trust, Evelina London Children's Hospital, London, SE1 7EH, United Kingdom|Manchester University NHS Foundation Trust, Royal Manchester Children's Hospital, Manchester, M13 9WL, United Kingdom |
| Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease | The purpose of this study is to determine whether giving extra arginine to patients with sickle cell disease seeking treatment for vaso-occlusive painful events (VOE) will decrease pain scores, decrease need for pain medications or decrease length of hospital stay or emergency department visit. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's Healthcare fo Atlanta at Hughes Spalding, Atlanta, Georgia, 30303, United States|Children's Healthcare of Atlanta at Egleston, Atlanta, Georgia, 30322, United States |
| Sulfadoxine- Pyrimethamine Versus Weekly Chloroquine for Malaria Prevention in Children With Sickle Cell Anemia | Malaria is fatal and increases the risk of death among children with sickle cell anemia. Chemoprophylaxis significantly improves quality of life in these children. In Uganda Chloroquine is the drug of choice for prophylaxis and yet it's effectiveness is limited due to high levels of resistance throughout the country. Intermittent presumptive treatment with sulfadoxine - Pyrimethamine a new approach to malaria prevention, has shown great potential in reducing incidence of malaria and anaemia among high risk groups such as pregnant women and infants. However no studies have been done in Uganda to determine if presumptive treatment with sulfadoxine- pyrimethamine reduces the incidence of malaria in children with sickle cell anaemia. Hypothesis : Presumptive treatment with sulfadoxine- Pyrimethamine is better than weekly chloroquine in reducing incidence of malaria in children with sickle cell anaemia. | Sickle Cell Anemia|Malaria | ALL | CHILD | Mulago Hospital, Kampala, Central, 256, Uganda |
| Efficacy of Antioxidant Therapy Compared With Enalapril in Sickle Nephropathy | The purpose of this study is to determine whether enalapril or antioxidant therapy (N-Acetylcysteine) is effective in reducing microalbuminuria in children with sickle cell disease and and its progression to sickle nephropathy | Microalbuminuria|Sickle Cell Nephropathy | ALL | CHILD, ADULT | Sickle Cell Unit, Kingston, Jamaica |
| SMART Mobile Application Technology Utilization in the Treatment of Sickle Cell Disease Post Day Hospital Discharge | The purpose of this study is to test a web-aided, mobile-based PHR (Personal Health Reporting) service to enhance SCD outpatient treatment after discharge from an acute care setting, such as Duke University Medical Center's Day Hospital. SMART is a new mobile application created by SickleSoft to increase patient involvement in their treatment and improve patient to doctor communication. SMART is a self-monitoring and management service for SCD patients and their treatment doctors. This study will test whether or not use of the SMART mobile application will help develop the type of patient-doctor relationships that lead to better health outcomes and a decrease in readmission to an acute care facility. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Duke University Medical Center, Durham, North Carolina, 27710, United States |
| Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies | This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD) grafts using the CliniMACS device in patients with sickle cell disease (SCD) and beta thalassemia major (BTM). | Sickle Cell Disease|Thalassemia Major | ALL | CHILD, ADULT | Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States |
| Optimizing the Management of Sickle Cell Patients on Hydroxyurea: The Value of Therapeutic Pharmacological Monitoring | Brief Summary: \* A short description of the clinical study, including a brief statement of the clinical study's hypothesis, written in language intended for the lay public. Limit: 5000 characters. Severe forms of sickle cell syndrome are characterized by the occurrence of repeated vaso-occlusive crises (CVO), early complications and a high morbidity and mortality in these patients. Intensified management is then required, with the introduction of hydroxyurea treatment and then, if it proves ineffective, a transfusion program or even a haematopoietic stem cell allograft. These latter treatments present significant risks of adverse effects for the patient (haemochromatosis, erythrocyte alloimmunisation for the transfusion program, risk of GVH, chemotherapy-related toxicity, MVO for the allograft). Hydroxyurea (HU) is the first treatment based on the specific pathophysiology of sickle cell disease. It is the first line of therapeutic intensification for adult patients and children (age ≥ 2 years) with major sickle cell disease. By mainly increasing the percentage of fetal haemoglobin (HbF), HU decreases the frequency of CVO, complications, hospitalizations and prolongs the life expectancy of patients. The initial dose of HU, recommended by the ANSM, is 15 mg/kg/d once daily. However, the optimal dose cannot be predicted at the start of treatment, which is why a dosage adjustment is essential. The usual dose is between 15 and 35 mg/kg per day. Typically, the dose is increased every 3 months until a mild myelosuppression tolerated by the patient is reached, indicating that the maximum tolerated dose (MTD) has been reached. When the dose of HU has reached the MTD, the ratio of clinical (reduced frequency of vaso-occlusive attacks) and biological (better % of HbF) benefits to risk (toxicity) is optimal for the patient. The disadvantages of this practice are that: * dose escalation can be long (9-12 months) * clinicians may be reluctant to escalate HU to MTD * patients are treated sub-optimally during the therapeutic adaptation period. Recent work has shown that it is beneficial for the patient to adjust the initial dose using a pharmacological therapeutic approach in addition to monitoring haematological tolerance. Thus, by customizing the dose of HU using an area under the curve (AUC) measurement at the initial intake of HU at a standardized dose (20 mg/kg/day), the MTD would be achieved in a faster time frame of 6-9 months. The primary objective of our trial is to identify the methodology that will most effectively decrease the time to reach the MTD (therapeutic target). The immediate benefit will be a reduction in CVO which is the major clinical problem and leads to a risk of complications in sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT | Strasbourg University Hospital, Strasbourg, 67091, France |
| Treatment of Adult Patients With Hemoglobin SC Disease (SCYTHE) | Sickle cell disease (SCD), specifically hemoglobin SC disease (HbSC), is a subtype of sickle cell disease with typically higher hemoglobin and milder or later disease complications. Sickle cell disease is a disorder in which red blood cells (RBCs) are abnormally shaped. This can result in painful episodes, serious infections, and damage to body organs. One medication used to treat sickle cell disease is hydroxyurea. Hydroxyurea therapy offers significant benefits for infants, children, and adolescents with sickle cell anemia. These include a reduction in the frequency of pain crises and acute chest syndrome (inflammation of the lungs). Hydroxyurea has been given to many HbSC patients but HbSC patients were not included in the large clinical trials used to test hydroxyurea in SCD, so less is known about how HbSC patients respond to hydroxyurea. The purpose of this research study is to see if hydroxyurea, a medication given to many patients with the most common type of sickle cell, those who are homozygous for the sickle mutation (HbSS), helps individuals who have HbSC. The investigators will see if it helps by giving a questionaire when the medication is started, and then every two months at a clinic visit. The questionaire, called the AdultsQLTM 3.0 Sickle Cell Disease Module, measures quality of life. The investigators will also see how hydroxyurea changes laboratory test numbers, and blood thickness. | Hemoglobin SC Disease | ALL | ADULT, OLDER_ADULT | Baylor College of Medicine, Houston, Texas, 77030, United States|University of Texas Houston, Houston, Texas, 77030, United States |
| Integration of mHEALTH Into the Care of Patients With Sickle Cell Disease to Increase Hydroxyurea Utilization | This project proposes to develop, test and evaluate targeted interventions to improve clinical provider prescribing of and patient adherence to hydroxyurea (HU). Using a stepped-wedge design, The investigators will test two innovative interventions utilizing mobile health to address both patients' and providers' needs: 1) an mHealth application for patients (InCharge Health app) that includes multi-component features to address the memory, motivation, and knowledge barriers to hydroxyurea use, and 2) an mHealth toolbox application for providers (HU Toolbox app) that addresses clinical knowledge barriers in prescribing and monitoring hydroxyurea use. These two interventions will be tested through the following aims: Aim 1. Improve Patient Adherence to Hydroxyurea: Addressing Memory, Motivation, and Knowledge Barriers to Hydroxyurea Use. Primary hypothesis: The investigators hypothesize that among adolescents and adults with SCD, the adherence to hydroxyurea, as measured by percentage of days covered (PDC), will increase by at least 20% at 24 weeks after receiving the InCharge Health app, compared to their hydroxyurea adherence at baseline. Sub-aim 1.a. To examine and assess both patient engagement and behaviors related to use of the InCharge Health app, the investigators will evaluate consistent use of the app among enrolled patients, patient satisfaction, and continued use of the app beyond the study period. Sub-Aim 1.b. To examine the clinical influence of the use of the InCharge Health app on PDC, patients' clinical outcomes, perceived health literacy, health related quality of life, and perceived self-efficacy between baseline and 24 weeks. Aim 2. Improve Provider Hydroxyurea Awareness, Prescribing and Monitoring Behaviors. Sub-Aim 2.a. To examine and assess provider engagement and behaviors related to use of the HU Toolbox, the investigators will evaluate consistent use of the app among enrolled providers, providers' satisfaction, and continued use of the app beyond the study period. Sub-Aim 2.b. To assess the combined effects of the patient and provider mHealth interventions on hydroxyurea and health care utilization, the investigators will examine if the changes in hydroxyurea adherence are enhanced by the use of both provider and patient interventions compared to those not exposed to one or both interventions. Aim 3. Identify and Evaluate the Barriers and Facilitators to the use of mHealth Interventions. | Sickle Cell Disease | ALL | CHILD, ADULT | Georgia Regents University, Augusta, Georgia, 30912, United States|University of Illinois, Chicago, Illinois, 60612, United States|Washington University, Saint Louis, Missouri, 63110, United States|Icahn School of Medicine at Mount Sinai, New York, New York, 10029, United States|Duke University, Durham, North Carolina, 27710, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Survey in a Population of Sickle Cell Disease Patients to Evaluate the Transition Between the Queen Fabiola Children Hospital and the CHU Brugmann Hospital, and the Quality of the Hospital Care Within the CHU Brugmann Hospital. | Sickle cell disease is a genetic disease responsible for an abnormal hemoglobin.The anomaly has several consequences: a low hemoglobin rate (chronic anemia), plugs formed by red blood cells in blood vessels (extremely painful vaso-occlusive crises) and greater susceptibility to infections. Patients with this disease should be monitored medically continuously from birth. At adulthood, they will pass from a pediatric medical care system to an adult medical care system.This transition can be experienced with more or less ease, depending on the organization within the pediatric and adult hospitals. This questionnaire aims to assess the quality of the transition between pediatric and adult services.The investigators want to better estimate hospital work and improve the quality of care for this type of patients, throughout their entire medical history. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | CHU Brugmann, Brussels, 1020, Belgium |
| Zinc Supplementation in Sickle Cell Disease: A Precursor to the Think Zinc for Bones Trial | The goal of this short term prospective Phase II study is to compare the effects of two alternate daily doses of zinc (25 and 40 mg/day) in 34 randomly assigned homozygous Sickle Cell Disease (SCD-SS) patients aged 15-35 years old. The main question it aims to answer is: Which biomarkers are most responsive to zinc supplementation, and what is the maximum tolerated zinc dose that induces the desired changes in biomarkers of bone turnover? Participants will be recruited from 7 American Society Hematology Research Collaborative SCD Centers. Eligible SCD subjects will be invited to participate in the 16-week study, involving 2 baseline blood draws 4 weeks apart, followed by a 12-week zinc intervention. The findings from this study will be used to determine the dosage of zinc to be used in a larger, future study on the long term impact of zinc supplementation on bone health in SCD-SS. | Sickle Cell Disease | ALL | CHILD, ADULT | UCSF Benioff Children's Hospital Oakland, Oakland, California, 94609, United States |
| Study of Clotrimazole and Hydroxyurea in Patients With Sickle Cell Syndromes | OBJECTIVES: Determine the effectiveness of the combined use of clotrimazole and hydroxyurea on a specific panel of red cell characteristics in patients with sickle cell syndromes. | Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | Brigham and Women's Hospital, Boston, Massachusetts, 02115, United States|Children's Hospital - Boston, Boston, Massachusetts, 02115, United States |
| Efficacy of Designed Exercise Program on Pain and Quality of Sleeping in Patient With Sickle Cell Disease Anemia. | Background: Pain and sleep disturbance are the most common problems experienced by adult patients with sickle cell disease anemia. Aim: the aim of this study is to evaluate the efficacy of designed exercise program on pain, and quality of sleeping in adult patients with sickle cell disease anemia and how the program affects their quality of lives. Subjects and methods: Adults patients with sickle cell diseases aging over 18 years old. Data will be collected in face-to-face interviews. Eligible participants will be equally and randomized into two groups. Group-1: Twenty-five adult patients with SCD will receive a designed exercise program of physical therapy for relief pain and improve sleep quality (experimental group). The designed exercise program will be distributed on everyone. The recommendations will be to train from 30 to 45 minutes, three days per week for 6 weeks in addition to walking daily 30 minutes on the ground surface. Group-2: Twenty-five adult patients with SCD will participate as a control group they will not receive exercise program. Analysis: The collected data will be managed by using t -test and the repeated measures of ANOVA test to compare the significance within groups and between two groups. | Sickle Cell Disease | ALL | ADULT | IAU, Dammam, Estern, Saudi Arabia |
| A Pilot Study of Azithromycin Prophylaxis for Acute Chest Syndrome in Sickle Cell Disease | Acute chest syndrome (ACS), a lung complication in sickle cell disease (SCD), is the second most common cause of hospitalization and leading cause of death in SCD. ACS is associated with airway inflammation, and a major cause is pulmonary infection from atypical organisms. To date, there are no drugs available to reduce inflammation and risk of recurrent ACS. Macrolides are a group of antibiotics that exert immunomodulatory and anti-inflammatory actions both in vitro and in vivo. In addition, macrolides reduce bacterial burden in the airway of atypical organisms, all of which play an important role in the pathophysiology of ACS. Numerous studies have evaluated macrolide prophylaxis in conditions associated with lung inflammation, such as cystic fibrosis, asthma, bronchiectasis etc., and high quality evidence have found macrolides to be beneficial as a disease modifying agent that leads to improvement in airway inflammation, reduced pulmonary exacerbations and improved lung function. The investigators hypothesize that azithromycin prophylaxis is well tolerated and has the potential to reduce inflammation and improve lung outcome in children with SCD with a history of ACS. A prospective, single arm, open label feasibility study of azithromycin prophylaxis will be performed in children with SCD with a history ACS with the specific aim to examine the feasibility, safety and tolerability of azithromycin prophylaxis administration in participants with SCD , and to examine whether azithromycin prophylaxis has the potential to improve lung outcome. In addition, this study will determine whether azithromycin prophylaxis reduces inflammation in participants with SCD with a history of ACS. | Sickle Cell Disease|Acute Chest Syndrome | ALL | CHILD | University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States|Vanderbilt University, Nashville, Tennessee, United States |
| Safety of ICL670 vs. Deferoxamine in Sickle Cell Disease Patients With Iron Overload Due to Blood Transfusions | The purpose of this study is to determine if the new orally active iron chelator, ICL670, is as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood transfusions. | Anemia, Sickle Cell | ALL | CHILD, ADULT, OLDER_ADULT | U. of S. Alabama Medical Center, Mobile, Alabama, 36604, United States|Loma Linda University Medical Center, Loma Linda, California, 92354, United States|Children's Hospital Los Angeles, Los Angeles, California, 90027, United States|Children's Hospital & Research Center, Oakland, California, 94609, United States|Colorado Sickle Cell Treatment and Research Center, Denver, Colorado, 80262, United States|Howard University Hospital, Washington, D.C., District of Columbia, 20059, United States|Tampa Children's Hospital at St Joseph's, Tampa, Florida, 33607, United States|Georgia Comprehensive Sickle cell Center, Grady Hospital, Atlanta, Georgia, 30335, United States|Adult Sickle Cell Clinic, Medical College of Georgia, Augusta, Georgia, 30912, United States|University of Illinois at Chicago, Chicago, Illinois, 60612, United States|Children's Memorial Hospital, Chicago, Illinois, 60614, United States|Tulane University Sickle Cell Center, New Orleans, Louisiana, 70112, United States|Children's Hospital, Department of Hematology/Oncology, New Orleans, Louisiana, 70118, United States|Children's Hospital Boston, Division of Hematology/Oncology, Boston, Massachusetts, 02115, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|Karmanos Cancer Institute, Detroit, Michigan, 48201, United States|NY Methodist Hospital, Brooklyn, New York, 11215, United States|Weill Medical College of Cornell University, New York, New York, 10021, United States|U. Of Rochester Medical Center, Rochester, New York, 14642, United States|Sickle Cell Center, Montefiore Hospital, The Bronx, New York, 10467, United States|Wake Forest University School of Medicine, Winston-Salem, North Carolina, 27106, United States|Barrett Center, University of Cincinnati, Cincinnati, Ohio, 45219, United States|Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States|James Cancer Hospital, Columbus, Ohio, 43210, United States|Penn State Milton S Hershey Medical Center, Hershey, Pennsylvania, 17033, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, 15213, United States|Liberty Hematology Oncology Center, Columbia, South Carolina, 29203, United States|Palmetto Health Clinical Trials, Columbia, South Carolina, 29203, United States|Santee Hematology/Oncology, Sumter, South Carolina, 29150, United States|Baylor College of Medicine, Houston, Texas, 77030, United States|Texas Children's Hospital/Baylor College of Medicine, Houston, Texas, 77030, United States|Scott and White Memorial Hospital & Clinics, Temple, Texas, 76508, United States|Children's Hospital of the King's Daughter, Norfolk, Virginia, 23507, United States |
| Pharmacokinetics and Safety of Endari (L-glutamine) in Sickle Cell Disease Patients | L-glutamine has been approved in the US to reduce the acute complications of sickle cell disease (SCD) in adult and pediatric patients 5 years of age and older. The purpose of this single-center, open-label, phase 4 study is to evaluate the pharmacokinetic characteristics and safety of L-glutamine in patients with SCD. | Sickle Cell Disease|Pharmacokinetics | ALL | CHILD, ADULT, OLDER_ADULT | Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States |
| High Dose Vitamin D Supplementation in Children With Sickle Cell Disease | Suboptimal vitamin D status is well reported in sickle cell disease (SCD) patients and associated with a negative impact on health-related quality of life (HRQL). The investigators enrolled 42 SCD patients and 42 healthy controls, subjects within each group received monthly oral vitamin D3 dose according to the baseline status of vitamin D as follows: sufficient: 100,000 IU, insufficient: 150,000 IU, and deficient: 200,000 IU. The investigators assessed safety and efficacy on normalization of vitamin D level, bone mineral density (BMD), hand grip strength (HGS), and HRQL. | Sickle Cell Disease|Vitamin D Deficiency|Health Related Quality of Life|Hand Grip Strength|Bone Mineral Density | ALL | CHILD, ADULT | Zagazig university, Zagazig, Sharkia, 44519, Egypt |
| Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels | Background: Sickle cell disease can often be treated with blood stem cell transplants. But for some people the disease returns. This study will give a second transplant to people whose disease has returned but still have some donor cells in their body. Objective: To cure people s sickle cell disease by giving a second treatment that makes more room in their bone marrow for donor cells. Eligibility: People ages 4 and older with sickle cell disease who had a transplant but the disease returned, and their donor relatives. Donors can be 2 years of age or older. Design: Participants will be screened with medical history, physical exam, and blood tests. Recipients will also be screened with heart and breathing tests, x-rays, a bone marrow sample, and teeth and eye exams. They must have a caregiver. Donors will have 7-8 visits. They will take a drug for 5-6 days to prepare them for the donation. For the donation, blood is taken from a vein in the arm or groin. The stem cells are collected. The rest of the blood is returned. This may be repeated. Recipients will get a long IV line in their arm or chest for about 1-2 months. They will take drugs to help their body accept the donor cells. They will get the donor cells and red blood cell transfusions through the line. They will stay in the hospital about 30 days after the transfusion of donor cells. In first 3 months after the infusion, recipients will have many visits. Then they will have visits every 6 months to 1 year for 5 years. During those visits they will repeat some of the screening tests.... | Myeloid Chimerism | ALL | CHILD, ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| A New Reagent Assay Examining Natural Parvovirus B19 Infection in Sickle Cell Disease | Parvovirus B19 is a small virus that is the cause of "fifth" disease, a common infection in childhood. In people with sickle cell disease (SCD), parvovirus B19 infection causes the bone marrow to stop producing red blood cells temporarily, which can be life-threatening. A novel vaccine is currently in development for children with SCD. This study is the first step within a larger parvovirus B19 multi-institutional project that will help develop this new vaccine, as it will define the value and utility of using a novel assay for measurement of parvovirus-specific antibodies. The main objective is to investigate the relationship between the newly developed VP1u ELISA assay and the gold standard neutralization assay for parvovirus B19 infection. The most accurate test, called a neutralizing antibody assay, to see if a person has had or currently has the infection is very complex and expensive and would be very difficult to use in a large research study to test the new vaccine. A new and simpler test has developed. The main goal of this study, iSCREEN, is to find out if this new test works. There will be distinct labs performing the VP1u ELISA and the neutralization assays and the respective laboratories will not have access to each other's results for individual subjects. The VP1u ELISA will be performed at St. Jude Children's Research Hospital. Neutralization assays will be conducted at the National Heart, Lung and Blood Institute. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE) | A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Birmingham, Alabama, 35205, United States|Mobile, Alabama, 36693, United States|Little Rock, Arkansas, 72204, United States|Oakland, California, 94609, United States|Miami, Florida, 33136, United States|Atlanta, Georgia, 30342, United States|Chicago, Illinois, 60612, United States|Indianapolis, Indiana, 46260, United States|Baton Rouge, Louisiana, 70808, United States|New Orleans, Louisiana, 70112, United States|Baltimore, Maryland, 21287, United States|Bethesda, Maryland, 20817, United States|Boston, Massachusetts, 02115, United States|Boston, Massachusetts, 02118, United States|Detroit, Michigan, 48201, United States|Newark, New Jersey, 07112, United States|Bronx, New York, 11501, United States|New York, New York, 10032, United States|Chapel Hill, North Carolina, 27514, United States|Durham, North Carolina, 27710, United States|Greenville, North Carolina, 27834, United States|Oklahoma City, Oklahoma, 73112, United States|Philadelphia, Pennsylvania, 19107, United States|Pittsburgh, Pennsylvania, 15219, United States|Charleston, South Carolina, 29425, United States|Memphis, Tennessee, 38105, United States|Nashville, Tennessee, 37232, United States|Houston, Texas, 77030, United States|Richmond, Virginia, 23298, United States|Toronto, M5G 2C4, Canada|Alexandria, 21131, Egypt|Cairo, 11566, Egypt|Cairo, Egypt|Zagazig, 44519, Egypt|Créteil, 94010, France|Paris, 75743, France|Paris, 75908, France|Monza, Milano, 20900, Italy|Padova, 35128, Italy|Verona, 37134, Italy|Kingston, JMAAW15, Jamaica|Nairobi, 42325-00100, Kenya|Nairobi, 47855, Kenya|Nairobi, 59857-00200, Kenya|Siaya, 144-40600, Kenya|Beirut, 11072020, Lebanon|Beirut, 1136044, Lebanon|Tripoli, 1434, Lebanon|Amsterdam, 1105 AZ, Netherlands|Den Haag, 2545 CH, Netherlands|Rotterdam, 3015 AA, Netherlands|Muscat, 123, Oman|Adana, 01130, Turkey|Kayseri, 38039, Turkey|Mersin, 33342, Turkey|London, E11BB, United Kingdom|London, E96SR, United Kingdom|London, SE17EH, United Kingdom|London, SE59NU, United Kingdom|London, W12 0HS, United Kingdom|London, WC1N3BG, United Kingdom|Manchester, M13 9WL, United Kingdom |
| A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises | This Phase 3 study will assess the safety and efficacy of inclacumab, a P-selectin inhibitor, in reducing the frequency of vaso-occlusive crises (VOCs) in approximately 240 adult and adolescent participants (≥ 12 years of age) with sickle cell disease (SCD). Participants will be randomized to receive inclacumab or placebo. | Sickle Cell Disease|Vaso-occlusive Pain Episode in Sickle Cell Disease|Vaso-occlusive Crisis | ALL | CHILD, ADULT, OLDER_ADULT | University of South Alabama Children's and Women's Hospital, Mobile, Alabama, 36604, United States|University of South Alabama Mitchell Cancer Institute, Mobile, Alabama, 36604, United States|University of South Alabama Strada Patient Care Center, Mobile, Alabama, 36604, United States|Phoenix Children's Hospital, Phoenix, Arizona, 85016, United States|Arkansas Children's Hospital, Little Rock, Arkansas, 72202, United States|UCSF Benioff Children's Hospital, Oakland, Oakland, California, 94609, United States|UC Irvine Health, Orange, California, 92868-3201, United States|UCI Center for clinical research, Orange, California, 92868, United States|Uconn Health/Uconn John Dempsey Hospital/Neag Comprehensive Cancer Center/New England Sickle Cell, Farmington, Connecticut, 06030-1163, United States|University of South Florida, Tampa, Florida, 33606, United States|USF Health South Tampa Center for Advanced Healthcare, Tampa, Florida, 33606, United States|John S. Curran, MD.,Children's Health Center, Tampa, Florida, 33612, United States|USF Clinical Investigational Research Pharmacy, Tampa, Florida, 33612, United States|USF Health Carol & Frank Morsani Center for Advanced Healthcare, Tampa, Florida, 33612, United States|Children's Healthcare of Atlanta - Egleston, Atlanta, Georgia, 30322, United States|Children's Healthcare of Atlanta - Scottish Rite, Atlanta, Georgia, 30342, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30342, United States|Hospital Pharmacy Services- Investigational Drug Services, Chicago, Illinois, 60612-4333, United States|Rush University Medical Center Investigator Pharmacy, Chicago, Illinois, 60612, United States|Rush University Medical Center, Chicago, Illinois, 60612, United States|University of Illinois Clinical Research Center (CRC), Chicago, Illinois, 60612, United States|University of Illinois Hospital and Health Sciences System(UI Health), Chicago, Illinois, 60612, United States|Dana-Farber Cancer Institute IDS Pharmacy, Boston, Massachusetts, 02215, United States|Dana-Farber Cancer Institute, Boston, Massachusetts, 02215, United States|University of Michigan Hospitals - Michigan Medicine, Ann Arbor, Michigan, 48109, United States|Jacobi Medical Center, Bronx, New York, 10461, United States|Erie County Medical Center, Buffalo, New York, 14215, United States|Duke University Medical Center, Durham, North Carolina, 27705, United States|DUMC Investigational Drug Services Pharmacy, Durham, North Carolina, 27710, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|UT Physicians Comprehensive Sickle Cell Clinic, Houston, Texas, 77004, United States|McGovern Medical School/Health Science Center Houston, Houston, Texas, 77030, United States|Memorial Hermann - TMC Investigational Drugs, IDS Pharmacy, Houston, Texas, 77030, United States|Memorial Hermann Hospital, Texas Medical Center - Clinical Research Unit (CRU), Houston, Texas, 77030, United States|UT Physicians Comprehensive Sickle Cell Clinic, Houston, Texas, 77030, United States|Instituto D'Or de Pesquisa e Ensino - Hospital São Rafael, Salvador, Bahia, 41253-190, Brazil|Hospital das Clinicas da Universidade Federal de Minas Gerais, Belo Horizonte, MG, 30130-100, Brazil|Hemocentro de Belo Horizonte - Fundacao Hemominas, Belo Horizonte, MG, 30130-110, Brazil|Hospital das Clinicas da Universidade Federal de Minas Gerais, Belo Horizonte, Minas Gerais, 30130-100, Brazil|Multihemo Serviços Médicos S/A, Recife, Pernambuco, 50070-460, Brazil|Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, 90035-903, Brazil|Fundação Faculdade Regional de Medicina de São José do Rio Preto, Sao Jose do Rio Preto, SAO Paulo, 15090-000, Brazil|Comite de Etica em Pesquisa - CEP do Hospital Alemao Oswaldo Cruz/ SP, Sao Paulo, SP, 01323-903, Brazil|Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP, Ribeirão Preto, SÃO Paulo, 14051-140, Brazil|Instituto Estadual de Hematologia Arthur Siqueira Cavalcanti - HEMORIO, Rio de Janeiro, 20211-030, Brazil|Hospital Samaritano Higienópolis/Esho Empresa De Servicos Hospitalares S.A, São Paulo, 01232-010, Brazil|Hospital das Clinicas da Faculdade de Medicina da Universidade de São Paulo-HCFMUSP, São Paulo, 05403-000, Brazil|Casa de Saúde Santa Marcelina, São Paulo, 08270-070, Brazil|CEPEC-Centro de Pesquisa Clinica, São Paulo, 08270-120, Brazil|Clinica de la Costa Ltda., Barranquilla, Atlantico, 080020, Colombia|Organizacion Clinica Bonnadona Prevenir S.A.S., Barranquilla, Atlántico, 080020, Colombia|Sociedad de Oncologia y hematologia del Cesar S.A.S., Valledupar, Cesar, 200001, Colombia|Zagazig University Hospital, Zagazig, ASH Sharqia, 44519, Egypt|Al Kasr Al Ainy Cairo University Hospital, Elmanial, Cairo Governorate, 11562, Egypt|Cairo University Paediatric Hospital - abou el Reesh University Hospital, El-Rashidy Street, ElSayeda Zeinb, Cairo, 11617, Egypt|Faculty of Medicine Cairo University, Cairo, 11562, Egypt|AinShams University Hospital, Cairo, 11588, Egypt|Hôpital Avicenne, Bobigny, 93000, France|Hôpital Henri Mondor, Créteil, 94010, France|Institut Universitaire du Cancer de Toulouse-Oncopole, Toulouse Cedex 9, 31059, France|Universitätsklinikum Regensburg Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation, Regensburg, 93053, Germany|Sickle-Cell Office, Directorate of Child Health, Komfo Anokye Teaching Hospital, Kumasi, Ashanti, Ghana|Farmacia Interna Azienda Ospedaliero-Ente Ospedaliero Ospedali Galliera, Genova, 16128, Italy|S.S.D. Microcitemia, anemie congenite e dismetabolismo del ferro Azienda, Genova, 16128, Italy|DAI Materno-Infantile, UOC Clinica Pediatrica 1 Azienda Ospedaliera Universitaria, Napoli, 80138, Italy|DAI Materno-Infantile,- UOC Clinica Pediatrica 1 Azienda Ospedaliera Universitaria "Luigi Vanvitel, Napoli, 80138, Italy|UO di Farmacia Clinica, Dipartimento di Medicina Sperimentale Azienda Ospedaliera Universitaria, Napoli, 80138, Italy|UOC Patologia Clinica e Molecolare Azienda Ospedaliera Universitaria "Luigi Vanvitelli", Napoli, 80138, Italy|UOC Radiologia Azienda Ospedaliera Universitaria "Luigi Vanvitelli", Napoli, 80138, Italy|Dipartimento Strutturale Aziendale Salute della Donna e del Bambino Clinica Ginecologica, Padova, 35128, Italy|Farmacia Azienda Ospedale Universita Padova, Padova, 35128, Italy|U.O.C. Farmacia Istituto Oncologico Veneto, Padova, 35128, Italy|KEMRI/CRDR, Siaya, KEMRI Clinical Research Annex, Kisumu, Siaya, 40600, Kenya|International Cancer Institute, Eldoret, 30100, Kenya|Gertrude's Children Hospital, Nairobi, 00100, Kenya|Kenya Medical Research Institute- Center for Respiratory Disease Research, Nairobi, 00100, Kenya|Strathmore University Medical Center - Center for Research in Therapeutic Sciences(CREATES), Nairobi, 00200, Kenya|American University of Beirut Medical Center, Hamra, Beirut, Lebanon|Nini Hospital, Tripoli, North Lebanon, Lebanon|University of Calabar Teaching Hospital, Calabar, Cross River State, 540242, Nigeria|National Hospital Abuja, Abuja, FCT, 900211, Nigeria|University of Abuja Teaching Hospital, Gwagwalada, FCT, 902101, Nigeria|Ahmadu Bello University Teaching Hospital, Zaria, Kaduna, 1100011, Nigeria|University of Nigeria Teaching Hospital, Enugu, 460000, Nigeria|Barau Dikko Teaching Hospital/Kaduna State University, Kaduna, 800212, Nigeria|Aminu Kano Teaching Hospital, Kano, 700233, Nigeria|Department of Pediatrics, College of Medicine, Lagos University Teaching Hospital, Lagos, 100254, Nigeria|Sultan َQaboos University Hospital, Muscat, 123, Oman|Prince Mohammed bin Nasser Hospital, Jizan, Southern, 82943, Saudi Arabia|NIMR-Mbeya Medical Research Center, Mbeya, Tanzania|Hacettepe University, Ankara, Altindag/sihhiye, 06230, Turkey|Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Merkezi Hastanesi, Yenisehir, Mersin, 33343, Turkey|Baskent University Hospital, Adana, Yuregir, 01250, Turkey|Acibadem Adana Hastanesi Cocuk Hematoloji Onkoloji, Adana, 01130, Turkey|Guy's and St Thomas' NHS Foundation Trust, London, London CITY OF, SE1 9RT, United Kingdom|King's College Hospital NHS Foundation Trust, London, London CITY OF, SE5 9RS, United Kingdom|Arthur Davison Childrens's Hospital, Ndola, Copperbelt, 10101, Zambia|Matero Clinical Research Site,, Lusaka, 10101, Zambia|University Teaching Hospital- Children's Hospital, Lusaka, 10101, Zambia |
| A Study Evaluating the Safety and Efficacy of Lovo-cel in Severe Sickle Cell Disease | This is a non-randomized, open label, multi-site, single dose, Phase 1/2 study in approximately 50 adults and adolescents with severe SCD. The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using lovo-cel. | Sickle Cell Disease | ALL | CHILD, ADULT | Birmingham, Alabama, United States|Oakland, California, United States|Atlanta, Georgia, 30322, United States|Chicago, Illinois, United States|Bethesda, Maryland, United States|Hackensack, New Jersey, United States|New Hyde Park, New York, United States|New York, New York, United States|Chapel Hill, North Carolina, United States|Philadelphia, Pennsylvania, United States|Charleston, South Carolina, United States |
| Sickle Cell Trait in Football Players | This study will look at the five different types of sickle cell and their relation to self-reported ill health to determine whether or not one or two of the sickle cell haplotypes are correlated with worse health outcomes. Participants can do complete the study in under half an hour in the privacy of their own home. | Sickle Cell Trait | MALE | ADULT, OLDER_ADULT | University of South Florida, Tampa, Florida, 33620, United States |
| Collect of Cord Blood From Subjects at Risk for Sickle Cell Disease, for the Purpose of Laboratory Research | The study consists in collecting umbilical cord blood cells from newborns at risk of sickle cell disease, to perform laboratory experiments aiming to characterize the cells with HbS/HbS mutation, to develop methods to prepare, to gene-modify and to preserve these cells. | Sickle Cell Disease | FEMALE | ADULT | CHSF, Corbeil-Essonnes, 91106, France |
| US Phase I Study of ECT-001-CB in Patients With Sickle-Cell Disease | The application of experimental hematopoietic cell transplantation (HCT) therapy in sickle-cell disease (SCD) must strike a balance between the underlying disease severity and the possibility of a direct benefit of the treatment, particularly in pediatric populations. Clinical studies in adults with SCD have focused on interventions that prolong survival and improve the quality of life. Unlike children, adults with SCD are much more likely to have a debilitating complication. As a result, the risk/benefit ratio of HCT is very favorable in adults, particularly if an approach to HCT that defines an acceptable level of toxicity can be established. Whereas hematopoietic stem cell transplantation (HSCT) remains the only curative treatment currently available for patients with SCD, the morbidity, the frequent irreversible damage in target organs and the mortality reported in the natural course of patients with severe SCD are strong incentives to perform HSCTs in younger age groups. For those who lack a matched related donor, CB transplant is an appealing option, but despite been less problematic, CB accessibility related to cell dose of appropriately matched cord blood unit (CBU) remains a significant issue. Through a 7-day culture process of a CBU's hematopoietic stem cell HSCs with the UM171 compound, the total cell dose is increased mitigating this limitation. UM171-CB expansion (ECT-001-CB) allows a greater CB accessibility, the selection of better matched cords that might translate into favourable clinical outcomes as reported in previous trials, including a lower risk of graft-versus-host disease. After CB selection and ex-vivo expansion, ECT-001-CB transplant will follow a myeloablative reduced-toxicity conditioning regimen consisting of rATG, busulfan and fludarabine with doses of all agents optimized to the individual using model-based dosing and will be followed by standard supportive care and GVHD prophylaxis consisting of tacrolimus and MMF. | Sickle Cell Disease|Umbilical Cord Blood|Hematopoietic Cell Proliferation | ALL | CHILD, ADULT | Stanford University School of Medicine, Palo Alto, California, 94304, United States|University of California San Francisco, San Francisco, California, 94158, United States |
| Using Patient-Centered Guidelines in a Technology Platform to Improve Health Care in Adults With Sickle Cell Disease | SCD is an inherited disorder of hemoglobin that affects over 100,000 Americans, most of whom live in low-resourced neighborhoods. Acute SCD complications result in 230,000 emergency department visits and $1.5 billion annually in acute-care expenditures. Prior research indicates that increased disease-specific knowledge correlates with improved clinical outcomes in SCD. Thus, targeting strategies to improve disease-specific knowledge is a high priority in the care of individuals with SCD. Significant evidence describes how educational materials, including online educational programs, can be used to increase disease-specific knowledge. In this study, the investigators will evaluate a mobile phone technology intervention based on the prior evidence that technologies can improve SCD-specific knowledge. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | The Ohio State University Medical Center, Columbus, Ohio, 43212, United States|Vanderbilt University, Nashville, Tennessee, 37203, United States |
| A Study to Evaluate Safety, Pharmacokinetic, and Biological Activity of INCB059872 in Subjects With Sickle Cell Disease | The purpose of this study was to evaluate the safety and tolerability, and the pharmacokinetic and biologic activity of INCB059872 in participants with sickle cell disease. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Acevedo Clinical Research Associates, Miami, Florida, 33142, United States|Advanced Pharma, Miami, Florida, 33147, United States|Vita Health and Medical Center, Tamarac, Florida, 33319, United States|University of Illinois at Chicago, Chicago, Illinois, 60607, United States|Boston University, Boston, Massachusetts, 02215, United States|Virginia Commonwealth University, Richmond, Virginia, 23298, United States|Blood Centers of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| Prospective, Observational Study in Sickle Cell Disease Patients on Crizanlizumab Treatment in Middle East Countries and India | This is a multicenter, prospective, single-arm observational non-interventional study (NIS), which will be conducted in various countries in the Middle East and India. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Novartis Investigative Site, Manama, 20525, Bahrain|Novartis Investigative Site, Al Ahmadi, 52700, Kuwait|Novartis Investigative Site, Doha, 305, Qatar|Novartis Investigative Site, Dammam, 32263, Saudi Arabia|Novartis Investigative Site, Jazan, 82943, Saudi Arabia |
| HLA-Identical Sibling Donor Bone Marrow Transplantation for Individuals With Severe Sickle Cell Disease Using a Reduced Intensity Conditioning Regimen | Sickle cell disease (SCD) is the most common inherited blood disorder in Saudi Arabia . Its clinical severity is widely heterogeneous among patients who share the same genetic mutation . Severe frequent pain crisis, recurrent acute chest syndrome and stroke are features of severe SCD. Hydroxyurea is an effective treatment of SCD as it ameliorates the severity and frequency of pain crisis and acute chest syndrome and decreases mortality, however, it is less effective in the prevention and treatment of stroke and other end organ dysfunctions . The only readily available cure of SCD is by hematopoietic stem cell transplantation (HSCT) . Most children with SCD who are treated by HSCT receive myeloablative conditioning with excellent results. The application of reduced intensity (RIC) and non-myeloablative (NMA) conditioning regimens are reserved for patients older than 16 years of age because of the increased risks of morbidity and mortality after HSCT6. However, infertility and gonadal failure after myeloablative conditioning are important barriers to the willingness of patients and their families to undergo HSCT . The development of an effective RIC HSCT in SCD that might spare the fertility of SCD patients would have obvious merit. With the ultimate goal of expanding this curative therapy to SCD patients, we propose to investigate HSCT with a RIC conditioning regimen. We will carry out a pilot study of HSCT from HLA matched sibling donors using thymoglobulin/fludarabine/melphalan conditioning and sirolimus and mycophenolate mofetil (MMF) as GVHD prophylaxis in SCD patients with severe complications such as stroke and other severe complications. We hypothesize that HSCT from HLA matched sibling using thymoglobulin/fludarabine/melphalan conditioning in SCD will maintain a level of stable donor chimerism that is sufficient to cure SCD with minimal toxicity. | Sickle Cell Disease | ALL | CHILD, ADULT | King Abdul Aziz Medical City for National Guard, Riyadh, Saudi Arabia |
| An Assessment of Prasugrel on Healthy Adults and Sickle Cell Adults | The purpose of this study is to measure the exposure to prasugrel's active metabolite and the pharmacodynamic effects of prasugrel treatment in people with Sickle Cell Disease (SCD). | Anemia, Sickle Cell | ALL | ADULT | For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., London, UK, SE 1 1YR, United Kingdom |
| Development and Adaptation of I-STRONG for SCD | This study develops and tests the feasibility and acceptability of an adapted intervention, Integrative Strong Body and Mind Training (I-STRONG), in adolescents with pain from sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's Healthcare of Atlanta (CHOA), Atlanta, Georgia, 30322, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States |
| PK Study of Ticagrelor in Children Aged Less Than 24 Months, With Sickle Cell Disease (HESTIA4) | The purpose of this Phase I study is to investigate the pharmacokinetic properties of ticagrelor in pediatric patients from 0 to less than 24 months with sickle cell disease. Ticagrelor dose level adjustment will require a Protocol amendment and regulatory approval. | Sickle Cell Disease | ALL | CHILD | Research Site, Edegem, 2650, Belgium|Research Site, Genova, 16100, Italy|Research Site, Kisumu, 40100, Kenya|Research Site, Nairobi, 00100, Kenya|Research Site, Beirut, 11-0236, Lebanon|Research Site, Tripoli, 1434, Lebanon|Research Site, Madrid, 28007, Spain|Research Site, London, SE1 7EH, United Kingdom |
| Preservation and Transfer of HBV Immunity After Allogeneic HSCT for SCD | Sickle cell disease (SCD) patients ending with mixed mononuclear chimerism after non-myeloablative HSCT with alemtuzumab/TBI conditioning will probably preserve their immune response to vaccinations administered prior to the transplantation and will therefore not need to be revaccinated. Furthermore, SCD patients after haploidentical HSCT might benefit from adoptive transfer of immunity from their donors. To test the first hypothesis, patients undergoing alemtuzumab/TBI HSCT will be vaccinated with a hepatitis B virus (HBV) vaccine before the transplant. To test the second hypothesis, haploidentical and matched related donors will be vaccinated prior to stem cell donation against HBV. Neither the patient nor the donor may previously have been immunized against HBV in all cohorts. Post-transplantation, the investigators will be able to evaluate whether SCD patients preserve their pre-transplant immune response in the post-transplantation period. Furthermore, the investigators will determine whether donors transfer their immunity to HSCT recipients with SCD disease. | Sickle Cell Disease | ALL | CHILD, ADULT | Amsterdam Medical Centre, Amsterdam, 1105AZ, Netherlands |
| A Rehabilitation Program in Children With Sickle Cell Disease and Cognitive Deficits: a Pilot Study | The overall goal of this project is to determine the feasibility of conducting a cognitive intervention for children with sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT | Washington University School of Medicine/St. Louis Children's Hospital, Saint Louis, Missouri, 63110, United States |
| Sleep Study in Adult Patients With Major Sickle Cell Disease With Paroxysmal Nocturnal Events | Hypothesis is that the occurrence of nocturnal Vaso-Occlusive Crisis (VOC) and priapism in adults might be related to episodes of nocturnal desaturation secondary to a sleep apnea syndrome. Investigator hypothesize that chronic biological consequences of Obstructive Sleep Apnea (hypercoagulability, endothelial dysfunction ...) favour VOC and acute manifestations (nocturnal desaturation) favour nocturnal VOC. The confirmation of this hypothesis will lead investigator to propose a systematic screening of obstructive sleep apnea (OSA) in patients with nocturnal VOC. Moreover, systematic treatment of OSA in sickle cell patients could help significantly reduce the number and severity of nocturnal VOC. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Henri Mondor Hospital, Creteil, 94010, France |
| Topical Sodium Nitrite for Chronic Leg Ulcers in Adult Patients With Blood Disorders | Background: - Chronic leg ulcers are a complication of many blood disorders such as sickle cell disease, thalassemia, and other red blood cell disorders. In these disorders, red blood cells break down earlier than normal, which researchers suspect may cause or contribute to the development of leg ulcers; however, the exact cause is unknown, and current therapies are not very effective. Researchers are interested in determining if a research cream made with sodium nitrite, a substance that is known to increase blood flow by dilating blood vessels, may speed up the healing of skin ulcers. Objectives: - To evaluate the safety and effectiveness of topical sodium nitrite cream as a treatment for chronic leg ulcers in individuals with sickle cell disease or other red blood cell disorders. Eligibility: - Individuals at least 18 years of age who have sickle cell disease or another red cell disorder and have had a leg ulcer for more than 4 weeks. Design: * Participants will be screened with a physical examination, medical history, blood tests, and an examination of the ulcer, including x-ray of the leg(s) with the ulcer and swabs from the wound. * Participants will be scheduled for a 5-day inpatient stay at the Clinical Center, with the following procedures: * Days 1 and 2: Participants will have blood draws, a wound assessment, ultrasound of the affected leg, imaging studies (magnetic resonance imaging and infrared photography), thermo-patch application to monitor temperature changes, measurements of blood flow in the skin, and questionnaires about pain and quality of life. An optional skin biopsy may also be conducted with samples taken near the skin ulcer * Day 3: Participants will have one ulcer treated with the topical cream. Frequent blood draws will be conducted before application and then regularly for up to 6 hours after application of the cream. Thirty minutes after the research cream is applied, participants will have imaging studies of the treated leg and measurements of pain levels and blood flow. * Day 4: Participants will have a blood draw and temperature recordings taken. * Day 5: Participants will have the research cream applied and the same imaging studies as before, and will be discharged for care at home. * For the following 3 weeks, participants will come to the clinical center twice a week to have the research cream applied to the leg ulcer and tests performed by the study researchers. * For the fourth and final week, participants will return for additional cream treatment sessions, imaging studies, blood draws, and other tests as directed by the study researchers. * Study participation will end in the following week (week 5). Subjects will come for a final visit one month after the end of the study. | Sickle Cell Anemia|Sickle Cell Disease|Chronic Hemolytic Disorders | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| Pharmacokinetics and Pharmacodynamics Study of SEG101 (Crizanlizumab) in Sickle Cell Disease (SCD) Patients With Vaso- Occlusive Crisis (VOC) | The purpose of the CSEG101A2202 study was to characterize the Pharmacokinetic (PK) and Pharmacodynamic (PD) of SEG101/crizanlizumab and to evaluate the safety and efficacy of SEG101/crizanlizumab in sickle cell disease (SCD) patients. | Sickle Cell Disease (SCD) | ALL | CHILD, ADULT, OLDER_ADULT | Novartis Investigative Site, Orange, Florida, 32763, United States|Novartis Investigative Site, Tampa, Florida, 33606, United States|Childrens Healthcare of Atlanta ., Atlanta, Georgia, 30342, United States|Augusta University Georgia Patient Treatment, Augusta, Georgia, 30912, United States|University of Maryland Medical Ctr, Baltimore, Maryland, 21201, United States|Childrens Hospital at Montefiore, Bronx, New York, 10467, United States|Duke University Medical Center Patient Treatment, Durham, North Carolina, 27710, United States|East Carolina University East Carolina University, Greenville, North Carolina, 27858, United States|Childrens Hospital Of Philadelphia Patient Treatment, Philadelphia, Pennsylvania, 19104-4399, United States|Medical Uni of South Carolina Medical Univ of SC, Charleston, South Carolina, 29425, United States|M Francisco Gonzalez MD PA ., Columbia, South Carolina, 29203, United States|Carolina Blood and Cancer Care of South Carolina, Rock Hill, South Carolina, 29732, United States |
| Study to Determine the Maximum Tolerated Dose, Safety and Effectiveness of Pomalidomide for Patients With Sickle Cell Disease | The purpose of the study is to determine the maximum tolerated dose, safety and effect on induction of fetal hemoglobin of pomalidomide in patients with Sickle Cell Disease. | Anemia, Sickle Cell | ALL | ADULT | Karmanos Cancer Institute, Detroit, Michigan, 48201-2097, United States |
| Actigraphy Improvement With Voxelotor (ActIVe) Study | This is a study to evaluate the effect of voxelotor on daily physical activity and sleep quality, as measured by a wrist-worn device in participants with sickle cell disease (SCD) and chronic moderate anemia. | Sickle Cell Disease|Sickle Cell Anemia | ALL | CHILD, ADULT | UConn Health, Farmington, Connecticut, 06030, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30342, United States|Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|The Children's Hospital at Montefiore, Bronx, New York, 10476, United States|Icahn School of Medicine at Mount Sinai, New York, New York, 10029, United States|Duke Department of Pediatrics, Durham, North Carolina, 27710, United States|The Ohio State University Wexner Medical Center, Columbus, Ohio, 43210, United States|University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, 15213, United States|The University of Texas Health Science Center at Houston, Houston, Texas, 77030, United States|VCU Health, Richmond, Virginia, 23298, United States |
| A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP) | This clinical trial is a Phase 2/3 study that will determine the recommended dose of mitapivat and evaluate the efficacy and safety of mitapivat in sickle cell disease by testing how well mitapivat works compared to placebo to increase the amount of hemoglobin in the blood and to reduce or prevent the occurrence of sickle cell pain crises. In addition, the long-term effect of mitapivat on efficacy and safety will be explored in an open-label extension portion. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | University of California San Diego, La Jolla, California, 92037-1337, United States|UCLA Health, Los Angeles, California, 90095-1678, United States|Children's Hospital Oakland, Oakland, California, 94609, United States|University of Connecticut Health Center, Farmington, Connecticut, 06030-0001, United States|Yale University, New Haven, Connecticut, 06520, United States|Children's National Hospital, Washington, District of Columbia, 20010-2916, United States|MedStar Washington Hospital Center, Washington, District of Columbia, 20010, United States|Sylvester Comprehensive Cancer Center-Miami, Miami, Florida, 33101, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30342-3283, United States|University of Chicago Medical Center, Chicago, Illinois, 60637-1443, United States|Riley Hospital For Children, Indianapolis, Indiana, 46202-5109, United States|LSU Health Sciences Center - Shreveport, Shreveport, Louisiana, 71103-4228, United States|National Heart Lung and Blood Institute, Bethesda, Maryland, 20814, United States|Kaiser Permanente - Largo Medical Center, Largo, Maryland, 20774-5374, United States|Massachusetts General Hospital, Boston, Massachusetts, 02114-2621, United States|Boston Children's Hospital, Boston, Massachusetts, 02115-5724, United States|Boston Medical Center & Boston University School of Medicine, Boston, Massachusetts, 02118, United States|University of Michigan, Ann Arbor, Michigan, 48109-5000, United States|Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|Mississippi Center for Advanced Medicine, Madison, Mississippi, 39110-6115, United States|Cure 4 The Kids Foundation, A Division of Roseman University of Health Sciences, Las Vegas, Nevada, 89106, United States|East Carolina University - Brody School of Medicine, Greenville, North Carolina, 27834, United States|The Cleveland Clinic Foundation, Cleveland, Ohio, 44195-0001, United States|Penn State Health Milton S. Hershey Medical Center, Hershey, Pennsylvania, 17033, United States|Penn Medicine - University of Pennsylvania Health System, Philadelphia, Pennsylvania, 19104-5127, United States|St. Christopher's Hospital for Children, Philadelphia, Pennsylvania, 19134-1011, United States|Lifespan at Rhode Island Hospital, Providence, Rhode Island, 02903, United States|University of Texas Health Science Center of Houston, Houston, Texas, 77030-1501, United States|Texas Children's Hospital, Houston, Texas, 77030, United States|Virginia Commonwealth University, Richmond, Virginia, 23298-5058, United States|Seattle Cancer Care Alliance, University of Washington, Seattle, Washington, 98195, United States|Hôpital Erasme, Anderlecht, Brussels, 1070, Belgium|ZNA Stuivenberg, Antwerpen, Brussels, 2060, Belgium|Universitair Ziekenhuis Antwerpen, Edegem, Brussels, 2650, Belgium|CHR de la Citadelle, Liège, 4000, Belgium|Clinique CHC MontLégia, Liège, 4000, Belgium|Multihemo Servicos Medicos S/A, Recife, Pernambuco, 50070-460, Brazil|Hospital de Clinicas de Porto Alegre (HCPA) - PPDS, Porto Alegre, Rio Grande Do Sul, 90035-903, Brazil|Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS), Porto Alegre, Rio Grande Do Sul, 90619-900, Brazil|Hospital de Clínicas da Unicamp, Campinas, São Paulo, 13083-878, Brazil|Hospital Das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP, Ribeirão Preto, São Paulo, 14051-140, Brazil|Praxis Pesquisa Medica, Santo Andre, São Paulo, Brazil|HEMORIO Instituto Nacional de Hematologia, Rio De Janeiro, 20211-030, Brazil|Hospital das Clínicas da Faculdade de Medicina da Universidad de São Paulo, São Paulo, 05403-010, Brazil|McMaster University - St. Joseph's Healthcare Hamilton, Hamilton, Ontario, L8N 4A6, Canada|University Health Network, Toronto, Ontario, M5G2C4, Canada|CHU Montreal, Montreal, Quebec, H2X 3E4, Canada|McGill University Health Center, Montreal, Quebec, H3A 2B4, Canada|Hopitaux de La Timone, Marseille, Bouches-du-Rhône, 13005, France|Hôpital Pellegrin, CHU de Bordeaux, Bordeaux, Gironde, 33000, France|CHU Guadeloupe, Pointe-à-Pitre, Guadeloupe, 97159, France|Institut Universitaire du Cancer de Toulouse - Oncopole, Toulouse, Haute-Garonne, 31100, France|Hôpital Européen Georges Pompidou, Paris, Ile De France, 75015, France|CHU Hôpital Henri Mondor, Créteil, Val-de-Marne, 94000, France|Universitätsklinikum Essen, Essen, 45147, Germany|Universitätsklinikum Regensburg, Regensburg, 93053, Germany|Korle-Bu Teaching Hospital, Accra, Ghana|Komfo Anokye Teaching Hospital, Kumasi, Ghana|HaEmek Medical Center, Afula, 1834111, Israel|Rambam Medical Center, Haifa, Ḥeifā, 31096, Israel|Ziv Medical Center, Safed, Ḥeifā, 13100, Israel|A.O.R.N. "A. Cardarelli", Napoli, Campania, 80131, Italy|AOU dell'Università degli Studi della Campania Luigi Vanvitelli, Napoli, Campania, 80138, Italy|Azienda Ospedaliero Universitaria Di Modena Policlinico, Modena, Emilia-Romagna, 41100, Italy|IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN, Roma, Lazio, 00165, Italy|Ente Ospedaliero Ospedali Galliera, Genova, Liguria, 16128, Italy|Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello, Palermo, Sicilia, 90146, Italy|Kemri Usamru, Kisumu, Western, 40100, Kenya|Kondele Children's Hospital, Kisumu, 40100, Kenya|Victoria Biomedical Research Institute (VIBRI), Kisumu, 40100, Kenya|KEMRI CRDR Clinical Research Clinic Nairobi, Nairobi, 00100, Kenya|KEMRI/CRDR Siaya Clinical Research Annex, Nairobi, 00200, Kenya|Strathmore University, Nairobi, 00200, Kenya|Gertrude's Children's Hospital, Nairobi, 42325- 00100, Kenya|American University of Beirut Medical Center, Beirut, Beyrouth, Lebanon|Nini Hospital, Tarablus, Liban Nord, Lebanon|Hammoud Hospital University Medical Center, Sidon, Lebanon|Erasmus MC, Rotterdam, Zuid-Holland, 3015 GD, Netherlands|Universitair Medisch Centrum Utrecht, Utrecht, 3584 CX, Netherlands|National Hospital Abuja, Abuja, Abuja Capital Territory, Nigeria|University of Abuja Teaching Hospital, Abuja, Abuja Capital Territory, Nigeria|Lagos University Teaching Hospital, Suru-Lere, Lagos, 101014, Nigeria|University of Calabar Teaching Hospital, Calabar, Nigeria|The University of Nigeria Teaching Hospiatal, Enugu, Nigeria|Barau Dikko Teaching Hospital (BDTH), Kaduna, Kaduna, Nigeria|Sultan Qaboos University Hospital, Hematology Department, COM&HS, Muscat, Musqal, Oman|King Khalid University Hospital, Riyadh, Ar Riya, 11472, Saudi Arabia|King Abdullah International Medical Research Center, Riyadh, 1515 (KAIMRC), Saudi Arabia|Hacettepe University, Ankara, Adana, Turkey|Acibadem Adana Hospital, Seyhan, Adana, 01130, Turkey|Mersin University Medical Faculty, Yenisehir, Içel, 33110, Turkey|Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi, Istanbul, 34093, Turkey|Ege Universitesi Tip Fakultesi Hastanesi, Izmir, 35040, Turkey|Evelina Children's Hospital, London, City Of London, SE1 7EH, United Kingdom|Cambridge University Hospitals NHS Foundation Trust, Cambridge, CB2 0QQ, United Kingdom|Guy's and St Thomas' NHS Foundation Trust, London, SE1 7EH, United Kingdom|King's College Hospital NHS Foundation Trust, London, SE5 9RS, United Kingdom|Hammersmith Hospital, London, W12 0HS, United Kingdom|University College London Hospitals (UCLH), London, WC1E 6BT, United Kingdom|Manchester Royal Infirmary, Manchester University NHS Foundation Trust, Manchester, M13 9WL, United Kingdom |
| Haploidentical Hematopoietic Stem Cell Transplantation | The study is designed as a Pilot/Phase 1 trial of reduced intensity Haploidentical HSCT in patients with sickle cell disease and thalassemia. The purpose of the study is to assess the safety and toxicity of reduced intensity conditioning haploidentical hematopoietic stem cell transplantation. | Sickle Cell-thalassemia Disease|Thalassemia | ALL | CHILD, ADULT | Childrens National Medical Center, Washington, District of Columbia, 20010, United States |
| A Pilot Study on Neuroimaging in SCD: Part of The Boston Consortium to Cure Sickle Cell Disease | Sickle Cell Disease (SCD) impairs oxygen transport to tissue and causes endothelial injury. Thus, therapeutic interventions aim to improve both, but there is an unmet need for biomarkers to determine when intervention is necessary and evaluate the effectiveness of the chosen intervention in individual patients. This study proposes to monitor SCD and its treatment through their impact on cerebral hemodynamics, as the brain is one of the most vulnerable and consequential targets of the disease. Specifically, this study will optimize quantitative magnetic resonance imaging (MRI) and advanced optical spectroscopy techniques such as frequency-domain near-infrared and diffuse correlation spectroscopies (FDNIRS-DCS) to monitor 1) cerebral oxygen transport with measures of cerebral blood flow (CBF), cerebral oxygen extraction fraction (OEF) and cerebral metabolic rate of oxygen consumption (CMRO2) and 2) endothelial function with cerebrovascular reactivity (CVR). Additionally, this study aims to monitor baseline cerebral oxygen transport and CVR, as well as changes that occur with treatment (transfusion or genetic therapy to induce fetal hemoglobin) and assess hemoglobinopathy patients with known genotypes and phenotypes. The ultimate goal is to demonstrate the potential of this monitoring approach to select individual SCD subjects for interventions and evaluate individual responses to treatment. Success will help justify inclusion of these modalities in ongoing and future clinical trials of novel SCD therapies. | Sickle Cell Disease | ALL | CHILD, ADULT | Boston Children's Hospital, Boston, Massachusetts, 02115, United States |
| Moderate Dose Hydroxyurea for Secondary Stroke Prevention in Children With Sickle Cell Disease in Sub-Saharan Africa | The overall goal of the proposed study is to determine the effectiveness of hydroxyurea therapy for secondary stroke prevention and prevention of other neurological events in children with SCA with an acute overt stroke. | Sickle Cell Disease|Sickle Cell Anemia|Stroke | ALL | CHILD | Aminu Kano Teaching Hospital, Kano, Nigeria|Murtala Muhammad Specialist Hospital, Kano, Nigeria |
| PK and PD Responses to Oral L-Citrulline in Patients With Sickle Cell Disease | To evaluate PK and PD responses to L-citrulline given orally for four weeks to patients with sickle cell disease who are otherwise healthy. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital of Pittsburgh of the University of Pittsburgh Medical Center Health System, Pittsburgh, Pennsylvania, 15213, United States |
| ASH Research Collaborative Data Hub | Benign and malignant hematologic diseases are relatively rare conditions within the spectrum of medical practice in any one site of care. Nonetheless, recent research in hematologic conditions from basic, translational, clinical and population perspectives offer the possibility of improving the way that these diseases are treated, and the outcomes experienced by patients. A repository that aggregates and validates this data across institutions and other practice settings is needed in order to identify variation in care, new findings, and further research. | Sickle Cell Disease|Multiple Myeloma | ALL | CHILD, ADULT, OLDER_ADULT | ASH Research Collaborative, Washington, District of Columbia, 20036, United States |
| New, Previously Unknown, Uses of Optical Coherence Tomography Angiography (OCTA) | Sickle cell maculopathy was sparingly mentioned in the literature before despite the fact that sickle cell disease or sickle cell trait is common in people of many areas of the world. This study shows that OCTA is a very valuable, non invasive, office procedure that may well be used in diagnosing this disease. | Sickle Cell Retinopathy | ALL | CHILD, ADULT, OLDER_ADULT | |
| Optimizing Hydroxyurea Therapy in Children With SCA In Malaria Endemic Areas | The Novel use Of Hydroxyurea in an African Region with Malaria (NOHARM) study is the first placebo-controlled randomized clinical trial of hydroxyurea treatment in a malaria endemic region. NOHARM has now achieved full enrollment; all children have completed the blinded portion of the protocol and are in the open-label study treatment portion. This extension study of maximum tolerated dose (MTD), addresses the next critical set of questions about the optimal dosing and monitoring of hydroxyurea treatment for children with SCA in low-resource settings. By providing guidance about optimal hydroxyurea treatment, the NOHARM MTD Study will directly inform policies that can transform the health of African children living with SCA. | Sickle Cell Anemia|Sickle Cell Disease|Malaria | ALL | CHILD | Mulago Hospital Sickle Cell Clinic, Kampala, Uganda |
| Phosphodiesterase Type-5 Inhibitor Therapy in Sickle Cell People With Pulmonary Hypertension | Background: Sickle cell disease (SCD) is a common inherited blood disorder. Many people with SCD are at risk to get pulmonary hypertension (PH). PH means that the blood pressure in the blood vessels to the lungs is high, and is a serious disease and. Very few studies have looked at the success of treatments for PH in people with SCD. Researchers want to learn more about treating PH with a type of drug called phosphodiesterase type 5 inhibitors (PDE5-I). They will look at the records of people who have already joined other studies. Objective: To identify people who already joined NIH SCD protocols whose medical records should be reviewed. The review will look at the description of SCD patients with PH who have already taken PDE5-I and the outcomes for these people. Eligibility: Adults ages 18 and older with SCD and PH. They must have joined certain NIH studies and taken PDE5-I therapy for at least 16 weeks. Design: This study is a review of medical records. Researchers will collect data from databases of existing studies. They will identify people in those studies who have SCD and PH and took the study drug for at least 16 weeks. Researchers will review the full medical records of those people. From that review, researchers will find participants who meet the inclusion criteria. They will extract data from those records. Researchers will analyze the data. This includes results from heart and lung tests, imaging, and walking tests. It will also include results of a procedure called right heart catheterization. Demographic data and lab data will also be collected. Researchers will remove identifying information from the data, then share it in a database. | Pulmonary Hypertension|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | National Heart, Lung and Blood Institute (NHLBI), Bethesda, Maryland, 20892, United States |
| Absorption, Metabolism, and Excretion of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease | The objective of this study is to evaluate the pharmacokinetics of deferiprone and its 3-O-glucuronide metabolite following administration of a single 1500 mg dose of Ferriprox in patients with sickle cell disease. | Sickle Cell Disease | ALL | ADULT | CHUM-Hôpital Notre-Dame, Montreal, Quebec, H2L 4M1, Canada |
| Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease | Background: Sickle cell disease (SCD) is a genetic disease that causes the body to produce abnormal ( sickled ) red blood cells. SCD can cause anemia and life-threatening complications in the lungs, heart, kidney, and nerves. People with SCD are also at increased risk of forming blood clots in the veins and lungs, but the standard treatments for these clots can cause increased bleeding in people with SCD. Better treatments are needed. Objective: To test a drug (fostamatinib) in people with SCD. Eligibility: People aged 18 to 65 with SCD. Design: Participants will have 6 clinic visits over 12 weeks. Each visit will be 2 to 3 hours. Participants will be screened. They will have a physical exam with blood tests. They will tell the researchers about the medications they take. Fostamatinib is a tablet taken by mouth. Participants will take the drug at home, twice a day, for up to 6 weeks. Participants will have a clinic visit every 2 weeks while they are taking the drug. At each visit they will have a physical exam with blood tests. They will talk about any side effects the drug may be causing. If they are tolerating the drug well after the first 2 weeks, they may begin taking a higher dose. Participants will have a final visit 4 weeks after they stop taking the drug. They will have a physical exam and blood tests; they will be checked for any side effects of the drug. | Sickle Cell Disease|Hb-SS Disease|Hemoglobin S|Disease Sickle Cell Anemia|Sickle Cell Disorders|Hemoglobin Beta Thalassemia Disease | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Evaluation of Impact of Disease on Quality of Life, Education and Socio-professional Integration of Adults and Parents of Children Living With Sickle- Cell Disease in France | Sickle cell disease (SCD) is the most common genetic disease in France. Its consequences on patient's life-course and quality of life need to be precisely identified among French patients and their family to be able to improve patients care according to their specific needs. The aim of the study is to accurately describe the impact of SCD on quality of life of patients living in France, or their family (for minor patients). The consequences of the disease on professional life, education and material condition of patients or their parents will be described by the patients themselves. | Sickle-cell Disease (SCD) | ALL | ADULT, OLDER_ADULT | Centre de références syndromes drépanocytaires majeurs thalassémie et autres maladies rares du globule rouge et de l'érythropoïése, Créteil, France |
| Acceptability, Feasibility and Safety of a Yoga Program for Chronic Pain in Sickle Cell Disease | Chronic Pain is associated with morbidity and poor quality of life in patients with Sickle Cell Disease (SCD). Complementary therapies, such as yoga are beneficial in patients with non-SCD chronic pain conditions. Yoga was shown to be acceptable, feasible and helpful in one study in acute SCD pain. The purpose of the study is to assess the acceptability, feasibility, and safety of yoga for chronic pain in SCD. | Sickle Cell Disease | ALL | CHILD, ADULT | Emory University, Atlanta, Georgia, 30322, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30329, United States |
| A Study of SANGUINATE for the Treatment of Vaso-occlusive Crisis (VOC) in Adult Sickle Cell Disease Patients | Safety and effect of SANGUINATE on Sickle Cell Disease patients experiencing a vaso-occlusive crisis who are admitted to the hospital for treatment. | Anemia, Sickle Cell | ALL | ADULT, OLDER_ADULT | Bogota, Colombia|Santo Domingo, Dominican Republic|San Pedro Sula, Honduras|Panama City, Panama |
| Abciximab (ReoPro) as a Therapeutic Intervention for Sickle Cell Vaso-Occlusive Pain Crisis | The purpose of this study is to determine whether giving abciximab (ReoPro) to children with sickle cell disease who are hospitalized for acute pain crisis will improve their pain and shorten the time spent in the hospital, when compared with standard supportive care. | Sickle Cell Disease|Hb-SS Disease With Vasoocclusive Pain|Hemoglobin SS Disease With Vasoocclusive Crisis|Other Sickle Cell Disease With Vaso-Occlusive Pain|Hemoglobin SS Disease With Crisis | ALL | CHILD, ADULT | Cardinal Glennon Children's Medical Center, St. Louis, Missouri, 63104, United States |
| Prevalence of Problematic Use of Equimolar Mixture of Oxygen and Nitrous Oxide and Analgesics in the Sickle-cell Disease | The use of analgesics can lead to cases of drug abuse and dependence. It can also cause pseudo-addiction in patients suffering from pain. What is the actual situation in patients suffering from severe sickle-cell disease, exposed to acute pain during vaso-occlusive crises? Evaluation of the use of analgesics, on the basis of Diagnostic and Statistical Manual of Mental Disorders criteria for substance abuse and dependence, makes it possible to differentiate the symptoms occurring only in a context of pain, in the aim of managing the pain, and thus describing pseudo-addiction, from symptoms also occurring when there is no pain, and more in favour of true addiction. Currently there is no data available in France on this problem, and no studies have been carried out in children or adolescents with sickle-cell disease. The purpose of the study is to evaluate the prevalence of problematic use of equimolar mixture of oxygen and nitrous oxide and other analgesic drugs in a population of subjects with severe sickle-cell disease in France. PHEDRE (Pharmacodépendance Et DREpanocytose-drug dependence and sickle-cell disease) is an observational, descriptive and transversal study. Patients under the age of 26 with sickle-cell disease are included in the study by the doctors looking after them in sickle-cell disease centres. The patients are then contacted by a trained researcher for a telephone interview, including an evaluation of the Diagnostic and Statistical Manual of Mental Disorders criteria for abuse and dependence to equimolar mixture of oxygen and nitrous oxide and for each of the analgesic drugs taken by the patient. The data are also completed using the subject's medical record. This study will make it possible to provide an initial quantitative and qualitative evaluation of problematic use of equimolar mixture of oxygen and nitrous oxide and analgesic drugs in the sickle-cell disease population. The results will be used firstly to provide additional data essential for monitoring the risk of overdose, abuse, dependence and misuse of these products, and to begin awareness-raising and to provide information for health care professionals, in order to significantly improve the management of sickle-cell disease-related pain. | Sickle Cell Disease | ALL | CHILD, ADULT | |
| Trial of Zileuton CR in Children and Adults With Sickle Cell Disease | The purpose of this research study is to test the safety of Zileuton and see what effects (good and bad) it has on you, other children and adults with Sickle Cell Disease (SCD). The investigators also want to see how Zileuton is handled by your body at different doses. Zileuton is a drug that is approved by the Food and Drug Administration (FDA) for the treatment of asthma for people age 12 and older. The FDA has not approved Zileuton for the treatment of SCD, so it is being studied as an investigational drug for SCD through an application to the FDA. In asthma patients, Zileuton helps by reducing inflammation. This study will see if Zileuton helps to reduce inflammation associated with SCD. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States |
| Deferoxamine for Sickle Cell Chronic Leg Ulcer Treatment | Approximately 60 subjects will be enrolled into this double-blind, placebo-controlled study for the Deferoxamine Intradermal Delivery Patch (DIDP). Those subjects who pass Screening will enter into the 2-week Standard of Care (SOC) Run-In period. During this time, ulcers will be assessed to check healing based on digital planimetry, and qualitative features of the ulcer. Subjects who meet eligibility criteria at the end of the 2-week Run-in Period will be randomized into active and control groups (2 active to 1 placebo) and enter the 12-week Treatment Period. At each visit during the Treatment Period, the target ulcer will be measured by digital photographic planimetry, the Principal Investigator will assess the wound qualitative attributes, and the DIDP (or placebo patch) will be placed as the primary wound dressing. At each visit the subject will also receive/review a daily diary to document pain , study drug compliance, and analgesic use. | Chronic Cutaneous Ulcer|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of Alabama at Birmingham, Birmingham, Alabama, 35294, United States|University of Miami, Miami, Florida, 33136, United States|Sonar Clinical Research LLC, Atlanta, Georgia, 30315, United States|Montefiore Medical Center, Bronx, New York, 10467, United States |
| L-Arginine in Children Having Sickle Cell Disease With Increased Tricuspid Regurgitant Jet Velocity | This study aims to investigate the possible efficacy and safety of L-Arginine in children having Sickle Cell Disease with increased Tricuspid Regurgitant Jet Velocity | Sickle Cell Disease | ALL | CHILD, ADULT | Tanta university, Tanta, Other (Non U.s.), Egypt |
| Stroke Prevention in Sickle Cell Anemia (STOP 1) | To reduce episodes of first time stroke by 75 percent in children with sickle cell anemia by the administration of prophylactic transfusion therapy. | Anemia, Sickle Cell|Cerebral Embolism and Thrombosis|Cerebrovascular Disorders|Hematologic Diseases|Hemoglobinopathies | ALL | CHILD | |
| Nutritional Outcomes After Vitamin A Supplementation in Subjects With SCD | This study establishes the safety and efficacy of vit A supplementation doses (3000 and 6000 IU/d) over 8 weeks in children with SCD-SS, ages 9 and older and test the impact of vit A supplementation on key functional and clinical outcomes. Additionally, vitamin A status is assessed in healthy children ages 9 and older to compare to subjects with SCD-SS. | Sickle Cell Anemia in Children|Vitamin A Deficiency in Children | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19146, United States |
| Dexamethasone to Treat Acute Chest Syndrome in People With Sickle Cell Disease | People with sickle cell disease (SCD) may develop acute chest syndrome (ACS), which is a common and serious lung condition that usually requires hospitalization. Dexamethasone is a medication that may decrease hospitalization time for people with ACS, but it may also bring about new sickle cell pain. This study will evaluate the effectiveness of a dexamethasone regimen that includes a gradual dose reduction at decreasing hospitalization and recovery time in people with SCD and ACS. | Anemia, Sickle Cell | ALL | CHILD, ADULT, OLDER_ADULT | University of California - Davis, Sacramento, California, 95817, United States|Kosair Children's Hospital, Louisville, Kentucky, 40202, United States|Children's Hospital Boston, Boston, Massachusetts, 02115, United States|University of North Carolina, Chapel Hill, North Carolina, 27599, United States|St. Christopher's Hospital, Philadelphia, Pennsylvania, 19134, United States|Children's Medical Center of Dallas, Dallas, Texas, 75235, United States |
| The Longitudinal Relationship of HU Adherence to HRQOL, Barriers to Adherence and Habit in SCD. | The primary objective of this study is to better understand factors contributing to variations in hydroxyurea (HU) adherence behavior in adolescents and young adults (AYA) with sickle cell disease (SCD). To meet this objective, the researchers will conduct a prospective cohort study to determine the longitudinal relationship between HU adherence and health-related quality of life (HRQOL) overtime among AYA with SCD. The long-term goal of this research is to promote medication adherence behavior and improve health outcomes in AYA with SCD. | Sickle Cell Disease|Sickle B+ Thalassemia|Sickle Beta Zero Thalassemia|Sickle Cell Hemoglobin C | ALL | CHILD, ADULT | Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States |
| Implications of a Paediatrician-psychologist Tandem for Sickle Cell Disease Care and Impact on Cognitive Functioning | Sickle cell disease (SCD) necessitates a paediatric treatment plan that considers the influence of psychological, family and intercultural factors. At the Louis-Mourier Hospital (APHP) in Colombes, France, a paediatric-psychological partnership where a clinical psychologist accompanies the paediatrician at programmed consultations was introduced. The psychological repercussions of SCD were assessed among children and their parents treated in Colombes and in two other paediatric units without a paediatric-psychological partnership. | Sickle Cell Disease | ALL | CHILD, ADULT | |
| Regional Anesthesia for Sickle Cell Crisis Using Ultrasound in The Emergency Department: Phase I | The hypothesis of this study is: Femoral nerve blocks can feasibly be performed on patients with Sickle Cell Disease and painful crisis in the Emergency Department. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Icahn School of Medicine at Mount Sinai, New York, New York, 10029, United States |
| Evaluation of Hydroxyurea Plus L-arginine or Sildenafil to Treat Sickle Cell Anemia | Patients with sickle cell disease have abnormal hemoglobin (the protein in red blood cells that carries oxygen to the body). This abnormality causes red blood cells to take on a sickle shape, producing disease symptoms. Fetal hemoglobin, a type of hemoglobin present in fetuses and babies, can prevent red cells from sickling. The drug hydroxyurea increases fetal hemoglobin production in patients with sickle cell disease by making a molecule called nitric oxide. The drugs L-arginine and Sildenafil (Viagra) increase the amount or the effect of nitric oxide. This study will evaluate: * The safety of giving L-arginine or Sildenafil together with hydroxyurea in patients with sickle cell disease; * The effectiveness of L-arginine plus hydroxyurea or Sildenafil plus hydroxyurea in increasing fetal hemoglobin in patients with sickle cell disease; and * The effectiveness of L-arginine plus hydroxyurea or Sildenafil and hydroxyurea in lowering blood pressure in the lungs of patients with sickle cell disease. (Pulmonary blood pressure is elevated in about one-third of patients with sickle cell disease, and this condition increases the risk of dying from the disease.) Patients with hemoglobin S-only, S-beta-thalassemia, or other sickle cell disease genotype may be eligible for this study. Before starting treatment, patients will have a complete medical history and physical examination. All patients will take hydroxyurea once a day every day by mouth for at least 2 months. They will be admitted to the NIH Clinical Center to take their first dose of hydroxyurea, and will have blood drawn through a catheter (plastic tube placed in a vein) every hour for 6 hours for tests to determine nitric oxide levels. After discharge, they will return to the clinic once every 2 weeks to check for treatment side effects and for blood tests to monitor hemoglobin and fetal hemoglobin levels. After fetal hemoglobin levels have been stable for 2 months, patients will be admitted to the Clinical Center for their first dose of L-arginine (for men) or Sildenafil (for women). Again, blood samples will be collected through a catheter once an hour for 6 hours. If there are no complications, patients will be discharged and will continue taking hydroxyurea once a day and L-arginine or Sildenafil three times a day for at least 3 months until fetal hemoglobin levels have been stable for at least 2 months. Patients will return to the clinic for blood tests every week for 2 weeks and then every 2 weeks to monitor hemoglobin and fetal hemoglobin levels and to check for treatment side effects. Patients will have eye examinations before and during treatment. Some patients with sickle cell disease develop abnormalities in the blood vessels of the eye. Also, Sildenafil can cause temporary changes in color vision. Rarely, more serious eye problems can occur, such as bleeding from the eye blood vessels or damage to the retina a layer of tissue that lines the back of the eye. Patients will also have an echocardiogram (ultrasound of the heart) before beginning treatment, after hydroxyurea treatment, and after 1 and 3 months of combined treatment with hydroxyurea and L-arginine or Sildenafil to help measure blood pressure in the lungs. Patients who develop complications from L-arginine or Sildenafil may continue in the study on hydroxyurea alone. Patients whose fetal hemoglobin levels increase with the combination therapy of hydroxyurea and L-arginine or Sildenafil may continue to take them. | Sickle Cell Anemia | ALL | CHILD, ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| Comparison of Allogeneic Matched Related Haematopoietic Stem Cell Transplantation After a Reduced Intensity Conditioning Regimen With Standard of Care in Adolescents and Adults With Severe Sickle Cell Disease | Although the survival of children with sickle cell disease (SCD) has dramatically improved over the last decades in the US and Europe, mortality remains high in adults. Moreover, many children and most adults develop a chronic debilitating condition due to organ damage. Allogeneic hematopoietic stem cell transplantation (HSCT) is currently the unique curative approach; it allows the cure of more than 95% of children transplanted from a matched related donor (MRD) after a myeloablative conditioning regimen.To date, few studies have addressed the role of HSCT in SCD adults, due to the risk of graft versus host disease (GVHD) and to the toxicity expected in older patients with a higher risk of organ damage. The development of safe, non-myeloablative conditioning regimens that allow stable mixed chimerism and avoid GVHD appears as an attractive option for HSCT to cure adults with severe SCD. The investigators design a prospective multicenter trial targeting patients over 15 years with severe SCD, and compare non-myeloablative transplant (when a matched related donor (MRD) is identified) versus no HSCT (for patients lacking MRD). The main objective is to assess the benefit of HSCT on the 2-year event free survival compared to standard care. The primary endpoint is the 2-year event free survival. | Sickle Cell Disease | ALL | CHILD, ADULT | |
| Evaluation of Virtual Reality to Save Morphinic in the Treatment of Vaso-occlusive Seizures of Sickle Cell Patients Consulting in the Emergency Room | The management of pain in the emergency department is a major issue, especially for sickle cell patients who regularly consult for vaso-occlusive seizure (VOS). The place of virtual reality remains to be defined in a busy environment, in which the permanence of care generates a significant turn over of medical and paramedical personnel. With Its immersive nature, allowing the patient to detach from his immediate environment, wich is often stressful for patients, we can hope that in multimodal management, Virtual Reality (VR) can contribute to a faster reduction in pain with lower doses of morphine, but so far we have no data. Our pilot study aims to assess the effectiveness, feasibility and tolerance of adding virtual reality to the management of VOS in sickle cell patients in the ER. | Vaso-occlusive Crisis|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | HOPITAL TENON Service des urgences, Paris, 75020, France |
| Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells | This is a clinical trial of bone marrow transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor. Genetic diseases of blood cell include: Red blood cell defects e.g. hemoglobinopathies (sickle cell disease and thalassemia), Blackfan-Diamond anemia and congenital or chronic hemolytic anemias; White blood cells defects/immune deficiencies e.g. chronic granulomatous disease, Wiskott-Aldrich syndrome,Osteopetrosis, Kostmann's syndrome (congenital neutropenia), Hereditary Lymphohistiocytosis (HLH); Platelets defects e.g.Congenital amegakaryocytic thrombocytopenia; Metabolic/storage disorders e.g. leukodystrophies,mucopolysaccharidoses as Hurler disease;Stem cell defects e.g.reticular agenesis, among many other rare similar conditions. The study treatment plan uses a new transplant treatment regimen that aims to try to decrease the acute toxicities and complications associated with the standard treatment plans and to improve outcome The blood stem cells will be derived from either unrelated donor or unrelated umbilical cord blood. | Sickle Cell Disease|Thalassemia|Anemia|Granuloma|Wiskott-Aldrich Syndrome|Chediak Higashi Syndrome|Osteopetrosis|Neutropenia|Thrombocytopenia|Hurler Disease|Niemann-Pick Disease|Fucosidosis | ALL | CHILD, ADULT | Children Hospital Los Angeles, Los Angeles, California, 90027, United States |
| Hybrid Effectiveness-Implementation Trial of Guided Relaxation and Acupuncture for Chronic Sickle Cell Disease Pain | The investigators will conduct a hybrid type 1 effectiveness implementation trial to assess the effectiveness of acupuncture and guided relaxation on 360 people with Sickle Cell Disease (SCD), while observing and gathering information on implementation in three health systems: University of Illinois Hospital \& Health Sciences System, University of Florida Health, and Duke University Health Systems. Each serves a large population with SCD, uses EPIC as their electronic health record, and has a Clinical and Translational Science Award (CTSA), which will help speed the translation of discovery into improved patient care. During the UH3 Implementation Phase, the 3-arm, 3-site randomized controlled trial will follow a quantitative modified SMART design, a pragmatic trial that evaluates adaptive interventions where the guided relaxation and acupuncture interventions respond to patients' characteristics and evolving pain status. The investigators rely on the Consolidated Framework for Implementation Research (CFIR) to plan, execute, and evaluate associated implementation processes. The use of complementary and integrative health (CIH) therapies by those with SCD to reduce pain and opioid use, to help enable them to better cope with their pain, is well known, but there are few studies that evaluate the effectiveness of these therapies, and none that also evaluates the implementation across multiple health care systems and patient populations as this study will. Aim 1: Determine the effectiveness of guided relaxation and acupuncture as compared to usual care in decreasing pain and opioid use for SCD patients. Hypothesis: At 6-weeks, SCD patients randomized to either CIH intervention will have a greater decrease in pain, opioid use, sleep, anxiety, depressive symptoms, and pain catastrophizing compared to SCD patients randomized to usual care. Aim 2: Identify the best adaptive intervention for improved outcomes by documenting outcomes among adaptive intervention sequences: (1) initiate guided relaxation and switch to acupuncture for non-responders at midpoint; (2) initiate guided relaxation and continue with guided relaxation for non-responders at midpoint; (3) initiate acupuncture and switch to guided relaxation for non-responders at midpoint or (4) initiate acupuncture and continue with acupuncture for non-responders at midpoint. Aim 3: Explore differences in response to the adaptive interventions by age and sex. Aim 4: Identify implementation facilitators, challenges, and solutions for structures and processes that contribute to the seamless integration of CIH therapies into the 3 health systems by conducting individual interviews with participants in the intervention group who responded to the intervention and those who did not. The investigators will also conduct focus groups with hospital personnel at 4 timepoints. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of Florida, Gainesville, Florida, 32610, United States|Emory University, Atlanta, Georgia, 30322, United States|University of Illinois at Chicago, Chicago, Illinois, 60612, United States|Johns Hopkins University, Baltimore, Maryland, 21218, United States|Duke University, Durham, North Carolina, 22710, United States |
| Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients | This study will examine the long-term safety and efficacy of Deferasirox in patients with sickle cell disease and iron overload from repeated blood transfusions. | Sickle Cell Disease|Iron Overload|Hemolytic Anemia | ALL | CHILD, ADULT, OLDER_ADULT | University of Alabama Pediatric Hematology/Oncology, Birmingham, Alabama, 35233, United States|University of Alabama Medical center, Birmingham, Alabama, 35294, United States|University of South Alabama Medical Center, Mobile, Alabama, 36604, United States|University of South Alabama, Mobile, Alabama, 36617, United States|Loma Linda University Medical Center, Loma Linda, California, 92354, United States|Children's Hospital Oakland, Oakland, California, 94609, United States|Center for Cancer and Blood Disorders, Washington, District of Columbia, 20010-2970, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States|Howard University Hospital, Washington, District of Columbia, 20059, United States|Miami Children's Hospital, Miami, Florida, 33155, United States|Tampa Children's Hospital at St Joseph's, Tampa, Florida, 33607-6387, United States|Tampa Children's Hospital at St. Joseph's, Tampa, Florida, 33607-6387, United States|H. Lee Muffit Cancer Center and Research Institute/James A. Haley Veterans Hospital, Tampa, Florida, 33612, United States|Emory University School of Medicine, Atlanta, Georgia, 30303, United States|Children's Healthcare of Atlanta at Scottish Rite, Atlanta, Georgia, 30342, United States|Adult Sickle Cell Clinic, Augusta, Georgia, 30912-3128, United States|Backus Children's Hospital, Savannah, Georgia, 31403, United States|University of Illinois at Chicago, Chicago, Illinois, 60612, United States|Children's Memorial Hospital, Chicago, Illinois, 60614-3394, United States|Pediatric Sickle Cell Program/James Whitcomb Riley Hospital for Children, Indianapolis, Indiana, 46202, United States|St. Jude Children's Hospital Affiliate, Baton Rouge, Louisiana, 70808, United States|Tulane University Sickle Cell Center, New Orleans, Louisiana, 70112, United States|Children's Hospital, New Orleans, Louisiana, 70118, United States|LSU Health Sciences Center/Carroll W. Feist Professor of Cancer Research, Shreveport, Louisiana, 71130, United States|Brigham and Woman's Hospital/Harvard Medical School, Boston, Massachusetts, 02115, United States|Children's Hospital Boston, Boston, Massachusetts, 02115, United States|Children's Hospital, Boston, Massachusetts, 02115, United States|University of Michigan, Ann Arbor, Michigan, 48109-0238, United States|Karmanos Cancer Institute, Detroit, Michigan, 48201, United States|Washington University School of Medicine, St. Louis, Missouri, 63110, United States|Sickle Cell Center, Montefiore Hospital, Bronx, New York, 10467-2490, United States|SUNY Downstate Medical Center, Brooklyn, New York, 11203, United States|New York Methodist Hospital, Brooklyn, New York, 11215, United States|Weill Medical College of Cornell University, New York, New York, 10021, United States|Columbia University, New York, New York, 10032, United States|Carolinas Medical Transplant Center, Charlotte, North Carolina, 28232, United States|University of Cincinnati, Cincinnati, Ohio, 45219, United States|Children's Hospital Medical Center, Cincinnati, Ohio, 72764, United States|The University of Oklahoma, Oklahoma City, Oklahoma, 73104, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104-4399, United States|Pennsylvania Oncology/Hematology, Philadelphia, Pennsylvania, 19106, United States|Jefferson University, Philadelphia, Pennsylvania, 19107, United States|Drexel University College of Medicine, Philadelphia, Pennsylvania, 19134-1095, United States|Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, 15213, United States|Hillman Cancer Center, Pittsburgh, Pennsylvania, 15232, United States|Liberty Hematology Oncology Center, Columbia, South Carolina, 29203, United States|Palmetto Health Clinical Trials, Columbia, South Carolina, 29203, United States|Santee Hematology/Oncology, Sumter, South Carolina, 29150, United States|St Jude's Children's Research Hospital, Memphis, Tennessee, 38105-2794, United States|St. Jude's Children Research Hospital, Memphis, Tennessee, 38105, United States|Cooks Children's Hospital, Fort Worth, Texas, 76104-2724, United States|Texas Children's Hospital/Baylor College of Medicine, Houston, Texas, 77030-2399, United States|Scott and White Memorial Hospital & Clinics, Temple, Texas, 76508, United States|Children's Hospital of the King's Daughter, Norfolk, Virginia, 23507, United States|Medical College of Virginia, Richmond, Virginia, 23298-0306, United States|Virginia Commonwealth University, Richmond, Virginia, 23298-0646, United States|University of Alberta, Edmonton, Alberta, T6G 2H7, Canada|The Ottawa Hospital, Ottawa, Ontario, K1H 8L6, Canada|Hopital Ste-Justine, Montreal, Quebec, H3T 1C5, Canada |
| Clinical Study of BRL-101 in Severe SCD | This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (BRL-101). | Sickle Cell Disease | ALL | CHILD, ADULT | First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi, 530021, China |
| Safety of Single Doses of CSL889 in Adult Patients With Sickle Cell Disease | This is a phase 1, first-in-human, multi-center, open-label, single dose cohort study to evaluate the safety and tolerability, pharmacokinetics (PK), exploratory pharmacodynamics (PD), and biomarkers of target engagement of CSL889 following single intravenous (IV) doses in subjects with sickle cell disease (SCD). The study involves sequential dose escalation of cohorts with between-group assessments of key safety and PK variables. | Sickle Cell Disease | ALL | ADULT | University of Illinois Hospital and Health Science Systems, Chicago, Illinois, 60612, United States|The Johns Hopkins Hospital, Baltimore, Maryland, 21287, United States|University of Minnesota, Minneapolis, Minnesota, 55455, United States|Jacobi Medical Center, Bronx, New York, 10461, United States|Brody School of Medicine at East Carolina University, Greenville, North Carolina, 27834, United States|Ohio State University, Columbus, Ohio, 43201, United States|UPMC Hillman Cancer Center, Pittsburgh, Pennsylvania, 15232, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|Amsterdam UMC Academic Medical Center, Amsterdam, Netherlands|Erasmus University Medical Center, Rotterdam, Netherlands|Liverpool University Hospital, Liverpool, United Kingdom|Guys and St. Thomas, London, United Kingdom|University College London Hospital, London, United Kingdom|Manchester University Hospitals NHS Foundation Trust / Manchester Royal Infirmary, Manchester, M13 9WL, United Kingdom|Early Phase Unit, Manchester, United Kingdom |
| Comparing Individualized vs. Weight Based Protocols to Treat Vaso-Occlusive Episodes in Sickle Cell Disease | The purpose of this research study is to compare two different ways to give opioid pain medicine to treat sickle cell disease pain that is bad enough to go to the emergency department for treatment. One way uses your weight to decide how much pain medicine to give you while in the emergency department. This is called weight based treatment. The other way uses how much pain medicine you take at home and how much medicine you needed during past emergency department visits to decide how much medicine to give you. This is called patient specific treatment. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of Maryland, Baltimore, Maryland, 21201, United States|Henry Ford Health System, Detroit, Michigan, 48202, United States|Wayne State University, Detroit, Michigan, 48202, United States|Atrium Health, Charlotte, North Carolina, 28204, United States|Case Western University, Cleveland, Ohio, 44106, United States|University of Texas Southwestern Medical Center, Dallas, Texas, 75390, United States |
| Neuropsychological Studies of Children With Sickle Cell | To identify those factors that contributed to cognitive deficiencies in children with sickle cell disease (SCD) who had not demonstrated any overt or clinically apparent neurological abnormalities. | Blood Disease|Anemia, Sickle Cell|Neurologic Manifestations | MALE | CHILD, ADULT, OLDER_ADULT | |
| Feasibility and Efficacy of a Home-based, Computerized Cognitive Training Program in Pediatric Sickle Cell Disease | Disease-related neurocognitive deficits are common in pediatric sickle cell disease (SCD). These deficits can significantly disrupt otherwise normal trajectories toward academic and vocational achievement and negatively impact psychosocial outcomes. Despite widespread recognition of neurocognitive deficits, there are no treatments shown to maintain or recover functioning once a child with SCD endures neuronal damage. Cognitive training (CT) has been a standard intervention used to stabilize and recover functioning in individuals with accidental or disease-related brain injury. Recent advances in technology have led to the development of computerized CT programs. This study seeks to assess the feasibility and efficacy of using computerized CT with pediatric patients with SCD. Children and adolescents with SCD between the ages of 7 and 16 years old (n = 80) will be recruited to complete a randomized (intervention or waitlist-control) home-based computerized CT program (Cogmed). Feasibility will be assessed by examining participation, retention, and program completion rates, as well as feedback from a feasibility and acceptability questionnaire and a brief qualitative interview. Participants will also complete assessments of attention, working memory, and academic fluency at baseline and immediately following the intervention. A final assessment will be conducted 6 months after the conclusion of the intervention to evaluate the stability of treatment effects. | Anemia, Sickle Cell | ALL | CHILD | Children's National Health System, Washington, District of Columbia, 20010, United States |
| Building Adaptive Coping and Knowledge to Improve Daily Life | The purpose of this study is to find out how teenagers with chronic pain and sickle cell disease respond to a new training program called Back2Life and get their feedback about how to modify the program to best fit their needs. The Back2Life training program focuses on teaching pain coping skills (also known as cognitive-behavioral therapy). The program teaches skills and strategies that may help teens improve chronic pain management and get back into their everyday activities. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's Healthcare of Atlanta at Hughes Spalding, Atlanta, Georgia, 30303, United States|Chilldren's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States|Emory Children's Center, Atlanta, Georgia, 30322, United States |
| Hydroxyurea and Magnesium Pidolate to Treat People With Hemoglobin Sickle Cell Disease | Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause intense pain episodes. Hemoglobin SCD (HbSC) is a form of SCD that is characterized by dense red blood cells. The purpose of this study is to evaluate the safety and effectiveness of hydroxyurea and magnesium pidolate, alone and combined, at reducing red blood cell density and the frequency of pain episodes in people with HbSC. | Hemoglobin SC Disease | ALL | CHILD, ADULT, OLDER_ADULT | University of Alabama at Birmingham, Birmingham, Alabama, 35233, United States|Children's Hospital and Research Center, Oakland, California, 94609-1809, United States|University of California Davis, Sacramento, California, 95817, United States|University of Colorado, Aurora, Colorado, 80045, United States|University of Miami, Miami, Florida, 33136, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30342-1600, United States|University of Louisville, Louisville, Kentucky, 40202, United States|Johns Hopkins University, Baltimore, Maryland, 21205, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|Children's Hospital Boston, Boston, Massachusetts, 02118, United States|University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States|Montefiore Medical Center, Bronx, New York, 10463, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|Cincinnati Children's Hospital, Cincinnati, Ohio, 45229, United States|University of Oklahoma, Oklahoma City, Oklahoma, 73104, United States|Saint Christopher's Hospital, Philadelphia, Pennsylvania, 19134-1095, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19444, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|Children's Medical Center, Dallas, Texas, 75235, United States |
| To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease | This is an open-label study to understand the safety and tolerability of AXA4010, a novel composition of amino acids in adult and adolescent subjects with sickle cell disease over 12 weeks. The study also assesses the effects of this amino acid composition on the structure and function of the vascular system. Physiological effects on structure and function will be assessed by Magnetic Resonance Imaging (MRI) to assess blood flow in the brain and kidneys and the 6-Minute walk with pulse oximetry. Changes in blood biomarkers of inflammation will also be assessed. | To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease|Anemia, Sickle Cell|Sickle Cell Anemia|Sickle Cell Disorders|Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Foundation for Sickle Cell Disease Research, Hollywood, Florida, 33021, United States|Advanced Pharma CR, LLC, Miami, Florida, 33147, United States|Primary Care research, Atlanta, Georgia, 30312, United States|Newark Beth Israel Medical Center, Newark, New Jersey, 07112, United States |
| Clinical Use of Parental Support To Detect Single Gene Mutations | Gene Security Network has developed a novel technology called Parental Support (PS) which is used for Preimplantation Genetic Screening/Diagnosis (PGS/D) during in vitro fertilization (IVF). This technology allows IVF physicians to identify embryos, prior to transfer to the uterus, which have the best chance of developing into healthy children. | Single Gene Disorders | ALL | CHILD, ADULT, OLDER_ADULT | Gene Security Network, Redwood City, California, 94063, United States |
| Evaluation of Therapeutic Adherence to Inciting Spirometry in Sickle Cell Patients | Sickle cell disease is one of the most common genetic diseases in the world. (1) It is characterized by the production of abnormal hemoglobin mainly responsible for vaso-occlusive clinical manifestations and chronic hemolysis with anemia. (2) It is therefore a chronic disease with major acute complications, such as acute chest syndrome. The treatment for this syndrome will be based on oxygenation, hydration and analgesia. At the physiotherapy level, we will have an action on the prevention and treatment of the syndrome by incentive spirometry. (3,4) In fact, it is currently the only physiotherapy treatment that has proven its effectiveness and is recommended for sickle cell patients. (3) As part of prevention, it is recommended to prescribe incentive spirometry during vaso-occlusive crisis. It has been shown to reduce the risk of atelectasis and significantly limit the risk of developing ACS. (5) In treatment, it makes it possible to regain normal chest amplification and therefore to allow ventilation of unventilated areas. (3.4) However, in order to increase therapeutic efficacy, patient compliance is essential. Adherence to treatment is a major problem in chronic diseases. Currently, it is estimated that 80% of patients with chronic conditions do not sufficiently follow their therapy, which limits the optimization of benefits. (6) This is the case in sickle cell patients, especially with hydroxyurea which is their disease-modifying treatment. Lack of adherence is the most common cause of primary failure of this treatment. During various treatments, we noticed the patients' lack of compliance with spirometry. Indeed, we explained to the patient how to do the incentive spirometry, so that he could practice it several times a day as recommended. When we returned the next day, or after a weekend, most of the time the patients had little or no observance. So I wanted to know if this concerns a majority of patients with sickle cell disease. Indeed, it appears important to assess compliance in these patients in order to improve the effectiveness of treatment and reduce the risk of ACS. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Centre hospitalier intercommunal de Créteil, Créteil, 94000, France |
| Sickle Cell Trait and Exercise, Effect of Hot Environment | The heterozygous form of sickle cell disease is clinically asymptomatic. Nevertheless, it was observed that, the sickle cell trait is associated with serious medical complications especially during intense physical efforts. Moreover, the exposure to a hot environment (tropical climate) is suspected to be a determining factor in the occurrence of these medical complications. However, the relationship between sickle cell trait and death during effort is not well established. Furthermore, the cascade of events that usually cause sickle cell crisis such as red blood cells sickling and rhabdomyolysis and which affect microcirculation are not known. Our main objective in this study is to verify whether young healthy active men with sickle cell trait have reactive hyperemia to their hemoglobinemic condition during exercise; to identify the contribution of hot environment on these possible disturbances; and to determine underlying mechanisms. In addition, disturbances in the regulation of glucose metabolism in healthy subjects under hot environment have been reported, marked by a significant increase in postprandial blood glucose. Therefore, this project is also intended to assess the contribution of the disturbance of glycoregulation during exercise under hot environment in active sickle cell trait carriers. The imbalance of pro and anti oxidant agents, the adhesion and inflammation markers will also be evaluated. Results of this study will allow a better understanding of physio-pathological mechanisms leading to vascular accidents during exercise under tropical climate in young healthy sickle cell trait carriers; and to identify physical activity programs and nutritional interventions adapted to patients with sickle cell disease under hot environment. | Sickle Cell Trait|Environmental Exposure|Adverse Effect | MALE | ADULT | ACTES laboratory, Pointe-à-Pitre, 97157, Guadeloupe |
| Utilizing A Single Session Problem-Solving Intervention With Caregivers of Pediatric Patients Receiving Chronic Transfusion to Treat Sickle Cell Disease | This study seeks to utilize an innovative approach of a single session problem-solving intervention to address psychosocial factors affecting patient outcomes within the pediatric sickle cell population. The study will be a randomized control trial of a single session problem-solving intervention. This original research will investigate the feasibility and efficacy of utilizing a single-session problem-solving intervention to address problems affecting children and families receiving chronic blood transfusions for sickle cell disease in order to: 1) contribute to literature related to single session problem solving interventions with the chronic transfusion sickle cell population and 2) identify a model of sustainable care that reduces the burden of a multiple session intervention and increases access to services. Additionally, this research aims to provide relatively low burden and potentially highly effective intervention into regular care for this population in order to evaluate the feasibility of integrating a single-session problem solving intervention into routine clinical flow, thereby addressing needs more systematically that have been identified by families. Further, this research aims to identify potential utility of medical providers being trained on providing the intervention, which could be part of a future study. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Children's Medical Center, Dallas, Texas, 75235, United States |
| Investigation of Selected Patient Groups From The Cooperative Study of Sickle Cell Disease | To continue to follow the newborn cohort and the over-35 years of age cohort from the Cooperative Study of Sickle Cell Disease (CSSCD), a study of the natural history of sickle cell disease. | Anemia, Sickle Cell|Blood Disease | MALE | CHILD, ADULT, OLDER_ADULT | |
| Study of GMI-1070 for the Treatment of Sickle Cell Pain Crisis | GMI-1070 is a new drug that may reduce the stickiness of cells in the blood. The purpose of this study is to evaluate whether GMI-1070 can reduce the time it takes for pain to go away in patients with vaso-occlusive crisis (also known as a sickle cell pain crisis). The study will also collect information on the safety of GMI-1070, how much of the drug is in the blood and urine, and if there are any other effects when used in patients who are in the hospital for a sickle cell pain crisis. | Sickle Cell Disease|Vaso-occlusive Crisis|Pain Crisis | ALL | CHILD, ADULT | University of Alabama Hospital, Birmingham, Alabama, 35294, United States|Alta Bates Summit Medical Center, Berkeley, California, 94705, United States|University of California, Davis Medical Center, Sacramento, California, 95817, United States|University of Colorado, Aurora, Colorado, 80045, United States|University of Connecticut Health Center, Farmington, Connecticut, 06030, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States|University of Miami Miller School of Medicine, Miami, Florida, 33136, United States|Georgia Health Sciences University, Augusta, Georgia, 30912, United States|University of Illinois, Chicago, Chicago, Illinois, 60612, United States|The Johns Hopkins School of Medicine, Baltimore, Maryland, 21205, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|Karmanos Cancer Institute, Detroit, Michigan, 48201, United States|University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States|Children's Hospital at Montefiore, Bronx, New York, 10467, United States|New York Methodist Hospital, Brooklyn, New York, 11215, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|Cincinnati Childrens' Hospital, Cincinnati, Ohio, 45229, United States|The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, 15224, United States|UT Southwestern Medical Center at Dallas, Dallas, Texas, 75235, United States|Virginia Commonwealth University, Richmond, Virginia, 23298, United States|The Hospital for Sick Children, Toronto, Ontario, M5G 1XB, Canada |
| Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/βC Genotype | The purpose of the study is to evaluate the efficacy and safety of CTX001 (exa-cel) in adolescent and adult participants with severe sickle cell disease (SCD), βS/βC genotype (HbSC). | Sickle Cell Disease | ALL | CHILD, ADULT | |
| Evaluation of the Prevalence of Pulmonary Hypertension in Adult Patients With Sickle Cell Disease | Recent data show that pulmonary hypertension (PH), defined by a tricuspid regurgitation jet (TRJ) velocity \> or equal at 2.5m/s on Doppler echocardiography, is present in about 30% of adults with sickle cell disease (SCD) and is associated with poor prognosis. However in SCD the occurrence of PH (defined by mean pulmonary arterial pressure (mPAP)\> or equal at 25 mmHg) is related to at least 3 mechanisms: PH due to hyperkinetic state with high cardiac output (CO) but normal pulmonary vascular resistance (PVR \<160 dynes), or postcapillary PH (pulmonary capillary wedge pressure PCWP \>15 mmHg), or precapillary pulmonary arterial hypertension (PAH) defined by mPAP \> or equal at 25 mmHg, PCWP\< or equal at 15 mmHg and PVR \> or equal at 160 dynes.The aim of this study is to evaluate in a French population of adults with sickle cell disease the characteristics, prevalence and prognosis of pulmonary hypertension. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Hôpital Antoine Béclère, Clamart, 92141, France |
| The Feasibility of Text Messaging to Assess Secondhand Smoke Exposure Among Youngsters With Cancer or Sickle Cell Disease | Exposure to secondhand smoke is a leading preventable cause of child morbidity and mortality, and the adverse health consequences of secondhand smoke are magnified among youngsters with cancer and sickle cell disease. Current methods for measuring secondhand smoke exposure (SHSe) rely on retrospective reports over extended time periods that are subject to recall errors and systematic inaccuracies in reporting and often do not include the youngster as the primary informant. These methods may underestimate the extent of cumulative SHSe and are not well suited to capturing exposure over time and across settings where young people frequent. More appealing methods that engage youngsters to better monitor tobacco smoke in their environment are warranted. The study will examine the feasibility of cell phone texting to obtain measures of secondhand smoke exposure (SHSe) in children treated for cancer or sickle cell disease (SCD). | Cancer|Sickle Cell Disease | ALL | CHILD | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Endothelial Function in Patients With Sickle Cell Anemia Before and After Sildenafil | The investigators would like to study the endothelial function in sickle cell patients without pulmonary hypertension in an in vivo method during a steady state condition before and after sildenafil treatment for 1 month, and to study the effects of this nitric oxide donor by measuring the Flow Mediated Dilatation, by measuring endothelial progenitor stem cells colonies, and by measuring the effect of therapy on markers of inflammation (cell adhesion molecules and cytokines). | Sickle Cell Anemia | ALL | ADULT | Baruch Padeh Poria Medical Center, Tiberias, Lower Galilee, 15208, Israel |
| Transillumination Device for Peripheral Intravenous Placement in Patients With Sickle Cell Disease (PERFID) | The primary goal of this randomized, controlled, open-label study is to determine the efficacy of AccuveinV400 (a transillumination veins device) to facilitate peripheral intravenous (IVP) catheter placement during a vaso-oclusive crisis. The investigators hypothesized that the number of attempts to a successful placement of a peripheral IV, our primary outcome, would be shorter with the assistance of the Accuvein V400. It is also expected that AccuveinV400 will reduce the time of the procedure, the rate of failure, the technique-related pain. | Sickle Cell Crisis | ALL | ADULT, OLDER_ADULT | AP-HP - Hopital Europeen Georges-Pompidou Paris, France, Paris, Ile-de-France, 75908, France|CHU H. Mondor, Créteil, France |
| Fetal Hemoglobin Induction Treatment Metformin | The purpose of this study is to determine whether metformin is effective in the treatment for sickle cell anemia (SCA). | Sickle Cell Anemia|Sickle Cell Disease|Hemoglobin Disorder | ALL | CHILD, ADULT | Texas Children's Hospital, Houston, Texas, 77030, United States|The University of Texas Health Science Center at Houston, Houston, Texas, 77030, United States |
| Enhancing Preventive Therapy of Malaria In Children With Sickle Cell Anemia in East Africa (EPiTOMISE) | This is a randomized, three-arm, open-label, clinical trial of malaria chemoprevention in children with sickle-cell anemia (SCA) at a single site in Homa Bay, Kenya. The study will enroll 246 children under 10 years of age, randomize participants 1:1:1 to one of three malaria chemoprevention regimens, and follow participants monthly for 12 months in order to record clinical episodes of malaria or SCA-related morbidity. Analyses will compare the efficacy of each regimen to prevent malaria and SCA morbidity. | Malaria,Falciparum | ALL | CHILD | Homa Bay County Referral Hospital, Homa Bay Town, Homa Bay County, 40300, Kenya |
| Physiotherapy for Children With Sickle Cell Disease: A Randomized Controlled Trial | Primary aim to determine the effects of immersive VR on pain management in children with SCD. Secondary aim to determine the effects of immersive VR in musculoskeletal dysfunction in children with SCD and health-related quality of life. | Blood Disease | ALL | CHILD, ADULT | |
| Phase I/II Randomized Study of Hydroxyurea With or Without Clotrimazole in Patients With Sickle Cell Anemia | OBJECTIVES: I. Compare the efficacy of hydroxyurea with or without clotrimazole in terms of limiting the severity of anemia and the rate of hemolysis in patients with sickle cell anemia. | Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | University of North Carolina School of Medicine, Chapel Hill, North Carolina, 27599-7070, United States |
| Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients | The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sickle cell disease (SCD) patients with history of vaso-occlusive crisis (VOC) leading to healthcare visit. | Sickle Cell Disease (SCD) | ALL | CHILD, ADULT, OLDER_ADULT | Childrens Healthcare of Atlanta, Atlanta, Georgia, 30342, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|Levine Cancer Insitute Carolinas Healthcare System, Charlotte, North Carolina, 28204, United States|Univ of Tenn Health Sciences Ctr, Memphis, Tennessee, 38163, United States|University of Texas Health Science Center at Houston, Houston, Texas, 77030, United States|Novartis Investigative Site, Brussel, 1000, Belgium|Novartis Investigative Site, Bruxelles, 1070, Belgium|Novartis Investigative Site, Edegem, 2650, Belgium|Novartis Investigative Site, Salvador, BA, 41253-190, Brazil|Novartis Investigative Site, Belem, PA, 66033 000, Brazil|Novartis Investigative Site, Recife, Pernambuco, 50070-170, Brazil|Novartis Investigative Site, Rio de Janeiro, RJ, 20211-030, Brazil|Novartis Investigative Site, Ribeirao Preto, SP, 14048-900, Brazil|Novartis Investigative Site, Sao Paulo, SP, 01232-010, Brazil|Novartis Investigative Site, Sao Paulo, SP, 05403 000, Brazil|Novartis Investigative Site, Sao Paulo, SP, 08270-070, Brazil|Novartis Investigative Site, Porto Alegre, 90035-003, Brazil|Novartis Investigative Site, Toronto, Ontario, M5G 2C4, Canada|Novartis Investigative Site, Montreal, Quebec, H2X 1R9, Canada|Novartis Investigative Site, Valledupar, Cesar, 5602310, Colombia|Novartis Investigative Site, Barranquilla, 080020, Colombia|Novartis Investigative Site, Monteria, 230004, Colombia|Novartis Investigative Site, Helsinki, FIN 00290, Finland|Novartis Investigative Site, Creteil, 94010, France|Novartis Investigative Site, Marseille, 13885, France|Novartis Investigative Site, Paris, 75015, France|Novartis Investigative Site, Berlin, 13353, Germany|Novartis Investigative Site, Essen, 45147, Germany|Novartis Investigative Site, Koeln, 50937, Germany|Novartis Investigative Site, Stuttgart, 70376, Germany|Novartis Investigative Site, Accra, 0437-0594, Ghana|Novartis Investigative Site, Athens, 115 27, Greece|Novartis Investigative Site, Patras, 265 00, Greece|Novartis Investigative Site, Thessaloniki, GR 54636, Greece|Novartis Investigative Site, Bhubaneswar, Odisha, 751003, India|Novartis Investigative Site, Hyderabad, Telangana, 500082, India|Novartis Investigative Site, Genova, GE, 16128, Italy|Novartis Investigative Site, Milano, MI, 20122, Italy|Novartis Investigative Site, Verona, VR, 37126, Italy|Novartis Investigative Site, Napoli, 80138, Italy|Novartis Investigative Site, Irbid, 22110, Jordan|Novartis Investigative Site, Beirut, 1107 2020, Lebanon|Novartis Investigative Site, Tripoli, 1434, Lebanon|Novartis Investigative Site, Rotterdam, Zuid Holland, 3015 GD, Netherlands|Novartis Investigative Site, Amsterdam, 1105 AZ, Netherlands|Novartis Investigative Site, Den Haag, 2545 CH, Netherlands|Novartis Investigative Site, Muscat, 123, Oman|Novartis Investigative Site, Panama City, Republica De Panama, 0801, Panama|Novartis Investigative Site, Panama City, 0801, Panama|Novartis Investigative Site, Panama, 0801, Panama|Novartis Investigative Site, Soweto, Gauteng, 2013, South Africa|Novartis Investigative Site, Barcelona, Catalunya, 08035, Spain|Novartis Investigative Site, Madrid, 28034, Spain|Novartis Investigative Site, Madrid, 28046, Spain|Novartis Investigative Site, Adana, 01330, Turkey|Novartis Investigative Site, Antakya Hatay, 31100, Turkey|Novartis Investigative Site, Sheffield, South Yorkshire, S10 2JF, United Kingdom|Novartis Investigative Site, Cambridge, CB2 0QQ, United Kingdom|Novartis Investigative Site, London, E1 1BB, United Kingdom|Novartis Investigative Site, London, SE1 9RT, United Kingdom|Novartis Investigative Site, London, SE5 9RS, United Kingdom|Novartis Investigative Site, Sheffield, S10 2TH, United Kingdom |
| Allogeneic Genoidentical Stem Cell Transplantation in Children With Sickle-cell Anemia and Cerebral Vasculopathy | The aim of this study is to demonstrate that cerebral velocities assessed by transcranial doppler (TCD) are more significantly decreased by SCT than by long-term transfusion program A multicenter, national, non-randomized, prospective study of paired cohort will be conducted, with 2 groups of exposed (SCT) and non-exposed (TP) patients. | Sickle Cell Anemia|Cerebrovascular Accident | ALL | CHILD | CHIC, Créteil, France |
| The Impact of Oxidative Stress on Erythrocyte Biology | This study will address if red blood cells transfused to a sickle cell patient from a donor with a glucose-6-phosphate-dehydrogenase (G6PD) enzyme deficiency have a different lifespan as measured by the percentage of red blood cells that survive post-transfusion compared to red blood cells transfused to a sickle cell patient from a donor without a G6PD enzyme deficiency. | Sickle Cell Disease Without Crisis | ALL | ADULT | University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, 27599, United States |
| A Socio-ecological Approach for Improving Self-management in Adolescents With SCD | The goal of this clinical trial is to evaluate the impact of SCThrive (a behavioral self-management intervention) on patient activation, self-management behaviors, daily functioning, and emergency room visits in 260 adolescents and young adults with sickle cell disease (SCD) ages 13-21 receiving care at 1 of 4 pediatric SCD clinics. The main question\[s\]it aims to answer are: * Does SCThrive improve patient activation? * Does SCThrive improve self-management behaviors, daily functioning, and decrease emergency room visits? * Are any improvements maintained 3 months after treatment? Participants will complete self-management related surveys before, after, and 3 months following their participation in an 8- week, virtual group intervention with an accompanying mobile app (SCThrive). Researchers will compare outcomes for participants who receive SCThrive and participants who receive uniform standard care (SCHealthED which = standard of care plus SCD educational text messages) to see if there are differences in patient activation, self-management behaviors, daily functioning, and emergency room visits. | Anemia, Sickle Cell | ALL | CHILD, ADULT | |
| A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301 | The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301. | Sickle Cell Disease|Transfusion-dependent Beta-Thalassemia|Hemoglobinopathies | ALL | CHILD, ADULT | Cleveland Clinic, Cleveland, Ohio, 44195, United States |
| Study of SANGUINATE™ In the Treatment of Sickle Cell Disease Patients With Vaso-Occlusive Crisis | Safety and effect of SANGUINATE on Sickle Cell Disease patients experiencing a vaso-occlusive crisis | Anemia, Sickle Cell | ALL | ADULT, OLDER_ADULT | FSCDR, Hollywood, Florida, 33021, United States|Florida Health Tampa General Hospital, Tampa, Florida, 33606, United States|University of Maryland School of Medicine, Baltimore, Maryland, 21201-1559, United States|Johns Hopkins Univeristy School of Medicine, Baltimore, Maryland, 21205, United States|Newark Beth Israel Medical Center, Newark, New Jersey, 07112, United States|University of Rochester Medical Center, Rochester, New York, 14627-0140, United States|Ohio State University Medical Center, Columbus, Ohio, 43203, United States|Virginia Commonwealth University, Richmond, Virginia, 23298, United States|Blood Center of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia | The purpose of this study is to determine if hydroxyurea therapy is effective in the prevention of chronic end organ damage in pediatric patients with sickle cell anemia. | Hematologic Diseases|Anemia, Sickle Cell | ALL | CHILD | University of Alabama at Birmingham, Birmingham, Alabama, 35233, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States|Howard University, Washington, District of Columbia, 20060, United States|University of Miami, Miami, Florida, 33136, United States|Emory University School of Medicine, Atlanta, Georgia, 30342, United States|Johns Hopkins University, Baltimore, Maryland, 21287, United States|Children's Hospital of Michigan/Wayne State Univ., Detroit, Michigan, 48201, United States|University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States|SUNY Health Science Center, Brooklyn, Brooklyn, New York, 11203, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|Drexel University, Philadelphia, Pennsylvania, 19134, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|University of Texas SW Medical Center, Dallas, Texas, 75390, United States |
| Study of Decitabine and Tetrahydrouridine (THU) in Patients With Sickle Cell Disease | The purposes of this study are to observe if oral tetrahydrouridine and decitabine can increase fetal hemoglobin levels and improve the symptoms of sickle cell disease, and to monitor how patient's bodies react to oral tetrahydrouridine and decitabine. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of Illinois at Chicago, Chicago, Illinois, 60612, United States |
| ElectroNic Hydroxyurea AdhereNCE: A Strategy to Improve Hydroxyurea Adherence in Patients With Sickle Cell Disease | HU is an FDA approved medication for the treatment of SCD. Many studies have shown that HU can reduce SCD related symptoms, but only 50% of patients take it as often as they should. This limits how much HU can help reduce SCD symptoms. Researchers are interested to see if electronic directly observed therapy (Mobile DOT), a program that uses cell phone reminder messages, videos, feedback messages, and incentives will help patients with SCD take HU as prescribed. | Sickle Cell Disease | ALL | CHILD, ADULT | Nationwide Children's Hospital, Columbus, Ohio, 43205, United States |
| Gum Arabic as Fetal Hemoglobin Agent in Sickle Cell Anemia | The purpose of this study is to determine whether Gum Arabic is effective as fetal hemoglobin inducing agent for sickle cell anemia patients. | Sickle Cell Anemia | ALL | CHILD, ADULT | Military Hospital, Omdurman, Khartoum, 1113, Sudan |
| A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease | This study is being done to learn about etavopivat, a once a day medicine taken by mouth in adolescents with sickle cell disease. The main goals are to study safety and how long etavopivat stays in the bloodstream, while also studying if there are benefits from taking etavopivat. Eligible participants who enter the study will start a 96-week treatment period. At the end of the 96 weeks, participants will have an end of study visit that occurs 4 weeks later. The participants will receive etavopivat every day throughout the treatment period. | Sickle Cell Disease | ALL | CHILD, ADULT | The Hospital for Sick Children, Toronto, Ontario, M5G 1X8, Canada|KEMRI-Walter-Reed Kericho, Kericho, 20200, Kenya|Ahero Clinical Trials Unit, Kisumu, Kenya, Kisumu, 40100, Kenya|Kombewa Clinical Research Centre, Kisumu, 40100, Kenya|Kenya Medical Research Institute-Centre for Respiratory Disease Research, Siaya Clinical Research Annexe, Siaya, 40100, Kenya|American University of Beirut Medical center, Beirut, 1107 2020, Lebanon|Hospital Nini, Tripoli, 113-6044, Lebanon|University of Nigeria Teaching Hospital, Enugu, Enugu, 00000, Nigeria|Lagos University Teaching Hospital, Lagos, Lagos, 102215, Nigeria|Aminu Kano Teaching Hospital (AKTH), Tarauni, 700101, Nigeria|Hacettepe University pediatric hematology, Ankara, 06100, Turkey|Acibadem Adana Hospital, Seyhan, 1130, Turkey|Guys and St Thomas NHS Foundation Trust / Evelina Childrens Hospital, London, SE1 7EH, United Kingdom|Kings College Hospital NHS Foundation Trust, London, SE5 9RS, United Kingdom|Manchester Royal Infirmary, Manchester, M13 9WL, United Kingdom |
| Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia | The purpose of this study is to find out if using a lower dose of chemotherapy before stem cell transplantation can cure patients of sickle cell anemia or thalassemia while causing fewer severe side effects than conventional high dose chemotherapy with transplantation. | Hemoglobinopathies|Anemia, Sickle Cell|Hemoglobin SC Disease|Thalassemia|Thalassemia Major | ALL | ADULT, OLDER_ADULT | Dana-Farber Cancer Institute/Harvard Cancer Center, Brigham and Women's Hospital and Massachusetts General Hospital, Boston, Massachusetts, 02115, United States |
| Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of ALXN1820 in Adult Participants With Sickle Cell Disease | The primary objective of this study is to assess the safety and tolerability of ALXN1820 SC (subcutaneous) in participants with SCD (Sickle Cell Disease). | Sickle Cell Disease (SCD) | ALL | ADULT, OLDER_ADULT | Research Site, Hollywood, Florida, 33023, United States|Research Site, Indianapolis, Indiana, 46260, United States |
| Blood Collection for Research Related to Certain Diseases Involving Blood Vessels | The collection of human specimens from both patients and healthy volunteers is necessary for the development of laboratory assays to further basic and clinical research studies. This protocol defines the purposes for which specimens will be collected and establishes general conditions under which sample collection will be performed.... | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States|INOVA Fairfax Hosptial, Fairfax, Virginia, 22042, United States |
| BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease | This is an open-label, single-arm, multicenter, Phase 1/2 study evaluating the safety and efficacy of the administration of autologous base edited CD34+ HSPCs (BEAM-101) in patients with severe SCD | Sickle Cell Disease | ALL | ADULT | University of Alabama at Birmingham, Birmingham, Alabama, 35233, United States|Phoenix Children's Hospital, Phoenix, Arizona, 85016, United States|Lucile Packard Children's Hospital at Standford, Palo Alto, California, 94304, United States|Mayo Clinic Florida, Jacksonville, Florida, 32224, United States|University of Miami, Miami, Florida, 33136-1005, United States|Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center - Egleston Hospital, Atlanta, Georgia, 30329, United States|Boston Children's Hospital, Boston, Massachusetts, 02215, United States|Henry Ford Cancer Center, Detroit, Michigan, 48202, United States|University of Minnesota, Minneapolis, Minnesota, 55455, United States|Washington University School of Medicine in St. Louis, Saint Louis, Missouri, 63110, United States|Hackensack University Medical Center, Hackensack, New Jersey, 07601, United States|Columbia University Irving Medical Center, New York, New York, 10032, United States|Memorial Sloan Kettering Cancer Center, New York, New York, 10065, United States|University Hospitals Cleveland Medical Center, Cleveland, Ohio, 44106, United States|The Cleveland Clinic Foundation, Cleveland, Ohio, 44195, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|St Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|The Children's Hospital at TriStar Centennial, Nashville, Tennessee, 37203, United States|Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia | Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin. Haemoglobin is the protein that carries oxygen through the body. This study is looking into how safe treatment with etavopivat is and how well it works over a long period of time. The study will last for up to 264 weeks, but it will end earlier if etavopivat is approved in the participant's country. | Sickle Cell Disease, Thalassemia | ALL | CHILD, ADULT, OLDER_ADULT | Univ of Alabama Birmingham, Birmingham, Alabama, 35233, United States|Phoenix Children's Hsptl, Phoenix, Arizona, 85016, United States|University Of California Irvine, Irvine, California, 92697, United States|Children's Hospital Los Angeles - Endocrinology, Los Angeles, California, 90027, United States|UCSF Oakland Benioff ChildHosp, Oakland, California, 94609, United States|Children's Hosp Of Orange, Orange, California, 92868, United States|University of Connecticut, Farmington, Connecticut, 06030, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States|Univ Miami-Miller School Med, Miami, Florida, 33136, United States|Emory University School of Medicine, Atlanta, Georgia, 30303, United States|Children's Healthcare Atlanta, Atlanta, Georgia, 30342, United States|Children's Hosp-New Orleans, New Orleans, Louisiana, 70118, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|Jacobi Medical Center, Bronx, New York, 10461, United States|Montefiore Medical Center, Bronx, New York, 10467, United States|NYC Health+Hospitals, Brooklyn, New York, 11203, United States|Columbia University Medical Center_New York_0, New York, New York, 10032, United States|Weill Cornell Med Coll-NYPH, New York, New York, 10065, United States|Duke University_Durham, Durham, North Carolina, 27705, United States|East Carolina University_Greenville, Greenville, North Carolina, 27834, United States|Atrium Health-Wake Forest Bapt, Winston-Salem, North Carolina, 27157, United States|Cincinnati Child's Hsp Med Ctr, Cincinnati, Ohio, 45229, United States|Neuro-Behavioral Clinical Research, North Canton, Ohio, 44720, United States|Medical University Of South Carolina_Charleston, Charleston, South Carolina, 29425, United States|East Carolina University_Greenville_0, Greenville, South Carolina, 27858, United States|UTHSC-Memphis, Memphis, Tennessee, 38104, United States|UT Health University of Texas, Houston, Texas, 77030, United States|Virginia Comm Univ Medical Ctr, Richmond, Virginia, 23298, United States|Mary Bridge Children's Health, Tacoma, Washington, 98405, United States|Providence Hematolgy, Vancouver, British Columbia, V6E 1M7, Canada|The Hospital for Sick Children, Toronto, Ontario, M5G 1X8, Canada|University Health Network - Toronto General Hospital, Toronto, Ontario, M5G 2C4, Canada|CHU Ste-Justine, Montreal, Quebec, H3T 1C5, Canada|Ap-Hp-Hopital Henri Mondor, Créteil, 94000, France|Hospices Civils de Lyon-Hopital Edouard Herriot, Lyon Cedex 03, 69437, France|Ap-Hp-Hopital Robert Debre, Paris, 75019, France|Charité Campus Virchow Klinikum - Klinik für Pädiatrie mit Schwerpunkt Onkologie und Hämatologie, Berlin, 13353, Germany|Universitätsklinikum Freiburg, Kinder- und Jugendklinik, Freiburg, 79106, Germany|General Hospital Of Larissa Koutlibaneio And Triantafylleio, Larissa, Thessaly, 412 21, Greece|Hippokration Hospital, Athens, 11527, Greece|General University Hospital of Patras, Patra, 26504, Greece|'Ippokrateio' General Hospital of Thessaloniki, Thessaloniki, 54642, Greece|Azienda Ospedaliera Universitaria San Luigi Gonzaga, Orbassano, Torino, 10043, Italy|Azienda Ospedale Universita Padova, Padova, 35128, Italy|Fondazione IRCCS Policlinico San Matteo, Pavia, 27100, Italy|American University of Beirut Medical Centre, Hamra, Lebanon|Chronic Care Center, Hazmieh, 21211, Lebanon|Hospital Nini, Tripoli, 1434, Lebanon|Sultan Qaboos University Hospital, Muscat, 123, Oman|Prince Mohammad Bin Naser Hospital, Jizan, 82943, Saudi Arabia|King Khalid University Hospital, Riyadh, 12372, Saudi Arabia|Hospital Universitario de Cruces, Baracaldo, 48903, Spain|Hospital Vall d'Hebron, Barcelona, 08035, Spain|Hospital Universitario La Paz, Madrid, 28046, Spain|Baskent Universitesi Adana, Adana, 01250, Turkey|Hacettepe University Hematology, Ankara, 06230, Turkey|Mersin University Medical Faculty Hospital, Hematology, Mersin, 33110, Turkey|Mersin University Medical Faculty Pediatric Hematology, Mersin, 33110, Turkey |
| STRIDE Biorepository | The STRIDE Biorepository is an optional substudy available to participants in "Bone Marrow Transplantation vs Standard of Care in Patients with Severe Sickle Cell Disease (BMT CTN 1503) (STRIDE)". | Anemia, Sickle Cell | ALL | CHILD, ADULT | Benioff Children's Hospital at Oakland, Oakland, California, 94609, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States|University of Florida Gainsville, Gainesville, Florida, 32611, United States|Foundation for Sickle Cell Research/Florida Sickle Inc., Hollywood, Florida, 33021, United States|University of Miami, Miami, Florida, 33136, United States|Grady Hospital, Atlanta, Georgia, 30303, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States|Emory Children's Center, Atlanta, Georgia, 30322, United States|Emory University, Atlanta, Georgia, 30322, United States|Augusta University Medical Center, Augusta, Georgia, 30912, United States|University of Chicago, Chicago, Illinois, 60637, United States|University of Iowa, Iowa City, Iowa, 52242, United States|Children's Hospital of New Orleans, New Orleans, Louisiana, 70118, United States|Oschner Medical Center, New Orleans, Louisiana, 70121, United States|Dana Farber Cancer Institute/Brigham and Women's Hospital, Boston, Massachusetts, 02214, United States|Boston University, Boston, Massachusetts, 02215, United States|Dana Farber Cancer Institute/Massachusetts General Hospital, Boston, Massachusetts, 02215, United States|University of Michigan Medical Center, Ann Arbor, Michigan, 48105, United States|Barbara Ann Karmanos Cancer Institute, Detroit, Michigan, 48201, United States|Washington University/St. Louis Children's Hospital, Saint Louis, Missouri, 63110, United States|Hackensack University Medical Center, Hackensack, New Jersey, 07601, United States|Newark Beth Israel Medical Center, Newark, New Jersey, 07112, United States|Montefiore Medical Center/Albert Einstein School of Medicine, Bronx, New York, 10467, United States|New York Presbyterian Brooklyn Methodist Hospital, Brooklyn, New York, 11215, United States|Cohen Children's Medical Center, New Hyde Park, New York, 11040, United States|Icahn School of Medicine at Mount Sinai, New York, New York, 10029, United States|Weill Cornell Medical College, New York, New York, 10065, United States|University of North Carolina Hospital at Chapel Hill, Chapel Hill, North Carolina, 27516, United States|Duke University Medical Center, Durham, North Carolina, 27705, United States|Ohio State University, Columbus, Ohio, 43210, United States|University of Oklahoma, Oklahoma City, Oklahoma, 73104, United States|Oregon Health Sciences University, Portland, Oregon, 97239, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, 15224, United States|Medical University of South Carolina, Charleston, South Carolina, 29435, United States|University of Texas Health Sciences Center, Houston, Texas, 77004, United States|Baylor College of Medicine/The Methodist Hospital, Houston, Texas, 77030, United States|University of Texas/MD Anderson CRC, Houston, Texas, 77030, United States|University of Virginia, Charlottesville, Virginia, 22908, United States|Virginia Commonwealth University, Richmond, Virginia, 23298, United States |
| Expanded Access Protocol for Adults and Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options | The objective of this Expanded Access Protocol (EAP) is to provide voxelotor for the treatment of sickle cell disease (SCD): The study GBT440-041 is the EAP for pediatric patients aged 6 months to 11 years who have no alternative treatment options and are ineligible to participate in a clinical trial of voxelotor and the study C5341057 is the EAP for adults/adolescents 12 years of age and older for patients who cannot satisfactorily be treated with an authorized medicinal product | Sickle Cell Disease | ALL | CHILD | Phoenix Children's Hospital, Phoenix, Arizona, 85016, United States|Rady Children's Hospital San Diego, San Diego, California, 92123, United States|Nemours/Alfred I. duPont Hospital for Children, Wilmington, Delaware, 19803, United States|Children's National Hospital / Children's National Health System, Washington, District of Columbia, 20010, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States|Baptist Medical Center/Wolfson Children's Hospital, Jacksonville, Florida, 32207, United States|Nemours Children's Health, Jacksonville, Jacksonville, Florida, 32207, United States|Nemours Children's Hospital, Orlando, Florida, 32827, United States|Children's Healthcare of Atlanta Scottish Rite, Atlanta, Georgia, 30342, United States|AU Medical Center Clinical Research Pharmacy, Augusta, Georgia, 30912, United States|Augusta University, Augusta, Georgia, 30912, United States|Children's Hospital of Georgia, Augusta, Georgia, 30912, United States|Memorial Health University Medical Center, Savannah, Georgia, 31404, United States|Tulane Lakeside, Metairie, Louisiana, 70001, United States|Tulane University Hospitals and Clinics, New Orleans, Louisiana, 70112, United States|Children's Hospital, New Orleans, Louisiana, 70118, United States|Childrens Hospital of NOLA, New Orleans, Louisiana, 70118, United States|University Of Michigan Hospitals, Ann Arbor, Michigan, 48109, United States|Rutgers Cancer Institute of New Jersey, New Brunswick, New Jersey, 08901, United States|Newark Beth Israel Medical Center & Children's Hospital of New Jersey, Newark, New Jersey, 07112, United States|BronxCare Health System, Bronx, New York, 10457, United States|Jacobi Medical Center, Bronx, New York, 10461, United States|East Carolina University Brody School of Medicine(ECU), Greenville, North Carolina, 27834, United States|ECU Physicians, Brody Outpatient Clinic, Greenville, North Carolina, 27834, United States|University Hospitals Cleveland Medical Center, Cleveland, Ohio, 44106, United States|Medical University of South Carolina: Investigational Drug Services Pharmacy, Charleston, South Carolina, 29425, United States|Medical University of South Carolina: Shawn Jenkins Women's and Children's Hospital, Charleston, South Carolina, 29425, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|Prisma Health - Upstate, Greenville, South Carolina, 29605, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|Cook Children's Health Care System, Fort Worth, Texas, 76104, United States|Pediatric Specialists of Virginia (Inova Ashburn HealthPlex), Ashburn, Virginia, 20148, United States|Pediatric Specialists of Virginia (Schar Cancer Institute), Fairfax, Virginia, 22031, United States|Multihemo Serviços Médicos S/A, Recife, Pernambuco, 50070-460, Brazil|Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP, Ribeirão Preto, SAO Paulo, 14048-900, Brazil|HEMORIO - Hematologia Laboratorial, Rio de Janeiro, 20211-030, Brazil|ESHO Empresa de Serviços Hospitalares S.A/ Hospital Samaritano de Higienópolis, São Paulo, 01232-010, Brazil |
| Comparison of Two Methods of Transfusion for Stroke Prevention in Sickle Cell | Chronic blood transfusions are essential supportive care for sickle cell patients at high risk for morbidity and mortality due to stroke. These patients, however, are at risk for iron overload. In the investigator's comprehensive sickle cell center, the investigators support chronic transfusion with rapid manual partial exchange transfusions (RMPET) using a single access central line port. The investigators do not have a comprehensive adult sickle cell program but upon transition of patients the patients would be provided simple transfusion (ST) in an adult ambulatory infusion setting due to nursing acuity needed for RMPET. The investigators plan to study the institution's participants currently on chronic transfusion support and compare different transfusion modalities to better understand the effects from switching from RMPET to ST. To date, there are no such comparisons within and between sickle cell patients in the literature. | Anemia, Sickle Cell|Sickling Disorder Due to Hemoglobin S | ALL | CHILD, ADULT | Chidlren's Hospital at Erlanger, Chattanooga, Tennessee, 37403, United States |
| The Brigance Assessment Of Individual Neurodevelopment In Young Children With Sickle Cell Disease- 2 | A preliminary study was conducted involving 88 three-year-old children with sickle cell disease (SCD) who were followed at the St. Jude Children's Research Hospital Sickle Cell Center. They were offered developmental screening with the Brigance Preschool Screen-II test during their regular clinic visits from January 2006 to August 2008. Data from this work showed that 50% of 3 year old children with SCD had low developmental screening scores. In addition, the low scores were found to be associated with less parental education and with speech deficits. However they were not associated with sickle cell genotype and hemoglobin level. The primary goal of this study is to prospectively administer Brigance Preschool Screen -II to 3 year old children with SCD and 3 year old children without SCD who come from similar socioeconomic backgrounds and compare the results between the two groups. | Sickle Cell Disease | ALL | CHILD | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Hydroxyurea and Transfusion | This study will prospectively investigate the feasibility, safety, and transfusion requirements of adding hydroxyurea to simple chronic transfusions for patients with sickle cell anemia already on chronic transfusions. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's National Health System, Washington, District of Columbia, 20010, United States |
| A Stress and Pain Self-management m-Health App for Adult Outpatients With Sickle Cell Disease | Our long-term goal is to reduce stress and improve sickle cell disease (SCD) pain control with less opioid use through an intervention with self-management relaxation/distraction exercises (RDE), named You Cope, We Support (YCWS). Americans living with SCD suffer recurrent episodes of acute and chronic pain, both of which are exacerbated by stress. Building on the successes of our prior formative studies, we now propose a well-designed, appropriately powered study to test efficacy of YCWS on outcomes (pain intensity, stress intensity, opioid use) in adults with SCD. We propose to recruit 170 adults for a randomized controlled trial of the short-term (8 weeks) and long-term (6 months) effects of YCWS on outcomes (pain, stress, and opioid use). Stratified on worst pain intensity (\<=5; \>5), we will randomly assign patients to groups: 85 to Control (Self-monitoring of outcomes + alerts/reminders), and 85 to Experimental (Self-monitoring of outcomes + alerts/reminders + use of YCWS \[RDE + Support\]). We will ask participants to report outcomes daily. During weeks 1-8, we will send system-generated alerts/reminders to both groups via phone call, text, or email to facilitate data entry (both groups) and intervention use support (experimental). If the patient does not enter data after 24 hours, study support staff will contact him/her for data entry trouble-shooting (both groups) and YCWS use (experimental). We will time stamp and track participants' online activities to understand the study context and conduct exit interviews on acceptability of the staff and system-generated support. We will analyze data using mixed-effects regression models (short-term, long-term) to account for repeated measurements over time and utilize machine learning to construct and evaluate models predictive of outcomes. Specific aims are: Aim 1: To determine the short-term effects of YCWS. Aim 2: To determine the long-term effects of YCWS. Aim 3 (exploratory): Using machine learning, to develop and evaluate models that predict patient outcomes based on their group assignment and on their personal (e.g., self-efficacy, sex, education, family income, computer experience, etc.,) and environmental characteristics (e.g., distance from care, quality of cell connection, etc.). | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of Florida, Gainesville, Florida, 32610, United States |
| Dipyridamole/Magnesium To Improve Sickle Cell Hydration | The purpose of this study is to determine the benefits as well as side effects of giving drugs called dipyridamole and magnesium to patients with sickle cell anemia (SCA). | Anemia, Sickle Cell | ALL | CHILD, ADULT, OLDER_ADULT | Karmanos Cancer Institute, Detroit, Michigan, 48201, United States|University of Cincinnati, Cincinnati, Ohio, 45219, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States |
| Glutamine Role in Preventing Vaso-occlusive Crisis Among SCD Patients | Prospective phase IV interventional open label randomized controlled trial to assess safety and efficacy of glutamine in preventing vaso-occlusive crisis (VOC) episodes in sickle cell pediatrics and adolescents' patients | Sickle Cell Disease | ALL | CHILD, ADULT | Faculty of Medicine Ain Shams University Research Institute- Clinical Research Center, Cairo, Non-US, 11566, Egypt|Ain Shams University, Cairo, 11566, Egypt |
| Open-Label Extension of Voxelotor | Open-label extension study of voxelotor for participants with Sickle Cell Disease who have participated in voxelotor clinical trials. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's National Medical Center, Washington, District of Columbia, 20010, United States|Children's Healthcare of Atlanta Scottish Rite, Atlanta, Georgia, 30342, United States|Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States|Children's Mercy Hospital, Kansas City, Missouri, 64108, United States|ECU Physicians, Brody Outpatient Clinic, Greenville, North Carolina, 27834, United States|UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, 15224, United States|Zagazig University Hospital, Zagazig, Alsharkia, Egypt|Alexandria University Hospital - Clinical Research Center, Alexandria, 21131, Egypt|Cairo University Hospital, Abu El Rish Hospital, Cairo, 11562, Egypt|Ain Shams University Hospital - Clinical Research Center (MASRI-CRC), Cairo, 11566, Egypt|Ain Shams University Hospital - Clinical Research Center (MASRI-CRC), Cairo, Egypt|Cairo University Hospital, Abu El Rish Hospital, Cairo, Egypt|American University of Beirut - Medical Center, Beirut, Lebanon|Nini Hospital, Tripoli, Lebanon|University of Calabar Teaching Hospital, Calabar, Cross River, 540281, Nigeria|University of Calabar Teaching Hospital, Calabar, Cross River, PMB, Nigeria|College of Medicine, University of Ibadan, Ibadan, OYO State, 200212, Nigeria|University College Hospital Paediatric Haematology and Oncology Unit, Ibadan, OYO State, 200281, Nigeria|Barau Dikko Teaching Hospital/Kaduna State University, Kaduna, 800212, Nigeria|Aminu Kano Teaching Hospital, Kano, 700233, Nigeria|Lagos University Teaching Hospital, Lagos, 100254, Nigeria|University College Hospital NHS Foundation Trust, London, Greater London, NW1 2PG, United Kingdom|Barts Health NHS Trust , The Royal London Hospital, London, E1 1BB, United Kingdom|Guy's & St Thomas NHS Trust, St Thomas' Hospital, London, SE1 7EH, United Kingdom|Guy'S and St Thomas' Nhs Foundation Trust, London, SE1 7EH, United Kingdom |
| Computerized Cognitive Behavioral Therapy Assisted Life Management for Pain in Sickle Cell Disease | The primary aim of this study is to test the feasibility and acceptability of implementing a multimedia computerized cognitive behavioral therapy (cCBT) program for reducing SCD pain symptoms in a single-arm pilot pragmatic clinical trial. The investigators will recruit 40 SCD patients with chronic pain and/or on chronic opioid pain treatment and randomize them 3:1 to two groups (cCBT and e-Education respectively), randomizing unevenly in order to best gather feasibility data for the cCBT. Both groups will use a mobile app to track daily pain/mood. The cCBT group will receive sessions of the CALM-SCD program to complete via mobile device and will have weekly follow-up with a care coach. The Education group will receive online education modules to complete via mobile device and will also have weekly follow-up with a care coach. The primary outcomes of the trial include feasibility (recruitment, retention, provider and patient feedback) and acceptability (sessions completed) of the CALM-SCD program. | Sickle Cell Disease|Chronic Pain | ALL | ADULT, OLDER_ADULT | University of Pittsburgh, Pittsburgh, Pennsylvania, 15237, United States |
| Microvascular and Fibrosis Imaging Study | In this study, Laser Doppler Flowmetry (LDF), Laser Doppler Imaging (LDI), Orthogonal Polarization Spectral Imaging (OPSI), Nail fold video capillaroscopy (NVC) and Optical Coherence Tomography (OCT) will be used to assess differences in microvascular function and density of oral mucosa and skin in subjects with 1) autoimmune diseases with cutaneous involvement: systemic sclerosis (SSc), morphea, dermatomyositis, cutaneous lupus and vasculitis, 2) sickle cell disease (SCD) and 3) chronic graft-versus-host disease (GVHD) compared to healthy subjects. The microvascular changes will be compared to overall treatment response in patients with scleroderma and chronic GVHD as assessments will be made before and after the patients start treatment for their diseases and determine if these imaging techniques provide valuable and reproducible data when assessing a patient's response to treatment for those diseases. In addition, the application of Acoustic Radiation Force Impulse (ARFI) in determining cutaneous thickness in patients with SSc, GVHD and morphea will be evaluated. The investigators hypothesize that the vascular and dermal structures are altered in patients with autoimmune disease, SCD and chronic GVHD. In addition, they hypothesize that imaging modalities such as LDF, LDI, OCT, NVC, OPSI and ARFI can quantify such structural alterations and can be used to 1) detect early disease activity, 2) quantify and assess response to therapy and 3) quantify and correlate with overall disease activity. | Autoimmune Diseases|Sickle Cell Disease|Chronic Graft Versus Host Disease | ALL | ADULT, OLDER_ADULT | Duke University Medical Center, Durham, North Carolina, 27710, United States |
| Low-Dose Ketamine Infusion for Children With Sickle Cell Disease-Related Pain | Acute pain episodes associated with sickle cell disease (SCD) are very difficult to manage effectively. Opioid tolerance and side effects have been major roadblocks in our ability to provide these patients with adequate pain relief. This pilot study is designed to examine the safety and feasibility of using ketamine, an N-methyl-D-aspartate (NMDA) receptor antagonist, in the inpatient seeing with children and adolescents who have sickle cell vasoocclusive pain. Previous research suggests that in subanesthetic doses, ketamine may be able to prevent the development of opiate tolerance and facilitate better pain relief with lower opiate doses, allowing for less respiratory depression, less sedation, easier ambulation, less deconditioning, shorter hospital stays, and better quality of life. The goal of this pilot study is to evaluate the safety and feasibility of using a continuous infusion of ketamine, in conjunction with opiates, in the inpatient setting for sickle cell vasoocclusive pain. It is hypothesized that using a low dose ketamine infusion in conjunction with opiates will be a safe and feasible practice for the treatment of sickle cell pain. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | University of Connecticut Health Center, Farmington, Connecticut, 06030, United States|Connecticut Children's Medical Center, Hartford, Connecticut, 06106, United States |
| A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease | A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide). | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of Illinois at Chicago College of Medicine, Chicago, Illinois, 60612, United States |
| Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031 | Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical Trials | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Arkansas Primary Care Clinic, PA, Little Rock, Arkansas, 72204, United States|UCSF Benioff Children's Hospital Oakland, Oakland, California, 94609, United States|Jackson Memorial Hospital (Investigational Drug Services), Miami, Florida, 33136, United States|Jackson Memorial Hospital, Miami, Florida, 33136, United States|University of Miami Hospital & Clinics/SCCC, Research Pharmacy (Investigational Drug Services), Miami, Florida, 33136, United States|University of Miami, Miami, Florida, 33136, United States|Children's Healthcare of Atlanta at Hughes Spalding, Atlanta, Georgia, 30303, United States|Children's Healthcare of Atlanta - Scottish Rite, Atlanta, Georgia, 30322, United States|Children's Healthcare of Atlanta - Scottish Rite, Atlanta, Georgia, 30342, United States|University of Illinois at Chicago Clinical Research Center, Chicago, Illinois, 60612, United States|University of Illinois Hospital and Health Science System, Chicago, Illinois, 60612, United States|Indiana Hemophilia and Thrombosis Center, Indianapolis, Indiana, 46260, United States|St. Jude Affiliate Clinic Baton Rouge, Baton Rouge, Louisiana, 70808, United States|Our Lady of the Lake Physician Group-Medical Oncology, Baton Rouge, Louisiana, 70809, United States|University Medical Center New Orleans, New Orleans, Louisiana, 70112, United States|Johns Hopkins University/Sickle Cell Infusion Center, Baltimore, Maryland, 21287, United States|The Johns Hopkins Hospital, Baltimore, Maryland, 21287, United States|Brigham and Women's Hospital, Boston, Massachusetts, 02115, United States|Brigham and Women's Hospital Research Pharmacy, Boston, Massachusetts, 02215, United States|Dana Farber Cancer Institute, Boston, Massachusetts, 02215, United States|Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|Newark Beth Israel Medical Center, Newark, New Jersey, 07112, United States|Montefiore - Einstein Center for Cancer Care, Bronx, New York, 10461, United States|Columbia University Medical Center - Herbert Irving Pavilion, New York, New York, 10032, United States|UNC Hospitals, Chapel Hill, North Carolina, 27514, United States|Clinical and Translational Research Center (CTRC), Chapel Hill, North Carolina, 27599, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|Investigational Drug Service, Duke University Hospital, Durham, North Carolina, 27710, United States|Lynn Health Science Institute, Oklahoma City, Oklahoma, 73112, United States|Thomas Jefferson University, Philadelphia, Pennsylvania, 19107, United States|University of Pittsburgh Medical Center (UPMC), Pittsburgh, Pennsylvania, 15232, United States|Medical University of South Carolina - Comprehensive Sickle Cell Clinic, Charleston, South Carolina, 29425, United States|Medical University of South Carolina: Investigational Drug Services Pharmacy, Charleston, South Carolina, 29425, United States|Methodist Comprehensive Sickle Cell Clinic, Memphis, Tennessee, 38104, United States|Texas Children's Hospital - Investigational Pharmacy, Houston, Texas, 77030, United States|Texas Children's Hospital, Houston, Texas, 77030, United States|Clinical Research Services Unit- Virginia Commonwealth University, Richmond, Virginia, 23298, United States|Toronto General Hospital-University Health Network, Toronto, Ontario, M5G 2C4, Canada|Alexandria Clinical Research Center, Faculty of Medicine, Alexandria University, Alexandria, Egypt|Alexandria Clinical Research Center, Faculty of Medicine, Alexandria, Egypt|Zagazig University Hospital, Alsharkia, Egypt|Abu El Rich Hospital,Cairo University Hospital, Cairo, 11541, Egypt|Ain Shams University Hospital, Cairo, Egypt|The Egyptian Thalassemia Association ( E.T.A), Cairo, Egypt|Hôpital Européen Georges Pompidou - Medecine Interne, Paris, 75015, France|Hôpital Européen Georges Pompidou, Paris, Île-de-france, 75015, France|Azienda Ospedaliera di Padova, Padova, Padova Veneto, 35128, Italy|Centres for Disease Control and Prevention, Siaya, Kisumu, Kenya|KEMRI/CRDR - Kenya Medical Research Insititute - Center for respiratory Disease Research, Nairobi, 00101, Kenya|Gertrude's Children's Hospital, Nairobi, Kenya|American University of Beirut, Beirut, Lebanon|Nini Hospital, Tripoli, Lebanon|Academic Medical Center(AMC), Amsterdam, 1105 AZ, Netherlands|ErasmusMC, Rotterdam, 3015 CN, Netherlands|Sultan Qaboos University Hospital, Muscat, 123, Oman|Erciyes Universitesi Tip Fakultesi Hastanesi, Istanbul, İ̇stanbul, 38039, Turkey|Adana Acibadem Hospital, Adana, 01130, Turkey|Erciyes Universitesi Tip Fakultesi, Kayseri, 38039, Turkey|Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Hastanesi, Mersin, 33343, Turkey|Barts Health NHS Trust, London, Greater London, E1 1BB, United Kingdom|Homerton University Hospital NHS Foundation Trust, London, Greater London, E9 6SR, United Kingdom|Guys and St. Thomas Hospital NHS Foundation Trust, Great Maze Pond, London, SE1 9RT, United Kingdom|Guys and STt Thomas NHS Foundation Trust, Great Maze Pond, London, SE1 9RT, United Kingdom|King's College Hospital, London, SE5 9RS, United Kingdom|Imperial College Healthcare NHS Trust, Hammersmith Hospital, London, W12 0HS, United Kingdom|McMillan Cancer Centre, London, WC1E 6AG, United Kingdom |
| LCI-HEM-SCD-ST3P-UP-001: The Sickle Cell Trevor Thompson Transition Project (ST3P-UP Study) | This multi-center study will compare the effectiveness of adding virtual peer mentoring (PM) to a structured education-based (STE) transition program for emerging adults with sickle cell disease to determine its effect on decreasing the number of acute care visits per year, improving patient-reported outcomes, and reducing healthcare utilization among emerging adults with sickle cell disease (EA-SCD) | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | University of Alabama at Birmingham, Birmingham, Alabama, 35233, United States|University of South Alabama Health System, Mobile, Alabama, 36617, United States|University of Miami, Miami, Florida, 33136, United States|Johns Hopkins All Children's Hospital, Saint Petersburg, Florida, 33701, United States|Children's Healthcare of Atlanta/Emory University, Atlanta, Georgia, 30322, United States|University of Louisville, Louisville, Kentucky, 40202, United States|Montefiore Medical Center, Bronx, New York, 10467, United States|Levine Cancer Institute, Charlotte, North Carolina, 28204, United States|Novant Health, Charlotte, North Carolina, 28204, United States|Duke University, Durham, North Carolina, 27705, United States|East Carolina University, Greenville, North Carolina, 27834, United States|Wake Forest Baptist Hospital, Winston-Salem, North Carolina, 27157, United States|Greenville Health System, Greenville, South Carolina, 29605, United States|Virginia Commonwealth University (VCU), Richmond, Virginia, 23298, United States |
| SCD Fit Homebase Program | The purpose of this project is to develop novel approaches to promote health and longevity while enhancing quality of life among persons with Sickle cell disease (SCD). Therefore, investigators are aiming to adapt an evidence-based exercise intervention for adults with SCD informed by culturally- relevant and biologic factors. | Cardiovascular Diseases|Sickle Cell Disease|Exercise | ALL | ADULT, OLDER_ADULT | |
| Phase 1/2 Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 Administered Daily in Patients With Sickle Cell Disease | The purpose of this study is to assess the safety and tolerability of HQK-1001 administered for a total of 12 weeks (with one dosing break) in subjects with sickle cell disease. | Sickle Cell Disease | ALL | CHILD, ADULT | Trialogic Research, Madison, Alabama, 35758, United States|Children's Hospital and Research Center at Oakland, Oakland, California, 94609, United States|Century Clinical Research, Inc., Daytona Beach, Florida, 32117, United States|Medical College of Georgia, Augusta, Georgia, 30912, United States|University of Illinois at Chicago, Chicago, Illinois, 60612, United States|Johns Hopkins School of Medicine, Baltimore, Maryland, 21205, United States|UNC Comprehensive Sickle Cell Program, Chapel Hill, North Carolina, 27599, United States|Texas Children's Cancer Center and Hematology Service, Houston, Texas, 77030, United States|University of the West Indies, Mona, Kingston, Mona, 7, Jamaica |
| Endothelial Monocyte-activating Polypeptide-II in Egyptian Sickle Patients | This study objectives to assess the role of endothelial monocyte-activating polypeptide II (EMAP II) as a marker of endothelial dysfunction and disturbed angiogenesis in sickle cell disease and to identify its correlation With the oxidative status. | Sickle Cell Disease | ALL | CHILD, ADULT | |
| Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC) | The purpose of this study is to evaluate whether MST-188 can reduce the duration of vaso-occlusive crisis (VOC) in subjects with sickle cell disease. The study will also evaluate whether MST-188 can reduce the frequency of rehospitalization of subjects due to a recurrence of VOC. Additionally, this study will compare the development of acute chest syndrome during VOC in subjects who receive MST-188 to those who do not receive MST-188. | Vaso-occlusive Crisis|Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | University of South Alabama, Mobile, Alabama, 36688, United States|Phoenix Children's Hospital, Phoenix, Arizona, 85016, United States|UCSF Benioff Children's Hospital, Oakland, California, 94609, United States|University of California Davis Health System, Sacramento, California, 95817, United States|Rady Children's Hosptial, San Diego, California, 92123, United States|Harbor-UCLA Medical Center, Torrence, California, 90502, United States|Al DuPont Hospital for Children, Wilmington, Delaware, 19803, United States|Howard University, Washington, District of Columbia, 20060, United States|Children's Hospital of SouthWest Florida, Fort Myers, Florida, 33908, United States|Joe Dimaggio Children's Hospital, Hollywood, Florida, 33021, United States|University of Miami, Miami, Florida, 33136, United States|Miami Children's Hospital, Miami, Florida, 33155, United States|All Children's Hospital, St. Petersburg, Florida, 33701, United States|Tampa General Hospital, Tampa, Florida, 33606, United States|Grady Memorial Hospital, Atlanta, Georgia, 30303, United States|Georgia Regents University, Augusta, Georgia, 30912, United States|Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States|Children's Hospital at the University of Illinois, Chicago, Illinois, 60612, United States|Fort Wayne Lutheran Hospital, Ft. Wayne, Indiana, 46804, United States|Riley Hospital for Children, Indianapolis, Indiana, 46202, United States|University of Iowa-Children's Hospital, Iowa City, Iowa, 52242, United States|University of Louisville/Kosair Children's Hospital, Louisville, Kentucky, 40202, United States|Our Lady of the Lake Children's Hospital, Baton Rouge, Louisiana, 70808, United States|Children's Hospital of New Orleans, New Orleans, Louisiana, 70118, United States|Johns Hopkins, Baltimore, Maryland, 21205, United States|The Herman and Walter Samuelson Children's Hospital at Sinai, Baltimore, Maryland, 21215, United States|Children's Hospital of Michigan-Wayne State University, Detroit, Michigan, 48201, United States|Hurley Research Center, Flint, Michigan, 48503, United States|University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States|Rutger's University, New Brunswick, New Jersey, 08903, United States|Bronx Lebanon Hospital, Bronx, New York, 10457, United States|NY Methodist, Brooklyn, New York, 11215, United States|Cohen Children's Medical Center, New Hyde Park, New York, 11040, United States|Golisano Children's Hospital at URMC, Rochester, New York, 14642, United States|University of North Carolina-Chapel Hill, Chapel Hill, North Carolina, 27599, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|East Carolina University, Greenville, North Carolina, 27834, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States|Rainbow Babies and Children's Hospital, Cleveland, Ohio, 44106, United States|Randall Children's Hospital, Portland, Oregon, 97227, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, 15224, United States|University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, 15232, United States|The Medical University of South Carolina, Charleston, South Carolina, 29425, United States|T.C. Thompson Children's Hospital, Chattanooga, Tennessee, 37403, United States|Cook's Children Hospital, Fort Worth, Texas, 76104, United States|Texas Children's Hospital, Houston, Texas, 77030, United States|University of Virginia Medical Center, Charlottsville, Virginia, 22908, United States|Children's Hospital of the King's Daughters, Norfolk, Virginia, 23507, United States|Children's Hospital of Richmond, Richmond, Virginia, 23298, United States|Reearch Center, Antwerp, Belgium|Research Center, Brussels, Belgium|Research Center, Edgem, Belgium|Research Center, Liege, Belgium|Research Center, Montegnee, Belgium|Resaerch Center, Rio de Janerio, Brazil|Research Center, Sao Paulo, Brazil|Research Center, Santo Domingo, 10101, Dominican Republic|Research Center, Santo Domingo, 10514, Dominican Republic|Research Center, Kingston, Jamaica|Research Center, Irbid, Jordan|Research Center, Beirut, Lebanon|Research Center, Tripoli, Lebanon|Research Center - Child Health Department, Muscat, Oman|Research Center, Muscat, Oman|Research Center Hospital del Nino, Panama, Panama|Research Center Hospital of Pediatric Specialities, Panama, Panama|Research Center Metropolitan Hospital, Panama, Panama|Research Center, Madrid, Spain|Research Center, Adana, Turkey|Research Center, Istanbul, Turkey|Research Center, Mersin, Turkey |
| Nitrous Oxide Analgesia Vaso-occlusive Crisis | Patients who have sickle cell VOC are usually treated with opioids, such as morphine. However, this current way of treating them has not improved the health, medical outcomes, or rates of hospitalizations. In addition, since VOC can happen very frequently over a long period of time, giving opioids over and over again can cause both short-term and long-term problems. Nitrous oxide (N2O) is a way of treating pain that may provide a better alternative to repeatedly giving opioids over long periods of time. N2O has been shown to provide up to 3 hours of pain relief in inpatient patients with VOC whose pain did not improve with morphine infusions, and is used extensively in France, where almost half of 85 pediatric emergency departments use nitrous oxide to treat children with VOC whose pain did not get better with standard treatment with morphine. However, pain relief which N2O provides in the acute setting has not been well described. Therefore, the purpose of our study is to describe how well N2O can relieve the pain in patients with SCD who present to the emergency department and are experiencing a VOC. | Sickle Cell Disease|Vaso-occlusive Crisis | ALL | CHILD, ADULT | New York Presbyterian Morgan Stanley Children's Hospital, New York, New York, 10032, United States |
| Screening for Subjects to Participate in Studies of Blood Disorders | This study will determine eligibility for participation in research studies on blood disorders conducted by the National Heart, Lung and Blood Institute and the National Institute of Diabetes, Digestive, and Kidney Diseases. Healthy volunteers, patients with blood disorders under study by NHLBI and NIDDK and potential stem cell donors for patients with blood disorders who are 8 years of age and older may be eligible for this screening protocol. (Healthy volunteers who qualify for research protocols would serve as control subjects.) Participants undergo the following tests and procedures: Healthy Volunteers * Medical history, physical examination, blood tests and urine sample collection. * Buccal mucosa sample collection. (Cells are collected from the inside of the cheek by gentle scraping with a bristly brush.) * Bone marrow aspiration (only for volunteers 18 years of age and older). Potential Stem Cell Donor -Same as for healthy volunteers plus evaluations that may include electrocardiogram, echocardiogram, imaging studies (X-rays, CT scans, MRI scans and others), heart evaluation, and lung function tests. Patient with Blood Disorder * Same as for stem cell donors plus additional evaluations and treatments that may include radiation oncology evaluation, catheter placement, blood transfusions, kidney and liver biopsies. Short courses of drug treatment for induction of fetal hemoglobin in sickle cell patients, and/or iron chelation in patients receiving chronic red cell transfusions may be included as well. | Bone Marrow Transplant|Sickle Cell Disease|G-CSF | ALL | CHILD, ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| A Study to Evaluate GBT021601-012 Single Dose and Multiple Dose in Participants With Sickle Cell Disease (SCD) | The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK), and pharmacodynamics (i.e., how the body absorbs, distributes, breaks down, and excretes) of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in participants with SCD, following single and multiple ascending doses. | Sickle Cell Disease | ALL | ADULT | Advanced Pharma CR, LLC, Miami, Florida, 33147, United States|Visionaries Clinical Research LLC, Atlanta, Georgia, 30329, United States|Children's Healthcare of Atlanta AFLAC Center, Atlanta, Georgia, 30342, United States |
| Novel Use Of Hydroxyurea in an African Region With Malaria | Multiple studies have shown that hydroxyurea has clinical efficacy in preventing acute painful episodes and reducing the need for blood transfusions in children with sickle cell anemia (SCA), but no study has been conducted in malaria endemic regions of sub-Saharan Africa, the areas with the most children with SCA. The primary goal of this study is to investigate the safety and efficacy of hydroxyurea for children with SCA in a malaria endemic region within sub-Saharan Africa. | Sickle Cell Anemia|Sickle Cell Disease|Malaria | ALL | CHILD | Mulago Hospital Sickle Cell Clinic, Kampala, Uganda |
| Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC) | CADPT03A12001 is a prospective, multi-center study that is designed to follow all enrolled patients who have received treatment with OTQ923 for long-term safety and efficacy. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | St Jude Childrens Rsrch Hospital, Memphis, Tennessee, 38105-3678, United States |
| A Study of the Effect of IW-1701 (Olinciguat), a Stimulator of Soluble Guanylate Cyclase (sGC), on Patients With Sickle Cell Disease (SCD) | The primary objective of the 1701-202 STRONG SCD study is to evaluate the safety and tolerability of different dose levels of IW-1701 compared with placebo when administered daily for approximately 12 weeks to patients with stable SCD. Exploratory objectives include evaluation of pharmacokinetic (PK) as well as evaluation of the effect of IW-1701 on symptoms of SCD, health-related quality of life, and biomarkers of pharmacodynamic (PD) activity. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital of Orange County, Orange, California, 92868, United States|MedStar Health Research Institute, MedStar Washington Hospital Center, Washington, District of Columbia, 20010, United States|Howard University Center for Sickle Cell Disease, Washington, District of Columbia, 20060, United States|Innovative Medical Research of South Florida, Inc., Aventura, Florida, 33180, United States|Century Clinical Research, Inc., Fort Lauderdale, Florida, 32117, United States|Foundation for Sickle Cell Disease Research, Hollywood, Florida, 33021, United States|Omega Research Maitland, LLC, Orlando, Florida, 32810, United States|Grady Memorial Hospital, Atlanta, Georgia, 30303, United States|Atlanta Center for Medical Research, Atlanta, Georgia, 30331, United States|University of Illinois at Chicago, Chicago, Illinois, 60612, United States|Healthcare Research Network II, LLC, Flossmoor, Illinois, 60422, United States|Clinical Trials of SWLA, LLC, Lake Charles, Louisiana, 70601, United States|University of Maryland Medical Center, Baltimore, Maryland, 21201, United States|Johns Hopkins School of Medicine Children's Center, Baltimore, Maryland, 21205, United States|Boston Children's Hospital, Boston, Massachusetts, 02115, United States|Children's Hospital of Michigan-Detroit, Detroit, Michigan, 33021, United States|Healthcare Research Network, Hazelwood, Missouri, 63042, United States|Hackensack University Medical Center, Pediatric Hematology and Oncology, Hackensack, New Jersey, 07601, United States|Jacobi Medical Center, Bronx, New York, 10461, United States|New York Medical College, Valhalla, New York, 10595, United States|East Carolina University - Leo W. Jenkins Cancer Center, Greenville, North Carolina, 27834, United States|East Carolina University Brody School of Medicine, Department of Pediatrics, Division of Pediatric Hematology, Greenville, North Carolina, 27834, United States|Lynn Institute of Tulsa, Tulsa, Oklahoma, 74105, United States|The Clinical Trial Center LLC, Jenkintown, Pennsylvania, 19046, United States|University of Pittsburgh Medical Center Hillman Cancer Center, Pittsburgh, Pennsylvania, 15232, United States|Accurate Clinical Research, Baytown, Texas, 77521, United States|"UT Health Clinical Research Unit Center for Clinical and Translational Sciences, Houston, Texas, 77030, United States|Mays Cancer Center UT Health San Antonio, San Antonio, Texas, 78229, United States|Virginia Commonwealth University - Clinical Research Unit, Richmond, Virginia, 23298, United States|Blood Center of Wisconsin (BCW), Wauwatosa, Wisconsin, 53226, United States|Hammoud Hospital University Medical Center, Sidon, Lebanon|Nini Hospital, Tripoli, Lebanon|Royal London Hospital, London, E1 2ES, United Kingdom|Whittington Hospital, London, N19 5NF, United Kingdom|Guys and St Thomas NHS Foundation Trust - Evelina London Childrens Hospital, London, SE1 7EH, United Kingdom|Guy's Hospital, London, SE1 9RT, United Kingdom|Hammersmith Hospital, London, W12 0NN, United Kingdom |
| A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease | This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001. | Sickle Cell Disease|Hematological Diseases|Hemoglobinopathies | ALL | CHILD, ADULT | Lucile Packard Children's Hospital of Stanford University, Palo Alto, California, 94304, United States|Ann & Robert Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States|University of Illinois at Chicago Hospitals and Health Systems, Chicago, Illinois, 60612, United States|Columbia University Medical Center (21+ years), New York, New York, 10032, United States|Columbia University Medical Center (≤21 years), New York, New York, 10032, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers, Nashville, Tennessee, 37203, United States|Methodist Children's Hospital/Texas Transplant Institute, San Antonio, Texas, 78229, United States|Hopital Universitaire des Enfants Reine Fabiola (HUDERF), Brussels, Belgium|The Hospital for Sick Children, Toronto, Canada|Hopital Necker Enfants Malades, Paris, France|University Hospital Duesseldorf, Dusseldorf, Germany|Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine, Paediatric Haemotology, Oncology and Stem Cell Transplantation, Regensburg, Germany|Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Ospedale Pediatrico Bambino Gesu - IRCCS, Rome, Italy|Imperial College Healthcare NHS Trust, Hammersmith Hospital, London, United Kingdom|Royal London and St Bartholomew's Hospital, Pathology and Pharmacy Building, London, United Kingdom |
| Hyperbaric Oxygen Therapy in Sickle Cell Pain | Hyperbaric oxygen therapy in acute sickle cell pain crisis. The purpose of this study is to explore if hyperbaric oxygen therapy would decrease hospital length of stay and pain associated with acute sickle cell pain crisis. Eligibility criteria include both female and males age 19 years or older with sickle cell who are in an acute pain crisis. Exclusions include pregnancy and a sickle cell crisis complicated by any acute significant concomitant factors/conditions (i.e., acute chest syndrome, acute myocardial infarction/stroke). Interventions would be 1-3 hyperbaric oxygen sessions depending on response to the therapy. Each treatment session will be approximately two hours in length. Evaluation would be through patients' self assessment via the visual analog scale for pain level before and after treatments as well as tracking length of stay in the hospital. | Vaso-occlusive Crisis|Sickle Cell Anemia Crisis | ALL | ADULT, OLDER_ADULT | Unversity of Nebraska Medical Center, Omaha, Nebraska, 68198, United States |
| Adjuvant Low-dose Ketamine in Pediatric Sickle Cell Vaso-occlusive Crisis | Acute vaso-occlusive episodes (VOEs) in sickle cell disease (SCD) are primarily managed with opioids. Tolerance and hyperalgesia to opioids develops due to N-methyl-D-aspartate (NMDA)-receptor mediated activation of the nociceptive system, and as a receptor antagonist, ketamine mitigates this. Intravenous (IV) ketamine has demonstrated efficacy in reducing post-operative, chronic, and cancer-related pain in pediatrics, as well as in reducing time to pain control in the emergency department (ED) in adults. Limited studies suggest efficacy in adult opioid-refractory SCD patients. This study is investigating the safety and tolerability of adjuvant low-dose IV ketamine bolus for pediatric SCD VOE in the ED, as well as its efficacy in improving pain control and reducing hospitalization. | Sickle Cell Disease|Vaso-Occlusive Crisis | ALL | CHILD, ADULT | UCSF Benioff Children's Hospital and Research Center Oakland, Oakland, California, 94609, United States |
| Stroke Prevention With Hydroxyurea Enabled Through Research and Education (SPHERE) | This study will 1) Evaluate the prevalence of elevated (conditional or abnormal) transcranial Doppler (TCD) velocities in a cross-sectional analysis of children with Sickle Cell Anemia (SCA) living in Tanzania; 2) Obtain longitudinal data on TCD velocities in this population; and 3) Measure the effects of hydroxyurea therapy on TCD velocities and associated primary stroke risk. | Sickle Cell Anemia in Children | ALL | CHILD | Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States|Bugando Medical Centre, Mwanza, Tanzania |
| Parent Willingness to Participate in Tobacco Trials in the Pediatric Clinical Setting | Little is known about what factors influence parental decisions to participate or to decline participation in tobacco trials offered in the pediatric clinical setting. Further, it is unclear what proportion of parents treated in our setting would elect to receive formal assistance with quitting smoking or consider alternative approaches that could facilitate eventual smoking cessation. While the recommendation to parents is generally to quit smoking, some may be unwilling or unable to quit and prefer more achievable alternative treatment goals. Some parent smokers may be unlikely to participate in an intervention aimed only at cessation but would be willing to participate in an intervention focused on establishing smoke-free environments for their child. Parents are typically not offered a choice regarding the type of intervention they receive and many interventions are not tailored to their readiness to quit smoking or designed to reach multiple family members in the home who may also smoke. Quitting smoking and establishing smoke-free homes and cars are distinct, yet challenging, goals for parents and families. Both approaches can directly, or indirectly, help parents to quit smoking, reduce the child's exposure to second-hand smoke (SHS), and initiate an important dialogue with families about tobacco control. How parental acceptability of smoking interventions is affected by the context of their child's treatment for cancer or SCD, as well as survivorship, warrants further study. | Smoking|Cancer|Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Intranasal Fentanyl Versus Intravenous Morphine in the Treatment of Severe Painful Sickle Cell Crises in Children | Sickle cell anaemia is an inherited blood disorder which results in abnormal sickle shaped red blood cells which do not fit well through small blood vessels. These blockages prevent oxygen (in blood) from reaching different parts of the body resulting in painful crisis. This study will compare the effectiveness of two types of pain medication, one given through a vein and one squirted up the nose. | Pain|Sickle Cell Disease | ALL | CHILD, ADULT | Our Lady's Children's Hospital, Crumlin, Dublin, Ireland |
| Peer Support for Adolescents and Emerging Adults with Sickle Cell Pain | The study, known as the Peer suppoRt for adolescents and Emerging adults with Sickle cell pain: promoting ENgagement in Cognitive behavioral thErapy (PRESENCE), aims to determine the effectiveness of digital CBT in reducing pain, opioid use, and healthcare utilization among AYAs with SCD. It also seeks to understand the role of personalized peer support in enhancing engagement and outcomes of digital CBT interventions. By leveraging existing infrastructure for delivering virtual peer support interventions, tailored digital CBT programs for individuals with SCD, and partnerships with CBOs, the study aims to provide valuable insights into the feasibility and effectiveness of digital CBT as a pain management strategy for this vulnerable population. | Pain|Sickle Cell Disease | ALL | CHILD, ADULT | |
| A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease | This Phase 2a, multicenter, open-label, dose-escalation study is designed to assess the safety and biologic activity of daily oral administration of 4 escalating doses of sapropterin dihydrochloride over 16 weeks in subjects with sickle cell disease. During an optional extension phase, the study will assess the safety, tolerability, and efficacy of extended treatment with sapropterin dihydrochloride, for a total of up to 2 years; The extension phase of this study was terminated. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Washington, District of Columbia, 20060, United States|Augusta, Georgia, 30912, United States|Savannah, Georgia, 31404, United States|Indianapolis, Indiana, 46260, United States|Detroit, Michigan, 48201, United States|Flint, Michigan, 48503, United States|Hackensack, New Jersey, 07601, United States|Chapel Hill, North Carolina, 27599, United States|Philadelphia, Pennsylvania, 19134, United States|Galveston, Texas, 77555, United States|Norfolk, Virginia, 23507, United States|Richmond, Virginia, 23298, United States |
| Ameliorating Attention Problems in Children With Sickle Cell Disease (SCD) | The purpose of this study is to assess whether methylphenidate is effective in enhancing the cognitive performance of children with the HbSS or HbSC genotype of SCD who have sustained neurological complications on laboratory-based measures of sustained attention, reaction time, and executive functions, and indirectly, verbal short-term and long-term memory. | Sickle Cell Disease | ALL | CHILD | Temple University, Philadelphia, Pennsylvania, 19140, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States |
| Lidocaine Intravenous in the Emergency Department For Sickle Cell Crisis | Sickle cell crisis continues to be a frequent presentation to emergency departments. Patients presenting will often require immediate treatment for their pain and often times this will include opioids. The opioid epidemic has cost thousands of lives; and continues to be a significant problem posing several challenges when treating patients presenting with sickle cell disease. Primarily, opioids remain the mainstay of treatment for these patients and the push to address the opioid crisis may present challenges for adequate opioid administration in patients suffering from a sickle cell crisis while hospitals find ways to curb the opioid crisis overall. Opioid treatment for patients in acute vaso-occlusive crisis has significantly contributed to quality of life and life expectancy of patients with this diagnosis. Measures should continue to attempt to administer a multi-model approach to sickle cell patients to minimize the morphine milligram equivalents in these patients while also successfully addressing the patient's pain. IV lidocaine is a pain medication that has been evaluated in several painful experiences, such as in renal colic. A few case reports have shown IV lidocaine use in sickle cell can be a potential effective adjunct medication to opioids to treat pain and reduce further opioid requirements. Currently, no prospective controlled trial exists to evaluate the true benefit of IV lidocaine in this population. Our study aims to evaluate IV lidocaine as an adjunct to opioid treatment in the emergency department to determine if improved pain is achieved and if there is a reduction in overall morphine milligram equivalents throughout the emergency department visit. | Sickle Cell Disease|Sickle Cell Crisis|Pain, Acute | ALL | ADULT, OLDER_ADULT | Monmouth Medical Center, Long Branch, New Jersey, 07740, United States|Robert Wood Johnson University Hospital, New Brunswick, New Jersey, 08901, United States|Newark Beth Israel Medical Center, Newark, New Jersey, 07112, United States |
| Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease | The purpose of this interventional, phase II, national, multicentric, non-randomised, open-label study is to evaluate the pharmacokinetics (PK), efficacy and safety of Hydroxycarbamide Paediatric dispersible tablets with a twice daily dosing regimen in children with Sickle Celle Disease between 9 months to 11 years of age. Participants will: * Take Hydroxycarbamide twice a day every day for 12 months * Visit the clinic at screening, baseline, 1, 3, 6, 9 and 12 months | Sickle Cell Disease | ALL | CHILD | Centre Hospitalier Intercommunal Créteil, Créteil, France|Hôpital Bicêtre, Le Kremlin-Bicêtre, France|Hôpital Necker-Enfants malades, Paris, France|Centre hospitalier de Cayenne, Cayenne, French Guiana |
| Low Dose Ketamine and Acute Pain Crisis | BACKGROUND: Current treatment standard for acute pain crisis in sickle cell disease (SCD) is largely supportive care: opioid analgesics, hydration, oxygen, and blood transfusion. Sickle cell disease (SCD) is a chronic condition associated with serious and disabling acute consequences such as a vaso-occlusive (VOC) or pain crisis. Uncontrolled pain is the hallmark of a VOC, and often results in acute unscheduled care in the patient's clinic or hospital emergency department (ED). During these pain crises, patients sometimes require high doses of opioids for analgesia. Opioid analgesics are fraught with challenges including the development of tolerance, dependence, and opioid-induced hyperalgesia (whereby the use of opioids actually makes patients more sensitive to pain). Finding non-opioid alternatives for intravenous analgesia is problematic based on the limited availability this class of drugs. Ketamine is a potent N-methyl-D-aspartate (NMDA) receptor antagonist that even at low doses has demonstrated efficacy as an adjunct to opioids for acute pain control. OBJECTIVE: The investigators will determine the comparative efficacy of low doses of ketamine as an adjunct to opioids versus standard care (opioids alone) for the treatment of acute severe pain in patients with sickle cell related pain crisis. METHODS: The investigators propose a double-blinded, randomized, placebo-controlled pilot study to determine the efficacy of ketamine 0.3mg/kg vs. placebo for the treatment of acute pain crisis. The investigators will include all eligible emergency department ≥18 years. The investigators will stratify 42 patients by location, 21 patients per site. Numeric Rating Scale (NRS) will be recorded as a part of the study log at 0, 1, 2 and 3hrs after the study drug administration. HYPOTHESIS: The investigators hypothesize that the ketamine will decrease overall pain intensity, visit length of stay, and hospitalizations. | Sickle Cell Crisis | ALL | ADULT, OLDER_ADULT | Rhode Island Hospital, Providence, Rhode Island, 02905, United States|The Miriam Hospital, Providence, Rhode Island, 02906, United States |
| Stroke Prevention in Sickle Cell Anemia (STOP 2) | To determine how long blood transfusions are needed for primary stroke prevention. Also, to determine the duration of risk associated with abnormal transcranial Doppler ultrasound (TCD) and to determine the specificity of the stroke risk model developed in STOP 1 in patients with abnormal TCD measurements. | Blood Disease|Cerebrovascular Accident|Anemia, Sickle Cell | ALL | CHILD, ADULT | |
| Clinical Trial to Study the Safety and Tolerability of Memantin Mepha® in Sickle Cell Disease Patients | Symptomatic sickle cell disease is worldwide the most frequent cause for hereditary hemolytic anemia with recurrent pain crisis. Hemolysis, vaso- occlusive and pain crises are hallmarks of this disease and are causative for an important socio-economic burden worldwide, especially in Africa. Aside from allogenic stem cell transplantation, which is rarely available and very expensive, at present there is no curative treatment for patients with sickle cell disease (SCD). The current standard of care includes treatment with hydroxycarbamide and symptomatic care such as transfusions, antibiotic/analgesic treatment. This study has the aim to study the safety and tolerability of Memantin in patients with sickle cell disease. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University Hospital Zürich, Zürich, 8091, Switzerland |
| A Single Dose Study of the Safety, Blood Levels and Biological Effects of Aes-103 Compared to Placebo in Subjects With Stable Sickle Cell Disease | The purpose of this study is to assess the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of Aes-103 (active ingredient 5-hydroxymethyl-2-furfural \[5-HMF\]) compared with placebo in subjects with stable sickle cell disease (SCD). Safety will be measured by monitoring adverse events (AEs), electrocardiograms (ECGs), vital signs, and laboratory values. Pharmacokinetics of Aes-103 will be measured over time in plasma, red blood cell hemolysate and binding of Aes-103 to hemoglobin. Pharmacodynamic effects will be assessed by measuring partial pressure of oxygen at which 50% of hemoglobin is saturated with oxygen (p50) while breathing normal air, blood oxygen levels (SpO2), ex-vivo antisickling effects in a hypoxic environment, and by imaging related changes in tissue blood flow and oxygen levels. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | US National Institutes of Health - National Heart, Lung, and Blood Institute, Bethesda, Maryland, 20892, United States |
| Quality of Life of Children With Sickle Cell Disease Who Are Getting Chronic Transfusions With a Lifeport | This study is being done to see what impact having a Lifeport device has on quality of life for children with sickle cell who are getting chronic transfusions, from the child's perspective. | Anemia, Sickle Cell | ALL | CHILD, ADULT | The Children's Mercy Hospital, Kansas City, Missouri, 64108, United States |
| A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease | The purpose of this study is to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of BMS-986470 in healthy volunteers and participants with sickle cell disease. | Anemia, Sickle Cell|Healthy Volunteers | ALL | ADULT, OLDER_ADULT | Local Institution - 0008, Birmingham, Alabama, 35294-3300, United States|Local Institution - 0021, La Jolla, California, 92037, United States|Local Institution - 0003, Oakland, California, 94609, United States|Local Institution - 0022, New Haven, Connecticut, 06510, United States|Local Institution - 0017, Atlanta, Georgia, 30322, United States|ICON, Lenexa, Kansas, 66219, United States|Local Institution - 0016, Boston, Massachusetts, 02118, United States|Local Institution - 0007, Philadelphia, Pennsylvania, 19107, United States|Local Institution - 0013, Fairfax, Virginia, 22031, United States|Local Institution - 0014, Richmond, Virginia, 23298, United States|Local Institution - 0015, Marseille, 13385, France|Local Institution - 0006, London, London, City Of, SE5 9RL, United Kingdom|Local Institution - 0005, Leeds, LS9 7TF, United Kingdom |
| Hydroxyurea for Children and Young Adults With Sickle Cell Disease and Pulmonary Hypertension | The goal of this study is to test the hypothesis that hydroxyurea is effective for the specific treatment of secondary pulmonary hypertension found on screening in children and young adults with sickle cell disease. | Sickle Cell Disease|Pulmonary Hypertension | ALL | CHILD, ADULT | Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States |
| Role of Virtual Reality (VR) in Patients With Sickle Cell Disease (SCD) | Patients with sickle cell disease (SCD) and cancer often have complicated courses while hospitalized and often deal with pain, anxiety and depression. Advances in the field of technology provide potential avenues for innovative and improved care models for our patients. Virtual reality (VR) has been recently utilized to improve anxiety and pain in a variety of patient populations including children undergoing elective surgery and children experiencing intravenous cannulation in the Emergency Department. Patients with SCD and cancer, both adults and children, are a group of patients that can benefit from VR as part of their care. Over the past four years, our team has successfully implemented several self-developed mobile applications ("apps") for our patients, in addition to integrating objective data (heart rate, activity, stress) from wearable activity trackers. The investigators now propose implementing a feasibility study followed by a pilot study and randomized-controlled trial of the use of VR in patients with SCD and cancer. The investigators plan to assess pain and anxiety prior to the session as well as following the session in hospitalized patients and outpatients with SCD and cancer. The sessions will include a ten-minute relaxation response introductory narrative segment (deep breathing and mindfulness) followed by a ten-minute narrated and immersive VR. Heart rate will be tracked using an Apple iWatch for 30 minutes prior to the session, during the session, and following the session. We anticipate VR will not only be a feasible method to provide non-pharmacologic treatment, but will also significantly reduce pain and anxiety. | Sickle Cell Disease|Virtual Reality|Anxiety|Depression|Cancer | ALL | CHILD, ADULT, OLDER_ADULT | Duke University Medical Center, Durham, North Carolina, 27710, United States |
| Inhaled Mometasone to Promote Reduction in Vasoocclusive Events 2 | The study team proposes a triple-blind, placebo-controlled, phase II clinical trial of once-daily inhaled mometasone for 48 weeks (with 4-week washout at study completion) in individuals with Sickle Cell Disease (SCD) who report episodic cough or wheeze (ECW) but do not have asthma. Patients will be recruited from and followed in SCD clinics at participating sites. The primary endpoint will be a reduction in sVCAM level of 20% or more in comparison to placebo. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Mount Sinai St Luke's, New York, New York, 10025, United States|Icahn School of Medicine at Mount Sinai, New York, New York, 10029, United States |
| Ketamine Infusion for Sickle Cell Pain Crisis | The purpose of this study is to prospectively study the efficacy of low dose ketamine infusions in treating patients who are admitted to the hospital with a sickle cell pain crisis. Participants will be prospectively randomized in unblinded fashion in the first 12 to 24 hours of an inpatient admission for sickle cell pain crisis to receive pain management without ketamine infusion (Group A) versus pain management that includes low-dose ketamine infusion starting at 0.2mg/kg/h (Group B). The effect of this intervention on various pain management and healthcare utilization outcome measures will be recorded and analyzed to determine whether or not there is a measurable benefit of using ketamine infusions in this patient population. | Anemia; Sickle-Cell, With Crisis|Acute Pain | ALL | ADULT, OLDER_ADULT | Duke Hospital, Durham, North Carolina, 27710, United States |
| SACRED A Prospective Research Study to Reduce Stroke in Children With Sickle Cell Anemia | Prospective screening and treatment study for children with Sickle Cell Anemia and increased stroke risk living in the Dominican Republic. | Sickle Cell Anemia | ALL | CHILD | Encargada del Servicio de Hematología-Oncología Hospital Infantil Dr. Robert Reid Cabral, Santo Domingo, Dominican Republic |
| Long-Term Effects of Hydroxyurea in Children With Sickle Cell Anemia (The BABY HUG Follow-up Study) | Sickle cell anemia (SCA) is an inherited blood disorder that can cause organ damage. The BABY HUG study is evaluating the use of the medication hydroxyurea at preventing organ damage in children with SCA. The purpose of this follow-up study is to evaluate the long-term effects of hydroxyurea in children who have participated in the BABY HUG study. | Anemia, Sickle Cell | ALL | CHILD | University of Alabama at Birmingham, Birmingham, Alabama, 35233, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States|Howard University College of Medicine, Washington, District of Columbia, 20060, United States|University of Miami School of Medicine, Miami, Florida, 33136, United States|Emory University School of Medicine, Atlanta, Georgia, 30342, United States|Johns Hopkins University School of Medicine, Baltimore, Maryland, 21205, United States|Children's Hospital of Michigan/Wayne State University, Detroit, Michigan, 48201, United States|University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States|Downstate Medical Center, Brooklyn, New York, 11203, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|Drexel University, Philadelphia, Pennsylvania, 19134, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|University of Texas Southwestern Medical Center at Dallas, Dallas, Texas, 75390, United States |
| Sickle Cell Improvement: Enhancing Care in the Emergency Department | Sickle cell disease (SCD) is an inherited blood disorder affecting approximately 36,000 children in the United States, approximately 90% of whom are Black. The disease is characterized by recurrent, severe pain crises which result in high rates of emergency department visits and hospitalizations, and decreased quality of life. The National Heart, Lung and Blood Institute, as well as the American Society of Hematology, have endorsed pain management guidelines regarding the timeliness of care for children presenting with these acute pain crises. These evidence-based guidelines are infrequently followed, resulting in increased pain and hospitalizations. In additional to other barriers to following the guideline, structural racism has been proposed as a significant contributor and the New England Journal of Medicine recently called for the institution of SCD-specific pain management protocols to combat structural racism and reduce time to opioid administration. The investigators' long-term goal is to improve the care and health outcomes of children with acute painful vaso-occlusive crisis treated in the emergency department. The overall aim of the investigators is to test a care pathway using multifaceted implementation strategies to increase guideline adherent care for children in the emergency department with acute painful vaso-occlusive crisis. | Sickle Cell Crisis | ALL | CHILD, ADULT | Children's Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| Ketamine as an Adjuvant Therapy for Acute Vaso Occlusive Crisis in Pediatric Patients With Sickle Cell Disease | The primary objective of the proposed study is to determine the potential role of Ketamine as an analgesic agent in pediatric sickle cell disease patients with refractory symptoms in acute (VOC). | Sickle Cell Disease | ALL | CHILD | |
| Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children | Acute chest syndrome is a severe sickle cell disease complication in children requiring blood transfusion therapy to prevent acute respiratory failure and death. Nitric oxide is a potent vasodilator that could reverse pulmonary vascular occlusion and restore normal oxygenation. The randomized trial will test that hypothesis. | Acute Chest Syndrome|Sickle Cell Disease | ALL | CHILD, ADULT | Hoipital Robert Debre, Paris, 75019, France |
| Hydroxyurea Optimization Through Precision Study | Hydroxyurea Optimization through Precision Study (HOPS) is a prospective, multi-center, randomized trial that will directly compare a novel, individualized dosing strategy of hydroxyurea to standard weight-based dosing for children with SCA. The primary objective of the study is to evaluate whether a pharmacokinetics-based starting hydroxyurea dose thieves superior fetal hemoglobin response to to standard weight-based initial dosing. Patients will be recruited from the pediatric sickle cell clinic at Cincinnati Children's Hospital Medical Center and from additional pediatric sickle cell centers within the United States. | Sickle Cell Disease|Sickle Cell Anemia | ALL | CHILD, ADULT | Phoenix Children's Hospital, Phoenix, Arizona, 85016, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30342, United States|Children's Hospital of Illinois, Peoria, Illinois, 61637, United States|Carle Foundation Hospital, Urbana, Illinois, 61801, United States|Riley Hospital for Children at Indiana University Health, Indianapolis, Indiana, 46202, United States|Indiana Hemophilia & Thrombosis Center, Inc. (IHTC), Indianapolis, Indiana, 46260, United States|Boston Children's Hospital, Boston, Massachusetts, 02215, United States|Children's Hospitals and Clinics of Minnesota, Minneapolis, Minnesota, 55404, United States|Cohen Children's Medical Center/Northwell Health, New Hyde Park, New York, 11040, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States|Rainbow Babies / University Hospitals Cleveland Medical Center, Cleveland, Ohio, 44106, United States|Cleveland Clinic Children's, Cleveland, Ohio, 44195, United States|Nationwide Children's Hospital., Columbus, Ohio, 43205, United States|Children's Hospital of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| Prevalence Of Microalbuminuria Among Children Suffering From Sickle Cell Nephropathy and Sickle Cell/Beta-Thalassemia | Sickle cell nephropathy is a known complication of sickle cell anemia (SCA) manifested by increase in glomerular filtration rate (glomerular hyperfiltration) and results in proteinuria and chronic renal failure. Our goal is to examine the prevalence of proteinuria and microalbuminuria as an early predictive factor of glomerular injury, among young people who suffer from SCA as well as those who suffer from combined sickle cell/beta-thalassemia. | Sickle Cell Anemia|Beta-Thalassemia|Microalbuminuria | ALL | CHILD, ADULT | |
| Study of Efficacy, Safety and Tolerability of ACZ885 (Canakinumab) in Pediatric and Young Adult Patients With Sickle Cell Anemia | The study assesses the efficacy, safety and tolerability of ACZ885 (canakinumab) in pediatric and young adult patients with sickle cell anemia (SCA). | Sickle Cell Anemia | ALL | CHILD, ADULT | Novartis Investigative Site, Atlanta, Georgia, 30329, United States|Novartis Investigative Site, Augusta, Georgia, 30912, United States|Novartis Investigative Site, Greenville, North Carolina, 27834, United States|Novartis Investigative Site, Toronto, Ontario, M5G 1X8, Canada|Novartis Investigative Site, Hamburg, 20246, Germany|Novartis Investigative Site, Afula, 1834111, Israel|Novartis Investigative Site, Johannesburg, Guateng, 2193, South Africa|Novartis Investigative Site, Adana, 01330, Turkey|Novartis Investigative Site, Ankara, 06100, Turkey|Novartis Investigative Site, Mersin, 33343, Turkey|Novartis Investigative Site, Wolverhampton, Staffordshire, WS11 5XY, United Kingdom|Novartis Investigative Site, London, E1 1BB, United Kingdom|Novartis Investigative Site, London, NW1 2BU, United Kingdom|Novartis Investigative Site, London, SE1 7EH, United Kingdom|Novartis Investigative Site, London, SE5 9RS, United Kingdom |
| sPLA2 in EBC During Acute Chest Syndrome | Secretory phosholipases A2 (sPLA2) are significantly elevated in the plasma of sickle cell disease patients with acute chest syndrome (ACS), and similar enzymes have been measured in exhaled breath condensate (EBC), which is collected easily and non-invasively. The investigators hypothesize that sPLA2 will be measurable in EBC samples from sickle cell patients with acute chest syndrome. | Sickle Cell Disease|Acute Chest Syndrome | ALL | CHILD, ADULT | Virginia Commonwealth University, Richmond, Virginia, 23298, United States |
| European Rare Blood Disorders Platform (ENROL) | ENROL, the European Rare Blood Disorders Platform has been conceived in the core of ERN-EuroBloodNet as an umbrella for both new and already existing registries on Rare Hematological Diseases (RHDs). ENROL aims at avoiding fragmentation of data by promoting the standards for patient registries' interoperability released by the EU RD platform. ENROL's principle is to maximize public benefit from data on RHDs opened up through the platform with the only restriction needed to guarantee patient rights and confidentiality, in agreement with EU regulations for cross-border sharing of personal data. Accordingly, ENROL will map the EU-level demographics, survival rates, diagnosis methods, genetic information, main clinical manifestations, and treatments in order to obtain epidemiological figures and identify trial cohorts for basic and clinical research. To this aim, ENROL will connect and facilitate the upgrading of existing RHD registries, while promoting the building of new ones when / where lacking. Target-driven actions will be carried out in collaboration with EURORDIS for educating patients and families about the benefits of enrolment in such registries, including different cultural and linguistic strategies. The standardized collection and monitoring of disease-specific healthcare outcomes through the ENROL user-friendly platform will determine how specialized care is delivered, where are the gaps in diagnosis, care, or treatment and where best to allocate financial, technical, or human resources. Moreover, it will allow for promoting research, especially for those issues that remain unanswered or sub-optimally addressed by the scientific community; furthermore, it will allow promoting clinical trials for new drugs. ENROL will enable the generation of evidence for better healthcare for RHD patients in the EU as the ultimate goal. ENROL officially started on 1st June 2020 with a duration of 36 months. ENROL is co-funded by the Health Programme of the European Union under the call for proposals HP-PJ-2019 on Rare disease registries for the European Reference Networks. GA number 947670 | Anemia|Bone Marrow Failure|Bleeding Disorder|Iron Metabolism Disorders|Myeloma|Lymphoid Neoplasm|Myeloma, Malignant|Leukemia|Anemia, Sickle Cell|Thalassemia|Blood Cancer|Red Cell Membrane and Enzyme Abnormalities | ALL | CHILD, ADULT, OLDER_ADULT | María del Mar, Barcelona, Catalunya, 08035, Spain |
| Risk-based Therapy for Sickle Cell Anemia: A Feasibility Study | Sickle cell anemia (SCA) patients experience organ damage that begins at an early age and results in significant morbidity and early mortality. Although all SCA patients share the same genetic mutation, the clinical complications are highly variable with some patients experiencing frequent and severe complications, while others have few serious complications. If SCA severity could be predicted early in life, those patients at greatest risk for complications could receive treatment prior to the onset of organ damage. No general SCA severity predictor or one that can be informative early in life exists. The investigators preliminary research has identified the absolute reticulocyte count (ARC) as a potential early predictive risk marker for SCA complications in pediatric patients. A higher ARC between ages 2 and 6 months of age is associated with an increased risk of hospitalization in the first 3 years of life; the mean ARC for the 36 patients who were hospitalized for SCA complications was significantly higher than that of the remaining 23 in those who were not hospitalized. Moreover, total hospitalizations were nearly three times higher by age 2 years in those infants who had an ARC of \> 200 than for those infants whose ARC was \<200. The proposed study will determine if ARC can be used as a risk-stratifier in asymptomatic infants with SCA and ascertain its value in targeting hydroxyurea therapy to those infants at highest risk of SCA sequelae. | Sickle Cell Anemia | ALL | CHILD | Children's National Medical Center, Washington, D.C., District of Columbia, 20010, United States |
| Identifying Barriers and Strategies to Support Self-efficacy for Medication Adherence With Text Messaging | The investigators will explore barriers to improving self-efficacy, or the ability to feel in control of their disease, and medication adherence with text messaging through surveys and interviews with adolescents and adults with SCD cared for at the Vanderbilt Meharry Center of Excellence (VMCE) in Sickle Cell Disease (SCD). The investigators will identify preferences to improve and sustain adherence to daily medication through selection of investigator-proposed or patient-generated text messaging strategies. Finally, the investigators will fill in the literature gaps by describing barriers to self-efficacy and medication adherence among adults with SCD as well as adolescents with SCD who are transitioning to adult care. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Vanderbilt University, Nashville, Tennessee, 37203, United States |
| A Self-Management Intervention for Youth With Sickle Cell Disease and Their Families: Phase I | This study is being conducted to test an intervention for children and adolescents ages 8-17 years with sickle cell disease and their families. In the first phase of this study, key informant interviews are being conducted with health care providers and children ages 8-17 with sickle cell disease and their primary caregivers. Participants are asked to review the intervention and provide feedback that will inform revision to the intervention. | Anemia, Sickle Cell | ALL | ADULT, OLDER_ADULT | Medical University of South Carolina, Charleston, South Carolina, 29425, United States |
| Therapeutic Response Evaluation and Adherence Trial (TREAT) | The primary objectives of this prospective study of hydroxyurea for children with sickle cell anemia are 1) Develop and prospectively evaluate a population pharmacokinetic/pharmacodynamics model to predict the maximum tolerated dose (MTD); 2) Identify urine biomarkers of hydroxyurea adherence using a novel metabolomics approach; 3) Identify pharmacogenomics modifiers of hydroxyurea MTD; and 4) Longitudinal monitoring of the effect of hydroxyurea upon organ function and quality of life. | Anemia, Sickle Cell | ALL | CHILD, ADULT | Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States |
| A Program to Increase Sickle Cell Trait Knowledge Among Parent of Young Children Identified in Newborn Screening | This is a study for parents of young children with Sickle Cell Trait (SCT) identified by newborn screening who are referred and present for in person SCT education at the Institution. The study will determine the feasibility of implementing a SCT education program (SCTaware) that is appropriate for all parents, including those with low base knowledge and low health literacy and then test if results in high and sustained SCT knowledge. | Sickle Cell Trait | ALL | ADULT, OLDER_ADULT | Nationwide Children's Hospital, Columbus, Ohio, 43205, United States |
| Lung Ultrasound in Pediatric Acute Chest Syndrome | Sickle-cell disease is a common disease with serious complications, in particular acute chest syndrome (ACS), which can be life threatening. The pathophysiology of ACS is poorly understood, but alveolar hypoventilation appears to play an important role. Pulmonary ultrasound is increasingly used in pediatrics to diagnose ACS. The management of ACS is complex, including oxygen therapy, antibiotics, spirometry, transfusions and ventilatory support. ACS and acute vaso-occlusive pain are the main reasons for hospitalisation in pediatric intensive care units. The aim of this study was to identify the pulmonary indicators correlated with ventilation time in these children, and to study the correlations between the results of lung ultrasound (LUS) and the clinical severity of the episode. The inclusion criteria for this study are the presence of an ACS in a child aged between 1 month and 17 years hospitalised in the pediatric intensive care unit at Robert-Debre Hospital who has not expressed any opposition and without opposition from their legal representative. The study will run for 2 years, with a target of 60 patients. Each patient included in the study will have multiple LUS during their care, in accordance with a protocol, and their clinical, biological and radiological data will be collected during their stay in the department. | Sickle Cell Disease | ALL | CHILD | Robert Debré hOSPITAL, Paris, 75019, France |
| Evaluation of Repeat Administration of Purified Poloxamer 188 | The purpose of this study is to evaluate the safety of repeat administration of MST-188 during vaso-occlusive crisis of sickle cell disease. Additionally, this study will evaluate the development of acute chest syndrome during VOC and re-hospitalization for recurrence of VOC. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Rady Children's Hospital, San Diego, California, United States|Children's Hospital of Southwest Florida, Fort Myers, Florida, 33908, United States|Joe Dimaggio Children's Hospital, Hollywood, Florida, 33021, United States|Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States|University of Iowa Children's Hospital, Iowa City, Iowa, United States|Our Lady of the Lake Children's Hospital, Baton Rouge, Louisiana, 70808, United States|Rutgers University, New Brunswick, New Jersey, United States|Medical University of South Carolina, Charleston, South Carolina, United States|T. C. Thompson Children's Hospital, Chattanooga, Tennessee, United States |
| Adherence to HU and HRQOL in Patients With Sickle Cell Disease: An Intervention Study Using HU-Go App | This project addresses three important research questions. First, adolescents and young adults (AYA) with sickle cell disease (SCD) and their parents/caregivers will be engaged to inform the (1) domains of health-related quality of life (HRQOL) most important to them, (2) frequency at which they are willing to complete them, and (3) other procedures related to the use, uptake and effect of the HU-Go app as a tool to improve hydroxyurea (HU) adherence. Second, this study seeks to utilize novel modern mobile technology using a multi-functional personalized platform to improve adherence to HU and measure HRQOL in youth with SCD, using NIH-endorsed PROMIS® measures, based on a conceptual model with predefined behavioral targets and mediators. Third, we plan to assess HRQOL changes and identify modifiable behavioral strategies that could serve as surrogates or predictors for HU adherence. This real-time feedback might empower self-directed changes in behavior that could improve adherence to HU. | Sickle Cell Disease|Sickle B+ Thalassemia|Sickle Beta Zero Thalassemia|Sickle Cell Hemoglobin C | ALL | CHILD, ADULT | Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States |
| High-flow Oxygen for Vaso-occlusive Pain Crisis | Sickle cell disease (SCD) is characterized by recurrent vaso-occlusive pain crisis (VOC), which may evolve to acute chest syndrome (ACS), the most common cause of death among adult patients with SCD. Currently, there is no safe and effective treatment to abort VOC or prevent secondary ACS. Management of VOC mostly involve a symptomatic approach including hydration, analgesics, transfusion, and incentive spirometry, which was investigated in a very limited number of patients (\<30). The polymerisation of HbS is one major feature in the pathogenesis of vaso-occlusion. Among factors determining the rate and extent of HbS polymer formation, the hypoxic stimulus is one of the most potent and readily alterable. Current guidelines recommend oxygen therapy in patients with VOC in order to maintain a target oxygen saturation of 95%. Low-flow nasal oxygen (LFNO) is routinely used to achieve this normoxia approach, particularly in patients at risk of secondary ACS because they may experience acute desaturation. In contrast, various case series suggest a potential beneficial role of intensified oxygen therapy targeting hyperoxia for the management of VOC, particularly with the use of hyperbaric oxygen, but the latter is difficult to implement in routine clinical practice. A recent high-flow nasal oxygen (HFNO) technology allows the delivery of humidified gas at high fraction of inspired oxygen (FiO2) through nasal cannula. The FiO2 can be adjusted up to 100% (allowing hyperoxia that may reverse sickling) and the flow can be increased up to 60 L/min (which generates positive airway pressure and dead space flushing, that may prevent evolution of VOC towards ACS by alleviating atelectasis and opioid-induced hypercapnia). In patients with acute respiratory failure, HFNO has been shown to improve patient's comfort, oxygenation, and survival as compared to standard oxygen or non-invasive ventilation. The aim of the present study is to test the efficacy and safety of HFNO for the management of VOC and prevention of secondary ACS. The investigators will use a multi-arm multi-stage (MAMS) design to achieve these goals. HFNO will be delivered through AIRVO 2 (Fisher and Paykel Healthcare, New Zealand), a device that incorporates a turbine allowing its use in hospital wards. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Henri Mondor, Créteil, 94000, France |
| Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease | An open-label, single arm study in patients 12 to 21 years of age with SCD to evaluate the effects of etavopivat on cerebral and muscle hemodynamics. | Sickle Cell Disease | ALL | CHILD, ADULT | Emory University Children's Healthcare of Atlanta, Atlanta, Georgia, 30342, United States |
| tDCS Associated With Peripheral Electrical Stimulation for Pain Control in Individuals With Sickle Cell Disease | So far, no study investigated the safety and efficacy analgesic of transcranial direct current stimulation (tDCS) associated to peripheral electrical stimulation (PES) in individuals with SCD who suffer from chronic pain. Several studies have reported a decrease in O²Hb concentration in the regions below the electrodes and in other cortical areas during anodic or cathodic tDCS, which implies a risk factor for vasoocclusive events in individuals with SDC due to polymerization of hemoglobin when exposed to these low O²Hb concentrations. For this reasion, the aim main of this study is to assess the effect of a single session of transcranial direct current stimulation (tDCS) associated to peripheral electrical stimulation (PES) on safety and efficacy analgesic in individuals with sickle cell disease (SCD). Others aims sencondaries are evaluate the effect of a single session of transcranial direct current stimulation (tDCS) associated to peripheral electrical stimulation (PES) on biomarkers neurophysiological and inflammatory. | Anemia, Sickle Cell|Chronic Pain | ALL | ADULT | Functional Electrical Stimulation Laboratory, Salvador, Bahia, 40110-902, Brazil |
| Decompression Coring Versus Conservative Therapy in Patients With Avascular Necrosis of the Hip Related to Sickle Cell Disease | OBJECTIVES: I. Phase II trial to determine surgical morbidity of decompression coring, including any adverse events in the perioperative period and the rate of secondary medical or surgical interventions. II. Collect preliminary data to determine if decompression coring results in a substantial improvement in pain and mobility compared to conservative therapy in patients with avascular necrosis of the hip related to sickle cell disease. | Bone Avascular Necrosis|Sickle Cell Anemia | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital of Oakland, Oakland, California, 94609, United States|University of North Carolina School of Medicine, Chapel Hill, North Carolina, 27599-7070, United States |
| Pediatrics:Chlamydia, Sickle Cell Anemia and Stroke Risk - Ancillary to STOP II | To establish a link among Chlamydia infection, sickle cell anemia, and stroke risk. | Blood Disease|Anemia, Sickle Cell|Chlamydia Infections|Cerebrovascular Accident | ALL | CHILD, ADULT, OLDER_ADULT | |
| Different Treatment Modalities in the Management of the Painful Crisis in Pediatric Sickle- Cell Anemia | The aim of the present study is comparing the effectiveness of different treatment regimens for investigating the therapeutic potential for each one in management of Vaso-occlusive pain in pediatric sickle cell disease. In addition, investigators apply the Cost-effectiveness analysis (CEA) as a form of economic analysis that compares the relative costs and outcomes (effects) for different treatment regimens on vaso-occlusive painful crisis. | Vaso-occlusive Crisis|Sickle Cell Disease|Sickle Cell Anemia in Children | ALL | CHILD | Faculty of medicine, Beni-suef univeristy - Beni-Seuf university hospital, Banī Suwayf, Egypt|Faculty of Pharmacy, Beni-Suef university, Banī Suwayf, Egypt|Health insurance hospital, Banī Suwayf, Egypt|Maternity and Children hospital, Mecca, Saudi Arabia |
| A Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With SCD | HSCT is currently the only curative option for SCD but less than 20% of SCD patients have a MD donor available. So far, all curative approaches beyond a MSD HSCT at young age are non-satisfactory. With the lack of a suitable donor for the vast majority of patients, the major question of this trial is, if a haploidentical αß/CD19+ T-cell depleted HSCT can be a valid alternative to a MSD HSCT. The main challenge in non-malignant diseases is to offer a safe and GvHD-free HSCT without rejection. | HbS Disease|Hemoglobin S Disease|Sickle Cell Anemia|Sickle Cell Disorders|Sickling Disorder Due to Hemoglobin S|Sickle Cell Disease | ALL | CHILD, ADULT | St. Anna Kinderspital, Vienna, Austria|University Hospital Aachen, Children's Hospital, Aachen, Germany|Charité University medicine, Clinic for Hematology, Oncology, Berlin, Germany|University Hospital Duesseldorf, Clinic for Pediatric Oncology, - Hemtaology and Clinical Immunology, Düsseldorf, 40225, Germany|University Hospital of Frankfurt, Clinic for Paediatrics and Adolescent Medicine, Frankfurt, Germany|University Hospital Heidelberg, Department of Pediatric Hematology, Oncology and Immunology, Heidelberg, 69120, Germany|University Hospital Regensburg, Dept. of Ped. Hematology, Oncology and Stem Cell Transplantation, Regensburg, 93053, Germany|University Children's Hospital Tübingen, Tübingen, 72076, Germany|University Children's Hospital Würzburg, Würzburg, 97080 Würzburg, Germany |
| Primary Prevention of Stroke in Children With SCD in Sub-Saharan Africa II | The overall goal of this proposal is to conduct a partial double-blind randomized Phase III clinical trial for primary stroke prevention in children with sickle cell anemia (SCA) in sub-Saharan Africa. | Sickle Cell Disease|Stroke | ALL | CHILD | Barau Dikko Teaching Hospital/Kaduna State University, Kaduna, Nigeria|Aminu Kano Teaching Hospital, Kano, Nigeria|Murtala Muhammad Specialist Hospital, Kano, Nigeria |
| Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies | The purpose of this study is to determine if treatment with reduced-dose busulfex, fludarabine and alemtuzumab (CAMPATH) followed by sten cell infusion will allow for donor stem cells to grow in patients with hemoglobinopathies bone marrow and restore circulating blood counts. In addition the incidence and severity of side effects and of graft vs. host disease (GVHD) will be monitored. | Hemoglobinopathies|Sickle Cell Disease|Thalassemia | ALL | ADULT, OLDER_ADULT | Winship Cancer Institute-Emory University, Atlanta, Georgia, 30322, United States|Feist-Weiller Cancer Center-LSU, Shreveport, Louisiana, 71130, United States|Beth Israel Deaconess Medical Center, Boston, Massachusetts, 02115, United States|Dana-Farber Cancer Institute, Boston, Massachusetts, 02115, United States|Massachusetts General Hospital, Boston, Massachusetts, 02115, United States|Ohio State University College of Medicine, Columbus, Ohio, 43210, United States |
| Oxbryta® Product Registry An Observational Study Designed to Evaluate the Effect of Oxbryta in Individuals With SCD | This registry is an observational study designed to evaluate the effect of Oxbryta in individuals with SCD in a real-world setting. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | University of South Alabama, Mobile, Alabama, 36617, United States|Phoenix Children's Hospital, Phoenix, Arizona, 85016, United States|University of California, San Diego, La Jolla, California, 92093-0987, United States|Center for Inherited Blood Disorders, Orange, California, 92868, United States|Bass Center for Childhood Cancer and Blood Disorders (Stanford Lucile Packard Children's Hospital), Palo Alto, California, 94304, United States|Department of Pediatrics, Hematology section, Palo Alto, California, 94304, United States|Stanford Children's Hospital, Palo Alto, California, 94304, United States|University of Connecticut Health, Farmington, Connecticut, 06030-1163, United States|University of Connecticut Health, Farmington, Connecticut, 06030, United States|Nemours Alfred I duPont Hospital for Children, Wilmington, Delaware, 19803, United States|Nemours Children's Health, Wilmington, Wilmington, Delaware, 19803, United States|Foundation for Sickle Cell Disease Research, Hollywood, Florida, 33023, United States|Nemours Children's Specialty Care, Jacksonville, Florida, 32207, United States|University of Miami Hospital, Miami, Florida, 33136, United States|Augusta University - Clinical Trials Office (clinic), Augusta, Georgia, 30912, United States|Augusta University, Augusta, Georgia, 30912, United States|University of Illinois at Chicago (UIC) Clinical Research Center, Chicago, Illinois, 60612, United States|University of Illinois at Chicago (UIC) Sickle Cell Center, Chicago, Illinois, 60612, United States|University of Illinois Hospital and Health Sciences System(UI Health), Chicago, Illinois, 60612, United States|University of Illinois Hospital and Health Sciences System, Chicago, Illinois, 60612, United States|University of Maryland Medical Center, College Park, Maryland, 21201, United States|Boston University Medical Center, Boston, Massachusetts, 02118, United States|Mississippi Center for Advanced Medicine, Madison, Mississippi, 39110, United States|Newark Beth Israel Medical Center, Newark, New Jersey, 07112, United States|Montefiore Medical Center, Bronx, New York, 10467, United States|Levine Cancer Institute, Charlotte, North Carolina, 28204, United States|Duke University Hospital, Durham, North Carolina, 27710, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|East Carolina University, Greenville, North Carolina, 27834-4300, United States|ECU Health Medical Center Laboratory, Greenville, North Carolina, 27834, United States|ECU Health Medical Center, Greenville, North Carolina, 27834, United States|UPMC Montefiore Hospital, Pittsburgh, Pennsylvania, 14213, United States|UPMC Sickle Cell Center, Pittsburgh, Pennsylvania, 15123, United States|UPMC Presbyterian, Pittsburgh, Pennsylvania, 15213, United States|University of Pittsburgh Medical Center (UPMC), Pittsburgh, Pennsylvania, 15261, United States|Medical University of South Carolina Shawn Jenkins Women's and Children's Hospital, Charleston, South Carolina, 29425, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|Children's Blood and Cancer Center at Dell Children's Medical Center, Austin, Texas, 78723, United States|Dell Children's Medical Center, Austin, Texas, 78723, United States|University of Texas Health Science Center at Houston, Houston, Texas, 77030, United States|Inova Schar Cancer Institute, Fairfax, Virginia, 22031, United States|INOVA Health, Falls Church, Virginia, 22042-2325, United States |
| A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke | The study will test a new medicine, etavopivat, for sickle cell disease and see if it is safe and help-ful for participants with sickle cell disease who are at an increased risk of stroke. Participants will be divided into two cohorts depending on their transcranial doppler (TCD) ultrasound results and whether or not they receive hydroxyurea (medication that they may already be taking). In one cohort, participants with conditional transcranial doppler (TCD) or participants with abnormal TCD who are not able to receive hydroxyurea will be included. The study doctor will determine if the TCD result is conditional or abnormal. In another cohort, participants with conditional TCD or participants with abnormal TCD who are receiving a stable dose of hydroxyurea will be included. The study doctor will determine if the TCD result is conditional or abnormal. The participant will start a 52-week (1 year) treatment period. The participant will take 400 milligrams (mg) of etavopivat once a day for the 52 weeks. The dose of 400 mg will be taken as 2 tablets by mouth, each containing 200 mg of etavopivat. Etavopivat may be taken with or without food. Each dose should be taken with a glass of water. As part of the study, the participants will be asked to visit the clinic frequently. The participant will have the opportunity to participate in a 48-week optional extension treatment period. The optional extension treatment period will allow continued as-sessment of safety of etavopivat in paediatric patients. At the end of the study, if deemed appro-priate the participant, the caregiver, and the study doctor, the participant may be offered the op-portunity to participate in a separate study to continue receiving etavopivat. If/when this separate study becomes available, the participant may only transfer to the new study after completion of the 52-week primary treatment period and at any time during the 48-week optional extension treatment period. | Sickle Cell Disease | ALL | CHILD | All India Institute of Medical Sciences (AIIMS), Raipur, Raipur, Chhattisgarh, 492099, India|All India Institute of Medical Sciences-Delhi, Delhi, 110029, India|Nirmal Hospital Pvt. Ltd., Gujarat, 395002, India|Suretech Hospital and Research Centre Ltd., Nagpur, 440012, India|Indira Gandhi Government Medical College & Hospital, Nagpur, 440018, India|University of Ibadan, University College Hospital, Ibadan, 200285, Nigeria|Aminu Kano Teaching Hospital (AKTH), Kano, 700101, Nigeria|Lagos University Teaching Hospital, Lagos, Lagos, 102215, Nigeria|Sultan Qaboos University Hospital, Muscat, 123, Oman |
| Developing the Family Map: Looking at Communal Coping | Background: - Knowing one s family medical history is a part of staying healthy. Some health risks run in families, and knowing these risks can promote more healthy behavior. Different social and cultural factors may affect how family members share this information. Genetic risk information that is shared in one family may not be shared in the same way in another. This information may also be shared differently between spouses, siblings, or parents and children. It may even be shared with more distant relatives. Knowing the information that family members share and how they share it may help researchers improve genetic disease treatment and support plans. Family surveys of people who have genetic health risks may help provide this information. Objectives: - To study how family members affected by genetic-related diseases share health information with each other. Eligibility: * Individuals at least 18 years of age who can read English or Spanish. * Participants affected by a genetic disease or be related or married to someone who has the disease. Design: * Participants will be screened with an initial questionnaire. They will identify their genetic disease and provide a basic health history. * Participants who have the disease will complete an online survey or participate in a personal interview. The questions will take about 45 minutes to 1 hour to answer. The survey will ask about family health history and family support. Participants will also provide referrals to a spouse or relatives who will participate in the study. * The spouse or relative will answer a similar survey. The survey will ask about health history and support for the spouse/relative with the disease. * A gift card will be given as thanks for participating in the study. | Sickle Cell|Diabetes|Cancer|Cardiovascular Disease|Genetic Screening | ALL | ADULT, OLDER_ADULT | National Human Genome Research Institute (NHGRI), 9000 Rockville Pike, Bethesda, Maryland, 20892, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229-3039, United States|Geisinger Autism & Developmental Medicine Institute, Lewisburg, Pennsylvania, 17837, United States |
| Pharmacokinetics (PK) of Liquid Hydroxyurea in Pediatric Patients With Sickle Cell Anemia | Hydroxyurea (HU) is approved by the United States Food and Drug Administration (FDA) to treat adults with sickle cell anemia. Hydroxyurea has also been tested and used with children with sickle cell anemia. However, there are not many studies describing the disposition of drug in children less than 5 years old. The FDA has requested this study to better understand how children ages 2 to 17 years with sickle anemia absorb and eliminate the drug (this is called pharmacokinetics). The investigators will measure how much Hydroxyurea (HU) gets into the bloodstream at different time points after taking this medication. | Sickle Cell Anemia | ALL | CHILD | Children's Hospital of Alabama, Birmingham, Alabama, 35233, United States|Children's Memorial Hospital (Northwestern University), Chicago, Illinois, 60614-3363, United States|Columbia University Medical Center, New York, New York, 10032, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|UT Southwestern University Hospital, Dallas, Texas, 75390-9063, United States|Children's Hospital of Wisconsin, Wauwatosa, Wisconsin, 53226, United States |
| Effects of Transfusion of Older Stored Red Cells | The purpose of this study is to determine the effects of transfusion of fresh and stored blood on patients. The investigators hope to test: * whether a similar effect (older stored blood is associated with worse outcomes) is seen in chronically transfused patients with hemoglobinopathies. This patient population will also allow the investigators to test whether iron- chelation therapy is beneficial in this setting. * whether washing or cryopreserving the red blood cells has any effect on this outcome. These findings may explain the immunomodulatory effects of older stored blood in patients and will help us develop safer transfusion products for patients. | Sickle Cell Disease|Thalassemia | ALL | CHILD, ADULT, OLDER_ADULT | Columbia University Medical Center, New York, New York, 10032, United States |
| A Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics and Efficacy of Crovalimab for the Management of Acute Uncomplicated Vaso-Occlusive Episodes (VOE) in Participants With Sickle Cell Disease (SCD). | The purpose of this study is to evaluate crovalimab for the treatment of a sickle cell pain crisis (also known as a VOE) that requires hospitalisation in adult and adolescent participants with SCD. The primary objective of this study is safety and will additionally evaluate pharmacokinetics (how crovalimab is processed by your body), pharmacodynamics (how your body reacts to crovalimab) and the preliminary efficacy of crovalimab compared with placebo. | Sickle Cell Disease | ALL | CHILD, ADULT | Children'S Healthcare of Atlanta, Atlanta, Georgia, 30329, United States|Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|Icahn School of Medicine at Mount Sinai, New York, New York, 10029, United States|East Carolina University, Greenville, North Carolina, 27834, United States|Hospital Sao Rafael - HSR, Salvador, Bahia, 41253-190, Brazil|Hospital das Clinicas - UFRGS, Porto Alegre, Rio Grande Do Sul, Brazil|CHU Henri Mondor, Créteil, 64010, France|Azienda Ospedaliera di Verona-Policlinico G.B. Rossi, Verona, Veneto, 37134, Italy|International Cancer Institute (ICI), Eldoret, 30100, Kenya|Gertrude's Children Hospital, Nairobi, Kenya|American University of Beirut - Medical Center, Beirut, 1107 2020, Lebanon|Hopital Nini, Tripoli, Lebanon|Amsterdam UMC Location VUMC, Amsterdam, 1081 HV, Netherlands|Charlotte Maxeke Johannesburg Hospital, Johannesburg, 2193, South Africa|Hospital General Univ. Gregorio Maranon, Madrid, 28009, Spain|Hospital Universitario Virgen del Rocio, Sevilla, 41013, Spain|Hospital Universitario Miguel Servet, Zaragoza, 50009, Spain|Hammersmith Hospital, London, W12 OHS, United Kingdom |
| Mobilization and Handling of Stem Cells for Transplant From Healthy Volunteers With Sickle Cell Trait | This study will examine the effects of granulocyte colony-stimulating factor (G-CSF) on bone marrow stem cells in healthy volunteers with sickle cell trait and determine if cells collected for transplantation from donors with sickle cell trait require special handling. Stem cells, which the bone marrow produces, are responsible for making all the different kinds of blood cells. They are the cells used in bone marrow, or stem cell, transplantation. The drug G-CSF, which is a naturally occurring hormone, causes stem cells to mobilize-that is, to be released from the bone marrow and enter the blood stream. This drug is given to stem cell donors to increase the amount of cells that can be collected. Stem cell donors for patients with sickle cell disease are often healthy siblings of the patient who have a matching bone marrow type. Some siblings carry the sickle cell trait, however, and, even though they do not have sickle cell disease and their blood and bone marrow are normal, it is not known how their cells will react to G-CSF stimulation. Nor is it known if their stem cells require special methods of removal, processing or storing. Healthy volunteers 18 years or older with sickle cell trait who have no history of sickle cell disease and no known medical problems may be eligible for this study. Participants will have a medical history and physical examination, including blood tests and urinalysis. They will receive injections of G-CSF under the skin once a day for 5 days. On the fifth day, stem cells will be collected through leukapheresis. In this procedure, whole blood is drawn from an arm vein, similar to donating whole blood. The blood then circulates through a cell separator machine, the stem cells are removed, and the rest of the blood is transfused back to the donor through a vein in the other arm. The information gained from this study will be used to ensure the safety of stem cell donors with sickle cell trait and to better prepare stem cells for transplantation in sickle cell patients. | Sickle Cell Trait | ALL | CHILD, ADULT, OLDER_ADULT | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), Bethesda, Maryland, 20892, United States |
| Role of Placenta Growth Factor in Sickle Acute Chest Syndrome | The purpose of this research study is to find out whether Placenta Growth Factor (PlGF) and related tests can predict the development of acute chest syndrome (ACS) in patients with sickle cell disease (SCD) during a period where patients are well and during admission to the hospital for an acute sickle event to see if these measures can predict the development of ACS. Understanding events precipitating ACS may lead to preventative and interventional therapies which will improve patient outcomes and quality of life. | Anemia, Sickle Cell | ALL | CHILD, ADULT | Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States |
| Implementing an Individualized Pain Plan (IPP) for ED Treatment of VOE's in Sickle Cell Disease | The overall purpose of this proposed study is to improve management of vaso-occlusive episodes (VOEs) in adult EDs. We aim to implement NHLBI recommendations for VOE treatment by embedding Individualized Pain Plans (IPPs) in the electronic health record (EHR). The EHR-embedded IPP will serve as a record of patients' SCD genotype and will include analgesic medication recommendations developed by the SCD provider. In this project, we will provide access to the IPP for both adult patients with SCD and ED providers. The proposed multisite study will use a pre-post study design, with a core set of mandatory intervention components and strategies for each participating site and optional components and strategies to allow for intervention adaptation to local needs and resources. The EHR-embedded IPP will be available for all adult ED providers to use as their routine practice, and patients will be invited to participate and enroll in the study. We will use a simplified Technology Acceptance Model to explain the use of the IPP and the RE-AIM framework to assess the Reach, Effectiveness, Adoption, Implementation, and Maintenance of the intervention. | Sickle Cell Disease|Genetic Disease|Hematologic Diseases|Anemia, Sickle Cell | ALL | ADULT | University of California San Francisco, Oakland, California, 94609, United States|Georgia Regents University, Augusta, Georgia, 30912, United States|University of Illinois, Chicago, Illinois, 60612, United States|Washington University, Saint Louis, Missouri, 63110, United States|Icahn School of Medicine at Mount Sinai, New York, New York, 10029, United States|Duke University, Durham, North Carolina, 27710, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|St. Jude's, Memphis, Tennessee, 38105, United States |
| Expanded Access to Voxelotor for Patients With Sickle Cell Disease Who Have No Alternative Treatment Options | The intent of this open-label, multicenter expanded access program (EAP) is to provide early access to voxelotor prior to market authorization | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Alabama Oncology, Birmingham, Alabama, 35211, United States|University of South Alabama, Mobile, Alabama, 36693, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30342, United States|Augusta University, Augusta, Georgia, 30912, United States|Our Lady of the Lake Physician Group, Baton Rouge, Louisiana, 70809, United States|University Medical Center New Orleans, New Orleans, Louisiana, 70112, United States|The John Hopkins Hospital, Baltimore, Maryland, 21287, United States|Rutgers Cancer Institute of New Jersey, New Brunswick, New Jersey, 08901-1914, United States|Montefiore Medical Center PRIME, Bronx, New York, 10461, United States|Queens Hospital Center, Jamaica, New York, 11432, United States|University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, 27514, United States|Duke Department of Pediatrics, Durham, North Carolina, 27710, United States|The Ohio State University Wexner Medical Center, Columbus, Ohio, 43210, United States|Thomas Jefferson University Hospital, Philadelphia, Pennsylvania, 19107, United States|University of Pittsburgh Medical Center Health System, Pittsburgh, Pennsylvania, 15232, United States|Cook Children's Medical Center, Fort Worth, Texas, 76104, United States|Texas Children's Hospital, Houston, Texas, 77030, United States|VCU Health, Richmond, Virginia, 23298, United States |
| The Afolabi Stroke Registry for Children and Young Adults With SCD in Northern Nigeria | Sickle Cell Anemia (SCA) occurs in 300,000 newborns per year in the world, with 150,000 affected births in Nigeria, alone. With improvement in survival for children with SCA in both high- and low-resource countries, neurological morbidity is an emerging significant public health challenge, particularly in countries with a high rate of sickle cell disease (SCD). Both silent cerebral infarcts (SCI) and overt strokes result in significant neurological morbidity and premature death. Five NIH-funded randomized controlled trials (RCT) demonstrated that regular blood transfusion or hydroxyurea therapy are efficacious treatments for primary and secondary stroke prevention in children with SCA. Despite the observation that at least 99% of children with SCA in high-resource settings reach adulthood, and approximately 60% of adults will experience one or more strokes (\~50% with SCI and \~10% with overt strokes) and the high disease-burden in Nigeria, the prevalence and incidence rates of new and recurrent stroke (overt and silent strokes)have not been collected systematically in children and young adults (16-25 years old) with SCA. In the last decade, there has been growing use of stroke registries in economically advanced nations, particularly for epidemiological purposes of trend analysis, clinical effectiveness, compliance to guidelines, assessment of implementation, adoption of novel techniques, and quality improvement process. For the first time in clinical centers in Nigeria, the Investigators will conduct an observational epidemiological study to document the prevalence and track the incidence of new and recurrent strokes in children and young adults with SCD. The Investigators will create a stroke registry referred to as the Afolabi Stroke Registry for Children and Young Adults with Sickle Cell Disease in Nigeria. The overall purpose of the stroke registry is to document the natural history of SCD in a low-resource setting and to improve the quality of the care of children and young adults with SCD living in Nigeria. | Sickle Cell Disease|Stroke|Neurologic Manifestations|Neurological Morbidity|Sickle Cell Anemia in Children|Sickle Cell Anemia | ALL | CHILD, ADULT | Vanderbilt University Medical Center, Nashville, Tennessee, 37232-9000, United States|Jamil Galadanci, Kano, PMB 3452, Nigeria|Aminu Kano Teaching Hospital, Kano, Nigeria |
| Albuminuria Reduction With Renin Angiotensin System Inhibitors in SCA Patients | The prevalence of Sickle Cell Associated Nephropathy (SCAN) is increasing and is a growing concern. Microalbuminuria is detected in the early onset of SCAN. Noteworthy, as in diabetic nephropathy, hyperfiltration seems to be a frequent finding, with, in our series, an overall incidence of 57 % and suggests a pathological links between glomerular hyperpressure and glomerulosclerosis which occurs several years after. Nitric oxide (NO) deficiency and the renin angiotensin system (RAS) are likely to be involved in the glomerular hyperpressure leading to hyperfiltration. Renin angiotensin antagonists are currently given for NEPHROPROTECTION in numerous nephropathy including SCAN despite few available reports. The percentage of decrease of albuminuria or the percentage of responders (ie patient normalizing albuminuria) has never been reported to our knowledge in SCAN patients at the time of hyperfiltration. The focus of our study is therefore to 1) Quantify albuminuria reduction after 6 months RAS treatment (primary end point); 2) Quantify glomerular filtration rate (GFR) reduction after 6 months of RAS treatment, and to test the hypothesis of a beneficial effect of RAS inhibitors on several biomarkers assessing hemolysis, NO inhibition and the endothelial damages (secondary end points). The ultimate aim of our study is to identify relevant (new) biomarkers associated to hyperfiltration and/or albuminuria decrease (/normalization). | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Centre de la Drépanocytose, Service de Médecine Interne. Hôpital Tenon, 4 Rue de la Chine, Paris, 75020, France |
| Phase II Randomized Trial:Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers | OBJECTIVES: I. Compare the efficacy of local care alone vs local care plus arginine butyrate in terms of healing rate in patients with refractory sickle cell ulcers. II. Determine the effect of arginine butyrate therapy on tissue factors related to promotion or inhibition of wound healing in these patients. III. Determine whether the regimen used in this study is appropriate for testing in pivotal trials. | Skin Ulcers|Sickle Cell Anemia | ALL | CHILD, ADULT | University of Illinois College of Medicine, Chicago, Illinois, 60612, United States|Boston University School of Medicine, Boston, Massachusetts, 02118, United States|Mount Sinai School of Medicine, New York, New York, 10029, United States|University of Tennessee, Memphis Cancer Center, Memphis, Tennessee, 38103, United States |
| Effects of Nitric Oxide and Nitroglycerin in Patients With Sickle Cell Anemia | Sickle cell anemia is the most common genetic disease affecting African-Americans. About 1 in every 1000 African-Americans has the disease and 1 in every 12 carry the genes that could be passed on to their children. People with sickle cell anemia have abnormal hemoglobin, the molecules responsible for carrying oxygen in the blood. The abnormal hemoglobin can cause damage to the red blood cells. The damaged red blood cell may then stick in the blood vessels and cause pain and injury to organs. Some of the complications caused by the sticking of blood cells are called acute pain crisis and acute chest syndrome (ACS). Nitric oxide (NO) is a gas that has been proposed as a possible therapy for the ACS complication of sickle cell anemia. Studies have shown that NO may favorably affect sickle cell hemoglobin molecules, thereby improving blood flow through small vessels. This study is designed to evaluate the effects of NO, when taken in combination with a drug called nitroglycerin on patients with sickle cell anemia and normal volunteers. The effects of these two drugs only last while the patient is receiving them. Researchers hope the information learned from this study will help to develop new therapies for sickle cell anemia. | Chest Pain|Sickle Cell Anemia | ALL | CHILD, ADULT, OLDER_ADULT | Warren G. Magnuson Clinical Center (CC), Bethesda, Maryland, 20892, United States |
| Is the Preoperative Preparation of Sickle Cell Patients Optimal: Assessment of Practices and Post-operative Complications | Children with sickle cell disease systematically receive a transfusion 2 to 5 days before scheduled surgery (with the exception of minor surgeries) in order to avoid post-operative complications of which the vaso-occlusive crisis and acute thoracic syndrome are the most frequent. This standardized preoperative protocol was established on the basis of the results of large-scale randomized studies, most of which date back over ten years, and which have demonstrated the beneficial effects of transfusion (or transfusion exchange) preoperatively. To date, several other more recent studies (but not controlled) have questioned this type of systematic management. The purpose of this study is to review retrospectively data of sickle cell children who have undergone elective surgery at the Huderf in the last ten years and to identify the eventual complications encountered. The most common procedures in these patients are: tonsillectomy with or without associated adenoids, splenectomy and cholecystectomy. General data on sickle cell disease (history, genotype, G6PD deficiency, biology and previous complications), pre-surgical preparation, surgery and post surgical management and complications will be collected and analyzed. This retrospective analysis will allow an objective assessment of the current quality of care and will provide useful data to improve patient management. | Sickle Cell Disease | ALL | CHILD, ADULT | Hôpital Universitaire Des Enfants Reine Fabiola, Brussels, Brussles, 1020, Belgium |
| Algorithm for Apherisis Monitoring and Prescription Assistance in Sickle Cell Patients (ALGODREP) | The main objective of this study is to prove the superiority of a procedure which calculates the volume of RBCs to transfuse and the time between apheresis based on this algorithm, compared to the current procedure. The primary endpoint would be the number of patients with individually achieved objectives in terms of % HbS before each apheresis (which reflects the effectiveness of the previous apheresis) over a period of 12 months. The secondary objectives would be to compare the volume differences of transfused RBCs in both groups over a period of 12 months, the occurrence of clinical events and the satisfaction of patients and physicians. The investigators hope that this study would improve the efficiency and the performance of apheresis in sickle cell patients. The investigators also hope to facilitate the organization of procedures with the flexibility that would allow the use of this algorithm. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | EFS Rhône-Alpes-Auvergne, Saint-Priest-en-Jarez, Auvergne Rhône-Alpes, 42277, France|Centre de Santé EFS, Besançon, Bourgogne Franche-Comté, 25000, France|Centre de Santé EFS, Rennes, Bretagne, 35016, France|Centre de Santé EFS, Tours, Centre-Val De Loire, 37206, France|CHU Kremlin Bicêtre, Le Kremlin-Bicêtre, Ile De France, 94270, France|Hôpital Henri Mondor, Créteil, Ile-de-France, 94010, France|Centre de Santé EFS, Bordeaux, Nouvelle-Aquitaine, 33035, France|CHU de Martinique, Le Lamentin, 97232, Martinique |
| Observational Prospective Study Measuring the Impact of the Use of a Hypnotic Script Associated With Virtual Reality on the Pain of the Child's Sickle Cell During a Vaso-occlusive Crisis | The purpose of this study is to evaluate the effect of Virtual Reality induced Hypnosis on patients' pain scores, anxiety and the use of analgesics during a vaso-occlusive crisis | Sickle Cell Disease | ALL | CHILD, ADULT | Trousseau Hospital, General pediatric departement, Paris, 75012, France |
| National Exhaustive Cohort of Hereditary Stomatocytoses and Other Channelopathies Affecting the Red Blood Cell | Hereditary stomatocytosis is a heterogeneous group of rare constitutional diseases of dominant transmission in the vast majority of cases. The data concerning their clinical and biological presentation, and their evolution are few, and come from about thirty clinical cases. The constitution of an exhaustive French cohort of hereditary stomatocytosis will improve the establishment of the diagnosis and the management of patients | Stomatocytosis | ALL | CHILD, ADULT, OLDER_ADULT | AP-HP, Bicêtre Hospital, Pediatrics - Hematology - Reference center for Sickle cell anemia, Thalassemia and other constitutional diseases of the red blood cell, Le Kremlin Bicêtre, 94275, France |
| Multicenter Observational Study on Myocardial Iron Overload in 3 Multitransfused Populations | The investigators' primary objective is to study prevalences of myocardial iron overload, defined as a cardiac T2\*\< 20 ms, in 3 populations of multiply transfused patients, affected with thalassemia, sickle cell disease, and myelodysplasia. | Thalassemia|Sickle Cell Disease|Myelodysplasia | ALL | CHILD, ADULT, OLDER_ADULT | Hôpital Necker Enfants Malades, Paris, 75015, France |
| Increasing Documentation and Disclosure of Sickle Cell Trait Status: an Implementation Science Approach | The hemoglobinopathy newborn screen (NBS) performed on all neonates in the U.S. allows for early life-saving medical care for infants with sickle cell disease (SCD), an autosomal recessive genetic disorder. Because of its detection method, the NBS incidentally reveals hemoglobinopathy traits including sickle cell trait (SCT). In an effort to uphold the rights of the newborn to their medical data and preserve autonomy in medical decision making, pediatric and genetic society guidelines recommend disclosure and documentation of SCT results during infancy. Despite this guidance, a large guideline-to-practice gap exists: SCT status is grossly under-documented in the pediatric electronic health record and few adults report knowing their SCT status despite universal screening. We plan to evaluate the effect of a toolkit of SCT Documentation and Disclosure (SCT-DD) strategies on documentation and disclosure of SCT by pediatric primary care providers in a 2-arm randomized interrupted time series trial. | Sickle Cell Trait | ALL | CHILD, ADULT, OLDER_ADULT | Nemours Children's Hospital, Delware, Wilmington, Delaware, 19803, United States |
| Pilot Study on the Effects of Intravenous Ketamine on Acute Pain Crisis in Patients With Sickle Cell Disease | The purpose of this pilot study is to provide a preliminary assessment of the feasibility and efficacy of intravenous ketamine in controlling pain in patients with sickle cell disease (who are admitted to the hospital with severe, acute pain crisis, and who have been resistant to intravenous narcotics). | Sickle Cell Disease | ALL | CHILD, ADULT | The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States |
| Long Term Follow up in Sickle Cell Patients Treated by Hydroxyurea | Hydroxyurea was found to be a good treatment in adult patients with sickle cell anemia with significant decrease in the frequency of vaso-occlusive crises and other crises related to SCA. Several studies were published with relative short term follow up in pediatric and young adult age. The purpose of this study is to assess the long term follow up in a group of patients that initiated Hydroxyurea treatment in childhood. | Sickle Cell Anemia|Sickle Cell Thalassemia | ALL | CHILD, ADULT | Pediatric Hematology Unit - HaEmek Medical Center, Afula, 18101, Israel |
| Comparison of Patient Centered Outcomes for People With Sickle Cell Disease in the Acute Care Setting | The Emergency Department has been the standard location where patients with Sickle Cell Disease (SCD) go to seek care for the treatment of acute painful events. Vaso- Occlusive Crisis (VOC) is the most common complication of SCD, The purpose of this study is to compare patient centered outcomes for patients being treated for an uncomplicated VOC in Infusion Centers (IC) and Emergency Departments (ED) in four locations around the United States. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Our Lady of the Lake Hospital, Baton Rouge, Louisiana, 70809, United States|Cleveland Medical Center at University Hospitals, Cleveland, Ohio, 44106, United States|Medical College of Wisconsin, Blood Center, Milwaukee, Wisconsin, 53201, United States |
| Evaluation of the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration of ICL670 Relative to Deferoxamine(DFO). | The safety, tolerability, effects on liver iron concentration and pharmacokinetics of ICL670 is studied in sickle cell disease patients with transfusional hemosiderosis. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | University of South Alabama College of Medicine, Mobile, Alabama, 36604, United States|Loma Linda University Medical Center, Loma Linda, California, 92354, United States|Children's Hospital Los Angeles, Los Angeles, California, 90027, United States|Children's Hospital & Research Center at Oakland, Oakland, California, 90609, United States|University of Colorado Health Science Center, Denver, Colorado, 80262, United States|Howard University Hospital, Washington, District of Columbia, 20059, United States|St Joseph Children's Hospital of Tampa, Tampa, Florida, 33607, United States|Grady Hospital, Georgia Comprehensive Sickle Cell Center, Atlanta, Georgia, 30303, United States|Medical College of Georgia, Augusta, Georgia, 30912, United States|University of Illinois at Chicago, Chicago, Illinois, 60612, United States|Children's Memorial Hospital, Chicago, Illinois, 60614, United States|Tulane University Medical Center, New Orleans, Louisiana, 70112, United States|LSUHSC Dept of Pediatrics, Shreveport, Louisiana, 71130, United States|Children's Hospital Boston, Boston, Massachusetts, 02118, United States|Karmanos Cancer Institute, Detroit, Michigan, 48201, United States|Montefiore Medical Center, Bronx, New York, 10467, United States|New York Methodist Hospital, Brooklyn, New York, 11215, United States|New York Presbyterian Hospital, New York, New York, 10021, United States|Wake Forest University Health Sciences, Winston-Salem, North Carolina, 27157, United States|Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States|University of Cincinnati, Cincinnati, Ohio, 45267, United States|Milton S. Hershey Medical Center, Hershey, Pennsylvania, 17033, United States|Yasin, Philadelphia, Pennsylvania, 19104, United States|Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, 15213, United States|Palmetto Health Richland, Columbia, South Carolina, 29203, United States|Santee Hematology/Oncology, Sumter, South Carolina, 29150, United States|St. Jude's Children Research Hospital, Memphis, Tennessee, 38105, United States|Texas Children's Hospital, Houston, Texas, 77030, United States|The Methodist Hospital, Houston, Texas, 77030, United States|Scott & White Memorial Hospital, Temple, Texas, 76508, United States|Children's Hospital of the King's Daughters, Norfolk, Virginia, 23507, United States|Novartis Investigative Site, Toronto, Canada|Novartis Investigative Site, Creteil, France|Novartis Investigative Site, Paris, France|Novartis Investigative Site, Catania, Italy|Novartis Investigative Site, Genova, Italy|Novartis Investigative Site, Milano, Italy|Novartis Investigative Site, Roma, Italy|Novartis Investigative Site, London, United Kingdom |
| Effectiveness of New Analgesic Strategy Compared to the Usal Antalgic Strategy | Quality of life of adult patients with sickle cell disease is deeply impaired by severe adverse medical events that inadvertently occur throughout their time life. Indeed, patients not presenting a life threatening condition often present to the emergency department with sickle cell disease crisis related pain. Currently, the effectiveness of specific analgesic strategies for treating sickle cell disease crisis related pain are mostly based on acetaminophen and opioid derivates combination along with oxygen delivery. Those strategies are effective but may last up to half an hour to obtain pain relief. This delay mostly depends on the availability of venous access and on individual patient response to treatment. Nitrous oxide is a volatile efficient analgesic therapy that has been repeatedly shown to allow rapid analgesia in the emergency department setting. The investigators hypothesise that a new analgesic strategy (rapid optimized analgesic strategy) including nitrous oxide and nefopam would be as safe and more rapidly effective than current analgesic strategy. | Sickle Cell Anemia | MALE | ADULT, OLDER_ADULT | Henri Mondor Hospital, Emergency Department, Créteil, 94000, France |
| Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients | The purpose of the Phase 2 CSEG101B2201 study is to confirm and to establish appropriate dosing and to evaluate the safety in pediatric participants ages 6 months to \<18 years with a history of VOC with or without HU/HC, receiving crizanlizumab for 2 years. The efficacy and safety of crizanlizumab was already demonstrated in adults with sickle cell disease. The approach is to extrapolate from the PK/pharmacodynamics (PD) already established in the adult population. The study is designed as a Phase II, multicenter, open-label study. | Sickle Cell Disease (SCD) | ALL | CHILD | University Of Alabama, Birmingham, Alabama, 35233, United States|Childrens National Hospital, Washington, District of Columbia, 20010, United States|Univ of Florida College of Medicine, Gainesville, Florida, 32610, United States|Joe DiMaggio Childrens Hospital, Hollywood, Florida, 33021, United States|Childrens Healthcare of Atlanta, Atlanta, Georgia, 30342, United States|Childrens Hosp Boston Dept of Hematology, Boston, Massachusetts, 02115, United States|Childrens Hospital at Montefiore, Bronx, New York, 10467, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|East Carolina University, Greenville, North Carolina, 27834, United States|Childrens Hospital Of Philadelphia, Philadelphia, Pennsylvania, 19104-4399, United States|Medical Uni of South Carolina, Charleston, South Carolina, 29425, United States|Cook Childrens Medical Center, Fort Worth, Texas, 76104, United States|Novartis Investigative Site, Brussel, 1000, Belgium|Novartis Investigative Site, Laeken, 1020, Belgium|Novartis Investigative Site, Liege, 4000, Belgium|Novartis Investigative Site, Salvador, BA, 41253-190, Brazil|Novartis Investigative Site, Ribeirao Preto, SP, 14048-900, Brazil|Novartis Investigative Site, Sao Paulo, SP, 01232-010, Brazil|Novartis Investigative Site, Montreal, Quebec, H3T 1C5, Canada|Novartis Investigative Site, Cali, Valle Del Cauca, 760012, Colombia|Novartis Investigative Site, Monteria, 230004, Colombia|Novartis Investigative Site, Paris 15, 75015, France|Novartis Investigative Site, Heidelberg, 69120, Germany|Novartis Investigative Site, Nagpur, Maharashtra, 440009, India|Novartis Investigative Site, Padova, PD, 35128, Italy|Novartis Investigative Site, Orbassano, TO, 10043, Italy|Novartis Investigative Site, Beirut, 1107 2020, Lebanon|Novartis Investigative Site, Tripoli, 1434, Lebanon|Novartis Investigative Site, Muscat, 123, Oman|Novartis Investigative Site, Esplugues De Llobregat, Barcelona, 08950, Spain|Novartis Investigative Site, Barcelona, Catalunya, 08035, Spain|Novartis Investigative Site, Palma De Mallorca, Islas Baleares, 07120, Spain|Novartis Investigative Site, Madrid, 28009, Spain|Novartis Investigative Site, Adana, 01330, Turkey|Novartis Investigative Site, Mersin, 33110, Turkey|Novartis Investigative Site, London, SE1 7EH, United Kingdom |
| A Study to Evaluate the Safety, Tolerability, and Immunogenicity of V114 in Children With Sickle Cell Disease (V114-023/PNEU-SICKLE) | This study is designed to describe the safety, tolerability, and immunogenicity of V114 in children with sickle cell disease. | Pneumococcal Infections | ALL | CHILD | Nemours/Alfred I. duPont Hospital for Children ( Site 0113), Wilmington, Delaware, 19803, United States|Children's Healthcare of Atlanta ( Site 0100), Atlanta, Georgia, 30303, United States|Children's Hospital of Michigan ( Site 0111), Detroit, Michigan, 48201, United States|Newark Beth Israel Medical Center ( Site 0115), Newark, New Jersey, 07112, United States|University of Rochester Medical Center ( Site 0105), Rochester, New York, 14642, United States|Cincinnati Children's Hospital Medical Center ( Site 0101), Cincinnati, Ohio, 45229, United States|Santa Casa de Misericordia de Belo Horizonte ( Site 0200), Belo Horizonte, MG, 30150-221, Brazil|Hospital Santo Antonio - Obras Sociais Irma Dulce ( Site 0205), Salvador, 40420-000, Brazil|Santa Casa de Misericordia de Sao Paulo ( Site 0202), Sao Paulo, 01221-900, Brazil|Clinica de la Costa Ltda. ( Site 0300), Barranquilla, Atlantico, 080020, Colombia|Centro de Estudios en Infectologia Pediatrica SAS ( Site 0301), Cali, Valle Del Cauca, 760045, Colombia|Fundacion Dominicana de Perinatologia PRO BEBE INC ( Site 0402), Distrito Nacional, Santo Domingo, 10204, Dominican Republic|Clinical Research Republica Dominicana ( Site 0401), Santo Domingo, 10122, Dominican Republic|Caimed Dominicana S.A.S ( Site 0400), Santo Domingo, 10205, Dominican Republic|Agia Sophia Children s Hospital ( Site 0700), Athens, 115 27, Greece|Hippokration General Hospital of Thessaloniki ( Site 0701), Thessaloniki, 546 42, Greece|Ospedale San Martino ( Site 0800), Genova, 16132, Italy|Cevaxin ( Site 0500), Panama, 0816-00383, Panama|Cevaxin ( Site 0502), Panama, 0816-00383, Panama |
| Quality of Life Study for Sickle Cell Patients Treated With Jobelyn (Sorghum Bicolor Extract) | The purpose of this study is to determine the antioxidant effect of prolonged use of sorghum bicolor (jobelyn) to increase the level of plasma superoxide dismutase and glutathione reductase in patients with sickle cell disease and to determine if there is any improvement in the quality of life of the patients. | Other Sickle-cell Disorders With Crisis, Unspecified | ALL | CHILD, ADULT | Lagos State University Teaching Hospital, Ikeja, Lagos, 100001, Nigeria |
| A Study to Assess the Safety and Pharmacokinetics of HBI-002, an Oral Carbon Monoxide Therapeutic, in Healthy Volunteers | This is a single center, open label Phase 1 clinical trial in normal adult subjects to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO), with single ascending doses (SAD), followed by multiple dose with doses daily for 7 days. | Anemia, Sickle Cell | ALL | ADULT | Contact Company, San Diego, California, 92121, United States |
| Hydroxyurea in the Emergency Room to Lessen Pain in Sickle Cell Crisis | This study will investigate the safety, tolerability and potential for the use of up to three daily doses of 30-40 mg/kg HU (daily) upon hospitalization for painful vaso-occlusive crises . | Anemia, Sickle Cell|Anemia; Sickle-Cell, With Crisis | ALL | ADULT | Hemorio, Rio de Janeiro, Brazil |
| A Dose Escalation Study of Intravenous L-citrulline in Steady-state Sickle Cell Disease | The purpose of this study is to assess the maximum tolerated dose, safety and pharmacokinetics of an investigational drug, intravenous (IV) citrulline, in subjects in steady-state sickle cell disease. | Sickle Cell Disease | ALL | ADULT | University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States |
| Dose Escalation Study to Evaluate the Safety, Tolerability, PK and PD of Voxelotor in Patients With SCD | Study participants will undergo up to four periods of voxelotor administered orally at progressively higher dose levels from 1500 mg until either a maximum tolerated dose (MTD) or 3000 mg/day dose is reached, whichever occurs first | Sickle Cell Disease | ALL | ADULT | Guy's and St Thomas' NHS Foundation Trust, London, United Kingdom|Guy's Hospital, London, United Kingdom|Hammersmith Hospital, London, United Kingdom|Homerton University, London, United Kingdom|King's College Hospital, London, United Kingdom|Royal London Hospital, Barts Health NHS Trust, London, United Kingdom |
| Applying Directly Observed Therapy to Hydroxyurea to Realize Effectiveness | This study is for caregivers of young children with sickle cell disease and adolescents with sickle cell disease who are currently prescribed hydroxyurea and are receiving care at one of the study sites. The study will assess retention and engagement during a pilot randomized control trial comparing video directly observed therapy (VDOT) to attention control. We also hope to understand more about patient and family preferences longer-term adherence monitoring and intervention. Participants will use an electronic adherence monitor (provided by the study team) to measure how often they are taking their hydroxyurea. Participants will also be asked to complete questionnaires throughout the study period to provide information about their expectations for, experience with, and satisfaction with the study materials. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Lurie Children's Hospital, Chicago, Illinois, 60611, United States|Hasbro Children's Hospital, Providence, Rhode Island, 02903, United States |
| Voxelotor Brain Oxygenation and Neurocognitive Study | This is an open label, single arm multicenter trial to evaluate the effect of voxelotor treatment on cerebral blood flow (CBF) and neurocognitive function in adolescent and young adult participants (12-30 years of age) with sickle cell disease (SCD). | Sickle Cell Disease | ALL | CHILD, ADULT | Washington University School of Medicine, Saint Louis, Missouri, 63110, United States|The Children's Hospital at Montefiore, Bronx, New York, 10467, United States |
| Expanded Access to CD34+ Selection Utilizing Miltenyi CliniMACS Prodigy® for Patients Receiving Peripheral Blood Stem Cell Transplantations and Stem Cell Boosts | Allogeneic stem cell transplantation (alloSCT) is utilized for various underlying diseases. AlloSCT is limited by graft versus host disease (GVHD), graft rejection, viral infections, and post-transplant lymphoproliferative disorders. To mitigate graft versus host disease, graft manipulation has been taking place with CD34+ selection to decrease T-cells entering into the patient, thus lowering the risk of GVHD. Historically CD34+ manipulation has been performed under a humanitarian use device by utilizing the Miltenyi CliniMACs CD34 Reagent System. This was used for patients with AML in first remission. This approach has additionally been used for patients with sickle cell disease, immune deficiencies, and poor graft function with excellent efficiency. The purpose of this protocol is to create expanded access of CD34+ manipulation for various underlying diseases utilizing the Miltenyi CliniMACS Prodigy® device. | Severe Combined Immunodeficiency|Fanconi Anemia|Dyskeratosis Congenita|Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | |
| Preventing Acute Chest Syndrome by Transfusion Feasibility Study | Acute chest syndrome (ACS) is similar to severe pneumonia and is a common cause of hospitalizations for people with sickle cell disease (SCD). Blood transfusions are one treatment option for ACS. High levels of an enzyme called secretory phospholipase A2 (sPLA2) may be present in people before they develop ACS. This study will determine how well sPLA2 levels can predict the onset of ACS and whether identifying high sPLA2 levels allows enough time to prevent ACS with blood transfusions. Results from this study will help to determine the feasibility of conducting a larger study that would further examine the use of sPLA2 levels and blood transfusions to prevent ACS in people with SCD. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital and Research Center, Oakland, California, United States|A.I. duPont Hospital for Children, Wilmington, Delaware, United States|Children's National Medical Center, Washington, District of Columbia, United States|Howard University Hospital, Washington, District of Columbia, United States|Emory University School of Medicine, Atlanta, Georgia, United States|Medical College of Georgia, Augusta, Georgia, United States|Children's Memorial Hospital, Chicago, Illinois, United States|University of Illinois Sickle Cell Center, Chicago, Illinois, United States|Kosair Children's Hospital, Louisville, Kentucky, United States|Johns Hopkins, Baltimore, Maryland, United States|Boston Medical Center, Boston,, Massachusetts, United States|Brigham & Women's Hospital, Boston,, Massachusetts, United States|Children's Hospital Boston, Boston, Massachusetts, United States|University of Mississippi Medical Center, Jackson, Mississippi, United States|Interfaith Medical Center, Brooklyn, New York, United States|New York Methodist Hospital, Brooklyn, New York, United States|The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States|Duke University Medical Center, Durham, North Carolina, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States|Nationwide Children's Hospital, Columbus, Ohio, United States|Ohio State University, Columbus, Ohio, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States|St. Christopher's Hospital for Children, Philadelphia, Pennsylvania, United States|Virginia Commonwealth University Health Systems, Richmond, Virginia, United States |
| Virtual Reality as an Adjuvant Therapy for Sickle Cell Vaso-Occlusive Crisis in the Pediatric Emergency Department | Vaso-occlusive crisis (VOC) is the most common complaint in patients with sickle cell disease presenting to the emergency room. VOC is most commonly treated with opioids and NSAIDs. However, new research is demonstrating that opioids in addition to virtual reality (VR) is more effective at reducing the experience of pain and pain nerve signals compared to opioids alone. Numerous research studies have demonstrated that VR reduces the experience of pain during painful medical procedures in children, such as venipuncture and burn wound dressing changes. The study aims to add VR to standard of care medical treatment for pediatric patients with sickle cell disease who present to the pediatric emergency department in VOC. Investigators will conduct a retrospective chart review of patients aged 6 to 21 years with sickle cell disease who present to the pediatric emergency department with VOC for the historical control arm. Investigators will also conduct a prospective convenient sampling of patient who receive VR plus standard medical care in patients aged 6 to 21years with sickle cell disease who present to the emergency department with VOC. Investigators hypothesize that VR, in addition to standard medical care, will reduce the experience of pain and hospital admissions compared to the historical control group (standard medical treatment). | Sickle Cell Crisis | ALL | CHILD, ADULT | University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States |
| DSUVIA in Patients With SCD VOC Present in the ED | This is an observational study to improve the treatment of patients with Sickle Cell Disease Vaso-Occlusive Crisis by administering pain medications more quickly after the patient arrives in the emergency department. Specifically, we are using a sublingual opioid called sufentanil \[Dsuvia\] that has already been approved by the Food and Drug Administration (FDA) for the treatment of acute pain. It is being studied as part of this research study to find out if we can relieve the patients pain more quickly and decrease the amount of time the patient needs to spend in the hospital by avoiding a hospital admission after the patients emergency department encounter if the patients pain is adequately controlled. | Sickle-Cell Disease With Crisis | ALL | ADULT, OLDER_ADULT | Tampa General Hospital, Tampa, Florida, 33606, United States |
| Ranibizumab for Neovascularization in Sickle Cell Retinopathy | The purpose of this study is to determine the ocular and non-ocular safety of a single dose of ranibizumab in treating neovascularization secondary to sickle cell retinopathy. | Sickle Cell Anemia|Retinopathy | ALL | ADULT, OLDER_ADULT | Kresge Eye Institute, Detroit, Michigan, 48201, United States |
| Health Literacy - Neurocognitive Screening in Pediatric SCD | The purpose of this study is to determine feasibility and potential benefits of providing a passport card with a summary of neurocognitive feedback results to families of patients with sickle cell disease. Given recent literature suggesting the need to be conscious of health literacy in populations with low socioeconomic status, this project is intended to provide a more health-literate appropriate format of neurocognitive evaluation feedback in the context of a routine screening program offered as a standard of care in the CHW pediatric sickle cell disease clinic. The specific aims is (1) to evaluate differences in caregiver understanding of neurocognitive report findings when provided with a health-literate passport card compared to control group and (2) to evaluate differences in follow-through on neurocognitive report recommendations when provided with a health-literate passport card compared to control group. | Sickle Cell Disease | ALL | CHILD | Children's Wisconsin, Milwaukee, Wisconsin, 53201, United States|Medical College of Wisconsin, Milwaukee, Wisconsin, 53266, United States |
| A Phase - IIa - IIb, Trial to Study the Safety, Tolerability and Efficacy of Memantine as a Long-term Treatment of SCD | Symptomatic sickle cell disease (SCD) is worldwide the most frequent cause for hereditary hemolytic anemia with recurrent pain crises. Hemolysis, vaso- occlusive and pain crises are hallmarks of this disease and are causative for an important socio-economic burden worldwide, especially in Africa. Aside from allogenic stem cell transplantation, which is rarely available and very expensive, at present there is no curative treatment for patients with SCD. The current standard of care includes treatment with Hydroxyurea and symptomatic care such as transfusions, antibiotic/analgesic treatment. Recent findings allowed the investigators to come up with a novel pharmacological target for prophylactic treatment of this group of patients. The investigators showed that N-methyl D-aspartate receptors (NMDARs) are substantially up-regulated in circulating red blood cells (RBCs) of SCD patients. Ca2+ uptake via these non-selective cation channels has major impact on RBC hydration and facilitates polymerization of deoxygenated hemoglobin S variant in RBCs of patients. In vitro observations shows that inhibition of NMDARs with Memantine caused re-hydration and largely prevented hypoxia-induced sickling in RBCs. A pilot trial MemSID (NCT02615847) was conducted in August 2015-March 2017 at the Hematology Division of University Hospital Zurich. A small cohort of adult SCD patients was treated with 20 mg Memantine daily to test safety, tolerability and efficacy of this drug and to assess the effect of Memantine on hemolytic activity and RBC stability. Pilot data reveal safety and an impressive therapeutic potential of Memantine in treating SCD patients. Due to a small number of SCD patients in Switzerland, an extended trial including larger number of adult and adolescent patients will be performed at the Pediatric Hematology Unit of the Emek Medical Center in Afula, Israel | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Emek Medical Centre, Afula, 18101, Israel |
| Genotype -Phenotype Correlation of PKLR Variants With Pyruvate Kinase, 2,3-Diphosphglycerate and Adenosine Triphosphate Activities in Red Blood Cells of People With Sickle Cell Disease | Background: Some people with the same disorder on a genetic level have more complications than others. Researchers want to look for a link between the PKLR gene and sickle cell disease (SCD) symptoms. The PKLR gene helps create a protein, called pyruvate kinase that is essential in normal functioning of the red blood cell. Differences in the PKLR gene, called genetic variants, may cause some changes in the pyruvate kinase protein and other proteins, that can affect functioning of the red blood cell adding to the effect of SCD. Researchers can study these differences by looking at DNA (the material that determines inherited characteristics). Objective: To study how the PKLR gene affects sickle cell disease. Eligibility: Adults ages 18-80 of African descent. They may have sickle cell disease or not. They must not have had a transfusion recently or have a known deficiency of pyruvate kinase. They cannot be pregnant. Design: Participants will be screened with questions. Participants will have blood drawn by needle in an arm vein. The blood will be genetically tested. Not much is known about how genes affect SCD, so the test results will not be shared with participants or their doctors. ... | Sickle Cell|PKLR Variants|Adenosine Triphosphate Activities | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Long-term Comparative Cerebrovascular Outcome After Transplantation vs Standard Care in Sickle Cell Anemia | The purpose of the present observational study is to remotely reevaluate the cohort of 67 sickle cell patients with transcranial Doppler-detected cerebral vasculopathy included in the national "Sickle Cell Transplant" protocol and whose 1- and 3-year results were published in JAMA (Journal of the American Medical Association) in 2019 and in BHJ in 2020. | Sickle Cell Disease|Cerebral Ischemia|Stenosis | ALL | CHILD, ADULT, OLDER_ADULT | Hôpital Robert Debré AP-HP, Paris, IDF, 75019, France|Groupe hospitalier Pellegrin, Bordeaux, 33000, France|Centre Hospitalier Intercommunal de Créteil, Créteil, 94000, France|Hôpital Henri Monr - APHP, Créteil, 94000, France|CHU de la Guadeloupe, La Guadeloupe, 97110, France|Hôpital Bicêtre AP-HP, Le Kremlin-Bicêtre, 94270, France|IHOPe, Lyon, 69008, France|CHU de Lyon, Lyon, 69737, France|Hôpital de la Timone, Marseille, 13005, France|CHU de Montpellier, Montpellier, 34295, France|Hôpital Necker - AP-HP, Paris, 75015, France|CH de Pau, PAU, 64000, France|CHU de Rennes, Rennes, 35033, France|Hôpital Hautepierre, Strasbourg, 67200, France |
| Impact on the Length of Stay in Incentive Spirometry and Pain in the Decompensation of Sickle Cell Disease: . | In this prospective observational study among sickle cell children aged 7 to 17 years, who face many experience of pain, pain will be assessed during incentive spirometry sessions. Then a relation between, inspiratory volume, pain and the length of hospital stay will be identified . Currently, there is no scientific data regarding the correlation between acute pain during vaso-occlusive crisis, incentive spirometry and the impact on length of hospital stay. In fact, physiotherapist experience's in the pediatric department suggests that the pain expressed by the child is not always correlated with inspiratory capacity. The absence of pain is one of the reasons for hospital discharge after decompensation in patients with sickle cell disease. However, no scientific study has linked incentive spirometry, pain and length of hospital stay. Investigator assume that these children underestimate the real pain and its impact on breathing pattern, and presume that the maximal inspiratory volume during spirometry sessions will be a better reflect of pain than standard pain scale. The aim of this study is to show that inspiratory volume would be a better indicator of discharge from hospitalization than actual pain scales. | Sickle Cell Disease|Pediatric | ALL | CHILD | CHR d'Orleans, Orléans, 45067, France |
| Pediatrics HOT COVID-19 Database in NY Tristate | New York City (NYC) has become the epicenter of the worldwide pandemic caused by severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2). By collecting and summarizing the experience with other major health care providers in the tristate (New York (NY), New Jersey (NJ) and Connecticut (CT)) are, the investigators are uniquely positioned to inform the rest of the country about what to expect and how to manage children and young adults with hematological, oncological or stem cell transplant diagnoses during the pandemic. | Pediatric Cancer|Immune System Disorder|COVID-19|Hemoglobinopathies | ALL | CHILD, ADULT | Columbia University Irving Medical Center, New York, New York, 10032, United States |
| A Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab as Adjunct Treatment in Prevention of Vaso-Occlusive Episodes (VOE) in Sickle Cell Disease (SCD) | This study is designed to evaluate the efficacy, safety and pharmacokinetics of crovalimab compared with placebo as adjunct therapy in the prevention of VOEs in participants with SCD. | Sickle Cell Disease | ALL | CHILD, ADULT | Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|Mississippi Center for Advanced Medicine, Madison, Mississippi, 39110, United States|Icahn School of Medicine at Mount Sinai, New York, New York, 10029, United States|East Carolina University, Greenville, North Carolina, 27834, United States|Hospital Sao Rafael - HSR, Salvador, Bahia, 41253-190, Brazil|Hospital das Clinicas - UFRGS, Porto Alegre, Rio Grande Do Sul, DUMMY_VALUE, Brazil|UNESP - Faculdade de Medicina da Universidade Estadual Paulista - Campus Botucatu, Botucatu, São Paulo, 18618-970, Brazil|Hospital das Clínicas Faculdades Médicas de Ribeirão Preto, Ribeirao Preto, São Paulo, 14051-140, Brazil|Hospital de Base de Sao Jose do Rio Preto, Sao Jose do Rio Preto, São Paulo, 15090-000, Brazil|Beneficencia Portuguesa de Sao Paulo, Sao Paulo, São Paulo, 01323-900, Brazil|HEMORIO, Rio de Janeiro, 20211-030, Brazil|Hospital Samaritano, São Paulo, 01232-010, Brazil|CHU Henri Mondor, Créteil, 64010, France|Università degli Studi della Campania Luigi Vanvitelli, Napoli, Campania, 80138, Italy|Azienda Ospedaliera di Verona-Policlinico G.B. Rossi, Verona, Veneto, 37134, Italy|International Cancer Institute (ICI), Eldoret, 30100, Kenya|Gertrude's Children Hospital, Nairobi, DUMMY_VALUE, Kenya|American University of Beirut - Medical Center, Beirut, 1107 2020, Lebanon|Hopital Nini, Tripoli, DUMMY_VALUE, Lebanon|Amsterdam UMC Location VUMC, Amsterdam, 1081 HV, Netherlands|Charlotte Maxeke Johannesburg Hospital, Johannesburg, 2193, South Africa|Hospital General Univ. Gregorio Maranon, Madrid, 28009, Spain|Hospital Universitario Virgen del Rocio, Sevilla, 41013, Spain|Hospital Universitario Miguel Servet, Zaragoza, 50009, Spain|Adana Acibadem Hospital; Pediatric Hematology, Adana, 01130, Turkey|Cukurova University Medical Faculty Balcali Hospital, Adana, 1330, Turkey|Mersin Universitesi Tip Fakultesi Hastanesi, Mersin, 33343, Turkey|UCL Hospital NHS Trust, London, NW1 2PG, United Kingdom|Central Middlesex Hospital, London, NW10 7NS, United Kingdom|Hammersmith Hospital, London, W12 0HS, United Kingdom |
| Prevention of Vaso-occlusive Painful Crisis by Using Omega-3 Fatty Acid Supplements | 140 SCD patients \[70 on Hydroxyurea\] will receive Omega-3 capsules whereas another 140 SCD patients \[70 on Hydroxyurea\] will receive placebo and will be recruited from the Sultan Qaboos University Hospital \[SQUH\] haematology specialty clinics. Patients will be randomized in a 1:1 ratio to receive placebo or Omega-3 for 52 weeks. The aim is to investigate the therapeutic potential of omega-3 fatty acids in the prevention of vaso-occlusive crisis in Omani patients with sickle cell disease\[SCD\]. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Department of Haematology, Sultan Qaboos University, Muscat, 123, Oman |
| Study of Crizanlizumab for Prevention of Silent Cerebral Infarcts in SCA | In this prospective, single-arm, open-label, imaging and treatment study, the investigator will test the hypothesis that crizanlizumab will prevent the progression of silent cerebral infarcts in patients with sickle cell disease. Study participants will undergo brain MRI before initiation of crizanlizumab and at 6 and 30 months after starting crizanlizumab infusions. The crizanlizumab cohort will be compared to a matched, observational cohort of patients not receiving crizanlizumab. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Barnes-Jewish Hospital, Saint Louis, Missouri, 63110, United States |
| Dissemination and Implementation of Stroke Prevention Looking at the Care Environment | The Dissemination and Implementation of Stroke Prevention Looking at the Care Environment (DISPLACE) study is a multi-center, national, National Heart, Lung and Blood Institute (NHLBI)-funded grant to look at the real-world implementation of stroke prevention guidelines (STOP Protocol) in which transcranial Doppler (TCD), a measure of cerebral blood vessel velocity, is used to screen for stroke risk in children ages 2-16 with sickle cell anemia (SCA). Part 3 of the DISPLACE study is an implementation clinical trial designed to test novel implementation strategies with the goal of improving adherence and implementation of stroke screening. 16 of the lowest scoring implementation rates from DISPLACE Part 1 will participate in DISPLACE Part 3. All original 28 sites from DISPLACE Parts 1 and 2 will receive a patient and provider educational intervention including a re-branding of the TCD as "Sickle Stroke Screen" with a new infographic and educational materials. The 16 sites with moving to Part 3 will be provided a Provider reminder strategy, which is a web based application designed to remind providers of when patients are due for their Sickle Stroke Screen. These 16 sites will be randomized and 8 will be given an additional Patient Communication Strategy. These sites will have a single designed coordinator with whom patients will communicate with about scheduling, rescheduling, and any other questions regarding their Sickle Stroke Screen. Upon completion, data will be analyzed to compare those who have had TCD screenings done appropriately and those who did not as well as the overall effect of the multi level interventions on the changes in TCD rates. | Sickle Cell Disease | ALL | CHILD | Arkansas Children's Research Institute, Little Rock, Arkansas, 72202, United States|UCSF Benioff Children's Hospital Oakland, Oakland, California, 94609, United States|Nemours Center for Cancer & Blood Disorders, Wilmington, Delaware, 19803, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States|Howard University Hospital, Washington, District of Columbia, 20060, United States|Broward Health Medical Center, Fort Lauderdale, Florida, 33316, United States|University of Florida Health Shands Children's Hospital, Gainesville, Florida, 32608, United States|University of Miami, Miami, Florida, 33136, United States|Children's Heathcare of Atlanta, Atlanta, Georgia, 30322, United States|University of Illinois at Chicago, Chicago, Illinois, 60607, United States|SSM Health Cardinal Glennon Children's Hospital, Saint Louis, Missouri, 63104, United States|St. Louis Children's Hospital, Saint Louis, Missouri, 63110, United States|Columbia University Medical Center, New York, New York, 10032, United States|Vidant Medical Center, Greenville, North Carolina, 27834, United States|Vanderbilt Children's Hospital, Nashville, Tennessee, 37232, United States |
| HPV Vaccine Hesitancy Among Indiana Youth With Cancer and Blood Diseases | For the identified groups of patients (survivors of childhood cancer and youth with sickle cell disease) the investigators want to better understand the barriers to, and facilitators of, HPV vaccination. Through HCP interviews the investigators will also assess both attitudinal and logistical obstacles to HPV vaccination. Some subspecialty HCPs may believe, for instance, that it is the primary care provider's responsibility to vaccinate or they may be unfamiliar with the requirement to enter vaccination data into CHIRP. Also, in some cases HPV vaccine may not be readily available in subspecialty clinic locations and/or subspecialty HCPs may not be Vaccines for Children (VFC) providers. Participants: the investigators will focus on two patient groups: survivors of childhood cancer, which includes children aged 9-21 years who have completed active therapy for cancer and are eligible for vaccination, and sickle cell disease, which includes children aged 9-21 years with a diagnosis of sickle cell disease. The investigators will recruit parents of children aged 9-21 years and older adolescents aged 18-21 years. For pediatric patients, the investigators selected a lower age of 9 years because HPV vaccine is licensed down to 9 years of age, and an upper limit of 21 years, as that is considered the upper bound of adolescence by the American Academy of Pediatrics. For patients 9-17 years of age, only parents will be interviewed by video or phone because parents are the vaccine decision-makers. For patients 18-21 years of age, the investigators will interview both young adults and their parents, because, while the young adult has legal decision-making capacity, in reality, the decision is frequently made jointly by the young adult and parent, and older adolescents are frequently unwilling to go against their parents' wishes. All research procedures will be conducted in English. Participants will be excluded if they have an intellectual disability or severe medical illness such that they are unable to consent or to understand the questions. | Human Papilloma Virus Vaccine Hesitancy | ALL | CHILD, ADULT, OLDER_ADULT | Riley Hospital for Children - Indiana University, Indianapolis, Indiana, 46202, United States |
| Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients | Red Blood Cell - IMProving trAnsfusions for Chronically Transfused recipients (RBC-IMPACT) is an observational cohort study to assess donor, component, and recipient factors that contribute to RBC efficacy in chronically and episodically transfused patients. The objective of the study is to determine how specific genetic and non-genetic factors in donors and recipients may impact RBC survival after transfusion - in short, what factors on both the donor and recipient side may improve the efficacy of the transfusion. | Sickle Cell Disease|Thalassemia|Pediatric Cancer | ALL | CHILD, ADULT, OLDER_ADULT | UCSF Benioff Children's Hospital, Oakland, California, 94609, United States|Vitalant Research Institute, San Francisco, California, 94118, United States|Boston Children's Hospital, Boston, Massachusetts, 02115, United States|Weill Cornell Medical Collection (WCMC)/New York Presbyterian Hospital (NYPH), New York, New York, 10021, United States|Columbia University Irving Medical Center/New York Presbyterian Hospital (NYPH), New York, New York, 10032, United States|New York Blood Center (NYBC), New York, New York, 10065, United States|Children's Wisconsin, Milwaukee, Wisconsin, 53226, United States|Froedtert Hospital, Milwaukee, Wisconsin, 53226, United States|Versiti Wisconsin, Inc., Milwaukee, Wisconsin, 53233, United States|HEMOAM - Amazonas, Manaus, Amazonas, 69050-001, Brazil|HEMOMINAS - Minas Gerais, Belo Horizonte, Minas Gerais, 30622-020, Brazil|HEMOPE - Pernambuco, Recife, Pernambuco, 52011-000, Brazil|HEMORIO - Rio De Janeiro, Rio De Janeiro, 20211-030, Brazil|Childrens Institute and Adult Clinics at Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo, São Paulo, 05403-000, Brazil |
| Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400. | Primary Objectives: Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT) Secondary Objectives: * Long-term efficacy of the biological treatment effect of BIVV003 in SCD * Long-term efficacy of the clinical treatment effect of BIVV003 on SCD-related clinical events * Long-term efficacy of the biological treatment effect of ST-400 in TDT * Long-term efficacy of the clinical treatment effect of ST-400 in TDT | Blood and Lymphatic Diseases | ALL | ADULT | UCSF Benioff Children's Hospital, Oakland, California, 94609, United States|University of California Davis Health System, Sacramento, California, 95817, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30329, United States|Boston Children's Hospital, Boston, Massachusetts, 02115, United States|Karmanos Cancer Institute, Detroit, Michigan, 48201, United States|Henry Ford Health System, Detroit, Michigan, 48202, United States|University of Minnesota, Minneapolis, Minnesota, 55455, United States |
| The Use of Warmed Saline in Vaso-occlusive Episodes | The purpose of this study is to determine if warming the intravenous (IV) fluid given to patients with Sickle Cell Disease who are experiencing painful episodes known as Vaso-Occlusive Episodes; will decrease rates of hospital admission, decrease amounts of IV pain medications given, improve pain and improve global comfort. | Sickle Cell Disease|Sickle Cell Crisis | ALL | CHILD, ADULT | |
| Study of Methodologies to Measure Blood Flow and Oxygenation in Adults With Sickle Cell Disease | The purpose of the study is to determine whether imaging techniques, such as magnetic resonance imaging (MRI), near infrared spectroscopy (NIRS), laser speckle contrast imaging (LSCI), and optical imaging (OI), can detect differences in blood flow and oxygen levels in different organ systems of participants with sickle cell disease (SCD). Differences in blood flow and oxygen levels detected by these techniques will be evaluated to determine their utility as biomarkers of clinical disease pathophysiology. | Healthy|Sickle Cell Disease | ALL | ADULT | Research Site, Detroit, Michigan, 48201, United States |
| A Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease | This study is an open-label study to evaluate the safety of long-term administration of inclacumab in participants with sickle cell disease (SCD). Participants in this study will have completed a prior study of inclacumab. | Sickle Cell Disease|Vaso-occlusive Crisis|Vaso-occlusive Pain Episode in Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Strada Patient Care Center, Pediatric Hematology, Mobile, Alabama, 36604, United States|University of South Alabama Children's and Women's Hospital, Mobile, Alabama, 36604, United States|Arkansas Children's Hospital, Little Rock, Arkansas, 72202, United States|Children's Hospital and Research Center at Oakland, Oakland, California, 94609-1809, United States|UC Irvine Medical Center, Orange, California, 92868-3201, United States|UC Irvine Medical Center, Orange, California, 92868, United States|UConn-Neag Comprehensive Cancer Center, Farmington, Connecticut, 06030-0001, United States|University of South Florida, Department of Pediatrics, Tampa, Florida, 33606, United States|USF Health South Tampa Center for Advanced Healthcare (STC), Tampa, Florida, 33606, United States|St. Joseph's Hospital, Tampa, Florida, 33607, United States|John S Curran,MD, Children's Health Center/Children's Medical Center (CMS), Tampa, Florida, 33612, United States|USF Health Carol and Frank Morsani Center for Advanced Healthcare, Tampa, Florida, 33612, United States|University of Illinois at Chicago, Chicago, Illinois, 60612, United States|University of Illinois Hospital and Health Sciences System (UI Health), Chicago, Illinois, 60612, United States|University of Illinois Hospital and Health Systems, Chicago, Illinois, 60612, United States|Brigham And Woman's Hospital, Boston, Massachusetts, 02115, United States|Dana-Farber Cancer Institute IDS Pharmacy, Boston, Massachusetts, 02215, United States|Dana-Farber Cancer Institute, Boston, Massachusetts, 02215, United States|University of Michigan Hospitals - Michigan Medicine, Ann Arbor, Michigan, 48109, United States|Jacobi Medical Center, Bronx, New York, 10461, United States|Erie Country Medical Center, Buffalo, New York, 14215, United States|Duke University Medical Center, Durham, North Carolina, 27705, United States|DUMC Investigational Drug Services Pharmacy, Durham, North Carolina, 27710, United States|University of Texas Health Science Center at Houston, Houston, Texas, 77030-1501, United States|University of Texas Health Science Center of Houston -6410 Fannin St Ste 600, Houston, Texas, 77030-1501, United States|McGovern Medical School at UTHealth, Houston, Texas, 77030, United States|Instituto D'Or de Pesquisa e Ensino - Hospital São Rafael, Salvador, Bahia, 41253-190, Brazil|Hemocentro de Belo Horizonte - Fundacao Hemominas, Belo Horizonte, MG, 30130-110, Brazil|Hospital das Clinicas da Universidade Federal de Minas Gerais, Belo Horizonte, Minas Gerais, 30130-100, Brazil|Multihemo Servicos Medicos S/A, Recife, PE, 50070-460, Brazil|Hospital de Clínicas de Porto Alegre, Porto Alegre, RIO Grande DO SUL, 90035-903, Brazil|Fundação Faculdade Regional de Medicina de São José do Rio Preto, Sao Jose do Rio Preto, SAO Paulo, 15090-000, Brazil|Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP, Ribeirão Preto, SÃO Paulo, 14051-140, Brazil|Instituto Estadual de Hematologia Arthur Siqueira Cavalcanti - HEMORIO, Rio de Janeiro, 20211-030, Brazil|Hospital Samaritano Higienópolis/Esho Empresa De Servicos Hospitalares S.A, São Paulo, 01232-010, Brazil|Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo -HCFMUSP, São Paulo, 05403-000, Brazil|Casa de Saude Santa Marcelina, São Paulo, 08270-070, Brazil|CEPEC-Centro de Pesquisa Clinica, São Paulo, 08270-120, Brazil|Clinica de la Costa Ltda., Barranquilla, Atlantico, 080020, Colombia|Sociedad de Oncología y hematología del Cesar, Valledupar, Cesar, 200001, Colombia|Universitätsklinikum Regensburg Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation, Regensburg, 93053, Germany|Farmacia Interna Azienda Ospedaliero-Ente Ospedaliero Ospedali Galliera, Genova, 16128, Italy|S.S.D. Microcitemia, anemie congenite e dismetabolismo del ferro, Genova, 16128, Italy|DAI Materno-Infantile,- UOC Clinica Pediatrica 1 Azienda Ospedaliera Universitaria "Luigi Vanvitel, Napoli, 80138, Italy|UO di Farmacia Clinica,Dipartimento di Medicina Sperimentale, Napoli, 80138, Italy|UOC Patologia Clinica e Molecolare Azienda Ospedaliera Universitaria "Luigi Vanvitelli", Napoli, 80138, Italy|UOC Radiologia Azienda Ospedaliera Universitaria "Luigi Vanvitelli", Napoli, 80138, Italy|Dipartimento Strutturale Aziendale Salute della Donna e del Bambino Clinica Ginecologica, Padova, 35128, Italy|Farmacia Azienda Ospedale Universita Padova, Padova, 35128, Italy|U.O.C. Farmacia Istituto Oncologico Veneto, Padova, 35128, Italy|KEMRI/CRDR Siaya Clinical Research Annex, Kisumu, Siaya, 40600, Kenya|International Cancer Institute, Eldoret, 30100, Kenya|Gertrude's Children's Hospital, Nairobi, 00100, Kenya|Kenya Medical Research Institute - Centre for Respiratory Disease Research, Nairobi, 00100, Kenya|Strathmore University CREATES, Nairobi, 00200, Kenya|American University of Beirut Medical Center, Hamra, Beirut, Lebanon|Nini Hospital, Tripoli, North Lebanon, Lebanon|University of Calabar Teaching Hospital, Calabar, Cross River State, 540242, Nigeria|National Hospital Abuja, Abuja, FCT, 900211, Nigeria|University of Abuja Teaching Hospital, Gwagwalada, FCT, 902101, Nigeria|Ahmadu Bello University Teaching Hospital, Zaria, Kaduna, 810105, Nigeria|Ahmadu Bello University Teaching Hospital, Zaria, Kaduna, 810107, Nigeria|University of Nigeria Teaching Hospital, Enugu, 460000, Nigeria|Barau Dikko Teaching Hospital/Kaduna State University, Kaduna, 800212, Nigeria|Aminu Kano Teaching Hospital, Kano, 700223, Nigeria|Department of Pediatrics, College of Medicine, Lagos University Teaching Hospital, Lagos, 100254, Nigeria|Sultan Qaboos University Hospital, Muscat, 123, Oman|Prince Mohammed bin Nasser Hospital, Jizan, Southern, 82943, Saudi Arabia|NIMR-Mbeya Medical Research Center, Mbeya, Tanzania|Acibadem Adana Hastanesi Cocuk Hematoloji Onkoloji, Adana, 01130, Turkey|Baskent University Adana Appl. and Research Central, Yuregir Baskent Hospital Hematology, Adana, 01250, Turkey|Hacettepe University Ihsan Dogramaci Children Hospital, Ankara, 06430, Turkey|Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Merkezi Hastanesi, Mersin, 33343, Turkey |
| MRI Evaluation of Iron Overload in the Heart, Liver and Pancreas in Patients Receiving Multiple Blood Transfusions. | Sickle cell anemia and Sickle cell β thalassemia patients require multiple transfusions in order to avoid chronic anemia sequel. This regimen entails intrinsic deleterious effects, the majority of which are related to iron deposition in the reticuloendothelial system. Thus, iron is deposited in hepatic, myocardial and endocrine glands tissues. Tools available for iron load evaluation include serum ferrtin levels, liver biopsy and echocardiography, all are non specific. The purpose of this work is to compare iron overload in the liver, heart and pancreas in Sickle cell anemia and Sickle cell β thalassemia patients using T2\* MRI sequences. | Iron Overload | ALL | ADULT | Sheba Medical Center , Imaging Dept, Tel Hashomer, 52621, Israel |
| Novel Dose Escalation to Predict Treatment With Hydroxyurea | Sickle cell disease is a disorder in which red blood cells (RBCs) are abnormally shaped. This can result in painful episodes, serious infections, chronic anemia (a decrease in the number of red blood cells), and damage to body organs. Hydroxyurea therapy offers significant benefits for infants, children, and adolescents with sickle cell anemia. These include a reduction in the frequency of pain crises and acute chest syndrome (inflammation of the lungs) and an increase in hemoglobin (the oxygen-carrying protein) in the blood. Patients on hydroxyurea who receive a maximum tolerated dose (MTD) that is specific for them have greater clinical benefit than those who receive a standard lower dose. There is, however, no way currently to predict the MTD for individual patients. As such, MTD for each patient is currently determined by gradual increases in the dose over several months. This process is time-consuming, requires monthly clinic visits, and delays the benefits of hydroxyurea therapy. Our research group has come up with an equation that could be used to predict each patient's MTD using baseline clinical and laboratory measures before starting hydroxyurea treatment. The purpose of this research study is to compare the use of our equation for predicting MTD to the current standard practice of gradually increasing the hydroxyurea dose until MTD is reached. We want to see if the use of our predictive equation will allow us to achieve MTD faster and with no more side effects than with the standard practice. | Sickle Cell Disease | ALL | CHILD | Texas Children's Hospital, Houston, Texas, 77030, United States |
| Realizing Effectiveness Across Continents With Hydroxyurea (REACH) | REACH is a prospective, phase I/II open-label dose escalation trial of hydroxyurea for for pediatric patients with sickle cell anemia (SCA). The short-term goal is to obtain critical pilot data regarding the feasibility, safety, and benefit of hydroxyurea for children with SCA in multiple distinct research settings in Africa. Based on that information, the longer-term goal is to make hydroxyurea more widely available for children with SCA in Africa, particularly those identified with SCA through expanded newborn screening programs. | Sickle Cell Disease | ALL | CHILD | Hospital Pediátrico David Bernardino, Luanda, Angola|Centre Hospitalier Monkole, Kinshasa, Congo, The Democratic Republic of the|KEMRI/Wellcome Trust Research, Kilifi, Kenya|Ministry of Health Mbale Regional Hospital, Mbale, Uganda |
| Hydroxyurea Exposure Limiting Pregnancy and Follow-Up Lactation | The purpose of this research study is to document and understand the effects of hydroxyurea exposure for women with SCD and their babies, during both gestation and lactation. | Sickle Cell Disease|Sickle Cell Anemia | ALL | CHILD, ADULT, OLDER_ADULT | Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States |
| Safety of Rivipansel (GMI-1070) in the Treatment of One or More Vaso-Occlusive Crises in Hospitalized Subjects With Sickle Cell Disease | This is an open label extension study in subjects with Sickle Cell Disease (SCD) who have completed the double blind Phase 3 study (B5201002). | Sickle Cell Anemia|Sickle Cell Disease|Sickle Cell Disorders|Pain Crisis|Vaso-occlusive Crisis | ALL | CHILD, ADULT, OLDER_ADULT | University of South Alabama Women's and Children's Hospital, Mobile, Alabama, 36604, United States|Arkansas Children's Hospital Research Pharmacy, Little Rock, Arkansas, 72202, United States|Arkansas Children's Hospital, Little Rock, Arkansas, 72202, United States|UC Davis Medical Center Main Hospital, Sacramento, California, 95817, United States|University of California Davis Medical Center, Sacramento, California, 95817, United States|Howard University Center for Sickle Cell disease, Washington, District of Columbia, 20001, United States|MedStar Health Research Institute, Washington, District of Columbia, 20010, United States|Howard University Hospital, Washington, District of Columbia, 20060, United States|Golisano Childrens Hospital of Southwest Florida, Fort Myers, Florida, 33908, United States|Jackson Memorial Hospital, Miami, Florida, 33136, United States|University of Miami, Miami, Florida, 33136, United States|St. Mary's Medical Center, West Palm Beach, Florida, 33407, United States|Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center:, Atlanta, Georgia, 30303, United States|Grady Health System, Atlanta, Georgia, 30303, United States|Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center:, Atlanta, Georgia, 30322, United States|Emory Children's Center, Atlanta, Georgia, 30322, United States|Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center/, Atlanta, Georgia, 30342, United States|Children's Healthcare of Atlanta: Scottish Rite Campus, Atlanta, Georgia, 30342, United States|Memorial Family Medicine Center, Savannah, Georgia, 31404, United States|Memorial Health University Medical Center, Savannah, Georgia, 31404, United States|The University of Chicago/Comer Children's Hospital, Chicago, Illinois, 60637, United States|University of Chicago, Investigational Drug Service Pharmacy, Chicago, Illinois, 60637, United States|University of Maryland Medical System Investigational Pharmacy, Baltimore, Maryland, 21201, United States|University of Maryland Medical System, Baltimore, Maryland, 21201, United States|Johns Hopkins Department of Medicine Clinical Trials Unit, Baltimore, Maryland, 21205, United States|The Johns Hopkins University School of Medicine, Baltimore, Maryland, 21205, United States|The Johns Hopkins Hospital Department of Pharmacy Services, Baltimore, Maryland, 21287-6180, United States|Johns Hopkins Medicine, Baltimore, Maryland, 21287, United States|Boston Children's Hospital, Boston, Massachusetts, 02115, United States|Brigham and Women's Hospital, Boston, Massachusetts, 02115, United States|Center for Clinical Investigation, Brigham and Women's Hospital, Boston, Massachusetts, 02115, United States|Investigational Drug Services, Boston, Massachusetts, 02115, United States|Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|University of Mississippi Medical Center - Outpatient Clinical Research Unit, Jackson, Mississippi, 39216, United States|University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States|Center for Outpatient Health, Saint Louis, Missouri, 63108, United States|Barnes-Jewish Hospital Department of Pharmacy, Saint Louis, Missouri, 63110, United States|Barnes-Jewish Hospital, Saint Louis, Missouri, 63110, United States|Center for Advanced Medicine, Saint Louis, Missouri, 63110, United States|Washington University School of Medicine, Saint Louis, Missouri, 63110, United States|Bristol Myers Squibb Children's Hospital at Robert Wood Johnson University Hospital, New Brunswick, New Jersey, 08901, United States|Rutgers-Robert Wood Johnson Medical School, New Brunswick, New Jersey, 08901, United States|Rutgers Cancer Institute of New Jersey, New Brunswick, New Jersey, 08903, United States|Jacobi Medical Center, Bronx, New York, 10461, United States|Kings County Hospital Center, Brooklyn, New York, 11203, United States|State University of New York (SUNY) Downstate Medical Center, Brooklyn, New York, 11203, United States|SUNY Downstate Medical Center University Hospital of Brooklyn, Brooklyn, New York, 11203, United States|Columbia University Medical Center Research Pharmacy, New York, New York, 10032, United States|MS CHONY Pediatric Emergency Department, New York, New York, 10032, United States|MS CHONY Pediatric Hematology/Oncology Unit, New York, New York, 10032, United States|Duke University Hospital, Investigational Drug Service, Durham, North Carolina, 27710, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|East Carolina University Brody School of Medicine, Greenville, North Carolina, 27834, United States|East Carolina University, Brody School of Medicine, Greenville, North Carolina, 27834, United States|Leo W. Jenkins Cancer Center, Greenville, North Carolina, 27834, United States|Vidant Medical Center, Greenville, North Carolina, 27834, United States|University of Cincinnati - Hoxworth Building, Cincinnati, Ohio, 45219, United States|University of Cincinnati Medical Center / Investigational Pharmacy, Cincinnati, Ohio, 45219, United States|University of Cincinnati Medical Center / Research Office, Cincinnati, Ohio, 45219, United States|University of Cincinnati Medical Center, Cincinnati, Ohio, 45219, United States|University of Cincinnati Physicians Company LLC, Cincinnati, Ohio, 45219, United States|UC Health Ridgeway Hospital, Cincinnati, Ohio, 45229, United States|The Ohio State University Investigational Drug Services, Columbus, Ohio, 43203, United States|The Ohio State University Wexner Center East, Columbus, Ohio, 43203, United States|The Ohio State University James Comprehensive Cancer Hospital & Solove Research Institute, Columbus, Ohio, 43210, United States|The Ohio State University Wexner Medical Center, Columbus, Ohio, 43210, United States|UPMC Presbyterian, Pittsburgh, Pennsylvania, 15213, United States|UPMC Hillman Cancer Center, Pittsburgh, Pennsylvania, 15232, United States|Hasbro Children's Hospital, Providence, Rhode Island, 02903, United States|Rhode Island Hospital-Pharmacy Service, Providence, Rhode Island, 02903, United States|Rhode Island Hospital, Providence, Rhode Island, 02903, United States|The Miriam Hospital, Providence, Rhode Island, 02906, United States|Medical University of South Carolina Lifespan Comprehensive Sickle Cell Center, Charleston, South Carolina, 29425, United States|Medical University of South Carolina-Hospital, Charleston, South Carolina, 29425, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|MUSC Investigational Drug Services, Charleston, South Carolina, 29425, United States|Cook Children's Hematology and Oncology Center, Fort Worth, Texas, 76104, United States|Cook Children's Medical Center, Fort Worth, Texas, 76104, United States|Cook Children's Hematology and Oncology Center-Grapevine, Grapevine, Texas, 76051, United States|University of Texas Medical School, Houston, Texas, 77030, United States|Primary Children's Hospital Laboratory, Salt Lake City, Utah, 84113, United States|Primary Children's Hospital, Salt Lake City, Utah, 84113, United States|Main Hospital-VCU, Richmond, Virginia, 23298, United States|Virginia Commonwealth University - Investigational Drug Services, Richmond, Virginia, 23298, United States|Virginia Commonwealth University- Clinical Research Services Unit, Richmond, Virginia, 23298, United States|Royal Alexandra Hospital, Edmonton, Alberta, T5H 3V9, Canada|Miseracordia Community Hospital, Edmonton, Alberta, T5R 4H5, Canada|Kaye Edmonton Clinic 3C, Edmonton, Alberta, T6G 1Z1, Canada|University of Alberta Hospital, Pharmacy Services, Edmonton, Alberta, T6G 1Z1, Canada|Stollery Children's Hospital, Edmonton, Alberta, T6G 2B7, Canada|University of Alberta Hospital, Edmonton, Alberta, T6G 2B7, Canada|Research transition Facility, Edmonton, Alberta, T6G 2V2, Canada|Grey Nuns Community Hospital, Edmonton, Alberta, T6L 5X8, Canada|Children's Hospital of Eastern Ontario, Ottawa, Ontario, K1H 8L1, Canada|The Hospital for Sick Children, Toronto, Ontario, M5G 1X8, Canada|Centre Hospitalier Universitaire Sainte-Justine, Montreal, Quebec, H3T 1C5, Canada|The Montreal Children's Hospital / McGill University Health Centre, Montreal, Quebec, H4A3J1, Canada |
| Metabolic and Hemodynamic Reserve in Pediatric SCA | The purpose of this research study is to better understand how blood flow and metabolism change can influence brain development in the early decades of life. SCA participants and healthy controls are age and sex-matched for comparison. Within the SCA cohort, children with infarcts may have thinner cortices than those without, reflecting a greater loss. The investigators will examine brain blood flow and metabolism using magnetic resonance imaging (MRI). The brain's blood vessels expand and constrict to regulate blood flow based on the brain's needs. The amount of expanding and contracting the blood vessels may vary by age. The brain's blood flow changes in small ways during everyday activities, such exercise, deep concentration, or normal brain growth. Significant illness or psychological stress may increase the brain's metabolic demand or cause other bigger changes in blood flow. If blood vessels are not able to expand to give more blood flow when metabolic demand is high, the brain may not get all of the oxygen it needs. In extreme circumstances, if the brain is unable to get enough oxygen for a long time, a stroke may occur. Sometimes small strokes occur without other noticeable changes and are only detectable on an MRI. These are sometimes called "silent strokes." In less extreme circumstances, not having a full oxygen supply may cause the brain to grow and develop more slowly than when it has a full supply. One way to test the ability of blood vessels to expand is by measuring blood flow while breathing in carbon dioxide. Carbon dioxide causes blood vessels in the brain to dilate without increasing brain metabolism. During this study participants may be asked to undergo a blood draw, MRI, cognitive assessments, and brief questionnaires. The study team will use a special mask to control the amount of carbon dioxide the participants breathe in. | Child, Only|Brain Diseases|Sickle Cell Disease|Anemia, Sickle Cell | ALL | CHILD, ADULT | Washington University in St. Louis, Saint Louis, Missouri, 63110, United States |
| SIKAMIC (SIklos on Kidney Function and AlbuMInuria Clinical Trial) | The purpose of this phase IIb, international, multicentre, double-blind, randomised, placebo-controlled study is to determine the effect of hydroxycarbamide on albuminuria after 6 months of treatment in SCD adult patients. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Centre Suisse de Recherches Scientifiques en Côte d'Ivoire (CSRS), Abidjan, Côte D'Ivoire|CHU d'Angers, Angers, France|Hôpital Saint-André, Bordeaux, France|CHRU Brest, Brest, France|Hôpital Louis Mourier, Colombes, France|Pablo Bartolucci, Créteil, 94017, France|Hopital Edouard Herriot, Lyon, France|Hôpital de la Timone, Marseille, France|Hôpital européen Georges-Pompidou, Paris, France|Hôpital Saint-Antoine, Paris, France|Service de biothérapie, consultation hématologie-drépanocytose hôpital Necker, Paris, France|CHU la Miletrie, Poitiers, France|Hôpital Robert Debré CHU Reims, Reims, France|Hopital Pontchaillou, Rennes, France|CHU Charles Nicolle, Rouen, France|Centre Hospitalier Delafontaine, Saint-Denis, France|Institut Universitaire du Cancer de Toulouse - Oncopole, Toulouse, France|CHU Pointe-à-Pitre/Abymes, Pointe-à-Pitre, Guadeloupe|Centre de Recherche et de Lutte contre la Drépanocytose de Bamako (CRLD), Bamako, Mali|CHU Martinique, Le Lamentin, Martinique|Service d'Hématologie Clinique, Centre National de Transfusion sanguine, Université Cheikh Anta Diop, Dakar, Senegal |
| A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell Disease | The purpose of the study is to assess the safety and tolerability of AG-946 in healthy volunteers after oral administration of single ascending doses (SAD) and multiple ascending doses (MAD) of AG-946 over 14 or up to 28 days of dosing, and to identify a range of doses that are safe and pharmacologically active in participants with sickle cell disease. The SAD and MAD parts of the study will be randomized and double-blinded, and will assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of AG-946 as well as the effect of food (SAD only) on the pharmacokinetics (PK) of AG-946. The sickle cell disease (SCD) part of the study will be non-randomized and open-label, and is designed to identify 1 or more safe and tolerable dose(s) of AG-946 with potential activity in the treatment of participants with sickle cell disease (SCD). | Healthy Volunteers|Anemia, Sickle Cell | ALL | ADULT, OLDER_ADULT | University of California San Diego, La Jolla, California, 92037, United States|Massachusetts General Hospital, Boston, Massachusetts, 02114, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|New York Presbyterian Hospital - Weill Cornell Medicine, New York, New York, 10065, United States|University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, 15213, United States|PPD Development, LP, Austin, Texas, 78744, United States|Texas Oncology-Baylor Charles A. Sammons Cancer Center - USOR, Dallas, Texas, 75246, United States|University of Texas Health Science Center of Houston, Houston, Texas, 77030, United States|Rigshospitalet, Department of Hematology, Copenhagen, 2100, Denmark|Hospital Universitario Vall d'Hebron - PPDS, Barcelona, 08035, Spain |
| Shared-Decision Making for Hydroxyurea | The goal of the study is to understand how best to help parents of young children with sickle cell disease and their clinicians have a shared discussion about hydroxyurea (one that takes into account medical evidence and parent values and preferences). The study will compare two methods to help clinicians facilitate this-a clinician pocket guide and a clinician hydroxyurea shared decision making toolkit-in a group of parents of children ages 0-5 with sickle cell disease. The investigators hope that both methods lead to parents reaching a high-quality, well-informed decision. In addition, the team hopes to demonstrate that parents who experience a shared decision will have lower anxiety and decisional uncertainty. The researchers also expect these parents to be more likely to choose hydroxyurea and that their children will have less pain, fewer hospitalizations, better developmental outcomes, and higher quality of life. The project team hopes to show that the toolkit method is easy for clinicians to use and gives parents the support needed to make an informed decision. | Sickle Cell Anemia|Children, Only | ALL | CHILD | UCSF Beinoff Children's Hospital and Research Center at Oakland, Oakland, California, 94609, United States|Nemours Children's Health, Wilmington, Delaware, 19803, United States|Howard University, Washington, District of Columbia, 20060, United States|Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States|Indiana Hemophilia & Thrombosis Center, Indianapolis, Indiana, 46260, United States|Boston Children's Hospital, Boston, Massachusetts, 02118, United States|The Washington University, Saint Louis, Missouri, 63110, United States|Nationwide Children's Hospital, Columbus, Ohio, 43205, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Vanderbilt University Medical Center, Nashville, Tennessee, 37232, United States|Baylor College of Medicine, Houston, Texas, 77030, United States |
| Retrospective Real World Oxbryta® Data Collection and Analysis Study | The aim of this study is to collect and analyze retrospective data on Oxbryta in a real-world setting. This is a multicenter, retrospective data collection and analysis study to characterize health outcomes in approximately 300 patients with SCD who have been treated with Oxbryta as part of their usual care. Any patient with SCD who received Oxbryta treatment for at least 2 weeks as part of their usual care according to the Oxbryta US Prescribing Information (USPI) is eligible to participate. Study data from 1 year before and up to 1 year after the first dose of Oxbryta will be entered in case report forms (CRFs) via an electronic data capture (EDC) system by the study staff. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | University of Connecticut Health, Farmington, Connecticut, 06030, United States|Brigham and Women's Hospital, Boston, Massachusetts, 02115, United States|Dana-Farber Cancer Institute, Boston, Massachusetts, 02215, United States|Central Michigan University/Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|Rutgers Robert Wood Johnson Medical School Pediatric Clinical Research Center, New Brunswick, New Jersey, 08901, United States|Rutgers Cancer Institute of New Jersey, New Brunswick, New Jersey, 08903-2681, United States|Montefiore Medical Center, Bronx, New York, 10467, United States|Levine Cancer Institute, Charlotte, North Carolina, 28204, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|Parkland Health & Hospital System, Dallas, Texas, 75235, United States|UT Southwestern Medical Center, Dallas, Texas, 75390, United States|University of Texas Health Science Center at Houston, Houston, Texas, 77030, United States |
| Sparing Conversion to Abnormal TCD (Transcranial Doppler) Elevation (SCATE) | The primary goal of the Phase III SCATE trial is to compare 30 months of alternative therapy (hydroxyurea) to standard care (observation) in children with sickle cell anemia and conditional (170 - 199cm/sec) Transcranial Doppler (TCD) velocities. For the alternative regimen (hydroxyurea) to be declared superior to the standard treatment regimen (observation), the hydroxyurea-treated group must have a three-fold reduction in the incidence of conversion to abnormal TCD velocities (≥ 200 cm/sec), compared to the standard treatment arm. | Sickle Cell Anemia | ALL | CHILD | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti (HEMORIO), Centro, Rio de Janeiro, Brazil|Tropical Medicine Research Institute, University of the West Indies (UWI), Mona, Kingston, Jamaica |
| Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias | This research is being done so that we can look at the safety and efficacy of deferiprone in people with sickle cell disease or other anemias. Deferiprone is a drug that removes iron from the body. We will be comparing deferiprone with deferoxamine, another drug that removes iron from the body. | Iron Overload|Sickle Cell Disease|Other Anemias | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital Oakland, Oakland, California, 94609, United States|University of Illinois at Chicago, Chicago, Illinois, 60612, United States|Children's Hospital, New Orleans, Louisiana, 70118, United States|University of Michigan Comprehensive Cancer Center, Ann Arbor, Michigan, 48109, United States|Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|The Children's Hospital of Philadephia, Philadelphia, Pennsylvania, 19104-4399, United States|Thomas Jefferson University, Philadelphia, Pennsylvania, 19107, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|Centro Infantil Boldrini, Campinas, Brazil|Hospital de Clínicas de Porto Alegre-HCPA,, Rio Branco, 90035-903, Brazil|Instituto Estadual de Hematologia Arthur Siqueira Cavalcanti - HEMORIO, Rio de Janeiro, 20211-030, Brazil|Casa de Saúde Santa Marcelina, São Paulo, Brazil|Universidade Federal de São Paulo, São Paulo, Brazil|Hospital for Sick Kids, Toronto, Ontario, Canada|Alexandria University, Alexandria, Egypt|Zagazig University, Alexandria, Egypt|Ains Shams University, Cairo, Egypt|Cairo University, Cairo, Egypt|Pediatric Hospital of Cairo University, Cairo, Egypt|Mansoura University Children's Hospital, Mansoura, Egypt|King Abdulaziz University Hospital, Jeddah, Western Region, 80215, Saudi Arabia|Asser Central Hospital, Abha, Saudi Arabia|King Khalid University Hospital, Riyadh, Saudi Arabia|National Center for Bone Marrow Transplantation, Tunis, Bad Saadoun, Tunisia|Farhat Hached Hospital, Hematology Department, Sousse, Tunisia|Principal Military Hospital of Instruction of Tunis, Tunis, Tunisia|Cukurova University, Adana, Turkey|Hacettepe University, Ankara, Turkey|Istanbul University, Istanbul, Turkey|Hammersmith Hospital, London, W12 0HS, United Kingdom|Barts and The London, London, United Kingdom|Evelina Children's Hospital, London, United Kingdom|Imperial College Healthcare NHS Trust, London, United Kingdom |
| Cognitive Behavioral Therapy and Real-Time Pain Management Intervention for Sickle Cell Via Mobile Applications | The investigators are conducting a comparative effectiveness trial among adult patients with sickle cell disease (SCD) who report chronic pain (N = 350), randomized to receive either mobile phone-delivered computerized cognitive behavioral therapy (cCBT; n = 175) or digital education (m-Education; n = 175). Both intervention groups will receive weekly (more frequent if requested or needed) follow-up with a health coach for at least 3 months to reinforce learned materials. Both groups will also use their mobile device to track daily pain, mood, and medication used for two-week periods at baseline and each of the follow-up points (3, 6 and 12 months). Participants will also be given access to a study-associated online support group page where members can discuss with other patients, issues participants faced and what skills were or could be used to address them. Participants will continue all routine care including opioid pain management and novel therapies. | Chronic Pain|Depression, Anxiety|Opioid Use | ALL | ADULT, OLDER_ADULT | University of Illinous-Chicago, Chicago, Illinois, 60607, United States|Johns Hopkins, Baltimore, Maryland, 21287, United States|Duke University, Durham, North Carolina, 27708, United States|East Carolina University, Greenville, North Carolina, 27858, United States|Ohio State University, Columbus, Ohio, 43210, United States|University of Pittsburgh, Pittsburgh, Pennsylvania, 15213, United States|Vanderbilt University, Nashville, Tennessee, 37240, United States |
| An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001 | This is an open label, single arm study which enrolled 5 subjects with SCD who previously participated in the GBT440-001 study (NCT02285088). | Sickle Cell Disease | ALL | ADULT | The BRC Research Facility, Floor 15 The Tower Wing, London, SE1 9RT, United Kingdom |
| Multi-Center Study of Iron Overload: Pilot Study | The purpose of this study is to initiate pilot studies to demonstrate that a sufficient number of iron-overloaded thalassemia, SCD and DBA populations with similar duration of chronic transfusion, and age at start of transfusions would be available for a confirmatory study and to validate that proposed multicenter MRI and biochemical studies can be completed. The study will examine the hypothesis that a chronic inflammatory state in Sickle Cell Disease (SCD) leads to hepcidin- and cytokine-mediated iron withholding within the RES (reticuloendothelial system), lower plasma NTBI (non transferrin bound iron) levels, less distribution of iron to the heart in SCD. | Sickle Cell Disease|Thalassemia|Diamond-Blackfan Anemia | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital & Research Center Oakland, Oakland, California, 94609, United States|Universitätsklinikum Hamburg-Eppendorf, Hamburg-Eppendorf, Germany|UCL Cancer Institute, London, WC1E 6BT, United Kingdom |
| Sevuparin Infusion for the Management of Acute VOC in Subjects With SCD | A Multi-Centre, Phase II, Randomized, Double-Blind, Placebo-Controlled Study to investigate Efficacy and Safety of Sevuparin Infusion for the Management of Acute Vaso-Occlusive Crisis (VOC) in Subjects with Sickle-Cell Disease (SCD). | Sickle-Cell Disease | ALL | CHILD, ADULT | Salmaniya Hospital, Kingdom of Bahrain, Manama, Bahrain|Salmaniya Medical Complex, Bahrain, Manama, Bahrain|Annotto Bay Hospital, Annotto Bay, Jamaica|Kingston Public Hospital, Kingston, Jamaica|University Hospital of the West Indies, Kingston, Jamaica|Winchester Surgical and Medical Institute, Kingston, Jamaica|Mandeville Regional Hospital, Mandeville, Jamaica|May Pen Public Hospital Clarendon, May Pen, Jamaica|Cornwall Regional Hospital, Jamaica, Montego Bay, Jamaica|American University of Beirut Medical Center, Beirut, Cairo street, Beirut, Lebanon, Beirut, Lebanon|Nini Hospital, Tripoli, Lebanon|Dept of Haematology, Amsterdam, Netherlands|Erasmus MC, Rotterdam, Netherlands|Sultan Qaboos University Hospital Alkhodh, Oman, Muscat, Oman|Sultan Qaboos University, Muscat, Oman|King Fahd Medical City, As Sulimaniyah, Riyadh Saudiarabien, Riyadh, Saudi Arabia|King Saud University, Riyadh, Saudiarabien, Riyadh, Saudi Arabia|Mersin University Faculty of Medicine, Adana, Mersin, Turkey|Cukurova University Faculty Of Medicine Tıp Fakültesi, Adana, Turkey|Dr Antmen, Adana, Turkey |
| Transcranial Doppler (TCD) With Transfusions Changing to Hydroxyurea | The primary goal of the Phase III TWiTCH trial is to compare 24 months of alternative therapy (hydroxyurea) to standard therapy (transfusions) for pediatric subjects with sickle cell anemia and abnormally high (≥200 cm/sec) Transcranial Doppler (TCD) velocities, who currently receive chronic transfusions to reduce the risk of primary stroke. For the alternative treatment regimen (hydroxyurea) to be declared non-inferior to the standard treatment regimen (transfusions), after adjusting for baseline differences, the hydroxyurea-treated group must have a mean TCD velocity similar to that observed with transfusion prophylaxis. | Sickle Cell Anemia | ALL | CHILD | |
| Virtual Reality Devices As an Adjunct to Usual Care for Patients with Sickle Cell Disease Experiencing Vaso-Occlusive Crises | This study aims to evaluate the use of virtual reality as an adjunct to standard care for patients with sickle cell disease experiencing vaso-occlusive crises. | Sickle Cell Disease|Vaso-occlusive Pain Episode in Sickle Cell Disease|Acute Pain | ALL | ADULT, OLDER_ADULT | University of Maryland Medical Systems, Baltimore, Maryland, 21201, United States |
| Sickle Cell Trait and the Risk of Venous Thromboembolism | The purpose of this trial is to investigate D-Dimer levels, a surrogate marker of venous thromboembolism, in pregnant/postpartum white women as compared to pregnant/postpartum black women, and pregnant/postpartum women with sickle cell trait. The investigators will determine whether increased D-Dimer levels are reflected in a greater incidence of thrombosis in the postpartum patient, as well as the prevalence of symptomatic venous thrombosis in black patients as compared to pregnant white patients and women with sickle cell trait. The investigators will also investigate the effect of blood group on these parameters. If there is evidence that there is an increased risk of thrombosis in sickle cell trait, the investigators will plan a trial of prophylactic anticoagulation during the last trimester and the four weeks post partum for patients with sickle cell trait and compare this population to patients who do not receive prophylactic anticoagulation. | Thrombosis | FEMALE | ADULT | Montefiore Medical Center (Einstein), Bronx, New York, 10461, United States |
| HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity | This study will evaluate the use of reduced intensity conditioning regimen in patients with high risk hemoglobinopathy Sickle Cell and B-Thalassemia Major in combination with standard immunosuppressive medications, followed by a routine stem cell transplant in order to assess whether or not it is as effective as myeloablative high dose chemotherapy and transplant. | Sickle Cell Disease|Beta Thalassemia-Major | ALL | CHILD, ADULT | Cohen Children's Medical Center of New York, New Hyde Park, New York, 11040, United States |
| Non-Myeloablative Conditioning and Bone Marrow Transplantation | Allogeneic blood or marrow transplantation (alloBMT) is a curative therapy for a variety of hematologic disorders, including sickle cell disease and thalassemia. Even when it is clear that alloBMT can give to these patients an improvement in their disease, myeloablative transplants have important toxicities and mortalities associated. The lack of suitable donors continues to be a limit to access to transplantation. Substantial progress has been made recently in the development of pre-treatment regimens that facilitate the sustained engraftment of donor marrow with reduced toxicity. Most of these regimens incorporate highly immunosuppressive drugs, which allow the reduction or elimination of myeloablative agents or total body irradiation without endangering the sustained engraftment of HLA-identical allogeneic stem cells. Preliminary results of non-myeloablative allogeneic stem cell transplantation suggest that the procedure can be performed in patients who are ineligible for myeloablative alloBMT, and that sustained remissions of several hematologic malignancies can be obtained. | Sickle Cell Disease|Hemoglobinopathies | ALL | CHILD, ADULT, OLDER_ADULT | Vanderbilt-Ingram Cancer Center, Nashville, Tennessee, 37232, United States|Saint-Louis Hospital, Paris, France|St Mary's Hospital, London, United Kingdom |
| Cerebrovascular Reserve and White Matter Disease in Patients with Chronic Anemia | This is primarily an observational trial in patients with chronic anemia syndromes (sickle cell disease and thalassemia) and control subjects. The key purpose is to understand how brain blood flow reserve (the ability of the brain to increase its flow in response to stress) is altered in patients with chronic anemia. Since this parameter may depend on anemia severity, we will perform the MRI monitoring prior to and following clinically indicated transfusions in a subset of patients. Most patients will already be prescribed hydroxyurea as part of their standard of care. Since hydroxyurea could impact brain blood flow, there is also a small pilot study (20 patients, nonrandomized, open label) where MRI imaging will be performed prior to and following administration of hydroxyurea up to maximum tolerated dose. The study will enroll 90 adult subjects with transfusion independent sickle cell disease (70 SS, 10 SC, 10 Sβ0) and 60 patients with transfusion-dependent sickle cell disease. It will also include 10 transfusion independent thalassemia patients and 20 transfusion dependent thalassemia patients as well as 40 control subjects recruited from first degree relatives of the sickle cell disease population. All eligible subjects will be asked to provide informed consent before participating in the study. | Thalassemia|Sickle Cell Disease|Healthy Controls | ALL | CHILD, ADULT, OLDER_ADULT | CHLA, Los Angeles, California, 90027, United States |
| Clonidine With Morphine in Patient Controlled Analgesia Pump in Vaso-Occlusive Crisis in Sickle Cell Disease Patient | Vaso-occlusive crisis are highly painful in Sickle-cell patients. Morphine is the treatment of choice for this pain. Various adjuncts have been studied for the treatment of vaso-occlusive crisis. The investigators aimed to study the effect of clonidine associated with morphine in PCIA (patient controlled intravenous analgesia pumps) regimen. The investigators will compare it to the morphine alone in PCIA for the treatment of vaso-occlusive pain. The investigators will measure the morphine consumption of all patient, the impact on the apparition of the morphine secondary effect and on inflammation biomarkers and the biopsychosocial respond. Each patient will be hospitalized and follow by haematologist from the hospital, pain doctors and nurses. It will be a double blind randomised, prospective study. The randomisation will be done by the pharmacy. | Sickle Cell Disease|Vaso-occlusive Pain Episode in Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | CHU Saint-Pierre, Bruxelles, 1000, Belgium |
| Bone Marrow for Hemoglobinopathy Research | Human participants affected with sickle cell disease or thalassemia will donate bone marrow for use in experimental laboratory models to study potential new treatments. This is an observational study using bone marrow from human participants. The investigators will use sickle cell and thalassemia mouse models to observe and evaluate the possibility of correcting these disorders through genetic alterations or drug treatment. | Sickle Cell Anemia|Thalassemia | ALL | CHILD, ADULT, OLDER_ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants | Background: Blood disorders like sickle cell disease and malaria affect many people around the world. Researchers want to learn more about blood disorders. To do this, they need to collect biological samples from people with blood disorders. They also need to collect samples from healthy people. Objective: To collect samples to use for research on blood disorders. Eligibility: People ages 18-70 who have blood disorders. Healthy volunteers without blood disorders are also needed. Design: Participants will be screened with a medical history, physical exam, and blood and urine tests. Participants will give one or more samples. They will give them over 5 years. They can choose not to give any of the samples: Saliva: Participants will spit into a tube. They may also have the inside of their mouth swabbed. Urine: Participants will urinate into a cup. Blood: Blood will be taken through a needle in the participant s arm. Fat samples: An area on the participant s belly or buttock will be numbed. A small cut will be made into the skin and a small piece of fat removed. Mucus and cells from the lungs: The participant will be sedated. A flexible tube will be inserted through the nose or mouth into the lung airways. These participants will also have a physical exam, chest x-ray, and heart tests after the procedure. | Alpha and Beta Thalassemia|Sickle Cell Disease|Malaria|Human Physiology | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Carbon Monoxide Monitor for the Measurement of End-Tidal Carbon Monoxide Levels in Children With or Without Hemolysis | People who have Sickle Cell Anemia (HbSS) produce red blood cells with shorter lifespans. These red blood cells breakdown faster, and this is called hemolysis. When red blood cells breakdown, a tiny amount of Carbon Monoxide (CO) is released into the blood and is eliminated in exhaled breath. This research study will use a device called CoSense™, which will measure Carbon Monoxide (CO) levels in breath. The purpose of the study is to see how well the device measures the CO levels that an individual breathes out. | Sickle Cell Anemia | ALL | CHILD | Children's Hospital & Research Center Oakland, Oakland, California, 94609, United States |
| SCD-PROMIS: A Software Platform to Enhance Self-efficacy and Patient-provider Engagement for Patients With Sickle Cell Pain | The overall goal of the project is to reduce pain-related, 30-day readmission rates for sickle cell disease (SCD) patients. The investigators want to see if a mobile phone application (app) can help decrease the need for repeat admission to the hospital because of sickle cell pain. | Sickle Cell Disease|Anemia|Anemia, Hemolytic|Anemia, Sickle Cell|Hematologic Diseases | ALL | CHILD, ADULT | Children's National Hospital, Washington, District of Columbia, 20010, United States |
| Voxelotor Neurocognitive Function Study | This is a Phase 3b, randomized, double-blind, placebo-controlled, multicenter study to assess the treatment effect of voxelotor on neurocognitive function as assessed by the National Institute of Health (NIH) Toolbox Cognition Module of executive abilities in pediatric participants (8 to \< 18 years) with SCD. | Sickle Cell Disease | ALL | CHILD, ADULT | University of Maryland Medical Center, Baltimore, Maryland, 21201, United States |
| Systematic Psychological and Medical Care for Children With SCD | Sickle cell disease (SCD), a genetically transmitted blood disease, necessitates life-long care. In children, the disease may cause intense pain and other severe complications. Studies show that sources of stress, as well as complex psychological and intercultural issues associated with SCD, often aggravate symptoms. At Louis Mourier hospital, the treatment model used is systematic psychological and medical care. Our hypothesis is that this care is beneficial for both the children and their families as seen in improved quality of life and positive impact on medical symptoms. | Anemia, Sickle Cell | ALL | CHILD, ADULT | Hôpital Louis Mourier, Colombes, 92, France |
| Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies | This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer. | Sickle Cell Disease|Transfusion Dependent Alpha- or Beta- Thalassemia|Diamond Blackfan Anemia|Paroxysmal Nocturnal Hemoglobinuria|Glanzmann Thrombasthenia|Severe Congenital Neutropenia|Shwachman-Diamond Syndrome|Non-Malignant Hematologic Disorders | ALL | CHILD, ADULT | University of Minnesota Medical Center, Fairview, Minneapolis, Minnesota, 55455, United States |
| Analgesic Effect of Parecoxib Versus Morphine in SCD Patients Presenting to the Emergency Department | Introduction: This study focuses on the treatment of painful crises in Sickle Cell Disease (SCD) patients using Paracoxib, a non-opioid, compared to Morphine. It addresses the need for alternative medications that reduce opioid dependency while providing effective analgesia. Objectives: Primary: Evaluate the analgesic effect of Paracoxib versus Morphine in SCD vaso-occlusive crises. Secondary: Reduce opioid use/dependence, decrease the length of hospital stays, and monitor side effects related to Paracoxib. Methodology: A double-blinded randomized controlled trial, conducted in a tertiary care emergency department. The study includes adult SCD patients with moderate to severe crises, excluding non-VOC pain, certain medications, and specific medical conditions. The sample size is 226 patients, split equally into two groups. Intervention: Patients receive either Morphine or Paracoxib, with periodic assessment of vital signs and pain. Additional Morphine is administered if required. Data collection and analysis are meticulously planned. Expected Outcomes: Improvement in SCD pain management, reduction in opioid usage, and potential benefits in terms of hospital stays and patient satisfaction. | Sickle-Cell Disease With Crisis | ALL | ADULT, OLDER_ADULT | |
| Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies | Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies | Sickle Cell Disease & Thalassemia | ALL | CHILD, ADULT | Steven & Alexandra Cohen Children's Medical Center, New York, New York, New York, 11040, United States|Duke University Medical Center, Durham, North Carolina, 27705, United States|The University Of Texas M. D. Anderson Cancer Center, Houston, Texas, 77030-4009, United States |
| Hydroxyurea Therapy for Neurological and Cognitive Protection in Pediatric Sickle Cell Anemia in Uganda ( BRAINSAFE-II ) | Worldwide, an estimated 200,000 babies are born with Sickle Cell Anemia (SCA) annually. Affected children suffer chronic ill health with some having frequent hospitalization. The patients are also at a high risk of brain injury arising from small and large cerebral blood vessel damage in SCA, also called sickle cell vasculopathy (SCV). SCV is associated with the high risk of stroke. Such injury may manifest with neurological and cognitive impairment. An abnormal blood flow to the brain, as measured by a Doppler Ultrasound scan is a known risk factor for stroke. The hypothesis is that hydroxyurea therapy will prevent, stabilize or improve SCV and its effects. The study is an open label, single arm clinical trial to test the impact of hydroxyurea treatment in 270 children with SCA starting at ages 3-9 years. Following baseline assessments, all participants will begin hydroxyurea therapy starting at about 20mg/kg/day. Changes in the frequency and severity of each test (neurological and cognitive tests and cerebral blood flow velocity) will be compared with their baseline tests (prior to hydroxyurea) by repeating these tests at 18 and 36 months. In a randomly selected subset of 90 participants, an evaluation of the impact of hydroxyurea on structural brain vascular injury using magnetic resonance brain imaging (MRI) and magnetic vessel imaging ,also called angiography (MRA) at baseline and at 36 months. Lastly, an assessment of changes to biomarkers of anemia, inflammation and malnutrition from before and during hydroxyurea therapy and determine their relationship to the outcomes. The proposed intervention with hydroxyurea is the first Africa-based trial to broadly prevent or ameliorate manifestations of SCV. | Sickle Cell Anemia in Children | ALL | CHILD | Global Health Uganda, Kampala, +256, Uganda |
| A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises | This Phase 3 study will assess the safety and efficacy of a single dose of inclacumab, a P-selectin inhibitor, for a vaso-occlusive crisis (VOC) after an index VOC in participants with sickle cell disease (SCD). Participants will be randomized to receive either inclacumab or placebo. | Sickle Cell Disease|Vaso-occlusive Crisis|Vaso-occlusive Pain Episode in Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Strada Patient Care Center, Mobile, Alabama, 36604, United States|University of South Alabama Children's and Women's Hospital, Mobile, Alabama, 36604, United States|University of South Alabama Mitchell Cancer Institute, Mobile, Alabama, 36604, United States|Phoenix Children's Hospital, Phoenix, Arizona, 85016, United States|Arkansas Children's Hospital, Little Rock, Arkansas, 72202, United States|UCSF Benioff Children's Hospital, Oakland, Oakland, California, 94609, United States|Children's Hospital of Orange County, Orange, California, 92868, United States|St. Joseph's Hospital, Tampa, Florida, 33607, United States|University of Michigan Hospitals - Michigan Medicine, Ann Arbor, Michigan, 48109, United States|Functional Fluidics, Inc., Detroit, Michigan, 48202, United States|Alliance for Childhood Diseases dba Cure 4 The Kids Foundation, Las Vegas, Nevada, 89135, United States|Jacobi Medical Center, Bronx, New York, 10461, United States|Erie County Medical Center, Buffalo, New York, 14215, United States|Duke University Medical Center, Durham, North Carolina, 27705, United States|DUMC Investigational Drug Services Pharmacy, Durham, North Carolina, 27710, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|PPD Bioanalytical, Richmond, Virginia, 23230, United States|Instituto D'Or de Pesquisa e Ensino - Hospital São Rafael, Salvador, Bahia, 41253-190, Brazil|Multihemo Servicos Medicos S/A, Recife, PE, 50070-460, Brazil|Hospital de Clinicas de Porto Alegre, Porto Alegre, RS, 90035-903, Brazil|Fundação Faculdade Regional de Medicina de São José do Rio Preto, São José Do Rio Preto, SP, 15090-000, Brazil|Casa de Saude Santa Marcelina, São Paulo, SP, 08270-070, Brazil|Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti - HEMORIO, Rio de Janeiro, 20211-030, Brazil|Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo -HCFMUSP, São Paulo, 05403-000, Brazil|CEPEC-Centro de Pesquisa Clinica, São Paulo, 08270-120, Brazil|Clinica de la Costa Ltda., Barranquilla, Atlantico, 080020, Colombia|Organizacion Clinica Bonnadona Prevenir S.A.S., Barranquilla, Atlántico, 080020, Colombia|Hopital Avicenne, Bobigny, 93000, France|Hopital Henri Mondor, Créteil, 94010, France|Universitatsklinikum Regensburg Padiatrische Hamatologie, Onkologie und Stammzelltransplantation, Regensburg, 93053, Germany|Farmacia Interna Azienda Ospedaliero-Ente Ospedaliero Ospedali Galliera, Genova, Genoa, 16128, Italy|S.S.D. Microcitemia, anemie congenite e dismetabolismo del ferro, Genova, 16128, Italy|DAI Materno-Infantile, UOC Clinica Pediatrica 1 Azienda Ospedaliera Universitaria "Luigi Vanvitell, Napoli, 80138, Italy|UO di Farmacia Clinica, Dipartimento di Medicina Sperimentale Azienda Ospedaliera Universitaria, Napoli, 80138, Italy|UOC Patologia Clinica e Molecolare Azienda Ospedaliera Universitaria "Luigi Vanvitelli", Napoli, 80138, Italy|UOC Radiologia Azienda Ospedaliera Universitaria "Luigi Vanvitelli", Napoli, 80138, Italy|Kemri/Crdr, Siaya, Kemri Clinical Research Annex,, Kisumu, Siaya, 40600, Kenya|International Cancer Institute, Eldoret, 30100, Kenya|Kenya medical Research Institute-centre for Respiratory Disease Research, Nairobi, 00100, Kenya|Strathmore University Medical Center - Center for Research in Therapeutic Sciences(CREATES), Nairobi, 00200, Kenya|American University of Beirut Medical Center, Hamra, Beirut, Lebanon|Nini Hospital, Tripoli, North Lebanon, Lebanon|University of Calabar Teaching Hospital, Calabar, Cross River State, 540242, Nigeria|National Hospital Abuja, Abuja, FCT, 900211, Nigeria|University of Abuja Teaching Hospital, Gwagwalada, FCT, 902101, Nigeria|Ahmadu Bello University Teaching Hospital (ABUTH), Zaria, Kaduna, 810107, Nigeria|University of Nigeria Teaching Hospital, Enugu, 460000, Nigeria|Barau Dikko Teaching Hospital/Kaduna State University, Kaduna, 800212, Nigeria|Aminu Kano Teaching Hospital, Kano, 700223, Nigeria|Department of Pediatrics, College of Medicine, Lagos University Teaching Hospital, Lagos, 100254, Nigeria|Sultan Qaboos University Hospital, Muscat, 123, Oman|Prince Mohammed bin Nasser Hospital, Jizan, Southern, 82943, Saudi Arabia|Hacettepe University, Ankara, Altindag/sihhiye, 06230, Turkey|Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Merkezi Hastanesi, Yenischir, Mersin, Mersin, 33343, Turkey|Baskent University Hospital, Adana, Yuregir, 01250, Turkey|Acibadem Adana Hastanesi Cocuk Hematoloji Onkoloji, Adana, 01130, Turkey|Guy's & Thomas' NHS Foundation Trust, London, England, SE1 9RT, United Kingdom|Matero Clinical Research Site,, Lusaka, 10101, Zambia|University Teaching Hospital- Children's Hospital, Lusaka, 10101, Zambia |
| Voxelotor CYP and Transporter Cocktail Interaction Study | This research study is examining multiple doses of voxelotor (a study drug intended for treatment of sickle cell disease) and how it interacts with additional substrates (substrates are drugs or other substances that are metabolized by cytochrome enzymes. The substrates used in this study are FDA approved medications). The study will help to determine the safety and tolerability of the study drugs taken together, as well as the pharmacokinetics (PK) on how your body processes and responds to the combination of the study drug and substrates. Although these drugs are FDA approved, their use in this study is experimental. | Sickle Cell Disease | ALL | ADULT | ICON Early Phase Services, LLC, San Antonio, Texas, 78209, United States |
| Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia | This study will develop a national cord blood bank for siblings of patients with hemoglobinopathies and thalassemia. | Hematologic Diseases|Anemia, Sickle Cell|Beta-Thalassemia|Hematopoietic Stem Cell Transplantation | ALL | CHILD | Children's Hospital Oakland, Oakland, California, 94609, United States|Children's Hospital, Oakland, Oakland, California, 94609, United States|Children's National Medical Center, Washington, District of Columbia, United States|Nemours Children's Clinic, Jacksonville, Florida, 32207, United States|University of Miami Batchelor Children's Research Center, Miami, Florida, 33136, United States|Children's Memorial Hospital, Chicago, Illinois, 60614, United States|Louisiana State University Children's Medical Center, New Orleans, Louisiana, United States|University of Michigan, Ann Arbor, Michigan, 48109, United States|Hackensack University Medical Center, Hackensack, New Jersey, 07601, United States|Duke University Medical Center Children's Hospital, Durham, North Carolina, United States|Children's Hospital Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Medical University of South Carolina, Charleston, South Carolina, 29403, United States|University of Texas Southwestern Medical Center - Dallas, Dallas, Texas, 75235, United States|Texas Transplant Institute, San Antonio, Texas, 78229, United States|Hopital Ste-Justine, Montreal, Quebec, Canada |
| Evaluation of Left Ventricular Function by Speckle Tracking Echocardiography in Patient Hospitalised in Intensive Care Unit for Vaso-occlusive Crisis | Vaso-occlusive crisis in Sickle cell disease might alter myocardial function through micro vascular obstruction. Evaluation of strain alteration using speckle tracking echocardiography is a non invasive technique that may allow us to observe such myocardial dysfunction. No such study has yet been conducted in patient hospitalised in intensive care unit. Our hypothesis is that strain alteration during vaso-occlusive crisis, if they do exist, can be correlated with other markers of myocardial injury such as troponin level or thoracic pain. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Medical Intensive Care Unit, Le Kremlin-Bicêtre, France |
| Evaluation of the Impact of Renal Function on the Pharmacokinetics of SIKLOS ® (DARH) | The use of hydroxyurea in sickle cell disease patients with glomerular hyperfiltration and renal failure requires a specific monitoring and dose adjustment in order to remain within the therapeutic interval while limiting the risk of toxicity or therapeutic failure. For this reason the investigators propose to compare the pharmacokinetic parameters of hydroxyurea in normal-renal function sickle cell patients to those of patients with glomerular hyperfiltration or moderate renal failure. | Sickle Cell Disease|Renal Function Disorder | ALL | ADULT, OLDER_ADULT | Centre de Référence des Syndromes Drépanocytaires Majeurs, Hôpital Henri-Mondor, Creteil, France |
| A Study of the Absorption, Metabolism, and Excretion of GBT440 in Healthy Male Subjects | This study will provide information regarding the metabolic pathway of GBT440, the need for evaluation of potential drug-drug interactions, and the need for studies in special populations. The administration of radiolabeled drug is necessary to fully characterize the rates and routes of elimination of GBT440, providing further quantitative information on the disposition of GBT440. The results from this study will permit a comprehensive comparison between animal and human routes of elimination and metabolic profiles of GBT440. | Anemia, Sickle Cell | MALE | ADULT | Covance Early Clinical Services, Madison, Wisconsin, 53704, United States |
| Stem Cell Collection for Adult Volunteers | This study will examine the development of stem cells (very immature cells produced by the bone marrow) and their potential to change into cells of other organ types. These cells will be studied for their potential use in creating replacement tissue for diseases ranging from diabetes to Parkinson s. Healthy volunteers 18 years of age or older may be eligible for this study. Candidates will be screened with a medical history, physical examination, and blood tests. Participants will undergo a process called 'stem cell mobilization and apheresis' to collect bone marrow stem cells. For five days before the collection they will receive injections of a hormone called G-CSF, which stimulates release of stem cells from the bone marrow into the bloodstream. On the fifth day of the injections, stem cells will be collected through apheresis. For this procedure, blood is collected through a catheter (plastic tube) placed in an arm vein and directed into a cell separator machine. There, the white cells and stem cells are separated from the other blood components through a spinning process and collected in a bag inside the machine. The rest of the blood is returned to the donor through a catheter in the other arm. | Sickle Cell | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| Stroke With Transfusions Changing to Hydroxyurea | The purpose of this study is to compare standard therapy (transfusions and chelation) with alternative therapy (hydroxyurea and phlebotomy) for the prevention of secondary stroke and management of iron overload in children with sickle cell anemia (SCA). | Hemochromatosis|Cerebrovascular Accident|Anemia, Sickle Cell|Hematologic Diseases | ALL | CHILD, ADULT | University of Alabama at Birmingham, Birmingham, Alabama, 35233, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States|Nemours Children's Clinic, Jacksonville, Florida, 32207, United States|University of Miami, Jackson Memorial Hospital, Miami, Florida, 33136, United States|Nemours Children's Clinic, Orlando, Florida, 32806, United States|Children's Healthcare of Atlanta at Egleston, Atlanta, Georgia, 30322, United States|Children's Healthcare of Atlanta at Grady, Atlanta, Georgia, 30322, United States|Children's Healthcare of Atlanta at Scottish Rite, Atlanta, Georgia, 30342, United States|Children's Memorial Hospital, Chicago, Illinois, 60614, United States|Boston Children's Hospital, Boston, Massachusetts, 02115, United States|Wayne State University, Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States|The Children's Mercy Hospital, Kansas City, Missouri, 64108, United States|St. Joseph's Children's Hospital, Paterson, New Jersey, 07503, United States|Montefiore Medical Center, Bronx, New York, 10467, United States|State University of New York/Downstate Medical Center, Brooklyn, New York, 11203, United States|Schneider Children's Hospital, New Hyde Park, New York, 11040, United States|Columbia University Medical Center, Morgan Stanley Children's Hospital of New York-Presbyterian, New York, New York, 10032, United States|East Carolina University, Greenville, North Carolina, 27834, United States|Cincinnati Children's Hospital, Cincinnati, Ohio, 45229, United States|The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, 15213, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|University of Texas Southwestern Medical Center at Dallas, Dallas, Texas, 75390-9063, United States|Baylor College of Medicine, Houston, Texas, 77030-2399, United States|Eastern Virginia Medical School, Norfolk, Virginia, 23510, United States|Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| Clinical and Laboratory Characteristics of Sickle Cell Anemia Patients Admitted With Fever | This study will summarized the clinical and laboratory data and the outcome of all the patients suffering from Sickle Cell Anemia (Including Sickle cell thalassemia) admitted to the pediatric ward. | Sickle Cell Anemia|Sickle Cell Thalassemia | ALL | CHILD, ADULT, OLDER_ADULT | Pediatric Hematology Unit - Ha'Emek Medical Center, Afula, 18101, Israel |
| Quality of Care of Children With Sickle Cell Disease (SCD) Screened at Birth in France | The main objective of this study is to evaluate at the national level (France) * the early healthcare practices for children with sickle cell disease screened at birth, * the adequacy of theses practices with the national recommendations, * their variability over time and according the characteristics of treatment centers. Will be studied in particular the diffusion of the latest preventive measures (practice of trans-cranial Doppler and pneumococcal conjugate vaccine) and their link with the residual risks of death, stroke and invasive pneumococcal infections. The study includes all patients born in France between 01/01/2006 and 31/12/2010. Events are recorded and analysed only during the first 5 years of life. | New-borns Screening|Sickle Cell Disease | ALL | CHILD | |
| Tricuspid Regurgitant Jet Velocity as an Independent Marker for Mortality in Sickle Cell Anemia | The purpose of this study is to evaluate patients with pulmonary hypertension and sickle cell disease who have had multiple echocardiograms. Previous studies have shown that an elevated tricuspid jet (TR) regurgitant velocity on echo in this population is a predictor of mortality. This initial data only examined an isolated TR jet velocity. It was presumed that the mortality was related to pulmonary hypertension. It is the aim of this study to retrospectively evaluate patients who have had multiple echocardiograms and to determine if patients who had either a normalization of their TR jet velocity on a subsequent echo or had no evidence of pulmonary hypertension on right heart catheterization had a similar mortality rate to those with persistently elevated TR jet velocity. | Sickle Cell Anemia|Pulmonary Hypertension | ALL | ADULT, OLDER_ADULT | The Ohio State University Medical Center, Columbus, Ohio, 43221, United States |
| Fertility Preservation in Women Who Will Have Gonadotoxic Therapy or Hematopoetic Stem Cell Transplantation, and in Women With Sickle Cell Disease | Background: - Some treatments for cancer or other diseases can lead to infertility in women. These treatments include chemotherapy, some stem cell transplants, and pelvic radiotherapy. They are called gonadotoxic therapies. Women can now have their eggs frozen before they have these treatments. This may allow them to get pregnant later. Researchers want to learn more about this technology and processes. Objectives: - To provide egg freezing for women having gonadotoxic therapies at NIH. To learn more about the effects of these therapies. Eligibility: - Women at least 18 years old who are past puberty and before menopause. They must be scheduled to have gonadotoxic therapies. Design: * Participants will be screened with medical history and blood and hormone tests. They will also have a physical exam and transvaginal ultrasound. * Ovary stimulation: participants will have medications injected under the skin. These increase the chance of fertility. This phase will take about 8 20 days. Participants will have blood drawn and transvaginal ultrasound daily or every other day. Some participants will also have blood thinner injected daily. * Egg retrieval: participants will check in to the hospital. Eggs will be removed with a needle during a short surgery. Participants will be awake but sedated. * Participants may stay overnight in the hospital. * They will return every 1 3 days for 1 3 weeks for blood tests. * Mature eggs will be frozen after egg retrieval and immature eggs (which cannot be fertilized for clinical use) will be used for research. Participants can use their eggs in the future at outside, private fertility clinics to try to become pregnant. If the eggs are stored for more than 5 years, participants must pay for storage. | Hemoglobin|Stem Cell Transplant|Aplastic Anemia | ALL | CHILD, ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| A GBT021601 ADME Microtracer Study in Healthy Volunteers | An Open-label Study of GBT021601 in 8 to 10 healthy male or female participants to evaluate the absorption, distribution, metabolism, and excretion (ADME) of GBT021601. | Sickle Cell Disease | ALL | ADULT | ICON Early Phase Clinic, LLC, Groningen, 9728, Netherlands |
| Gum Arabic as Anti-oxidant, Anti-inflammatory and Fetal Hemoglobin Inducing Agent in Sickle Cell Anemia Patients | To study the efficacy of Gum Arabic as an anti-oxidant, anti-inflammatory and Fetal Hemoglobin-inducing agent among Sickle Cell Disease children. Half of participants will receive Gum Arabic and the other half will receive placebo | Sickle Cell Anemia in Children | ALL | CHILD, ADULT | Military Hospital, Omdurman, Khartoum, 1113, Sudan |
| Pharmacokinetics of Oral Hydroxyurea Solution | An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e. to the day before 18th birthday), with a 12 to 15 month treatment period for each participant. The study treatment duration will be for 6 months at the maximum tolerated dose \[MTD\], which is usually reached by 6 months after initiation of treatment. For patients in whom time to MTD is longer than 6 months or not achieved at all, the maximum duration of study treatment will be 15 months. | Sickle Cell Disease|Sickle-Cell; Hemoglobin Disease, Thalassemia|Sickle Cell-beta-thalassemia|Sickle Cell Hemoglobin C | ALL | CHILD | Dr Angela E Rankine- Mullings, Kingston, Jamaica|Birmingham Women's and Children's NHS Foundation Trust, Birmingham, United Kingdom|Alder Hey Children's NHS Foundation Trust, Liverpool, United Kingdom|Evelina London Children's Hospital, London, United Kingdom|King's College Hospital NHS Foundation Trust, London, United Kingdom|The Royal London Children's Hospital, Barts Health NHS Trust, London, United Kingdom |
| Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II Protocol | The BABY HUG Treatment Study was designed to see if treatment with the drug hydroxyurea (also called HU) in children with sickle cell disease could prevent organ damage, especially in the spleen and kidneys. There was also a chance that treatment could prevent painful crises, lung disease, stroke, and blood infection. | Sickle Cell Anemia | ALL | CHILD, ADULT | University of Alabama at Birmingham, Birmingham, Alabama, 35233, United States|Children's National Medical Center Center for Cancer and Blood Disorders, Washington, District of Columbia, 20010, United States|Howard University College of Medicine, Washington, District of Columbia, 20060, United States|University of Miami School of Medicine, Miami, Florida, 33136, United States|Emory University School of Medicine, Atlanta, Georgia, 30342, United States|Johns Hopkins University School of Medicine, Baltimore, Maryland, 21205, United States|Sinai Hospital of Baltimore Alfred I Coplan Pediatric Hematology Oncology Outpatient Center, Baltimore, Maryland, 21215, United States|Children's Hospital of Michigan/Wayne State University, Detroit, Michigan, 48201, United States|University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States|Downstate Medical Center, Brooklyn, New York, 11203, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|University of Texas Southwestern Medical Center at Dallas, Dallas, Texas, 75390, United States |
| Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy | Determine knowledge, attitudes, and beliefs among adult patients, and parents of pediatric patients, with transfusion dependent beta-thalassemia and sickle cell disease toward gene therapy to treat their or their child's illness, and to assess the likely impact of gene therapy on patients' quality of life. | Transfusion Dependent Beta Thalassemia|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Georgetown University Medical Center, Washington, District of Columbia, 20007, United States |
| THE IMPROVE TRIAL: Improving Pain Management and Outcomes With Various Strategies of Patient-Controlled Analgesia (PCA) | Patient-Controlled Analgesia (PCA) means that the patient is in control of his/her pain medicine. In this study two (2) different treatment plans of Patient-Controlled Analgesia will be used to treat people with sickle cell disease who are admitted to the hospital for a pain crisis. The purpose of this study is to find out if one plan is better than the other in controlling sickle cell pain. If you are eligible for the study, you will be assigned by chance (like flipping a coin) to either get a higher continuous amount of the pain medicine with a smaller amount for pain as you need it, OR to get a smaller continuous amount of pain medicine with a larger amount of pain medicine as you need it. You or your study doctor can not choose which plan you receive, and you will not be told which one you have been assigned to. The doctors and nurses taking care of you will know which plan you are assigned to so they can safely and effectively take care of your pain. Some members of the study team will not know which plan you are on. We will give you morphine sulfate or hydromorphone (dilaudid) for your pain. These medicines are approved by the Food and Drug Administration (FDA) and have been used for a long time to relieve pain. If you have been treated for pain before with hydromorphone (dilaudid) and you prefer it to morphine, then you may choose to get it during the study. If you have not received hydromorphone (dilaudid) before or you do not have a preference then you will be given morphine for pain. The pain medicine will be given through the IV in your arm. You will receive morphine or hydromorphone continuously through the IV and will also be able to use the PCA machine to give yourself extra pain medicine as you need it for pain. You will need to push a button to give yourself extra medicine for pain. The amount of pain medicine you get on these plans is based on how much you weigh. | Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital and Research Center, Oakland, California, United States|Yale-New Haven Medical Center,, New Haven, Connecticut, United States|A.I. duPont Hospital for Children, Wilmington, Delaware, United States|Children's National Medical Center, Washington, District of Columbia, United States|Howard University Hospital, Washington, District of Columbia, United States|Emory University School of Medicine, Atlanta, Georgia, United States|Medical College of Georgia, Augusta, Georgia, United States|Children's Memorial Hospital, Chicago, Illinois, United States|University of Illinois Sickle Cell Center, Chicago, Illinois, United States|Kosair Children's Hospital, Louisville, Kentucky, United States|Children's Hospital at Sinai, Baltimore, Maryland, United States|Johns Hopkins, Baltimore, Maryland, United States|National Institutes of Health Clinical Center, Bethesda, Maryland, United States|Boston Medical Center, Boston,, Massachusetts, United States|Children's Hospital Boston, Boston, Massachusetts, United States|University of Mississippi Medical Center, Jackson, Mississippi, United States|Interfaith Medical Center, Brooklyn, New York, United States|New York Methodist Hospital, Brooklyn, New York, United States|The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States|Duke University Medical Center, Durham, North Carolina, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States|Nationwide Children's Hospital, Columbus, Ohio, United States|Ohio State University, Columbus, Ohio, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States|St. Christopher's Hospital for Children, Philadelphia, Pennsylvania, United States|Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States|Texas Children's Hospital, Houston, Texas, United States|Virginia Commonwealth University Health Systems, Richmond, Virginia, United States |
| Study Evaluating 13-valent Pneumococcal Conjugate Vaccine (13vPnC) in Children With Sickle Cell Disease | This study is to evaluate the safety, tolerability and immunogenicity of 13-valent pneumococcal Conjugate Vaccine in children with Sickle Cell Disease who have already been vaccinated with 23-valent polysaccharide vaccine. The study will measure the amount of antibodies (the proteins that fight off germs) produced by children with Sickle Cell Disease after they have been given the 13-valent pneumococcal vaccine between 6 and less than 18 years of age. They will be given the vaccination twice, each vaccination separated by approximately 6 months. | Pneumococcal Conjugate Vaccine | ALL | CHILD | Pfizer Investigational Site, Birmingham, Alabama, 35233, United States|Pfizer Investigational Site, Louisville, Kentucky, 40202-3830, United States|Pfizer Investigational Site, Louisville, Kentucky, 40202, United States|Pfizer Investigational Site, Bronx, New York, 10467, United States|Pfizer Investigational Site, Nashville, Tennessee, 37232, United States|Pfizer Investigational Site, Cairo, 11361, Egypt|Pfizer Investigational Site, Cairo, 11511, Egypt|Pfizer Investigational Site, Paris, 75015, France|Pfizer Investigational Site, Orbassano (TO), 10043, Italy|Pfizer Investigational Site, Roma, 00165, Italy|Pfizer Investigational Site, Beirut, 2833-7401, Lebanon|Pfizer Investigational Site, Beirut, Lebanon|Pfizer Investigational Site, Riyadh, Saudi, Saudi Arabia|Pfizer Investigational Site, Manchester, Cheshire, M27 4HA, United Kingdom|Pfizer Investigational Site, London, E1 1BB, United Kingdom|Pfizer Investigational Site, London, SE1 7RH, United Kingdom|Pfizer Investigational Site, London, SE1 9RT, United Kingdom|Pfizer Investigational Site, London, SE5 9RS, United Kingdom|Pfizer Investigational Site, London, SW17 0RE, United Kingdom |
| Central Nervous System Vascular Changes in Adult Sickle Cell Disease and the Effect of Treatment With Simvastatin | Stroke is a frequent complication of sickle cell disease (SCD), with varying levels of central nervous system (CNS) involvement. The summation of several ischemic events, even when silent, can lead to devastating consequences, from reduced academic performance to physical dependence. Despite knowledge that brain flow velocities evaluated by Doppler ultrasound identify pediatric SCD patients at a greater stroke risk (Adams et al, NEJM 1998; 339:5-11), this method is not able to predict the occurrence of strokes in adults. There is also no consensus on the management of adult patients in relation to primary and secondary prevention. The aim of this study is to evaluate the effects of the administration of Simvastatin on CNS structural and functional vascular changes in 30 adult patients with SCD (SS and Sβ), above 35 years of age, observed through Magnetic Resonance Imaging (MRI). The data on the effect of simvastatin on disease manifestations is quite scarce, however this drug reportedly significantly reduces plasma concentrations of adhesion molecules and inflammatory markers, such as E-selectin, VEGF, CRP and IL-6 (Hoppe et al, BJH 2011; 153:655-663; Hoppe et al, BJH 2017;177:620-629). Thus, in addition to the search for early diagnostic markers and risk stratification for primary or recurrent stroke, we will also compare CNS images before and 12 months after the administration of Simvastatin. The drug alter stroke recurrence rates in the general adult population, but their effects on vascular changes in patients with SCD have not yet been adequately elucidated. This is particularly important because these are low cost drugs which present good tolerability, and could be part of the therapeutic arsenal of SCD, even in low income settings. Concomitantly with the CNS evaluation, this study also intends to investigate molecular pathways that may be affected by the drugs. We will evaluate microvesicle release patterns, as well as the content of microRNAs possibly involved in the occurrence of stroke, in addition to metabolomic studies and plasma cytokine profile. | Sickle Cell Disease|Stroke | ALL | ADULT, OLDER_ADULT | Hematology and Transfusion Medicine Center, Campinas, São Paulo, 13083-870, Brazil |
| Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells | Many genetic diseases of lymphohematopoietic cells (such as sickle cell anemia, thalassemia, Diamond-Blackfan anemia, Combined Immune Deficiency (CID), Wiskott-Aldrich syndrome, chronic granulomatous disease, X-linked lymphoproliferative disease, and metabolic diseases affecting hematopoiesis) are sublethal diseases caused by mutations that adversely affect the development or function of different types of blood cells. Although pathophysiologically diverse, these genetic diseases share a similar clinical course of significant progressive morbidity, overall poor quality of life, and ultimate death from complications of the disease or its palliative treatment. Supportive care for these diseases includes chronic transfusion, iron chelation, and surgery (splenectomy or cholecystectomy) for the hemoglobinopathies; prophylactic antibiotics, intravenous immunoglobulin, and immunomodulator therapies for the immune deficiencies; and enzyme replacement injections and dietary restriction for some of the metabolic diseases. The suboptimal results of such supportive care measures have led to efforts to implement more aggressive therapeutic interventions to cure these lymphohematopoietic diseases. The most logical strategies for cure of these diseases have been either replacement of the patient's own hematopoietic stem cells (HSC) with those derived from a normal donor allogeneic bone marrow transplant (BMT) or hematopoietic stem cell transplant (HSCT), or to genetically modify the patient's own stem cells to replace the defective gene (gene therapy). | Stem Cell Transplantation|Bone Marrow Transplantation|Peripheral Blood Stem Cell Transplantation|Allogeneic Transplantation|Genetic Diseases|Thalassemia|Pediatrics|Diamond-Blackfan Anemia|Combined Immune Deficiency|Wiskott-Aldrich Syndrome|Chronic Granulomatous Disease|X-linked Lymphoproliferative Disease|Metabolic Diseases | ALL | CHILD, ADULT, OLDER_ADULT | |
| Drug Interaction Study of GBT440 With Caffeine, S-warfarin, Omeprazole, and Midazolam in Healthy Subjects | The purpose of this study to evaluate the effect of concomitant administration of GBT440 on caffeine (a CYP1A2 probe substrate), S warfarin (a CYP2C9 probe substrate), omeprazole (a CYP2C19 probe substrate), and midazolam (a CYP3A4 probe substrate) plasma concentrations. | Sickle Cell Disease | ALL | ADULT | ICON Early Phase Services, LLC Clinical Research Unit, San Antonio, Texas, 78209, United States |
| A Study to Evaluate the Safety and Efficacy of Crizanlizumab in Sickle Cell Disease Related Priapism | The goal of the study is to evaluate the efficacy and safety of crizanlizumab in SCD patients with priapism. | Priapism | MALE | CHILD, ADULT, OLDER_ADULT | University Of Alabama ., Birmingham, Alabama, 35294, United States|University of Connecticut Health Center ., Farmington, Connecticut, 06030, United States|Childrens National Hospital SC, Washington, District of Columbia, 20010, United States|Foundation for Sickle Cell Disease Research, Hollywood, Florida, 33021, United States|Emory University School of Medicine ., Atlanta, Georgia, 30303, United States|LSU Medical Center, Shreveport, Louisiana, 71130, United States|Childrens Hospital Boston, Boston, Massachusetts, 02115, United States|Montefiore Medical Center ., Bronx, New York, 10461, United States|Duke University Medical Center ., Durham, North Carolina, 27710, United States|Brody School of Medicine ., Greenville, North Carolina, 27834, United States|University of Pittsburgh ., Pittsburgh, Pennsylvania, 15213-2548, United States|Prisma Health Upstate ., Greenville, South Carolina, 29615, United States|University of Texas Medical School CFTY720D2399E1, Houston, Texas, 77030, United States |
| Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Study Of PF-04447943, Co-Administered With And Without Hydroxyurea, In Subjects With Stable Sickle Cell Disease | This study is being conducted to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of an investigational drug, PF-04447943, in subjects with stable sickle cell disease with and without co-administration with hydroxyurea. This study will also aid in selecting the doses for future studies and evaluation of substances in the blood which may help access the effectiveness of the drug. | Phase 1 Sickle Cell | ALL | ADULT, OLDER_ADULT | University of Illinois Hospital and Health Sciences System, Chicago, Illinois, 60612-5836, United States|University of Illinois at Chicago Clinical Research Center, Chicago, Illinois, 60612, United States|University of Illinois Hospital and Health Sciences System, Chicago, Illinois, 60612, United States|Boston Medical Center E7E, Boston, Massachusetts, 02118, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|Boston University Medical Center, Boston, Massachusetts, 02118, United States|Interfaith Medical Center, Brooklyn, New York, 11213, United States|Interfaith Medical Center, Brooklyn, New York, 11238, United States|UNC Hospitals' Investigational Drug Service Pharmacy, Chapel Hill, North Carolina, 27514, United States|UNC School of Medicine Clinical and Translational Research Center, Chapel Hill, North Carolina, 27599, United States|Investigational Drug Services, Richmond, Virginia, 23298, United States|Virginia Commonwealth University, Richmond, Virginia, 23298, United States|Pfizer Clinical Research Unit, Brussels, B-1070, Belgium|Fondazione IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milano, 20122, Italy|A.O.O.R Villa Sofia - V. Cervello, Palermo, 90146, Italy|Centre for Human Drug Research, Leiden, 2333 CL, Netherlands|Royal Liverpool and Broadgreen University Hospital Trust, Liverpool, Merseyside, L7 8XP, United Kingdom|Guy's and St Thomas' NHS Foundation Trust, London, SE1 9RT, United Kingdom|King's College Hospital NHS Foundation Trust, London, SE5 9RS, United Kingdom|Imperial College Healthcare NHS Trust, London, W12 0HS, United Kingdom|Central Manchester University Hospitals NHS Foundation Trust, Manchester, M13 9WL, United Kingdom|Manchester Royal Infirmary, Manchester, M13 9WL, United Kingdom|Oxford University Hospitals NHS Trust, Oxford, OX3 7LE, United Kingdom |
| Inhaled Corticosteroid Use to Prevent Acute Chest Syndrome Recurrence in Children Between 1 and 4 With Sickle Cell Disease: a Feasibility Trial | Acute and chronic pulmonary complications with concomitant inflammatory response are a leading cause of morbidity and mortality in children with sickle cell disease (SCD). Acute chest syndrome (ACS), defined broadly as an increase in respiratory effort, fever and new radiodensity on chest x-ray, is a major cause of death in children and adults with SCD. There is a high rate of ACS in children between 1 and 4 years of age that is associated with an asthma diagnosis, and children with ACS events before 4 years of age have a 50% rate of being hospitalized for either ACS or pain within 1 year of admission. For children with SCD that develop ACS, the investigators propose that the use of budesonide inhalation suspension (BIS) will attenuate pulmonary inflammation after an ACS episode and will decrease future vaso-occlusive pain and ACS episodes. Through a single-arm prospective feasibility trial and in preparation for a limited-institution randomized trial, the investigators plan to test the following primary hypothesis for a phase III definitive trial: In children with SCD admitted to the hospital for an ACS episode between 1 and 4 years of age, low dose BIS for 6 months will result in a 50% reduction in the recurrent incidence rate of ACS or pain requiring hospitalization. Through this trial, the investigators will determine the acceptability of and adherence to BIS in the study population. The investigators will track respiratory symptoms in cases versus controls over 6 months. Finally, the investigators will explore the impact of BIS on biological correlates (sVCAM-1). | Sickle Cell Disease|Asthma|Acute Chest Syndrome | ALL | CHILD | Vanderbilt University Medical Center, Nashville, Tennessee, 37232-9000, United States |
| The American Society of Hematology (ASH) Research Registry: A Multicenter Research Registry of Patients With Hematologic Disease | This is a multicenter, retrospective and prospective, long-term registry of patients with benign or malignant hematologic diseases, whether or not these patients were or were not treated with disease-specific treatments. Information will be collected on patient demographics, disease characteristics, genomic and molecular data, laboratory data, pathology, radiographic reports, clinical status, quality of life, medications, and dosing information. Where appropriate, these data structures may be based on a combination of Fast Healthcare Interoperability Resources (FHIR) , Consolidated-Clinical Data Architecture (C-CDA) and/or client-specific structure definitions. | Benign and Malignant Hematologic Diseases | ALL | CHILD, ADULT, OLDER_ADULT | American Society of Hematology, Washington, District of Columbia, 20036, United States |
| A Study to Compare if the Uptake of Ticagrelor in the Body Differs When Different Tablets Are Administered | To evaluate the relative bioavailability of ticagrelor for the different formulations. A randomized cross-over design has been chosen to minimize the effects of between-subject variability and any period effects on the overall results. | Sickle Cell Disease | ALL | ADULT | Research Site, Berlin, 14050, Germany |
| A Study to Evaluate GBT021601 in Single and Multiple Doses in Healthy Participants | This first in human study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and food effect of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in healthy participants. | Sickle Cell Disease | ALL | ADULT | ICON Early Phase Services, LLC, San Antonio, Texas, 78209, United States|Harry Perkins Institute of Medical Research, Nedlands, Western Australia, 6009, Australia|Linear Clinical Research, Nedlands, Western Australia, 6009, Australia|Oxford Compounding, North Perth, Western Australia, 6006, Australia |
| Use of a Mobile-based App for SCD Patients | The study will seek to enroll 100 sickle cell or thalassemia patients who are age 12 or older who have access to a smartphone or tablet with Internet access daily. The study will evaluate patient-reported comfort level with using a mobile device to record their pain levels, as well as adherence to recording these levels daily. The study will track patients' assessment of their pain, actions taken, and outcomes related to pain management and provider involvement. This study will attempt to collect information about differences in the use of two traditional pain assessment modes (verbal scale and paper) versus the use of a pain assessment tool on a mobile device in the form of a smartphone, tablet, or iPad with an Android or iOS operating system. | Sickle Cell Disease|Chronic Pain | ALL | CHILD, ADULT, OLDER_ADULT | Duke University Medical Center, Durham, North Carolina, 27710, United States |
| Hydroxyurea Management in Kids: Intensive Versus Stable Dosage Strategies | This is a pilot study, single-blind, randomized, multicenter, therapeutic clinical trial designed to evaluate the feasibility of enrolling infants and toddlers (9 months to 36 months) with sickle cell anemia (SCA; HbSS or HbSβ\^0thalassemia), regardless of disease severity, to a therapeutic trial. A prior clinical trial at St. Jude Children's Research Hospital (SJCRH) (BABYHUG, NCT01783990) demonstrated that a fixed dose (20 mg/kg/day) of hydroxyurea was safe and effective in decreasing SCA-related complications in very young children (9-18 months), and largely due to these findings, hydroxyurea is recommended to be offered to all children (≥9 months old) with SCA, independent of disease severity. Nevertheless, children in the treatment arm of BABYHUG continued to experience vaso-occlusive symptoms and to incur organ damage. In clinical trials of older children with SCA, intensification of hydroxyurea to a maximum tolerated dosage (MTD), defined by mild to moderate myelosuppression, may be associated with improved laboratory parameters compared to fixed lower-dosing, but the clinical benefits gained from dose intensification have not been described. Therefore, in this trial, children in the standard treatment arm will receive a fixed dose of hydroxyurea (20 mg/kg/day), and participants in the experimental arm will receive hydroxyurea intensified to MTD, defined by a goal absolute neutrophil count (ANC) of 1500-3000 cells/µL. This trial aims to establish a multicenter infrastructure that will identify, enroll and randomize very young children (9-36 months) to receive fixed dose versus intensified-dose hydroxyurea in a single blinded manner, and to obtain prospective pilot data comparing the clinical and laboratory outcomes between the treatment arms to facilitate design of a definitive phase III trial. | Sickle Cell Anemia | ALL | CHILD | Emory University/Children's Health Care of Atlanta, Atlanta, Georgia, 30322, United States|University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|University of Texas Southwestern Medical Center at Dallas, Dallas, Texas, 75390-9063, United States |
| Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) | This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001). | Sickle Cell Disease|Hydroxyurea Failure|Hydroxyurea Intolerance|Hemoglobinopathies|Hematological Diseases | ALL | CHILD | Atrium Health Levine Children's Hospital, Charlotte, North Carolina, 28203, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers, Nashville, Tennessee, 37203, United States|University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology, Dusseldorf, Germany|Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Ospedale Pediatrico Bambino Gesu - IRCCS, Rome, Italy|St Mary's Hospital, London, United Kingdom |
| Imatinib and Carvedilol for High Blood Pressure in the Lungs in Adults With Sickle Cell Disease | Background: - About one-tenth of adults with sickle cell disease have pulmonary hypertension (high blood pressure in the lungs). This condition can cause shortness of breath, pain crisis, and congestive heart failure. It may even lead to death. Researchers want to test the drugs imatinib and carvedilol to see if they can treat high blood pressure in the lungs. Both drugs have been used to treat other types of heart problems, but they have not been tested as a treatment for high blood pressure related to sickle cell disease. Objectives: - To see if imatinib and carvedilol are safe and effective treatments for high blood pressure in the lungs in adults with sickle cell disease. Eligibility: - Adults at least 18 years of age who have sickle cell disease and have or may have high blood pressure in the lungs. Design: * Participants will be screened with a physical exam and medical history. They will also have different tests of heart and lung function, including a walking test and imaging studies. Blood and urine samples will also be collected. * Participants who meet specific criteria will take one of two possible study drugs. Those who receive imatinib will take it daily. Those who receive carvedilol will take it twice a day. * Participants will have weekly study visits for blood tests and other exams. The study drug dose will be adjusted at each weekly visit. It will be increased slowly to reach a target dose(based on the participant s weight) or to find a stable effective dose. * Participants may continue to take their study drug for up to 24 weeks, with weekly study visits. Regular blood samples and heart and lung function tests will be performed. * After 24 weeks, qualified participants may continue to take their study drug for up to 6 more months. They will have regular study visits to monitor the treatment. | Pulmonary Hypertension | ALL | ADULT, OLDER_ADULT | |
| Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies | This clinical trial studies the effect of pre-transplant immunosuppression (PTIS) and donor stem cell transplant in treating patients with severe blood diseases (hemoglobinopathies). PTIS helps prepare the body for the transplant and lowers the risk of developing graft versus host disease (GVHD). Hematopoietic cells are found in the bone marrow and produce blood cells. Hematopoietic cell transplantation (HCT) injects healthy hematopoietic cells into the body to support blood cell production. PTIS and HCT may help to control severe hemoglobinopathies. | Beta Thalassemia Major|Sickle Beta 0 Thalassemia|Sickle Beta Plus Thalassemia|Sickle Beta Thalassemia|Sickle Cell Disease|Sickle Cell-SS Disease | ALL | CHILD, ADULT | |
| Efficacy and Safety of Bosentan in Sickle Cell Disease (SCD) Patients Diagnosed With Pulmonary Hypertension (PH) | The study will assess the effect of bosentan on pulmonary vascular resistance and exercise capacity in Sickle Cell Disease (SCD) patients diagnosed with Pulmonary Hypertension. It consists of 3 phases: screening, treatment and follow-up. During the screening visit, the study doctor will decide if patients meet the study requirements. All potential patients will have a diagnosis of increased pulmonary artery pressures that is shown by right heart catheterization conducted shortly prior to start of study treatment. Patients will be asked to perform exercise capacity test (walking as far as possible for 6 minutes). Following the baseline visit the treatment phase consists of 4 additional clinic visits during which the good and bad effects of the drug are reviewed and exercise capacity test will be repeated. Patients will be treated for 16 weeks. Blood samples will be collected every month, or more often, if needed. At the end of the study some of the patients will be asked to repeat the right heart catheterization. All patients will repeat an exercise capacity test. After completion of the study, patients will have the option of enrolling in a long-term follow-up study where all patients will receive active drug. Patients electing not to participate in the extension study will be followed up for safety assessments for about 28 days after the end of the study treatment. | Pulmonary Hypertension | ALL | CHILD, ADULT, OLDER_ADULT | University of Alabama, Birmingham, Alabama, 35294, United States|Alta Bates Medical Center, Berkeley, California, 94705, United States|Harbor -UCLA Medical Center, Torrance, California, 90502, United States|University of Colorado Health Sciences Center, Denver, Colorado, 80262, United States|Howard University Hospital, Washington, District of Columbia, 20060, United States|University of Illinois Medical Center, Chicago, Illinois, 60612, United States|National Institutes of Health, Bethesda, Maryland, 20892, United States|Boston Medical Center/Boston University School of Medicine, Boston, Massachusetts, 02118, United States|Harper University Hospital/Wayne State University, Detroit, Michigan, 48201, United States|Henry Ford Hospital; Dept. of Pulmonology, Detroit, Michigan, 48202, United States|SoLUtions/Saint Louis University, St. Louis, Missouri, 63104, United States|Columbia University Medical Center; Pediatric Cardiology, New York, New York, 10032, United States|UNC Comprehensive Sickle Cell Program, Chapel Hill, North Carolina, 27599, United States|Duke University Medical Center; Duke University Health Systems, Durham, North Carolina, 27710, United States|University Hospitals of Ohio, Cleveland, Ohio, 44106, United States|Ohio State University, Columbus, Ohio, 43210, United States|Temple University Lung Center, Philadelphia, Pennsylvania, 19140, United States|University of Tennessee Health Science Center, Memphis, Tennessee, 38163, United States|The Methodist Hospital/Baylor College of Medicine; Pulmonary and Critical Care Medicine, Houston, Texas, 77030, United States|University of Texas Medical School; Division of Pulmonary and Critical Care Medicine, Houston, Texas, 77030, United States|Virginia Commonwealth University Medical Center, Richmond, Virginia, 23298-5028, United States|CHU de Fort de France, Fort de France, La Martinique, 97200, France|Hopital Antoine Beclere, Clamart, 92141, France|CHU Henri Mondor, Creteil, 94010, France|Amsterdam Medical Center, Department of Hematology, Amsterdam, 1105 AZ, Netherlands|Royal Free Hospital, Rheumatology Department, London, NW3 2QG, United Kingdom|Royal Hallamshire Hospital, Pulmonary Vascular Medicine, Sheffield, S10 2JF, United Kingdom |
| Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease | This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001. | Beta-Thalassemia|Thalassemia|Hematologic Diseases|Genetic Diseases, Inborn|Hemoglobinopathies|Sickle Cell Anemia|Sickle Cell Disease | ALL | CHILD, ADULT | Columbia University Medical Center, New York, New York, 10032, United States|Atrium Health Levine Children's Hospital, Charlotte, North Carolina, 28203, United States|SCRI at the Children's Hospital at TriStar Centennial, Nashville, Tennessee, 37203, United States|Universitätsklinikum Düsseldorf Hospital Duesseldorf, Duesseldorf, Germany|Ospedale Pediatrico Bambino Gesù, IRCCS, Rome, Italy|King Faisal Specialist Hospital and Research Centre, Al Mathar Ash Shamali, Saudi Arabia |
| Management of Severe Acute Malnutrition in SCD, in Northern Nigeria | Except for children with HIV, all recommendations for treatment of childhood malnutrition are for children \< 5 years of age. The overall goal of this randomized controlled nutrition feasibility trial is to identify whether families of children with sickle cell disease (SCD) 5 years and older agree to participate over a 12-week period. The investigators will also establish a safety protocol for monitoring potential complications associated with treating severe malnutrition in children 5 years and older with and without SCD, in a low-resource setting. | Sickle Cell Anemia|Severe Acute Malnutrition | ALL | CHILD | Vanderbilt University Medical Center, Nashville, Tennessee, 37232-9000, United States|Aminu Kano Teaching Hospital, Kano, Nigeria|Murtala Mohammad Specialist Hospital, Kano, Nigeria |
| Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP | Patients are being asked to participate in this study because they have severe sickle cell anemia (SCD) with or without the beta thalassemia trait. Sickle cell anemia is an illness where the red blood cells change shape and can clog up blood vessels. This keeps the body from getting the oxygen it needs. Thalassemia is when the body does not make enough hemoglobin, something that helps the oxygen get to the places it needs to go in the body. The patient may or may not need to get regular blood transfusions (getting more blood) to improve their quality of life (feel better) and prevent organ damage (problems with the brain, heart, lung, kidney, and gonad, for example.). The transfusions can also cause problems, including iron overload (too much iron in the blood), which can be fatal (patients can die) without regular deferoxamine shots. Even with the best usual treatments, people with thalassemia or SCD die sooner. There is no proven cure. We would like to treat patients using bone marrow transplantation, a treatment that has been used for people with SCD. The transplant uses healthy "matched" bone marrow. This comes from a brother or sister who does not have sickle cell disease or severe thalassemia. If the treatment works, the sickle cell disease or thalassemia may be cured. This treatment has been used to treat patients with sickle cell disease or thalassemia. It has worked in most cases. We hope, but cannot promise, that the transplanted marrow will make healthy cells, and patients will not have sickle cell disease or severe thalassemia anymore. We do not know what effect this treatment will have on the damage that has already been done by the disease. Finding that out is the main reason for this study. Currently, very little has been reported about organ function after bone marrow transplants in patients with sickle cell anemia. | Sickle Cell Disease|Hemoglobin SC | ALL | CHILD, ADULT | Methodist Hospital, Houston, Texas, 77030, United States|Texas Children's Hospital, Houston, Texas, 77030, United States |
| Intranasal Ketamine for Pain Control in Patients with Sickle Cell Disease and Vaso-occlusive Episode (VOE) in the PED | This will be a descriptive cohort study of intranasal ketamine as the initial analgesic for children with sickle cell disease who present to the pediatric emergency department with vaso-occlusive crisis and are awaiting intravenous line placement. | Sickle Cell Disease|Vaso-Occlusive Pain Episode in Sickle Cell Disease|Vaso-occlusive Crisis|Ketamine|Intranasal Ketamine|Analgesia | ALL | CHILD, ADULT | Jacobi Medical Center, Bronx, New York, 10461, United States|JACOBI, Bronx, New York, 10461, United States |
| Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases | The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD. | Primary Immune Deficiency Disorders|Hemophagocytic Lymphohistiocytosis|Inherited Bone Marrow Failure Syndrome|Hemoglobinopathies|Metabolic Disorders | ALL | CHILD, ADULT | Fred Hutchinson Cancer ResearchCenter, Seattle, Washington, 98109, United States |
| Validation of a Predictive Score of Acute Chest Syndrome | Vaso-Occlusive Crisis (VOC), the most common manifestation of sickle cell disease (SCD), is the first cause of death, particularly when complicated by an acute chest syndrome (ACS). The PRESEV score could help the physicians to better manage VOC and could be used for future therapeutic trials. This predictive score of secondary ACS has to be validated in a multicenter international study. | Vaso Occlusive Crisis|Acute Chest Syndrome | ALL | CHILD, ADULT, OLDER_ADULT | Henri Mondor Hospital, Creteil, 94000, France |
| Screening for Hemoglobinopathies in Pregnant Women | This prospective monocentric study project is to identify hemoglobinopathies in pregnant women in order to optimize antenatal care and to investigate the prevalence of hemoglobinopathies in pregnant women in Switzerland. | Hemoglobinopathies | FEMALE | CHILD, ADULT, OLDER_ADULT | Frauenklinik University Hospital Basel, Basel, 4031, Switzerland |
| Efficacy and Safety of Bosentan in Sickle Cell Disease (SCD) Patients With Pulmonary Arterial Hypertension (PAH) | The study will assess the effect of bosentan on pulmonary vascular resistance and exercise capacity in sickle cell disease (SCD) patients diagnosed with pulmonary arterial hypertension. It consists of 3 phases: Screening, Treatment and Follow-up. During the Screening visit, the study doctor will decide if patients meet the study requirements. All potential patients will have a diagnosis of increased pulmonary artery pressures that is shown by right heart catheterization conducted shortly prior to start of study treatment. Patients will be asked to perform exercise capacity test (walking as far as possible for 6 minutes). Following the Baseline visit, the treatment phase consists of 4 additional clinic visits during which the good and bad effects of the drug are reviewed and exercise capacity test will be repeated. Patients will be treated for 16 weeks. Blood samples will be collected every month, or more often, if needed. At the end of the study, patients will be asked to repeat the right heart catheterization and exercise capacity test. After completion of the study, patients will have the option of enrolling in a long-term follow-up study where all patients will receive active drug. Patients electing not to participate in the extension study will be followed up for safety assessments for about 28 days after the end of the study treatment. | Pulmonary Hypertension | ALL | CHILD, ADULT, OLDER_ADULT | University of Alabama, Birmingham, Alabama, 35294, United States|Alta Bates Medical Center, Berkeley, California, 94705, United States|Harbor -UCLA Medical Center, Torrance, California, 90502, United States|University of Colorado Health Sciences Center, Denver, Colorado, 80262, United States|Howard University Hospital, Washington, District of Columbia, 20060, United States|University of Illinois Medical Center, Chicago, Illinois, 60612, United States|National Institutes of Health, Bethesda, Maryland, 20892, United States|Boston Medical Center/Boston University School of Medicine, Boston, Massachusetts, 02118, United States|Harper University Hospital/Wayne State University, Detroit, Michigan, 48201, United States|Henry Ford Hospital; Dept. of Pulmonology, Detroit, Michigan, 48202, United States|SoLUtions/Saint Louis University, St. Louis, Missouri, 63104, United States|Columbia University Medical Center; Pediatric Cardiology, New York, New York, 10032, United States|UNC Comprehensive Sickle Cell Program, Chapel Hill, North Carolina, 27599, United States|Duke University Medical Center; Duke University Health Systems, Durham, North Carolina, 27710, United States|University Hospitals of Ohio, Cleveland, Ohio, 44106, United States|Ohio State University, Columbus, Ohio, 43210, United States|Temple University Lung Center, Philadelphia, Pennsylvania, 19140, United States|University of Tennessee Health Science Center, Memphis, Tennessee, 38163, United States|The Methodist Hospital/Baylor College of Medicine; Pulmonary and Critical Care Medicine, Houston, Texas, 77030, United States|University of Texas Medical School; Division of Pulmonary and Critical Care Medicine, Houston, Texas, 77030, United States|Virginia Commonwealth University Medical Center, Richmond, Virginia, 23298-5028, United States|CHU de Fort de France, Fort de France, La Martinique, 97200, France|Hopital Antoine Beclere, Clamart, 92141, France|CHU Henri Mondor, Creteil, 94010, France|Amsterdam Medical Center, Department of Hematology, Amsterdam, 1105 AZ, Netherlands|Royal Free Hospital, Rheumatology Department, London, NW3 2QG, United Kingdom|Royal Hallamshire Hospital, Pulmonary Vascular Medicine, Sheffield, S10 2JF, United Kingdom |
| Designing an Implementation Strategy for Delivering Routine Mental Health Screening and Treatment | African Americans living with chronic health conditions are more likely to experience depression and other mental health disorders than their healthy counterparts, and are more likely to experience severe depression than whites, but less likely to be diagnosed or receive treatment. One especially vulnerable group is patients with sickle cell disease (SCD), a genetic blood disorder that primarily affects people of African descent, many of whom live in disadvantaged circumstances and are cared for in under-resourced settings. SCD causes severe acute and chronic pain, end-organ damage, and early mortality. Patients transitioning from adolescence to adulthood (ages16-30) are at high risk for mental health disorders and suicide. Using mobile technology, the investigators can provide high-quality, evidence-based behavioral mental health treatment that reaches patients in different settings. Digital cognitive behavioral therapy (CBT) is effective for treating depression and anxiety and can be brought to scale at low cost. Despite the promise of digital CBT, there are barriers to its widespread use, particularly in low-resource settings serving minorities. Qualitative data show that cultural factors-lack of relatability, representation, and perceived stigma regarding mental health treatment-limit engagement with digital CBT programs. Population-and setting-specific adaptations to interventions can lead to their successful implementation and wider use. The investigators will work with a digital CBT program to decrease stigma and make it more relatable and relevant to young adults with SCD, by devising changes to advertising and promotion, and tailoring communication with an integrated health coach, Aim 1: Use implementation science (ImS) and human-centered design methods to define the barriers to delivering routine mental health screening and digital CBT to adolescents and young adults with SCD. Aim 2: Rapidly iterate, test, and evaluate adaptations to the implementation strategy for a coach-enhanced digital mental health service. Aim 3: Demonstrate that a population-specific implementation strategy improves engagement with a digital CBT-based mental health service. The investigators will capitalize on our mobile technology tools, interdisciplinary expertise, and community-based partnerships to investigate the implementation of digital CBT into low-resource clinics and community-based organizations serving adolescents and adults with sickle cell disease. | Sickle Cell Disease|Depression|Anxiety | ALL | CHILD, ADULT | University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, 15213, United States |
| A Long-term Follow-up Study in Participants Who Received CTX001 | This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161 (NCT05477563) (transfusion-dependent β-thalassemia \[TDT\] studies) or Study 121 (NCT03745287) or 151 (NCT05329649), 161(NCT05477563),171 (NCT05951205) (severe sickle cell disease \[SCD\] studies). | Beta-Thalassemia|Thalassemia|Sickle Cell Disease|Hematologic Diseases|Hemoglobinopathies|Genetic Diseases, Inborn|Sickle Cell Anemia | ALL | CHILD, ADULT, OLDER_ADULT | Lucile Packard Children's Hospital, Palo Alto, California, 94304, United States|Ann & Robert Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States|Columbia University Medical Center (21+ years), New York, New York, 10032, United States|Columbia University Medical Center, New York, New York, 10032, United States|Atrium Health Levine Children's Hospital, Charlotte, North Carolina, 28203, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers, Nashville, Tennessee, 37203, United States|Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's Hospital, San Antonio, Texas, 78229, United States|Hopital Universitaire des Enfants Reine Fabiola (HUDERF), Brussels, Belgium|The Hospital for Sick Children, Toronto, Canada|Toronto General Hospital, University Health Network, Toronto, Canada|St. Paul's Hospital, Vancouver, Canada|University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology, Dusseldorf, Germany|Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine, Regensburg, Germany|University Hospital Tuebingen, Tuebingen, Germany|Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Ospedale Pediatrico Bambino Gesu - IRCCS, Rome, Italy|Imperial College Healthcare NHS Trust, Hammersmith Hospital, London, United Kingdom|University College London Hospitals NHS Foundation Trust, London, United Kingdom |
| A Study of the Safety, Blood Levels and Biological Effects of GBT440 in Healthy Subjects and Subjects With Sickle Cell Disease | The purpose of this study is to assess the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of GBT440 compared with placebo in healthy subjects and subjects with sickle cell disease (SCD). | Healthy Subjects|Sickle Cell Disease | ALL | ADULT | Guy's Hospital, London, SE1 9RT, United Kingdom |
| Low Dose Aspirin for Preventing Intrauterine Growth Restriction and Preeclampsia in Sickle Cell Pregnancy (PIPSICKLE) | Pregnancy in sickle cell disease (SCD) is fraught with many complications including preeclampsia (PE) and intrauterine growth restriction (IUGR). Previously, the investigators found an abnormality in prostacyclin-thromboxane ratio in sickle cell pregnant women, a situation that is also found in non-sickle pregnancies with PE and unexplained IUGR. Low dose aspirin (LDA) has been found to reduce the incidence of PE and IUGR in high-risk women due to its reduction of vasoconstrictor thromboxane whilst sparing prostacyclin, in effect "correcting" the ratio. It has been found to be safe for use in pregnancy and is recommended in obstetric guidelines for this use but has not been tested in sickle cell pregnancy. The investigators hypothesize that LDA would reduce the incidence of IUGR and PE in pregnant haemoglobin (Hb)SS women. The investigators also plan to build a machine-learning model to predict severe maternal outcomes in them. The investigators propose a multi-site, randomized, controlled, double blind trial comparing a daily dose of 100mg aspirin with placebo, from 12 - 28 weeks gestation until 36 weeks. The study sites are three teaching hospitals in Lagos and Ile-Ife, and twelve general hospitals and one federal medical centre within Lagos state, with the coordinating centre at the College of Medicine, University of Lagos (CMUL), Idi-Araba, Lagos. A total of 476 eligible pregnant HbSS and HbSC women will be recruited consecutively and randomly assigned to either group using a web-based app, sealed envelope. Each study group will comprise 238 pregnant women with SCD. All participants will be followed from recruitment till six weeks postpartum. They will have their body weight, blood pressure and haematocrit checked at each antenatal visit. Their full blood count, vital signs and oxygen saturation will be checked and recorded at each visit. Primary outcome measure will be birth weight below 10th centile for gestational age on INTERGROWTH 21 birthweight charts, and incidence of miscarriage or perinatal death. Analysis will be by intention to treat, and the main treatment effects will be quantified by relative risk with 95% confidence intervals, at a 5% significance level. The investigators plan to develop a prediction model to predict the risk of complications in these women using machine learning. The prediction outcome will be severe maternal outcomes comprising maternal near miss or death. | Intrauterine Growth Restriction|Preeclampsia|Sickle Cell Disease|Pregnancy Complications | FEMALE | ADULT, OLDER_ADULT | Ajeromi General Hospital, Ajegunle, Lagos State, Ajegunle, Lagos, Nigeria|Federal Medical Centre, Ebute Metta, Lagos State, Ebute-Metta, Lagos, Nigeria|Lagos University Teaching Hospital, Idi Araba, Lagos, 100254, Nigeria|Alimosho General Hospital, Igando, Lagos State, Igando, Lagos, Nigeria|Lagos State University Teaching Hospital (LASUTH, Ikeja, Lagos, Nigeria|General Hospital, Ikorodu, Lagos State, Ikorodu, Lagos, Nigeria|General Hospital, Isolo, Lagos State, Isolo, Lagos, Nigeria|General Hospital, Somolu, Lagos State, Somolu, Lagos, Nigeria|Randle General Hospital, Surulere, Lagos State, Suru Lere, Lagos, Nigeria|Obafemi Awolowo University Teaching Hospital OAUTH), Ife, Osun State, Ife, Osun, Nigeria|Lagos Island Maternity Hospital, Lagos, Lagos, 101001, Nigeria|General Hospital, Gbagada, Lagos State, Lagos, Nigeria|General Hospital, Ibeju-Lekki, Lagos State, Lagos, Nigeria|General Hospital, Ifako Ijaiye, Lagos State, Lagos, Nigeria|General Hospital, Orile-Agege, Lagos State, Lagos, Nigeria|Mother and Child Centre, Amuwo-Odofin, Lagos, Lagos, Nigeria |
| A Study of Patients Having Pulmonary Hypertension Associated With Sickle Cell Disease and Completing an ASSET Study | This study will assess the safety and efficacy of bosentan therapy (in a study known as ASSET) for patients who have high blood pressure in the lungs associated with sickle cell disease. That form of hypertension places people at risk for complications, including shortness of breath, pain, pneumonia, and death. Previous studies have shown that bosentan can be helpful in reducing pulmonary hypertension. Patients ages 16 and older who have completed the 16-week treatment in the ASSET 1 or ASSET 2 study and who are not pregnant or breastfeeding may be eligible for this study. The research will be conducted in about 25 hospitals in the United States and Europe. Up to 30 participants will be enrolled. The screening visit will involve a physical examination, blood sample of about 3 teaspoons for laboratory tests, and a pregnancy test. Patients' doctors will give them bosentan tablets (62.5 mg each), to take one in the morning and one in the evening. After 1 month, patients will be told whether the dose should be increased to 125 mg tablets to take twice a day. Two weeks after the increase in dose, a blood test will be done to analyze the drug's effects on the liver. After the start of treatment, patients will return for visits every 6 months, when there will be a 6-minute walking test to measure exercise capacity and evaluate shortness of breath. There will be follow-up for patients up to the end of the study and for 28 days after the last dose of bosentan is taken, to collect information about side effects. Some patients on bosentan have had changes in liver function and red blood cell count. Side effects commonly reported are headache, flushed appearance, inflammation of the throat and nasal passages, and gastrointestinal symptoms. If patients have sudden worsening in breathing in the first few weeks after taking bosentan, they should immediately tell their doctors, because it may be necessary to change the treatment. | Pulmonary Hypertension|Sickle Cell Anemia | ALL | CHILD, ADULT, OLDER_ADULT | University of Alabama, Birmingham, Alabama, 35294, United States|University of California, Los Angeles, Los Angeles, California, 90095, United States|University of Colorado, Denver, Colorado, 80220-3706, United States|University of Illinois, Chicago, Illinois, 60612, United States|University of Kansas, Kansas City, Kansas, 66160, United States|National Heart, Lung and Blood Institute (NHLBI), 9000 Rockville Pike, Bethesda, Maryland, 20892, United States|Boston University School of medicine, Boston, Massachusetts, 02118-2354, United States|Wayne State University Hutzel Hospital, Detroit, Michigan, 48201, United States|Henry Ford Health Systems, Detroit, Michigan, 48202, United States|St. Louis University, St. Louis, Missouri, 63104, United States|Albert Einstein College of Medicine, Bronx, New York, 10461, United States|Columbia University, New York, New York, 10032-3784, United States|University of North Carolina, Chapel Hill, North Carolina, 27599-7030, United States|Duke University, Durham, North Carolina, 27710, United States|University Hospitals of Ohio, Cleveland, Ohio, 44106, United States|Ohio State University, Columbus, Ohio, 43210-1240, United States|Thomas Jefferson University, Philadelphia, Pennsylvania, 19107-6541, United States|Temple University, Philadelphia, Pennsylvania, 19140, United States|University of Tennessee, Memphis, Tennessee, 38163, United States|Baylor College of Medicine, Houston, Texas, 77030, United States|University of Texas, Houston, Houston, Texas, 77030, United States|Virginia Commonwealth University Medical Center, Richmond, Virginia, 23298, United States|Amsterdam Medical Center, Amsterdam, Netherlands|Royal Free Hospital, London, United Kingdom |
| The BENeFiTS Trial in Beta Thalassemia Intermedia | Beta-thalassemias and hemoglobinopathies are serious inherited blood diseases caused by abnormal or deficiency of beta A chains of hemoglobin, the protein in red blood cells which delivers oxygen throughout the body.The diseases are characterized by hemolytic anemia, organ damage, and early mortality without treatment. Increases in another type of (normal) hemoglobin, fetal globin (HbF), which is normally silenced in infancy, reduces anemia and morbidity. Even incremental augmentation of fetal globin is established to reduce red blood cell pathology, anemia, certain complications, and to improve survival. This trial will evaluate an oral drug discovered in a high throughput screen, which increases fetal globin protein (HbF and red blood cells expressing HbF)and messenger ribonucleic acid (mRNA) to high levels in anemic nonhuman primates and in transgenic mice. The study drug acts by suppressing 4 repressors of the fetal globin gene promoter in progenitor cells from patients. The drug has been used for 50 years in a combination product for different actions - to enhance half-life and reduce side effects of a different active drug- and is considered safe for long-term use. This trial will first evaluate 3 dose levels in small cohorts of nontransfused patients with beta thalassemia intermedia. The most active dose will then be evaluated in larger subject groups with beta thalassemia and other hemoglobinopathies, such as sickle cell disease. | Beta Thalassemia Intermedia|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | UCSF Benioff Children's Hospital at Oakland, Oakland, California, 94609, United States|Massachusetts General Hospital, Boston, Massachusetts, 02114, United States|Susan Perrine, Weston, Massachusetts, 02493, United States|Weil Cornell Medicine, New York, New York, 10065, United States|University Health Network and Toronto General Hospital, Toronto, Ontario, M5G2C4, Canada |
| A SAD/MAD to Assess the Safety, PK/PD of FT-4202 in Healthy Volunteers and Sickle Cell Disease Patients | FT-4202 is an oral small-molecule agonist of pyruvate kinase red blood cell isozyme (PKR) being developed for the treatment of hemolytic anemias. This initial study will characterize the safety, tolerability and the pharmacokinetics/pharmacodynamics (PK/PD) of a single ascending dose and multiple ascending doses of FT-4202 in the context of Phase 1 studies in healthy volunteers and sickle cell disease patients. The effects of food on the absorption of FT-4202 will also be evaluated in healthy volunteers. | Healthy Volunteers|Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Woodland International Research Group (SCD subjects only), Little Rock, Arkansas, 72211, United States|Collaborative Neuroscience Research, LLC (SCD subjects only), Long Beach, California, 90806, United States|Pacific Research Partners (SCD subjects only), Oakland, California, 94607, United States|UCSF Benioff Children's Hospital Oakland (SCD subjects only), Oakland, California, 94609, United States|Advanced Pharma CR, LLC (SCD subjects only), Miami, Florida, 33147, United States|Children's Healthcare of Atlanta (SCD subjects only), Atlanta, Georgia, 30342, United States|Augusta University Medical Center (SCD subjects only), Augusta, Georgia, 30912, United States|University of Illinois at Chicago (SCD subjects only), Chicago, Illinois, 60612, United States|University of Maryland, Greenebaum Comprehensive Cancer Center (SCD subjects only), Baltimore, Maryland, 21201, United States|Columbia University Medical Center (SCD subjects only), New York, New York, 10032, United States|Levine Cancer Institute (SCD subjects only), Charlotte, North Carolina, 28204, United States|Duke University Medical Center (SCD subjects only), Durham, North Carolina, 27710, United States|University of Cincinnati Medical Center (SCD subjects only), Cincinnati, Ohio, 45219, United States|Medpace Clinical Pharmacology Unit (Healthy Volunteers only), Cincinnati, Ohio, 45227, United States|Cincinnati Children's Hospital Medical Center (SCD subjects only), Cincinnati, Ohio, 45229, United States|Lynn Institute of Tulsa (SCD subjects only), Tulsa, Oklahoma, 74135, United States|St. Jude Children's Research Hospital (SCD subjects only), Memphis, Tennessee, 38105, United States|The University of Texas Health Science Center at Houston (SCD subjects only), Houston, Texas, 77030, United States |
| Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis | This study consists of two parts: phase 2 (Part A) and phase 3 (Part B). It is a multicenter study designed to evaluate the safety, effectiveness, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with sickle cell disease (SCD) experiencing vaso-occlusive crises (VOC). The main objectives of the study are to assess how CSL889 affects the time it takes for VOC to resolve in participants with SCD, and to evaluate the safety and tolerability of CSL889 in study participants. | Sickle Cell Disease Vaso-occlusive Crisis | ALL | CHILD, ADULT, OLDER_ADULT | |
| Promoting Utilization and Safety of Hydroxyurea Using Precision in Africa | Sickle cell anemia (SCA) is among the world's most common and devastating blood disorders, affecting more than 300,000 newborns per year. Most infants with SCA are born in the low-resource settings of sub- Saharan Africa, where an estimated 50-90% will die before 5 years of age due to lack of early diagnosis and appropriate care. Hydroxyurea is a safe and effective once-daily oral medication that has become the standard of care for the treatment of children with SCA in high-resource settings. There is now a growing body of evidence to support the safety and clinical benefits of hydroxyurea for the treatment of SCA in sub-Saharan Africa. The requirement for frequent laboratory monitoring, uncertainties about appropriate, most effective dosing, and the concern for hematologic laboratory toxicities, however, will continue to limit widespread hydroxyurea utilization and real-world effectiveness. The investigators have recently developed and prospectively evaluated an individualized, pharmacokinetics-guided hydroxyurea dosing strategy for children with SCA that has demonstrated optimal clinical and laboratory benefits with minimal toxicity. In this research study, the investigators aim to extend this precision medicine approach to Africa. | Sickle Cell Anemia in Children|Sickle Cell Disease | ALL | CHILD | Hospital Geral dos Cajueiros, Luanda, Angola |
| Pathophysiological Explorations of Red Blood Cells | GR-Ex is a program labelled by Labex (Laboratory of Excellence) by the French Ministry of Higher Education and Research. This program aims to develop the means to improve knowledge in the physiology and pathologies of erythropoiesis, red blood cells and iron metabolism, and to develop new therapeutic protocols capable of providing added value in terms of innovation. | Red Blood Cell Disorder | ALL | CHILD, ADULT, OLDER_ADULT | Hopital Necker - Enfants malades, Paris, France |
| Cerebral Oxygen Metabolism in Children | The purpose of this research study is to better understand how blood flow and metabolism change can influence brain development in the early decades of life. We will examine brain blood flow and metabolism using magnetic resonance imaging (MRI). The brain's blood vessels expand and constrict to regulate blood flow based on the brain's needs. The amount of expanding and contracting the blood vessels can do varies by age. The brain's blood flow changes in small ways during everyday activities, such as normal brain growth, exercise, or deep concentration. Significant illness or psychological stress may increase the brain's metabolic demand or cause other bigger changes in blood flow. If blood vessels are not able to expand to give more blood flow when metabolic demand is high, the brain may not get all of the oxygen it needs. In extreme circumstances, if the brain is unable to get enough oxygen for a long time, a stroke may occur. Sometimes small strokes occur without other noticeable changes and are only detectable on an MRI. These are sometimes called "silent strokes." In less extreme circumstances, not having as much oxygen as it wants may cause the brain to grow and develop more slowly than it should. One way to test the ability of blood vessels to expand is by measuring blood flow while breathing in carbon dioxide. Carbon dioxide causes blood vessels in the brain to dilate without increasing brain metabolism. During this study participants may be asked to undergo a blood draw, MRI, and potential neuropsychological assessments. It is also possible that the study team will use a special mask to control the amount of carbon dioxide the participants breathe in so they don't breathe in too much. | Sickle Cell Disease|Cerebral Stroke | ALL | CHILD, ADULT | Washington University of St. Louis, Saint Louis, Missouri, 63110, United States |
| Genes Influencing Iron Overload State | Iron overload, which can be defined operationally as too much iron in the body, develops as a consequence of too many blood transfusions given, or due to genetic defects hereditary hemochromatosis). Iron accumulates in several organs in the body, such as the heart, liver, endocrine glands (pancreas, thyroid, etc.), and spleen. Excessive iron can damage organs and may even cause death. Iron overload needs to be appropriately monitored and treated to avoid unnecessary morbidity and mortality. The present study, GENIOS, proposes to test prospectively the hypothesis that genetic modifiers influence the iron overload status of patients receiving transfusions. To test this hypothesis, the study will perform genetic studies to investigate possible genetic influences for iron accumulation in the body and will study iron accumulation not only in the liver, but also in the heart, pancreas, kidneys, and spleen. In addition: the study will investigate if these same genes have any role during treatment of iron overload, in other words, if certain genetic mutations will influence how iron exits the body. This study will also investigate how substances that are known to control the trafficking of iron in and out of the body and its damaging effects to the tissues (hepcidin and non transferrin-bound iron) are linked to the accumulation of iron in the heart and liver. Iron in the body will be measured by R2\*MRI and no liver biopsies will be required. Genetic studies will be done by specialized tests using peripheral blood DNA. Iron accumulates differently in different people and in different organs of the body. Some people accumulate iron faster than others, even when receiving the same number of blood transfusions | Sickle Cell Disease|Thalassemia|Marrow Aplasia | ALL | CHILD, ADULT, OLDER_ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Realizing Effectiveness Across Continents With Hydroxyurea | REACH is a prospective, phase I/II open-label dose escalation trial of hydroxyurea for children with confirmed SCA between 3 and 10 years of age. The short-term goal is to obtain critical pilot data regarding the feasibility, safety, and benefit of hydroxyurea for children with SCA in multiple distinct research settings in Africa. Based on that information, the longer-term goal is to make hydroxyurea more widely available for children with SCA in Africa, particularly those identified with SCA through expanded newborn screening programs. | Sickle Cell Disease|Children | ALL | CHILD | Hospital Pediátrico David Bernardino, Luanda, Angola|Centre Hospitalier Monkole, Kinshasa, Congo, The Democratic Republic of the|KEMRI/Wellcome Trust Research, Kilifi, Kenya|Mbale Regional Hospital, Mbale, Uganda |
| A Single-Dose Relative Bioavailability Study Of GBT440 300 mg Capsules in Healthy Subjects | The purpose of this study is to evaluate the relative bioavailability of a single 300 mg dose of GBT440 administered as a high strength (1 × 300 mg) capsule versus a low strength (3 × 100 mg) capsule formulation in healthy fasted subjects. | Sickle Cell Disease | ALL | ADULT | ICON Early Phase Services, LLC Clinical Research Unit, San Antonio, Texas, 78209, United States |
| A Trial for Prevention of Recurrent Ischemic Priapism in Men With Sickle Cell Disease: A Pilot Study | To conduct a randomized controlled internal pilot feasibility trial for the prevention of recurrent ischemic priapism referred to as the Priapism in Nigeria (PIN) trial. The study team will enroll a minimum of 30 participants and a maximum of 200 participants. Study investigators hypothesize that hydroxyurea therapy combined with tadalafil is superior to a combination of hydroxyurea and placebo in the prevention of recurrent ischemic priapism. | Priapism Due to Sickle Cell Disease | MALE | ADULT | Aminu Kano Teaching Hospital, Kano, Nigeria|Murtala Mohammed Specialist Hospital, Kano, Nigeria |
| Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias | This is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more information about the safety and efficacy of deferiprone in patients with sickle cell disease or other anemias who suffer from iron overload caused by regular blood transfusions. | Iron Overload|Sickle Cell Disease|Other Anemias | ALL | CHILD, ADULT, OLDER_ADULT | UCSF Benioff Children's Hospital Oakland, Oakland, California, 94609, United States|University of Michigan Comprehensive Cancer Center, Ann Arbor, Michigan, 48109, United States|Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|The Children's Hospital of Philadephia, Philadelphia, Pennsylvania, 19104-4399, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|Hospital for Sick Kids, Toronto, Ontario, Canada|Zagazig University, Alexandria, Egypt|Ain Shams University, Cairo, Egypt|Cairo University, Cairo, Egypt|Pediatric Hospital of Cairo University, Cairo, Egypt|Asser Central Hospital, Abha, Saudi Arabia|Barts and The London, London, United Kingdom|Evelina Children's Hospital, London, United Kingdom |
| A Pharmacokinetic (PK) and Pharmacodynamic (PD) Dose-ranging Phase II Study of Ticagrelor in Paediatric Patients With Sickle Cell Disease | The purpose of this Phase II dose-ranging study is to investigate pharmacokinetic (PK) and pharmacodynamic (PD) properties of various doses of ticagrelor followed by 4 weeks of twice-daily treatment in paediatric patients with sickle cell disease | Investigation of Platelet Aggregation in Paediatric Patients With Sickle Cell Disease | ALL | CHILD | Research Site, Orange, California, 92868, United States|Research Site, Chicago, Illinois, 60612, United States|Research Site, Detroit, Michigan, 48201, United States|Research Site, Hershey, Pennsylvania, 17022, United States|Research Site, Philadelphia, Pennsylvania, 19104, United States|Research Site, Charleston, South Carolina, 29425, United States|Research Site, Toronto, Ontario, M5G 1X8, Canada|Research Site, Kisian, 100, Kenya|Research Site, Nairobi, Kenya|Research Site, Beirut, 1107 2020, Lebanon|Research Site, Beirut, 113-6044, Lebanon|Research Site, Tripoli, 1434, Lebanon|Research Site, Parow, 7500, South Africa|Research Site, Rondebosch, 7700, South Africa|Research Site, Cardiff, CF4 4XN, United Kingdom|Research Site, London, E1 1BB, United Kingdom|Research Site, London, SE1 7EH, United Kingdom|Research Site, Manchester, M13 9PT, United Kingdom |
| Nitric Oxide and Transfusion Therapy for Sickle Cell Patients With Pulmonary Hypertension | This study will test whether inhaling nitric oxide (NO) gas mixed with room air can improve pulmonary hypertension (high blood pressure in the lungs) in patients with sickle cell anemia. Patients with sickle cell disease 18 years of age or older may be eligible to participate in one or more parts of this three-stage study, as follows: Stage 1 Patients undergo the following tests to determine the cause of their pulmonary hypertension: blood tests; echocardiogram (heart ultrasound); asthma test; oxygen breathing study with measurement of arterial blood oxygen levels; chest X-ray; lung scans; MRI of the heart; 6-minute walk test; night-time oxygen measurement while sleeping; and exercise studies. Stage 2 Patients have a detailed MRI evaluation of the heart and are admitted to the NIH Clinical Center intensive care unit (ICU) for the following test: A plastic tube is placed in a vein in the patient's arm and another tube is placed in a deeper neck or leg vein. A third tube is inserted through the vein into the heart and the lung artery to measure blood pressures in the heart and lungs directly. Following baseline measurements, three medications (inhaled oxygen, infused prostaglandin, and inhaled NO) are delivered for 2 hours each, separated by a 30-minute washout period. A small blood sample is drawn during the NO administration. Patients who cannot be treated with nitric oxide or for whom the treatment does not work may receive monthly exchange transfusions for 3 months. For this procedure, 3 to 5 five units of the patient's blood is removed and replaced with 3 to 5 units that do not have sickle hemoglobin. Some patients who do not respond to NO or exchange transfusions may receive an alternative therapy, such as oxygen, prostacyclin, L-arginine, bosentan or sidenafil. Stage 3 Patients remain in the ICU with catheters in place for another 24 hours. During this time they breathe NO. Lung pressures are measured every 4 hours and blood is drawn every 8 hours. They then stay in the hospital 1 more day for observation. Patients then breathe nitric oxide continuously for 2 months using a tank of gas that delivers the NO through tubes placed in the nose. They may do this at home on an outpatient basis or may remain in the hospital for the 2 months. Patients have an echocardiogram and blood tests every week and do a 6-minute walk test every 2 weeks.... | Sickle Cell Anemia|Pulmonary Hypertension | ALL | ADULT, OLDER_ADULT | Suburban Hospital, Bethesda, Maryland, 20814, United States|National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| A Comprehensive Care Plan for Pediatric Patients With Vaso-Occlusive Crises | Sickle cell disease (SCD) is the most common inherited blood disorder affecting 80,000 to 90,000 individuals in the United States.\[10\] There are 13,000 hospital admissions for a sickle cell crises, costing $448 million dollars annually.\[10\] In our hospital, the sickle cell population is known to have some of the longest length of stays. Between October 2014 and September 2015, there were 89 admissions for a vaso-occlusive crisis with an average length of stay of 6 days and 12 admissions greater than 10 days and 5 admissions greater than 20 days. We propose to evaluate the feasibility of the new CPP in a pilot randomized control trial to determine if pain and length of stay can be reduced in patients with sickle cell disease. We also propose to evaluate a sleep regimen to determine if this can reduce the hospital stay and help with pain. We hypothesize increased physical activity and proper sleep, as implemented in the CPP, are correlated with decreased hospital length of stay and decreased pain. Additionally, we believe that creating a standardized nighttime environment at the hospital will help the children stay in their circadian rhythm thus promoting improved sleep and a more effective inpatient disease management. | Sickle Cell Disease|Sickle Cell Crisis|Vaso-Occlusive Crises | ALL | CHILD, ADULT | Phoenix Children's Hospital, Phoenix, Arizona, 85016, United States |
| Immunogenicity and Safety Study of Pneumococcal 7-Valent Conjugate Vaccine in Sickle Cell Disease Infants. | The primary objectives of this study were to assess the immunogenicity and the tolerance of the heptavalent pneumococcal conjugate vaccine (Prevenar) in young infants (2 months of age) with sickle cell disease when administred at 2,3, and 4 months of age. | Pneumococcal Infections | ALL | CHILD | Paris, 75019, France |
| Hydoxyurea Exposure in Lactation A Pharmacokinetics Study (HELPS) | To examine the pharmacokinetics and distribution of oral hydroxyurea when administered as a single dose to lactating women | Sickle Cell Anemia | FEMALE | ADULT, OLDER_ADULT | Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States |
| Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies | CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells. The overall study objectives are to evaluate the safety and efficacy of CordIn™. | Sickle Cell Disease|Thalassemia | ALL | CHILD, ADULT | UCSF Benioff Children's Hospital, Oakland, California, 94609, United States|Children's National, Washington, District of Columbia, 20010, United States|Hôpital Robert Debré, Paris, France |
| AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion | Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion. All other treatment is standard of care. | Acute Leukemia|Severe Aplastic Anemia|Non-hodgkin Lymphoma|Hodgkin Lymphoma|Kostmann|Diamond Blackfan Anemia|Amegakaryocytic Thrombocytopenia|Sickle Cell Disease|Beta-Thalassemia | ALL | CHILD, ADULT | New York Medical College, Valhalla, New York, 10595, United States |
| Preliminary Feasibility and Efficacy of Behavioral Intervention to Reduce Pain-Related Disability in Pediatric SCD | Pain is the primary complication of sickle cell disease (SCD), including vaso-occlusive crises and more persistent, chronic pain. SCD-related pain is associated with significant functional impairment, spanning poor school attendance, decreased quality of life, and stress and mood difficulties. Pharmacological approaches are the first-line treatment for SCD-related pain, but these can be costly and have unwanted side effects. Given limitations from pharmacological approaches and the influence that poor behavioral responses have on disease management and health outcomes suggest a critical need for alternative and adjunctive treatments. Due to gaps in available behavioral treatments specifically designed for addressing common challenges associated with pain management in pediatric SCD, the investigators developed a manualized behavioral therapy protocol by tailoring existing evidence-based treatments. The overall goal of the intervention is to reduce the impact of pain on daily functioning in pediatric SCD. This study will empirically test the feasibility and preliminary efficacy of this intervention for youth with SCD. Children and adolescents with SCD between the ages of 8 and 17 years old (n=20) will be recruited to complete the treatment protocol. Feasibility will be assessed by examining participation and program completion rates, as well as feedback from a treatment acceptability questionnaire and qualitative interview. Participants will complete baseline assessments, weekly questionnaires, and post-treatment assessments (post-intervention assessment, follow-up time points: 1-month following the intervention, and 3-months following the intervention). | Sickle Cell Disease|Pain | ALL | CHILD | Children's National Hospital, Washington, District of Columbia, 20010, United States |
| Use of Mobile Technology for Intensive Training in Medication Management | Purpose: Assess whether intensive training with education and daily remote monitoring with provider involvement has a lasting positive impact on adherence to medication management. The study will seek to enroll 25 subjects with sickle cell disease or thalassemia, and less than 100% compliance for taking iron chelators in the previous three month prior to participation in the study. Subjects will be asked to monitor their daily iron chelator administration by taking a video recording of preparing it and ingesting at least one sip. Subjects will also use a medication log to record daily administration of medication, and meet with study staff monthly for educational activities. The data collected will be analyzed to describe patient adherence and comfort level with the process of daily recording of medication management. Mean percent adherence in the pre-study periods and each of the study periods will be analyzed and compared. | Sickle Cell Disease|Thalassemia | ALL | CHILD, ADULT, OLDER_ADULT | Duke University Medical Center, Durham, North Carolina, 27710, United States |
| Stroke Prevention in Nigeria 2 Trial | The primary goal of this study is to complete a multicenter single-arm, type I hybrid trial to assess the effectiveness of hydroxyurea therapy for primary stroke prevention in high-risk children with sickle cell anemia (SCA) living in Nigeria in routine care settings. | Sickle Cell Disease|Stroke | ALL | CHILD | Aminu Kano Teaching Hospital, Kano, Nigeria|Murtala Muhammad Specialist Hospital, Kano, Nigeria |
| Medication Adherence and Non-adherence in Adults With Rare Disease | The purpose of this study is to use the Medication Adherence Reasons Scale (MAR-Scale) to determine the extent of non-adherence to specific medications indicated to treat cystic fibrosis, hemophilia (A or B), idiopathic pulmonary fibrosis, myasthenia gravis, and sickle cell disease, and to identify the top patient-reported reasons for non-adherence. Internal reliability of the MAR-Scale will also be assessed in each condition. | Cystic Fibrosis|Hemophilia A|Hemophilia B|Idiopathic Pulmonary Fibrosis|Myasthenia Gravis|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | |
| Hydroxyurea to Prevent Stroke in Children With Sickle Cell Anemia and Elevated TCD Flow Velocity | The purpose of this study is to assess prospectively the efficacy of hydroxyurea therapy in the setting of cerebrovascular disease, manifest as conditional or abnormal transcranial doppler ultrasonography (TCD) flow velocities, in children with sickle cell anemia (SCA). TCD is used to measure flow velocity in intracranial arteries as a marker of increased stroke risk in children with SCA. The primary objective of this protocol is to determine whether hydroxyurea reduces elevated TCD velocity. | Stroke | ALL | CHILD, ADULT | Duke University Medical Center, Durham, North Carolina, 27710, United States |
| A Relative Bioavailability Study of a Prasugrel Orally Disintegrating Tablet | This study compares the clinical tablet formulation of prasugrel taken orally with an orally disintegrating tablet (ODT) taken orally. The study will evaluate the amount of prasugrel active metabolite circulating in the blood for each treatment. | Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician., Honolulu, Hawaii, United States |
| CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation | This study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection, to assess event free survival and overall survival, to determine the time to neutrophil and platelet engraftment, to determine the time to immune reconstitution (including normalization of T, B and natural killer (NK) cell repertoire and Immunoglobulin G production), and to establish the incidence of infectious complications including bacterial, viral, fungal and atypical mycobacterial and other infections following CD34+ selection in children, adolescents and young adults receiving an allogeneic peripheral blood stem cell transplant from a family member or unrelated adult donor for a non-malignant disease. | Bone Marrow Failure Syndrome|Severe Aplastic Anemia|Severe Congenital Neutropenia|Amegakaryocytic Thrombocytopenia|Diamond-Blackfan Anemia|Schwachman Diamond Syndrome|Primary Immunodeficiency Syndromes|Acquired Immunodeficiency Syndromes|Histiocytic Syndrome|Familial Hemophagocytic Lymphocytosis|Lymphohistiocytosis|Macrophage Activation Syndrome|Langerhans Cell Histiocytosis (LCH)|Hemoglobinopathies|Sickle Cell Disease|Sickle Cell-beta-thalassemia | ALL | CHILD, ADULT | Morgan Stanley Children's Hospital, New York-Presbyterian, Columbia University, New York, New York, 10032, United States |
| A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease | This is a Phase 1/2, open label, safety, and efficacy study of the administration of LentiGlobin BB305 Drug Product to participants with either transfusion dependent beta-thalassemia (TDT) or sickle cell disease (SCD). | Beta-Thalassemia Major|Sickle Cell Disease | ALL | CHILD, ADULT | Paris, France |
| Transition From Paediatric Intensive Care to General Paediatrics and Pneumology Units : a Study of the Post-intensive Care Syndrome | In developed countries, mortality rates in pediatric intensive care units (PICUs) are around 4% and most children admitted to these units survive. However, some pediatric survivors experience long-term morbidity (cognitive, psychological and/or physical impairment) associated with their PICU stay and there is increasing awareness of the onset of post-intensive care syndromes (PICS) like in adults. However comprehensive descriptive data are still lacking regarding PICS in pediatrics (PICS-p). The aim of this study is to describe (in nature and frequency) the alterations in health defined by the WHO of children who have passed through the PICU and constitute a possible PICS-p. In order to do this, we will perform a prospective cohort study in Robert-Debré University Hospital including the PICU. We will include children with an unplanned hospitalization for more than 72 hours for acute complication of sickle cell disease (such as acute chest syndrome or vaso-occlusive crisis), acute asthma or sepsis and aged from 3 to 17 years. We plan to include 40 patients admitted to the PICU as well as 40 controls admitted to the general pediatrics unit or the pneumology unit without PICU admission, matched on diagnosis, age range and period. The primary endpoint will be the prevalence of children that had been admitted to PICU and reporting cognitive, psychological, physical and social impairments measured by questionnaire and medical record data collection on the day before discharge and at the routine post-hospitalization visit. Secondary objectives will be to study the risk factors for PICS-p, to compare alterations in cognitive, psychological, physical and social domains in children with the same diagnosis and age not admitted to the PICU during their hospitalization. In order to this, we will measure the association with the PICU stay characteristics, parental experience and social characteristics of families. We will also report the prevalence of children not admitted in intensive care and reporting cognitive, psychological, physical and social impairments measured by questionnaire and medical record data collection on the day before discharge and at the routine post-hospitalization visit. Patients and their parents will be given questionnaires the day before discharge and during the first follow-up consultation between 2 and 4 months after hospital discharge. Questionnaires will include the Pediatric Symptom Checklist long version (Assessment of cognitive, psychological and social domains - 35 items scored from 0 to 2) reported by parents for children under 8 years and by the patients for children older than 8 years. older), the physical items of Pediatric Quality of Life scale (8 items scored from 0 to 4) and a parent self-questionnaire (including relationship to child, annual income, household composition, understanding and use of the French language, highest diploma of mother and father, social support (""How many people can you really count on when you need help?"")) and a parental mental health self-assessment (PHQ-8). The analyses will be descriptive (description of the nature and frequency of alterations) and comparative (between children who have or have not been in PICU). Univariate tests will be performed to identify possible risk factors for post intensive care syndrome. Statistics will be carried out on SAS 9.4 software. | Pediatric Post-intensive Care Syndrome|Intensive Care Unit Syndrome | ALL | CHILD | Robert Debré Hospital, Paris, 75019, France |
| Hydroxyurea Therapy: Optimizing Access in Pediatric Populations Everywhere | Primary Objective 1. Define the pharmacokinetics of liquid-formulated HU in infants (9 months to \<2 years) 2. Assess the relative bioavailability of HU "sprinkles" compared to capsules in children and adolescents (≥2 to 18 years). Secondary Objective: Compare PK parameters in infants versus older children on this study and those from our previous "Pharmacokinetics and Bioavailability of a Liquid Formulation of Hydroxyurea in Pediatric Patients with Sickle Cell Anemia" (NCT01506544) trial. Exploratory Objectives: Capture information regarding the taste of HU sprinkles using palatability questionnaire. This trial is an open label, single center assessment of the pharmacokinetics of two formulations of hydroxyurea (HU) designed to (1) determine the pharmacokinetic profile of a liquid formulation in infants and to (2) determine the bioavailability of "sprinkles", a novel method of administration for older children. The study aims to generate data to facilitate FDA approval for HU in children and potentially validate a new mode of administration ("sprinkles") that will optimize access and adherence for children in the US and globally. | Sickle Cell Disease|Thalassemia | ALL | CHILD, ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Treatment of Sickle Cell Patients Hospitalized in Pain Crisis With Prophylactic Dose Low-molecular-weight Heparin (LMWH) Versus Placebo | Sickle cell disease (SCD) is one of the most common inherited diseases worldwide and exhibits highest frequency in people of African descent. Patients with SCD currently have few treatment options, with hydroxyurea being the only medication approved to reduce the frequency of vaso-occlusive crisis (VOC) and prevent other SCD complications such as acute chest syndrome. Once patients develop VOC, hospitalizations aim to alleviate pain; no specific therapy is currently available to otherwise affect the course of the VOC. However, there has been increasing interest in the role of coagulation in the pathogenesis of SCD. The investigators hypothesize that low dose anticoagulant therapy, such as prophylactic dose low-molecular-weight heparin (LMWH), could be a novel way to ameliorate the vaso-occlusive process and thereby hasten the resolution of pain. | Sickle Cell Disease|Vaso-occlusive Crisis | ALL | ADULT, OLDER_ADULT | Duke University, Durham, North Carolina, 27710, United States |
| Choosing Opioid Management for Pain and Analyzing Acute Chest Syndrome (ACS) Rates Equally | The pathophysiology of sickle cell disease (SCD) manifestations, are complex with interactions of intracellular hemoglobin, membrane and endothelial activation but the hallmark remains recurrent and painful vaso-occlusive episodes (VOC). These painful episodes are thought to result from ischemia caused when small blood vessels are occluded by misshapen, inflexible erythrocytes. Painful episodes are the most common cause of hospitalization, morbidity, and impairment for SCD patients. There is no therapy that completely prevents or directly aborts painful events for all patients. Consequently, treatment for acute VOC is primarily supportive using hydration and medicinal pain control. Every pain medication has the potential to relieve pain but is associated with significant limitations and side effects. The primary hypothesis to be tested in this double blind, randomized controlled trial is that Nalbuphine is equivalent to morphine for pain control and patients will suffer fewer episodes of acute chest syndrome. The investigators also expect subjects will report fewer side effects from respiratory depression, abdominal distention from reduced peristalsis, reduced histamine release causing pruritis and still be provided adequate pain control. Further hypotheses to be tested is ability to recruit patient participants while being treated in the Emergency Department and that continuous infusion of Nalbuphine with accompanying patient controlled analgesia (PCA) is safe and effective in controlling pain, requiring less total opiates consumption, while decreasing length of hospitalization. | Pain|Sickle Cell Disease | ALL | CHILD, ADULT | Children's Healthcare of Atlanta, Atlanta, Georgia, 30303, United States |
| IMPACTS Trial: Investigation of the Modulation of Phospholipase in Acute Chest Syndrome | The study will be conducted at 15-20 US centers in a randomized, placebo-controlled, double-blind fashion. Enrollees will be hospitalized sickle cell disease (SCD) patients at-risk for acute chest syndrome (ACS) based on the presence of vaso-occlusive crisis (VOC), fever (T ≥38.0°C) and serum sPLA2 concentration ≥50 ng/mL. | Sickle Cell Disease|Vaso-occlusive Crisis|Acute Chest Syndrome | ALL | CHILD, ADULT, OLDER_ADULT | Howard University Hospital, Washington, District of Columbia, 20060, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30342, United States|Children's Memorial Hospital, Chicago, Illinois, 60614, United States|SUNY Downstate Medical Center, Brooklyn, New York, 11203, United States|Duke University Comprehensive Sickle Cell Center, Durham, North Carolina, 27713, United States|Virginia Commonwealth University, Richmond, Virginia, 23219, United States |
| TAPS2 Transfusion Antenatally in Pregnant Women With SCD | Sickle Cell Disease (SCD) is a serious inherited blood disorder affecting red blood cells. When oxygen levels drop the red cells become abnormally shaped and unable to move through the blood vessels easily. Blood and oxygen do not reach body organs, resulting in episodes of severe pain and other complications. Pregnant women with SCD have an increased risk of both sickle and pregnancy complications, including raised blood pressure. Their babies may grow more slowly in the womb, are more likely to be born early and need special care, and have a higher risk of dying. The only treatments currently available for women with SCD are Hydroxycarbamide (which cannot be used during pregnancy) and blood transfusion. Currently, blood transfusion is only used during pregnancy to treat emergency complications. It has been suggested that giving blood transfusions throughout pregnancy could improve outcomes for both mother and babies. In Serial Prophylactic Exchange Blood Transfusion (SPEBT), sickle blood is mechanically removed and simultaneously replaced with donor red cells. A trial is needed to assess SPEBT given every 6-10 weeks, starting before 18 weeks of pregnancy, compared to standard care. This trial will evaluate outcomes for women (e.g. hospital admission, frequency of crisis) and their infants (e.g. early delivery, birthweight). However, the feasibility of such a study needs to be assessed before embarking on a large multicentre trial. This study is therefore a feasibility study in which we will randomly allocate participants to have either SPEBT or standard care. The study will be carried out in multiple maternity units in England and last two years. The willingness of eligible women to join the study will be assessed, along with how many participants remain part of the study until the end and if participants find the intervention acceptable. | Sickle Cell Disease|Pregnancy, High Risk|Blood Transfusion Complication | FEMALE | ADULT, OLDER_ADULT | Barts Health NHS Trust, London, United Kingdom|Guy's and St Thomas' NHS Foundation Trust, London, United Kingdom|King's College Hospital, London, United Kingdom|St George's University Hospitals NHS Foundation Trust, London, United Kingdom|Whittington Health NHS Trust, London, United Kingdom|Manchester University NHS Foundation Trust, Manchester, United Kingdom |
| Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat | Background: Sickle cell disease (SCD) is a disorder that causes episodes of acute pain and progressive organ damage. Ways to manage SCD have evolved slowly. Treatments do not always work. Researchers want to see if a drug called mitapivat can help people with SCD. Objective: To test the long-term tolerability and safety of mitapivat (or AG-348) in people with SCD. Eligibility: Adults age 18-70 with SCD who took part in and benefited from NIH study #19H0097. Design: Participants will be screened with a medical history and physical exam. They will give a blood sample. They will have an electrocardiogram to test heart function. Participants will repeat some of the screening tests during the study. Participants will complete 6-minute walk tests to measure mobility and function. They will have transthoracic echocardiograms to measure heart and lung function. They will have dual-energy X-ray absorptiometry scans to measure bone health. They will complete online questionnaires that measure their overall health and well-being. Participants will take the study drug in the form of a tablet twice a day. Participants will keep a study diary. They will record any symptoms they may have. Participation will last for about 54 weeks. After 48 weeks, participants can either keep taking the study drug for 48 more weeks or be tapered off of the study drug to complete the study. Those who are on the study for 1 year will have 10 study visits. Those who are on the study for 2 years will have 14 study visits. | Sickle Cell Disease|Hemolytic Anemia | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies | The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with hemoglobinopathies. | Anemia, Sickle Cell|Complex and Transfusion-dependent Hemoglobinopathies|Thalassemia|Diamond-Blackfan Anemia|Bone Marrow Failure Syndromes|Alpha-Thalassemia|Beta-Thalassemia | ALL | CHILD, ADULT | Northwestern Memorial Hospital, Chicago, Illinois, 60611, United States|University of Louisville, Louisville, Kentucky, 40202, United States|Duke University Medical Center, Durham, North Carolina, 27705, United States |
| Hyperbaric-oxygen Therapy (HBOT) Versus Placebo for Treating Vaso-Occlusive Crisis (VOC) in Sickle Cell Disease (SCD) | This is a randomised, controlled, double-blind, placebo trial of HBOT (intervention) superiority in the treatment of VOC in SCD, to demonstrate the effectiveness of HBOT for the decrease in pain level in the treatment of SCD-VOC. | Sickle Cell Disease|Hyperbaric Oxygen Therapy|Vaso-occlusive Crisis | ALL | CHILD, ADULT, OLDER_ADULT | Hospices civils de Lyon, Lyon, France|Centre de compétences Sd drépanocytaires, Toulouse, France|HUG, Geneva, 1211, Switzerland |
| L-citrulline Injection in Patients Aged 6-21 Years Old with Sickle Cell Disease Presenting with Vaso-Occlusive Crisis (VOC) | The purpose of this study is to determine if intravenous L-citrulline can abrogate an active vaso-occlusive crisis in sickle cell disease, resulting in decreased pain, reduction or elimination of opiate usage, and reduction or elimination of hospital admission. The applicant is developing intravenous L-citrulline (Turnobi™) for treatment of sickle cell disease (SCD). The current development program targets treatment of sickle cell-associated vaso-occlusive crisis (VOC) specifically. The aim of Part 1 is to identify the optimum dose regimens for the Part 2 of the trial which is a double-blind, placebo controlled adaptive 'pick-the-winner' design. This study will allow assignment of more subjects to the better treatment arm/s based on emerging data. The study, initially, will evaluate efficacy and tolerability of incremental doses of intravenous (IV) L-citrulline (Turnobi™) in patients with SCD while receiving standard of care therapy for VOC. | Acute Vaso Occlusive Crisis (VOC) | ALL | CHILD, ADULT | Children's National Hospital, Washington DC, District of Columbia, 20010, United States|The University of Mississippi Medical Center, Jackson, Mississippi, 39216, United States |
| Exploring Near Infrared Spectroscopy (NIRS) Technologies for Assessment of Muscle Physiology, Tissue Oxygenation, and Blood Flow in Patients With Sickle Cell Disease (SCD) | Sickle cell disease (SCD) is an inherited disorder of the blood. SCD can injure the smallest blood vessels, which can cause pain and damage organs all over the body. Some treatments are available, but researchers need better ways to monitor the effects of these treatments. An imaging technique called near infrared spectroscopy (NIRS) may be helpful. Objective: To test NIRS as a tool for measuring oxygen levels, blood flow, and the makeup of skin and muscle in patients with SCD. Eligibility: People aged 18 years and older with SCD. Healthy volunteers are also needed as a comparison for the changes in SCD patients. Design: Participants will be screened. They will have a physical exam, and 1 teaspoon of blood will be drawn. Participants will have NIRS testing on their second visit. Probes will be placed on their skin. A blood pressure cuff will be placed on their arm. The cuff will be filled with air for up to 5 minutes and then released. Participants may be asked to breathe at a certain rate or hold their breath during these measurements. At this visit, participants will also have an ultrasound exam to get images of their heart. They will be monitored while they walk for 6 minutes. They will have 1 tablespoon of blood drawn. Their height, weight, and vital signs will be measured. Participants may be asked to return for up to 4 additional visits for NIRS testing within 120 days, but this is optional. The visits must be at least 3 days apart. Each visit will last up to an hour.... | SCD | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Invasive Infections in Children With Hemoglobinopathies | Patients diagnosed as having hemoglobinopathies are exposed to serious bacterial infections, principally those patients that underwent splenectomy. Since the introduction of anti pneumococcal vaccine the incidence decreased significantly but other bacteria besides encapsulated bacteria takes place as principal cause of invasive infections. The purpose of this study is to analyse in a retrospective study the incidence of those infections in a group of patients suffering from thalassemia and sickle cell anemia treated in our clinic. | Thalassemia|Sickle Cell Anemia | ALL | CHILD, ADULT | Pediatric Hematology Unit and Pediatric Dpt B - HaEmek Medical Center, Afula, 18101, Israel |
| Intranasal Fentanyl for Initial Treatment of a Vaso-occlusive Crisis | The purpose of this study is to determine if intranasal fentanyl can decrease the pain of patients with sickle cell disease who present to the pediatric emergency department with a vaso-occlusive crisis. | Anemia, Sickle Cell|Pain | ALL | CHILD, ADULT | Children's Hospital at Montefiore, Bronx, New York, 10467, United States |
| Blood Transfusions in Thalassemia Patients, Complications and Adverse Effects | Patients suffering from Thalassemia or another hemoglobinopathies required regular blood transfusions. The complications and adverse effects of blood transfusions can be classified as immediate and late. Among the immediate effects the most common are allergic reactions and fever, besides congestive heart failure in patients with cardiomyopathy. The late effects are mostly related to blood transmitted infections like HIV or Hepatitis C infections. The purpose of this study is to summarize the data of those complications in a cohort of 100 patients receiving regular blood transfusion. | Thalassemia|Sickle Cell Anemia | ALL | CHILD, ADULT | Pediatric Hematology Unit and Pediatric Dpt B - HaEmek Medical Center, Afula, 18101, Israel |
| Feasibility and Preliminary Efficacy of Acceptance and Commitment Therapy (ACT) for Sleep Disturbances in Adults With Sickle Cell Disease (SCD) | Research Type: Clinical Trial Background: People with sickle cell disease (SCD) have many health challenges. Also, they often have trouble sleeping. Acceptance and commitment therapy (ACT) might help people with SCD to improve their sleep problems. Objective: To see how well ACT works in people with SCD and sleep problems and to find out how they feel about it. Eligibility: People between the ages of 18 and 55 with SCD and trouble sleeping. Design: The study is remote. Participants will not have to come to the NIH at all. They will need a device that has Bluetooth and can connect to the internet. Some participants will be in the study for 12 weeks. Others will participate for 20 weeks. Participants will video chat with an ACT coach once a week for 8 weeks. The coach will guide participants through mindfulness exercises and teach ACT ideas. Each session lasts about 45 minutes. Participants will be loaned an actigraph, a device worn on the wrist like a watch that measures and records movement. They will download a free app to upload data from the actigraph for the researchers. Participants will wear the actigraph on their nondominant wrist day and night for either 4 or 6 designated weeks. During these weeks, participants will complete a sleep diary each morning when they wake up. This takes about 2 minutes. Participants will be sent other surveys to complete from home during the study. They will answer questions about their physical and emotional health. These take 20-25 minutes. The last survey will be 4 weeks after participants finish the ACT treatment. They will answer questions about how helpful they thought ACT was and how easy or hard it was to wear the actigraph. | Sickle Cell Disease|Sickle Cell Anemia|Insomnia|Sleeplessness|Transient Insomnia|Nonorganic Insomnia|Chronic Insomnia | ALL | ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Virtual Reality As Adjunct Therapy for Vaso-Occlusive Pain | The goal of this randomized control clinical trial is to learn if virtual reality can be used to treat sickle cell pain in children. The main questions it aims to answer are: Does virtual reality reduce pain severity during a child's hospital stay for a vaso-occlusive pain crisis? Does virtual reality decrease the daily use of opiates? Researchers will compare standard therapy to the use of standard therapy plus a daily virtual reality experience to see if virtual reality works to treat sickle cell pain. All patients will: - Be asked to fill out a pain assessment survey three times daily for up to 3 days If randomized to intervention arm, patients will: * Participate in an immersive virtual reality experience once daily for up to 3 days * Fill out a survey twice daily to monitor for side effects from virtual reality experience * Fill out a satisfaction survey once during the study period | Sickle Cell Disease|Vaso-occlusive Pain Episodes | ALL | CHILD, ADULT | Children's Hospital of New Orleans, New Orleans, Louisiana, 70118, United States |
| Silent Cerebral Infarct Transfusion Multi-Center Clinical Trial | The goal of this study is to determine the effectiveness of blood transfusion therapy for prevention of silent cerebral infarct (stroke) in children with sickle cell anemia. | Sickle Cell Anemia|Stroke | ALL | CHILD | Washington University School of Medicine, St. Louis, Missouri, 63110, United States |
| Long Term Effects of Erythrocyte Lysis | In this prospective observational trial, participants with chronic hemolysis will be assessed with echocardiogram for elevated tricuspid jet velocity and other evidence of pulmonary hypertension. Participants will have laboratory studies evaluating: severity of hemolysis, splenic function, inflammation, endothelial dysfunction, and hypercoagulability. There will be 3 main categories of participants enrolled in this study: (1) pediatric participants with severe sickle cell disease (SCD) (HbSS, HbS/β° thalassemia ) who are not receiving treatment (e.g., hydroxyurea or chronic transfusions); (2) pediatric participants with other forms of SCD or severe SCD (HbSS, HbS/β° thalassemia) patients being treated with hydroxyurea or chronic transfusions; and (3) pediatric and adult participants with other non-sickling hematological disorders. | Sickle Cell Disease|Hemolytic Anemia | ALL | CHILD, ADULT, OLDER_ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38115, United States |
| Study to Evaluate Efficacy and Safety of Oral Treprostinil in Subjects With Pulmonary Hypertension (PH) Associated With Sickle Cell Disease (SCD) | This is a multicenter, randomized (2:1; oral treprostinil:placebo), double-blind, placebo-controlled event-driven (time to pulmonary hypertension \[PH\] clinical worsening) study in subjects with PH associated with sickle cell disease (SCD). Once enrolled, subjects will be evaluated at Weeks 6, 12, 24, and then every 12 weeks for the duration of the study. Subjects will be permitted to enter a 48-week open-label extension period if they experience a PH clinical worsening event. | Pulmonary Hypertension Associated With Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | |
| Effect of Exercise on Biomarkers in SCT | This study measures the effect of exercise on a variety of biomarkers in blood and urine selected to evaluate the physiological pathways of hemolysis, myolysis, thrombosis, inflammation, and renal function in subjects with sickle cell trait. These pathways have been shown to be associated with adverse events in athletes and warfighters with SCT upon protracted, repeated, strenuous exertion. Changes in biomarkers post-exercise compared to pre-exercise (and compared to healthy controls) suggest activation of the associated pathway(s) which may contribute to exercise-related events in athletes and warfighters and subclinical complications in non-athletes. | Sickle Cell Trait | ALL | ADULT, OLDER_ADULT | Saint Louis University, Saint Louis, Missouri, 63104-1111, United States |
| Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation | Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications. Much of the risk stems from the use of high dose radiation and chemotherapy for conditioning, the treatment administered just prior to transplant that eliminates the patients' marrow and immune system, effectively preventing rejection of the donors' cells. Attempts to make blood and marrow transplantation safer for children with non-malignant diseases by using lower doses of radiation and chemotherapy have largely failed because of a high rate of graft rejection. In many such cases, it is likely that the graft is rejected because the recipient is sensitized to proteins on donor cells, including bone marrow cells, by blood transfusions. The formation of memory immune cells is a hallmark of sensitization, and these memory cells are relatively insensitive to chemotherapy and radiation. Alefacept, a drug used to treat psoriasis, on the other hand, selectively depletes these cells. The investigators are conducting a pilot study to begin to determine whether incorporating alefacept into a low dose conditioning regimen can effectively mitigate sensitization and, thereby, prevent rejection of allogeneic blood and marrow transplants for multiply transfused children with non-malignant hematological diseases. | Thalassemia|Sickle Cell Disease|Glanzmann Thrombasthenia|Wiskott-Aldrich Syndrome|Chronic-granulomatous Disease|Severe Congenital Neutropenia|Leukocyte Adhesion Deficiency|Schwachman-Diamond Syndrome|Diamond-Blackfan Anemia|Fanconi Anemia|Dyskeratosis-congenita|Chediak-Higashi Syndrome|Severe Aplastic Anemia | ALL | CHILD, ADULT | Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States |
| Frequency of COVID-19 Antibodies in Patients With Hereditary Hematological Diseases | In Italy there are about 5000 patients with dependent transfusion thalassemia (source Italian Thalassemia and Hemoglobinopathies - SITE) and a smaller number, currently not definable, of patients with sickle cell anemia in chronic transfusion. A recent study in the Lombardy region identified the positivity of anti-Covid-19 antibodies in 4.5-7% of asymptomatic donors (Valenti L et al). As already known, a preliminary study conducted in Italy (Motta I et al, Hussain FA et al, Taher A et al) reported only 11 cases of symptomatic infection all with benign evolution. Currently there are 15 reported cases (12 thalassemias and 3 sickle cell anemias). 75% of the cases have been identified in Lombardy. Our hypothesis is that in a percentage of polytransfused patients a transmission of the virus may have developed that stimulated the production of protective antibodies. This could be an explanation of the low contagiousness and severity of the infection in polytransfused patients. Currently no data are available for this purpose. This study will be conducted in collaboration with the Microbiology Unit and involves the determination of SARS-CoV-2 antibodies (anti-s1 and s2) by CLIA method with a high sensitivity (94.7%) and specificity (98.5%). | Thalassemia Major|Covid19 | ALL | ADULT, OLDER_ADULT | ASST Monza - Ospedale San Gerardo, Monza, MB, 20900, Italy |
| Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload | This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot adequately be treated with locally approved iron chelators. | Thalassemia|Sickle Cell Disease|Diamond Blackfan Anemia|Myelofibrosis | ALL | CHILD, ADULT, OLDER_ADULT | Arkansas Children's Hospital, UAMS College of Medicine, Little Rock, Arkansas, 72202, United States|Alta Bates Comprehensive Cancer Center, Berkeley, California, 94704, United States|Children's Hospital of Orange County, Orange, California, 92868, United States|Children's Hospital and Health Center of San Diego, San Diego, California, 92123, United States|Stanford University, Stanford, California, 94305, United States|Alfred I. Dupong Hospital for Children, Wilmington, Delaware, 19803, United States|Osler Medical, Inc., Melbourne, Florida, 32901, United States|Hematalogy Oncology Associates, Pensacola, Florida, 32501, United States|Tampa Children's Hospital at St. Joseph's Hospital, Tampa, Florida, 33607, United States|James A. Haley Veterans Hospital, Tampa, Florida, 33612, United States|Backus Children's Hospital, Memorial Health University Medical Center, Savannah, Georgia, 31403, United States|Hematalogy Oncology Clinic, Baton Rouge, Louisiana, 70808, United States|Borgess Hospital, Kalamazoo, Michigan, 49048, United States|Children's Hospitals and Clinics of Minnesota, Minneapolis, Minnesota, 55405, United States|University of Mississippi Medical Center, Jackson, Mississippi, 39762, United States|Children's Mercy Hospital, Kansas City, Missouri, 64108, United States|The Cancer Institute of New Jersey, New Brunswick, New Jersey, 08903, United States|Schneider Children's Hospital, New Hyde Park, New York, 11040, United States|PCTI, Columbus, Ohio, 43205, United States|The Children's Medical Center of Dayton, Dayton, Ohio, 45404, United States|Oregon Health & Science University, Portland, Oregon, 97239, United States|Hershey Medical Center, Hershey, Pennsylvania, 17033-0850, United States|Children's Hospitals of Pittsburgh, Pittsburgh, Pennsylvania, 15213, United States|Texas Children's Hospital, Houston, Texas, 77030, United States|Children's Hospital of the Kings Daughters, Norfolk, Virginia, 23507, United States|VCU Pediatric Hematology/Oncology, Richmond, Virginia, 23219, United States|University of Washington Seattle Cancer Care Alliance, Seattle, Washington, 98195, United States|Ziekenhuisnetwerk Antwerpen-AZ Middelheim, Antwerpen, Belgium|Centre Hospitalier Notre Dame et Reine, Charleroi, Belgium|CHR de la Citadelle, Liege, Belgium|Centre Hospitalier Chretien-Clinique Saint-Joseph, Montegnee, Belgium|The Ottawa Hospital-General Campus, Ottawa, Ontario, Canada|University of Alberta, Edmonton, Canada|CHUM-Hopital-Notre-Dame, Montreal, Canada|MUHC- Montreal Children's Hospital, Montreal, Canada|MUHC- Royal Victoria Hospital, Montreal, Canada|Hopital de l'Enfant-Jesus, Quebec, Canada|The Hospital for Sick Children,, Toronto, Canada|Toronto General Hospital-Hemoglobinopathy, Toronto, Canada|Burrard Medical Building, Vancouver, Canada|Charite-Universitatsmedizin Berlin, Berlin, Germany|Universitaetsklinik Dusseldorf, Duesseldorf, Germany|Johann Wolfgang von Goethe Universitat, Frankfurt am Main, Germany|Georg-August-Universitat Gottingen, Gottingen, Germany|Universitatskrankenhaus Hamburg-Eppendorf, Hamburg, Germany|Medizinische Hochschule Hannover, Hannover, Germany|Universitatsklinikum Koln, Köln, Germany|Klinikum Stuttgart Bismarckstrasse 8, Stuttgart, Germany|Universitatsklinikum Ulm, Ulm, Germany|Agia Sofia Hospital of Athens, Athens, Greece|General Hospital of Athens, Athens, Greece|Ippokration Hospital of Athens, Athens, Greece|General Hospital of Heraklion Benizeleio-Pananeio, Heraklion, Greece|University Hospital of Ioannina, Ioannina, Greece|General Hospital of Kalamata, Kalamata, Greece|Agia Sofia Hospital of Athens, Karditsa, Greece|General Hospital of Karditsa, Karditsa, Greece|General Hospital of Athens, Kerkyra, Greece|General Hospital of Korinthos, Korinthos, Greece|General Hospital of Larisa Tsakalof 1, Larisa, Greece|General Hospital of Mytilini Vostaneio, Mytilini, Greece|General State Hospital of Nikaia St. Panteleimon, Nikaia, Greece|University Hospital of Patras, Patra, Greece|General Hospital of Thessaloniki Agios Pavlos, Thessaloniki, Greece|General Hospital Thessalonikis Hippokratio, Thessaloniki, Greece|General Hospital of Volos, Volos, Greece|General Hospital of Xanthi, Xanthi, Greece|Presidio Ospedale Muscatello, Augusta, Italy|A.O. Ospedale Policlinico Consorziale di Bari, Bari, Italy|Presidio Ospedaliero Antonio Perrino, Brindisi, Italy|Ospedale Regionale Microcitemie, Cagliari, Italy|Ospedale Di Venere, Carbonara di Bari, Italy|Azienda Ospedali Vittorio Emanuele, Ferrarotto e San Bambino, Catania, Italy|Presidio Ospedaliero S. Bambino, Catania, Italy|PresidioOspedaliero S. Luigi Curro, Catania, Italy|Azienda Ospedaliera Pugliese Cicaccio, Catanzaro, Italy|Ospedale Civile dell'Annunziata, Cosenza, Italy|Ospedale San Giuseppe, Empoli, Italy|Azienda Ospedaliera Universitaria di Ferrara, Ferrara, Italy|Azienda Ospedaliera A. Meyer, Firenze, Italy|E.O. Ospedali Galliera, Genova, Italy|Ospedale Regionale Microcitemie, Itala, Italy|Ospedale Madonna delle Grazie, Matera, Italy|Az. Ospedaliera Universitaria Policlinico G. Martino, Messina, Italy|Fondazione Ospedale, Milano, Italy|Azienda Ospedaliero-Universitaria di Modena, Modena, Italy|AORN A. Cardarelli, Napoli, Italy|Azienda Ospedaliera Universitaria Federico II, Napoli, Italy|Azienda Ospedaliera V. Cervello, Palermo, Italy|Azienda Ospedaliera Villa Sofia-CTO, Palermo, Italy|Presidio Ospedaliero Giovanni di Cristina, Palermo, Italy|IRCCS Policlinico San Matteo, Pavia, Italy|Azienda Ospedaliera San Salvatore, Pesaro, Italy|Azienda Ospedaliero Universitaria Pisana, Pisa, Italy|Azienda Ospedaliera Civile- Maria Paterno, Ragusa, Italy|Azienda Ospedaliera Bianchi-Melacrino-Morelli, Reggio Calabria, Italy|Ospedale S. Eugenio, Roma, Italy|Ospedale nostra Signora di Bonaria, San Gavino Monreale- CA, Italy|Presidio Ospedaliero di Sassari-Ospedale SS, Sassari, Italy|Azienda Ospedaliera Ospedali Civili Riuniti di Sciacca, Sciacca, Italy|Ospedale Umberto I, Talassemie, Italy|Presidio Ospedaliero Centrale, Taranto, Italy|Ospedale Infantile Regina Margherita, Torino, Italy|AMC, Amsterdam, Netherlands|Haga Ziekenhuis, Den Haag, Netherlands|Catharina-ziekenhuis, Eindhoven, Netherlands|Erasmus MC, Rotterdam, Netherlands|Erasmus Medisch Centrum, locatie Sophia, Rotterdam, Netherlands|Hospital de Torrecardenas, Almeria, Spain|Hospital Infanta Cristina, Badajoz, Spain|Hospital Cruces, Baracaldo, Spain|Hospital Puerta del Mar, Cadiz, Spain|Hospital De Gran Canaria, Canara, Spain|Hospital Universitario La Paz, Madrid, Spain|Althaia : Xarxa Assistencial de Manresa, Manresa, Spain|Hospital Universitario Marques de Valdecilla, Santander, Spain|Hospital Virgen de la Salud, Toledo, Spain|Hospital Universitario La Fe, Valencia, Spain|Hospital Xeral de Vigo, Vigo, Spain|Kaohsiung Medical University Hospital, Kaohsiung, Taiwan|Mackay Memorial Hospital, Taipei, Taiwan|National Taiwan University Hospital, Taipei, Taiwan|Chang Gung Children's Hospital, Taoyuan, Taiwan|Tao-yuan General Hospital, Tau-Yuan County, Taiwan|Phramongkutklao Hospital, Bangkok, Thailand|Ramathibodi Hospital, Bangkok, Thailand|Songklanagarind Hospital, Bangkok, Thailand|Srinagarind Hospital, Khon Kaen, Thailand|Cukurova Universitesi, Adana, Turkey|Hacettepe Universitesi, Ankara, Turkey|Akdeniz Universitesi, Antalya, Turkey|Gaziantep Universitesi, Gaziantep, Turkey|Suleyman Demirel, Isparta, Turkey|Istanbul Universitesi, Istanbul, Turkey|Ege Universitesi Tip Fakultesi, Izmir, Turkey|Erciyes Universitesi, Kayseri, Turkey|Evelina Hospital St. Thomas' Hospital, London, United Kingdom|North Middlesex University Hospital, London, United Kingdom|The Royal Hospital London, London, United Kingdom|Whittington Hospital, London, United Kingdom|St. George's Hospital, Tooting, United Kingdom |
| Does IV Acetaminophen Reduce Opioid Requirement in Pediatric Patients With Acute Sickle Cell Crises? | The purpose of this study is to determine whether IV acetaminophen can decrease the need for subsequent opioid administration in the acute management of sickle cell crisis pain in the pediatric emergency room. | Sickle Cell Anemia Crisis | ALL | CHILD | Newark Beth Israel, Newark, New York, 07112, United States |
| The Genetics and Functional Basis of Inherited Platelet, White Blood Cell, Red Blood Cell, and Blood Clotting Disorders. | Blood contains red blood cells, white blood cells, and platelets, as well as a fluid portion termed plasma. We primarily study blood platelets, but sometimes we also analyze the blood of patients with red blood cell disorders (such as sickle cell disease), white blood cell disorders, and disorders of the blood clotting factors found in plasma. Blood platelets are small cell fragments that help people stop bleeding after blood vessels are damaged. Some individuals have abnormalities in their blood platelets that result in them not functioning properly. One such disorder is Glanzmann thrombasthenia. Most such patients have a bleeding disorder characterized by nosebleeds, gum bleeding, easy bruising (black and blue marks), heavy menstrual periods in women, and excessive bleeding after surgery or trauma. Our laboratory performs advanced tests of platelet function and platelet biochemistry. If we find evidence that a genetic disorder may be responsible, we analyze the genetic material (DNA and RNA) from the volunteer, and when possible, close family members to identify the precise defect. | Glanzmann Thrombasthenia | ALL | CHILD, ADULT, OLDER_ADULT | Rockefeller University Hospital, New York, New York, 10021, United States |
| Diabetes and Heart Disease Risk in Blacks | It is unknown if obesity contributes to the development of heart disease in African American men and women. This study was created to determine whether there is a relationship between sex and body size and the incidence of heart disease in African American men and women. Researchers will attempt to associate obesity with the presence of heart disease risk factors. Risk factors that will be studied include; total body fat, body fat distribution, fat content of the blood (triglyceride concentration, low density lipoproteins \[LDL\], and high density lipoproteins \[HDL\]), how fast fat is removed from the blood, and how well insulin works in the body. Scientific studies have shown that obesity and increased levels of fat content in the blood are important risk factors for heart disease in Caucasian women. However, similar studies in African American women have failed to show the same correlation. In fact, it appears that African American women in all three body weight groupings, nonobese, overweight, and obese experience high death rates due to heart disease. In addition, prior research has shown that obese African American men tend to have elevated levels of fat in the blood while African American women have normal blood fat levels. Therefore, if high levels of triglycerides (fat found in the blood) are not seen in non-diabetic obese African American women, it cannot be considered a risk factor in this population. This suggests that studies conducted on Caucasian women may not provide insight into heart disease risk factors in African American women. The study will take 2000 healthy non-diabetic African American men and women (ages 18-70) and body mass index 3 subgroups; nonobese, overweight and obese. Diabetes undeniably increases the risk of heart disease. Therefore patients suffering from diabetes will not be included in the study. Candidates for the study will undergo a series of tests and examinations over 2 outpatient visits. Subjects will have body fat analyses, resting energy expenditure measurements, an EKG (electrocardiogram), and specific blood tests. Researchers believe this study will provide significant insight into the causes of obesity and heart disease in African Americans. | Cardiovascular Diseases|Diabetes|Obesity|Hypertension | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| The Feasibility of the Pray Until Something Happens (PUSH) Intervention-OUTPATIENT | The goal of this research study is to reduce stress and improve sickle cell disease (SCD) pain control and sleep quality with less opioid use by determining the feasibility of an intervention with self-management combined intercessory and petitionary prayer, named Pray Until Something Happens (PUSH) stress reduction intervention using a mobile smart device. Currently, opioid analgesics are primarily used to treat SCD pain while self-managed behavioral modalities such as PUSH, are rarely used. Little is known about the effects or mechanisms of PUSH on pain, stress, and sleep symptoms in adults with SCD. Emerging evidence from the hypothalamic pituitary adrenal (HPA) axis theory offer insights for understanding the mechanisms. Adding PUSH as a supplement to analgesic therapies will address the dearth of self-management strategies for controlling pain in SCD. PUSH is a simple and cost-effective non-drug intervention that could reduce pain and stress in inpatients with SCD. GR is an intervention where inpatients with SCD are directed to listen to the audio recordings of the PUSH prayer session. | Sickle Cell Disease|Stress|Pain | ALL | ADULT, OLDER_ADULT | |
| Effect of Inhaled Nitric Oxide in Acute Chest Syndrome (INOSTA Study) | Acute chest syndrome (ACS) is a frequent and potentially life-threatening pulmonary illness. It is a complication of sickle cell disease and is the leading cause of death from this disease in adults. Several pathologic processes are recognized causes of ACS, including infectious diseases, hypoventilation secondary to chest pain, in situ thrombosis and pulmonary fat embolism. Inhaled nitric oxide (iNO) has been shown to be a pulmonary vasodilatator with minimal systemic effects and has also been shown to improve gas exchange in both animal and human acute lung injury (ALI). The combined effects of iNO gas of improving pulmonary ventilation to perfusion matching, reducing alveolar and systemic inflammation, modulate the course of acute chest syndrome, which combine the physiopathology of vaso-occlusive crisis and acute lung injury. We hypothesise inhaled NO will improve oxygenation and clinical outcome of sickle cell disease patients with acute chest syndrome. | Sickle Cell Disease|Acute Chest Syndrome | ALL | ADULT, OLDER_ADULT | Réanimation Médicale, Hôpital A Chenevier-H Mondor, Creteil, 94 000, France |
| Awake Prone Positioning for Severe Acute Chest Syndrome | Acute chest syndrome (ACS) is the leading cause of admission to intensive care and the leading cause of death in patients with sickle cell disease. Irrespective of the cause of ACS, there is an heterogeneity in pulmonary ventilation/perfusion ratios, leading to worsening of the disease. Efficiency of awake prone positioning (APP) in acute respiratory failure (ARF) was particularly highlighted during the COVID-19 pandemic. Several physiological factors contribute to this benefit including an improvement in ventilatory drive and gas exchange. The investigator hypothesize that APP could lead to clinical improvement in ACS in terms of oxygenation and ventilatory drive, by improving the heterogeneity of ventilation | Acute Chest Syndrome|Sickle Cell Anemia | ALL | ADULT, OLDER_ADULT | Service de Médecine Intensive Réanimation TENON, Paris, 75020, France |
| Combined Use of a Respiratory Broad Panel Multiplex PCR and Procalcitonin to Reduce Antibiotics Exposure in Hospitalized Sickle-cell Adults With Acute Chest Syndrome. | Many patients with Sickle Cell Disease (SCD) may develop Acute Chest Syndrome (ACS). ACS is usually caused by a Lower respiratory tract infection (LRTI) which may be caused by either a bacterium or a virus. Antibiotics are usually used for 7 to 10 days with no microbiological workup. The hypothesis of the study is that the identification of the microorganisms might lead to a reduction of antibiotics exposure and a better care of the patients. We speculate that an early pathogen-directed strategy (respiratory broad panel multiplex PCR and early antibiotics interruption based on the PCT values decrease) might reduce the antibiotics exposure in SCD patients with ACS who are hospitalized and for whom an antibiotic treatment is indicated, as compared with usual care | Acute Chest Syndrome|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | Service de Réanimation et USC médico-chirurgicale, Paris, 75020, France |
| Oral Magnesium Pidolate, Hemoglobin SC Disease, MG Pidolate | Subjects have a form of sickle cell disease, called hemoglobin SC disease. This results in abnormally shaped red blood cells that get 'stuck' in blood vessels and then results in episodes of severe pain (pain crises). Patients with the more common form of sickle cell disease, called hemoglobin SS disease, also suffer from pain crises. Treatment with the drug hydroxyurea is available to help prevent the pain crises in hemoglobin SS disease, but there is no good treatment to help prevent the pain crises in hemoglobin SC disease. It has been shown that one of the reasons for the formation of the abnormally shaped red blood cells in patients with SC disease is the fact that these cells do not contain enough water; they are dehydrated. Drinking more water will not increase the amount of water in the cells. Certain salts and minerals can however have an effect on the amount of water in the red blood cells. One of the most important minerals influencing this is called magnesium. Magnesium is present in food and also in certain medications used to treat heartburn. Magnesium has been used successfully both in animals and people to increase the amount of water in the red blood cells and is very well tolerated by most people. Investigators are using a new form of magnesium known as magnesium pidolate because this form of magnesium may help with the symptoms of disease without causing diarrhea (a common side effect of magnesium products). Purpose The purpose of this study is to find out whether treatment with magnesium pidolate will increase the amount of water in the red blood cell and result in fewer painful crises in patients with hemoglobin SC disease while not causing diarrhea. The study will last for about 64 weeks (about 16 months). | HEMOGLOBIN SC DISEASE | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital, Boston, Massachusetts, 02115, United States|Texas Children's Hospital, Houston, Texas, 77030, United States |
| Cognitive Remediation Intervention to Prepare for Transition of Care | Randomized Controlled Trial (RTC) testing the efficacy of a telehealth adaptation of the Cognitive-Remediation of Executive and Adaptive Deficits in Youth (C-READY) intervention to prepare adolescents with sickle cell disease for transition of care. | Sickle Cell Disease|Cognitive Impairment|Adolescent Behavior|Self Efficacy|Health-Related Behavior|Coping Skills | ALL | CHILD, ADULT | University of Alabama at Birmingham, Birmingham, Alabama, 35233, United States |
| Transcranial Doppler (TCD) Ultrasound of Subjects Enrolled in BABY HUG - Ancillary to BABY HUG | To use transcranial Doppler (TCD) ultrasound to detect stroke risk in children with sickle cell disease. | Blood Disease|Anemia, Sickle Cell|Cerebrovascular Disorders|Cerebral Embolism and Thrombosis | ALL | CHILD | |
| Cognitive Behavioral Therapy and Real-time Self-management Intervention for SCD Via Mobile Applications | Patients with sickle cell disease (SCD) experience significant depressive symptoms that currently go unrecognized and under-treated. Further, depression in this patient population has the potential to contribute to high health care utilization and poor disease outcomes; however, there are currently no comparative effectiveness studies of evidenced-based mental health treatments for depression in SCD. The primary objective of this study is to test the effectiveness of an online computerized cognitive behavioral therapy intervention to address psychological and behavioral needs of patients with sickle cell disease, namely depression and pain symptoms. The investigators will implement an existing computerized cognitive behavioral therapy (CCBT) program called "Beating the Blues" into routine clinical care at the University of Pittsburgh Medical Center (UPMC) Adult Sickle Cell Clinic to determine the effectiveness of this intervention in decreasing depression and pain versus treatment as usual. Patients with significant distress-depression and/or anxiety symptoms-will be randomized to either eight sessions of a CCBT program and weekly follow-up with a care manager or treatment as usual where the treating physician is notified of the patient's symptoms. The investigators will evaluate patient acceptability, implementation and practicality of the online mental health intervention through patient use of the site (frequency and duration of visits), qualitative interviews, and surveys. The investigators hypothesize: 1) the CCBT will be an acceptable mental health treatment for patients and easily integrated into routine clinical care; 2) patients in the treatment arm will show a greater decrease in depression/anxiety symptoms and average daily pain than patients in usual care. | Sickle Cell Disease|Depression|Anxiety | ALL | ADULT, OLDER_ADULT | University of Pittsburgh, Pittsburgh, Pennsylvania, 15237, United States |
| Effect of Broccoli Sprouts Homogenate on SS RBC | The overall purpose of this study is to obtain a better understanding of the biological response of red blood cells to sulforaphane contained in fresh broccoli sprouts that have been put through a blending process. This study will use commercially available fresh broccoli sprouts certified by Brassica Protection Products LLC (BroccoSprouts®). This product can also be purchased at some local grocery stores in the produce section. It is believed that NRF2, a transcription factor encoded by the NFE2L2 gene, plays a role in the regulation of defense against oxidative stress. The detrimental accelerated breakdown of sickle cell disease (SCD) red blood cells (SS RBC) is partially due to reduced anti-oxidative capacity. Previous analysis of SS RBC microRNAs revealed that a reduced level of NRF2, the master regulator of anti-oxidative stress capacity, contributes to reduced resistance to oxidative stress and increased hemolysis; NRF2 also induces fetal hemoglobin (HbF), which is known to prevent SS RBC sickling. First, erythroid progenitors from normal and SCD subjects will be tested ex-vivo to find out how sulforaphane, a natural NRF2 activator, affects the oxidative stress capacity, HbF expression, and microRNA expression of red cells. Second, a pilot clinical trial will be conducted to determine the safety and physiological effects of 3 weeks of daily consumption of broccoli sprout homogenate in a cohort of Hb SS/SB0 thalassemia adult SCD patients. During this study, subjects RBCs will be assayed for changes in anti-oxidative stress capacity and microRNA composition in mature SCD red blood cells. | Sickle Red Blood Cell|Fetal Hemoglobin|Oxidative Stress | ALL | ADULT, OLDER_ADULT | Duke University Medical Center, Durham, North Carolina, 27710, United States |
| Comparison of Patient Controlled Analgesia (PCA) Versus Bolus Narcotic Therapy for the Treatment of Vaso-Occlusive Crisis (VOC) | This research is being done to find out the best way to give narcotics for pain relief in adults with sickle cell disease and painful crisis. This study is a comparison of two ways of giving narcotics. The first way is what occurs now in the Emergency Acute Care Unit (EACU) where patients are given a single intravenous (iv) dose of a narcotic which is repeated by the nurse as needed to control the pain. The second way is to provide a single iv dose of narcotic and then allow the patient to push a button and receive one or more additional doses of narcotic when he/she thinks it is needed. Our hypothesis is that PCA will be a more effective way of controlling pain. | Sickle Cell Disease|Vaso-occlusive Crisis | ALL | ADULT, OLDER_ADULT | Johns Hopkins Hospital, Baltimore, Maryland, 21205, United States |
| Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders | OBJECTIVES: I. Determine the efficacy of bone marrow transplantation using matched related donors in patients with nonmalignant hematologic disorders. II. Determine the quality of life, absence of adverse effects (e.g., graft versus host disease and B cell lymphoproliferative disease), and completeness of recovery of their underlying condition in these patients with this treatment regimen. | Neutropenia|Sickle Cell Anemia|Thalassemia Major|Red-Cell Aplasia, Pure | ALL | CHILD, ADULT, OLDER_ADULT | Fairview University Medical Center, Minneapolis, Minnesota, 55455, United States |
| Prognostic Factors of Acute Splenic Sequestration | Acute splenic sequestration is a frequent and life threatening complication occurring in approximately 10 % of homozygous children. Maximal incidence is between 6 and 18 months. The investigators formulate the hypothesis that there are clinical, biological and genetic markers predictive of severe complications notably acute splenic sequestration in SCD children. The present research project thus aims at analyzing in a forward-looking way the profile of severity by analysing clinical, biological and genetic characteristics in a multicentric cohort of 60 SCD children | Anemia; Drepanocytic | ALL | CHILD | Necker Hospital, Paris, 75015, France |
| Cardiopulmonary Function Assessment and NO-Based Therapies for Patients With Hemolysis-Associated Pulmonary Hypertension | This study will evaluate new treatments for people who have pulmonary hypertension, or high blood pressure in the lungs, caused by sickle cell anemia or thalassemia. Patients ages 18 and older with a diagnosis of sickle cell disease or thalassemia, who have mild to severe pulmonary hypertension, and who are not pregnant or breastfeeding may be eligible for this study. There are three stages in the study, with up to 200 participants in the screening. Patients will undergo pulmonary function tests, including those for asthma and measurement of oxygen levels in the arterial blood. They will have a chest X-ray, computed tomography (CT) scan of the lungs, ventilation perfusion lung scan to look for blood clots, echocardiogram, test to measure how far patients can walk in 6 minutes, nighttime oxygen measurement done while asleep, blood collection, magnetic resonance imaging (MRI) scan of the heart, and exercise test. About 3 to 4 days are needed for the tests, all of which can be done while patients are outpatients, except for the sleep study. For the CT scan, patients lie on a table while an X-ray beam takes images of the lungs and heart. The lung scan involves breathing of a small amount of a radioactive aerosol called Tc99m DTPA while pictures are taken of the lungs from various angles. Then an injection of albumin, a protein with a small amount of radioactivity, will be given, and more lung pictures will be taken. For the MRI scan, patients lie on a table that slides into a machine. A medication called gadolinium will be injected, to help improve images made through the scan. After the tests, patients will be admitted to the Clinical Center for 1 day. A small plastic catheter, or tube, will be placed in the vein of an arm. A longer catheter will go into a deeper vein (neck or leg), and a pulmonary artery catheter will be inserted to measure blood pressure in the blood vessels. Doctors will guide the catheter into the lung artery. Patients will be asked to pedal on a stationary bicycle while heart and lung pressure is measured. If pulmonary hypertension is present, patients will proceed to the second stage (up to 50 participants). While the catheter is still in place, patients will wear a face mask and breathe nitric oxide (NO) for 20 minutes. They will take 50 mg of sildenafil by mouth, and pressure in the heart and lungs will be monitored for about 4 hours. They will again receive NO for another 20 minutes. Blood samples will be taken, and the heart rhythm and pressure in the lungs will be monitored. Sildenafil can cause headache, flushing, and indigestion. Side effects of the lung scan involve allergic reactions to DTPA and albumin. Patients with an allergy to eggs should not have that test. Up to 25 patients can enter the third stage. They will breathe NO by using a tank of gas that delivers it through tubes to the nose, for a period of 6 weeks. They will continue taking sildenafil as previously prescribed and visit the clinic every 2 to 4 weeks for an echocardiogram, blood tests, and 6-minute walk test. After 6 weeks, patients will have catheterization of the heart again to measure pressure in the heart and lungs. Then NO will be stopped, and pressure in the lungs will be checked to see if NO has helped lower the blood pressure-and to make sure that the blood pressure does not increase when medication is stopped. Patients whose symptoms have improved as a result of breathing NO may wish to continue with that therapy. | Pulmonary Hypertension | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant | The purpose of this study is to learn more about the effects of (classification determinant) CD34+ stem cell selection on graft versus host disease (GVHD) in children, adolescents, and young adults. CD34+ stem cells are the cells that make all the types of blood cells in the body. GVHD is a condition that results from a reaction of transplanted donor T-lymphocytes (a kind of white blood cell) against the recipient's body and organs. Study subjects will be offered treatment involving the use of the CliniMACS® Reagent System (Miltenyi Biotec), a CD34+ selection device to remove T-cells from a peripheral blood stem cell transplant in order to decrease the risk of acute and chronic GVHD. This study involves subjects who are diagnosed with a malignant disease, that has either failed standard therapy or is unlikely to be cured with standard non-transplant therapy, who will receive a peripheral blood stem cell transplant. A malignant disease includes the following: Chronic Myeloid Leukemia (CML) in chronic phase, accelerated phase or blast crisis; Acute Myelogenous Leukemia (AML); Myelodysplastic Syndrome (MDS); Juvenile Myelomonocytic Leukemia (JMML); Acute Lymphoblastic Leukemia (ALL); or Lymphoma (Hodgkin's and Non-Hodgkin's). | Chronic Myeloid Leukemia (CML)|Acute Myelogenous Leukemia (AML)|Myelodysplastic Syndrome (MDS)|Juvenile Myelomonocytic Leukemia (JMML)|Acute Lymphoblastic Leukemia (ALL)|Lymphoma (Hodgkin's and Non-Hodgkin's) | ALL | CHILD, ADULT | Columbia University Irving Medical Center, New York, New York, 10032, United States |
| Oral Ketamine for Treatment of Vaso-Occlusive Pain | The purpose of this study is to learn more about the feasibility of oral ketamine for the treatment of painful sickle-cell crises in children and adolescents as a supplement to intravenous (IV) opioids. There is a need for improved non-opioid analgesia for patients experiencing sickle-cell crises in the hospital and prehospital setting, as children and adolescents with sickle cell disease who experience sickle-cell crises often have severe pain that is not well controlled by high dose opioids, leading to poor pain management and opioid-related side effects. The study will begin when patients are admitted to the Emergency Department of Boston Children's Hospital for treatment of a sickle-cell crisis. Oral ketamine will be administered every 8 hours for the next 48 hours. Patients will have continuous cardiorespiratory monitoring for the duration of the study, as per routine care, as well as monitoring by the hospital's Acute Pain Service at least twice daily for pain management and side effects of pain treatment. At the end of the 48-hour study duration, patients will discuss with the Pain Service and Hematology Service whether to continue oral ketamine, change to intravenous ketamine, or discontinue ketamine based on clinical indications such as level of pain and sedation while on opioids. | Vaso-occlusive Crisis | ALL | CHILD, ADULT | Boston Children's Hospital, Boston, Massachusetts, 02115, United States |
| Ledipasvir/Sofosbuvir Fixed-Dose Combination for 12 or 24 Weeks in Genotype 1 or 4 HCV Infected Adults With Sickle Cell Disease | The primary objectives of this study are to evaluate the antiviral efficacy, safety, and tolerability of ledipasvir/sofosbuvir (LDV/SOF) fixed-dose combination (FDC) administered for 12 or 24 weeks in adults with genotype 1 or 4 hepatitis C virus (HCV) infection with sickle cell disease (SCD). | Hepatitis C Virus Infection | ALL | ADULT, OLDER_ADULT | Lutherville, Maryland, United States |
| Fresh Versus Old Red Blood Cells for Transfusion | Packed red blood cell units destined for transfusion can be stored for up to 42 days prior to transfusion based on Food and Drug Administration (FDA) guidelines. Recent studies suggest that certain patients transfused with blood stored for longer duration have poorer outcomes than patients transfused fresher blood. The investigators' hypothesis is that the delivery of an immediate and substantial load of hemoglobin-associated iron from a stored unit of blood leads to changes that explain the differences in outcome between patients transfused old versus fresh blood. The investigators propose to test this hypothesis in humans by transfusing an individual's own blood, both fresh and after storage, and comparing levels of various outcome measures. | Iron, Abnormal Blood Level|Other Abnormal Blood Chemistry | ALL | ADULT, OLDER_ADULT | Columbia University Medical Center, New York, New York, 10032, United States |
| Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases | This is a Phase II study for the use of T-cell replete reduced intensity conditioning (RIC) haploidentical donor allogeneic hematopoietic cell transplantation (HaploHCT) for individuals with high-risk non-malignant diseases who lack a suitable HLA-matched sibling donor. | Sickle Cell Disease|Thalassemia|High Risk Hematologic Disorders|Cerebral Adrenoleukodystrophy|Inherited Metabolic Disorders | ALL | CHILD, ADULT | Masonic Caner Center at University of Minnesota, Minneapolis, Minnesota, 55455, United States |
| A Study of IMR-687 in Healthy Adult Volunteers | The purpose of this Phase 1a, first in human, randomized, double-blind, placebo-controlled study is to evaluate the safety, tolerability, PK and PD profile of the orally administered IMR-687 in healthy adult subjects. | Sickle Cell Disease|Sickle-Cell; Hb-SC|Sickle Beta 0 Thalassemia | ALL | ADULT | Quintiles, Overland Park, Kansas, 66211, United States |
| Bi-Level Positive Airway Ventilation for Acute Chest Syndrome | Acute chest syndrome (ACS) is a frequent complication of sickle cell disease and is diagnosed by having findings on a chest x-ray and one of the following: chest pain, fever, or trouble breathing. Patients with Acute Chest Syndrome can get very sick and require an exchange transfusion (special large blood transfusion) and mechanical ventilation. Bi-level Positive Airway Pressure (also known as BLPAP or BiPAP) is a device that blows air into a patients lungs via a mask that covers the nose. The goal of this study is to determine whether giving children BiPAP when they have ACS, in addition to providing standard clinical care for ACS, alters the clinical course of these patients. The investigators hypothesize that patients receiving effective BiPAP will have milder clinical courses resulting in shorter hospital stays and fewer transfers to the intensive care unit and exchange transfusions. | Sickle Cell Anemia|Acute Chest Syndrome | ALL | CHILD, ADULT | Children's Hospital @ Montefiore, Bronx, New York, 10467, United States |
| Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes | RATIONALE: Although used primarily to treat malignant disorders of the blood, allogeneic stem cell transplantation can also cure a variety of non-cancerous, inherited or acquired disorders of the blood. Unfortunately, the conventional approach to allogeneic stem cell transplantation is a risky procedure. For some non-cancerous conditions, the risks of this procedure outweigh the potential benefits. This protocol is designed to test a new approach to allogeneic stem cell transplantation. It is hoped that this approach will be better suited for patients with non-cancerous blood and bone marrow disorders. | Sickle Cell Anemia|Severe Aplastic Anemia|Paroxysmal Nocturnal Hemoglobinuria (PNH)|Pure Red Cell Aplasia | ALL | ADULT, OLDER_ADULT | Florida Hospital Cancer Institute, Orlando, Florida, 32804, United States|Duke Cancer Institute, Durham, North Carolina, 27710, United States |
| Test of the Safety, Effectiveness, & Acceptability of An Improvised Dressing for Sickle Cell Leg Ulcers in the Tropics | One in 300 Jamaicans have HbSS sickle cell disease, and of these, up to 70% will suffer from sickle cell leg ulcers (SCLUs). Of these, 24% will have a chronic SCLU (one lasting longer than 6 months). SCLUs heal very slowly, and sometimes they never close. SCLU patients would benefit from an economical, less painful, dressing option. In addition, because SCLUs often compromise education and employment opportunities, improving wound care for this population benefits their entire community. This three-armed evaluator-blinded randomized controlled trial will determine if a cut-to-fit food-grade plastic-based improvised dressing decreases pain, improves quality of life, and is safe, effective, and acceptable for managing SCLUs in Jamaica. The negative control will be usual practice, and the positive control will be the advanced wound dressing with the strongest evidence supporting its use in a tropical climate (polymeric membrane dressing). Patients with SCLUs will be actively recruited from three adjacent parishes. The first 120 SCLU patients meeting study criteria presenting to UHWI, Mona, will be randomized immediately after initial cleansing/debriding into group (1) current usual practice, group (2) improvised dressings, or group (3) advanced dressings. Data will be added to each participant's data collection tool weekly. Results will be reported using descriptive statistics and ANCOVA. The expected outcome is both improvised and advanced dressing superiority to usual practice. Because proposed improvised dressing materials are easily obtainable, their use would increase the capacity of wound patients to safely and effectively care for themselves. Signed informed consent will be obtained from patients/parents. Only principal research investigators will have access to participant confidential information. The literature review demonstrates that risks are not higher than usual practice. | Sickle Cell Leg Ulcer | ALL | CHILD, ADULT, OLDER_ADULT | University Hospital of the West Indies, Mona, Kingston, Jamaica |
| Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders | This phase II clinical trial studies how well treosulfan and fludarabine phosphate with or without low dose radiation before donor stem cell transplantation works in treating patients with nonmalignant (noncancerous) diseases. Hematopoietic cell transplantation has been shown to be curative for many patients with nonmalignant (noncancerous) diseases such as primary immunodeficiency disorders, bone marrow failure syndromes, hemoglobinopathies, and inborn errors of metabolism (metabolic disorders). Powerful chemotherapy drugs and/or radiation are often used to condition the patient before infusion of the new healthy donor cells. The purpose of the conditioning therapy is to destroy the patient's abnormal bone marrow which doesn't work properly in order to make way for the new healthy donor cells which functions normally. Although effective in curing the patient's disease, many hematopoietic cell transplantation regimens use intensive chemotherapy and/or radiation which can be quite toxic, have significant side effects, and can potentially be life-threatening. Investigators are investigating whether a new conditioning regimen that uses less intensive drugs (treosulfan and fludarabine phosphate) with or without low dose radiation results in new blood-forming cells (engraftment) of the new donor cells without increased toxicities in patients with nonmalignant (noncancerous) diseases. | Non-Neoplastic Hematologic and Lymphocytic Disorder | ALL | CHILD, ADULT | Children's Hospital Colorado, Aurora, Colorado, 80045, United States|Oregon Health and Science University, Portland, Oregon, 97239, United States|Vanderbilt University/Ingram Cancer Center, Nashville, Tennessee, 37232, United States|Seattle Children's Hospital, Seattle, Washington, 98105, United States|Fred Hutch/University of Washington Cancer Consortium, Seattle, Washington, 98109, United States|Children's Hospital of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| Hemoglobinopathy Nursing Program and Pediatric Nursing Students | Hemoglobinopathies are the most common life threatening, monogenic disorders in the world. The most common causes of hemoglobinopathies are sickle cell disease and thalassemia. Aim: This study aimed to evaluate the effect of a hemoglobinopathy nursing program on pediatric nursing students' performance. | Hemoglobinopathies|Thalassemia|Sickle Cell Disease|Nurse's Role | ALL | ADULT | Benha University, Banhā, Other, 13511, Egypt |
| Let's Get REAL: Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy | The investigators will conduct a pilot feasibility and efficacy trial of a newly developed family health communication tool (called Let's Get REAL) in increasing youth involvement in real-time stem cell transplant and cellular therapy decisions (SCTCT). The investigators will pilot the intervention among 24 youth and their parents, stratified by youth age (stratum 1, 8-12 years of age and stratum 2, 13-17 years of age). | Hematologic Malignancy|Solid Tumor|Sickle Cell Disease|Aplastic Anemia|Immune Deficiency|Metabolic Disorder | ALL | CHILD, ADULT, OLDER_ADULT | Washington University School of Medicine, Saint Louis, Missouri, 63110, United States |
| A Pilot Study of HSCT for Patients With High-risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen | Hypothesis 1: A novel nonmyeloablative condition regimen will be safe and efficacious in producing stable donor chimerism and cure of severe hemoglobinopathy. Hypothesis 2: Stable donor chimerism will result in amelioration of cerebral vasculopathy, improved cerebral perfusion and neurocognitive function. Specific Aim 1: Study the safety and efficacy of a novel non-toxic conditioning regimen for HSCT for patients with severe hemoglobinopathies and the kinetics of lineage specific chimerism after HSCT We will test our hypothesis that a novel nonmyeloablative condition regimen will be safe and efficacious in producing stable donor chimerism and cure of severe hemoglobinopathy: Specific Aim 2: Optimize the immunosuppressive regimen for HSCT patients through a thorough understanding of the pharmacokinetics of Busulfan (BU) and mycophenolate mofetil (MMF) in the patient population. This will involve: 1. Determine the pharmacokinetics of intravenously and orally administered MMF and intravenous BU in patients receiving HSCT. 2. Determine the relationship of Area under the curve (AUC) of BU and mean trough concentrations of mycophenolic acid (MPA) to engraftment and graft versus host disease (GVHD). 3. Determine the relationship of Area under the curve (AUC) and steady state concentration of BU to engraftment at day 30 and 1 year post HSCT. Specific Aim 3: Study the effect of complete or partial donor chimerism on silent and overt cerebral vasculopathy, and neurocognitive functioning in patients with SCD undergoing HSCT. We will test our hypothesis that stable donor chimerism will result in improvement in cerebral vasculopathy and neurocognitive function. This will include. 1. Determine effect of transplantation silent and overt cerebral vasculopathy by comparison MRA and TCD 1 year after HSCT to pre-HSCT studies. 2. Determine effect on HSCT on neurocognitive function. Specific Aim 4: To determine the rate of T cell immune reconstitution in children with sickle cell disease following myeloablative compared to nonmyeloablative stem cell transplantation, using immunophenotyping assays, CDR3 spectratyping TREC analysis, and measurement of T cell specific donor engraftment. | Sickle Cell Disease|Thalassemia|Hemoglobinopathies | ALL | CHILD, ADULT | Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, 15213, United States |
| Clinical Diagnosis of Teenagers Admitted to Pediatric Departments | This study intends to analyze the clinical characteristics of teenagers admitted to pediatric wards due to organic illness and non organic diagnosis, including patients suffering from chronic diseases and comorbidity. | Anorexia|Thalassemia|Sickle Cell Anemia|Chronic Renal Failure|Suicide Attempt | ALL | CHILD, ADULT | Pediatric Dpt B - Ha'Emek Medical Center, Afula, 18101, Israel |
| Red Blood Cell Exchange Transfusion as a Novel Treatment for GLUT1 Deficiency Syndrome | This proposal is an investigator-initiated, single-site proof of concept trial. Five patients will undergo isovolemic hemodilution-red cell exchange (IHD- RBCx) with up to 10 units of red cell antigens (Rh group, Kell, Duffy, Kidd blood group antigens) matched normal donor red cells to replace a target of 70% of the patient's red cells with donor red cells. The procedure will be performed as an outpatient according to protocols established for sickle cell anemia patients. One of the investigators is an expert on RBCx and will oversee the transfusion. Subjects will be assessed before and after transfusion, and at two months post transfusion. Outcome measures include neurological exam, electroencephalography (EEG), neuropsychological testing, and biochemical assays. | Glucose Transporter Type 1 Deficiency Syndrome|GLUT1DS1 | ALL | CHILD, ADULT | UT Southwestern, Dallas, Texas, 75390, United States |
| Becoming HIV-Exposed, Uninfected Children Born to HIV-Positive Mothers Followed At Bichat Claude Bernard Hospital | "The strategies for the Prevention of Mother-to-Child Transmission of the Human Immunodeficiency Virus (HIV) are very effective and have become common practices in many contexts. Some studies have identified clinical and biological anomalies in a small proportion of infants exposed to antiretrovirals. Beyond 2 years of age, there are few studies with low power describing the long-term consequences of perinatal exposure to antiretrovirals. It is necessary to understand the impact of antiretroviral treatment during the perinatal period in order to improve the management of children born to HIV-positive mothers. Retrospective cohort study with interviews of women living with HIV who were followed for a pregnancy in the Infectious diseases department of Bichat-Claude Bernard Hospital, PARIS, France. After recruitment during a follow-up consultation: * Information regarding the prenatal and perinatal management of HIV and the infant's outcomes up to 12 months will be collected retrospectively through the medical record * Information regarding the child's development and follow-up from the age of 1 to a maximum of 15 years will be gathered retrospectively through a self-administered questionnaire or a questionnaire filled out with assistance during a single 1-hour interview with the mother." | Virus-HIV | FEMALE | ADULT, OLDER_ADULT | Hôpital Bichat-Claude Bernard, Paris, 75018, France |
| Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension | The primary hypothesis of this study is that glutamine supplementation will improve the erythrocyte glutamine/glutamate ratio, a biomarker of oxidative stress, hemolysis and pulmonary hypertension (PH) in sickle cell disease (SCD) and thalassemia (Thal) patients with PH. PH is defined as a tricuspid regurgitant jet velocity (TRV) on Doppler echocardiography \> 2.5 m/s. We also predict that glutamine therapy will increase arginine bioavailability and subsequently alter sickle red cell endothelial interaction that can be identified using endo-PAT technology through nitric oxide (NO) generation, leading to changes in biological markers, and clinical outcome. Specifically our second hypothesis is that oral glutamine will decrease biomarkers of hemolysis and adhesion molecules, and improve the imbalanced arginine-to-ornithine ratio that occurs in hemolytic anemias, leading to improved arginine bioavailability and clinical endpoints of endothelial dysfunction and PH in patients with SCD and Thal. | Pulmonary Hypertension|Sickle Cell Disease|Thalassemia | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital & Research Center Oakland, Oakland, California, 94608, United States |
| Intranasal Sufentanil for Analgesia of Severe Sickle Cell Vaso-occlusive Pain Crisis in the Pediatric | Sickle cell disease (SCD) is characterized by an abnormal hemoglobin, the main protein in the red blood cell. From the first months of life, acute obstruction of the vessels of the microcirculation manifests as intense and unpredictable recurrent episodes of severe pain. In the Emergency Department (ED), patients presenting with a vaso-occlusive crisis (VOC) required a rapid evaluation and administration of pain relief therapies and hydration. this strategy is based on an intranasal (IN) administration of Sufentanil at the initial management of children with VOC, followed by morphine intravenous (IV) relay as soon as possible, compared to the usual care procedure with IV morphine as soon as possible. The hypothesis is that the use of this IN opioid at the beginning of the management of children with VOC can reduce the time before being pain relieved. Indeed, the IN administration make it easier and faster to administer. | Vaso-occlusive Crisis | ALL | CHILD, ADULT | |
| A Follow-up Trial of Proglucamune® in the Treatment of Protective Qi Deficiency, a TCM Condition | This is a follow-up Phase II study of a previously complete pilot trial (Protocol ID: 201875) with the same goal: evaluating the activity of Proglucamune on Protective Qi (PQi) Insufficiency. According to Traditional Chinese Medicine (TCM) principle, Protective Qi is a one specific concept of Qi that provides the vital energy of the body. It works primarily on the body surface as a defensive barrier. In this context, Protective Qi is analogous to anatomical barriers of the innate immune system located for example, at the skin surface and the mucosal surfaces of the respiratory and digestive tract. Individuals with PQi Insufficiency are predisposed to frequent cold and other symptoms caused by invasion of external pathogens ("Wai Xie" or "external evil" in TCM). ß-glucan is a polysaccharide that activates macrophage (Dectin-1) and neutrophil (CR3) receptors, and therefore enhances immune defense at digestive and respiratory mucosa. Clinical trials have shown its immune activity such as preventing upper respiratory tract infection (URTI) and Traveler's diarrhea. Notably, ß-glucan is a component of Ganoderma Lucidum (or Reishi / Lingzhi), one TCM ingredient well-known for improving Qi. Based on this connection, investigators hypothesized that ß-glucan is the active ingredient in Reishi that at least partially accounts for Reishi's activity on Qi. To test our hypothesis, investigators have conducted an uncontrolled pilot trial that investigated the effect of a commercially available, high ß-glucan containing product, Proglucamune®, on PQi status. Proglucamune contains ß-glucan from three different natural sources: Reishi mushroom, Shiitaki mushroom, and Bakers' yeast, each providing ß-glucan that differs slightly in their molecular structure. The data obtained demonstrated a statistically significant effect of Proglucamune on improving PQi in individuals with PQi Insufficiency. The current study is aimed to further determine this effect in a more stringent (placebo-controlled, randomized, and triple-blinded) way. | Protective Qi Insufficiency (a Condition Term From TCM) | ALL | ADULT, OLDER_ADULT | Elegant And Olive Health Clinic, Markham, Ontario, L3R 5V6, Canada |
| Study of the Role of Genetic Modifiers in Hemoglobinopathies | This study will investigate the role of genetic modifiers in hemoglobinopathies through a large-scale, multi-ethnic genome-wide association study (GWAS). | Sickle Cell Disease|Thalassemia, Beta|Thalassemia Alpha|Hemoglobinopathies | ALL | CHILD, ADULT, OLDER_ADULT | Boston Children's Hospital, Boston, Massachusetts, 02115, United States|Lucrecia Paím Maternity, Luanda, Angola|University of Buenos Aires, Buenos Aires, Argentina|University Hospitals Leuven, Leuven, Belgium|Universiti Brunei Darussalam, Brunei, Brunei Darussalam|Centre Hospitalier Monkole, Kinshasa, Congo, The Democratic Republic of the|Larnaca General Hospital, Larnaca, Cyprus|Limassol General Hospital, Limassol, Cyprus|Archbishop Makarios III Hospital, Nicosia, Cyprus|Paphos General Hospital, Paphos, Cyprus|Rigshospitalet, Copenhagen, Denmark|Hippokrateio Hospital of Athens, Athens, Greece|Laiko General Hospital, Athens, Greece|National and Kapodistrian University of Athens, Athens, Greece|General Hospital of Larissa, Larissa, Greece|Emek Medical Centre, Afula, Israel|University of Turin, Turin, Italy|Ampang Hospital, Ampang, Malaysia|Universiti Kebangsaan Malaysia, Bangi, Malaysia|Universiti Sains Malaysia, Kota Bharu, Malaysia|University of Abuja, Abuja, Nigeria|Kaduna State University, Kaduna, Nigeria|Ahmadu Bello University, Zaria, Nigeria|University of Lahore, Lahore, Pakistan|Centro Hospitalar e Universitário de Coimbra, Coimbra, Portugal|Hospital Clínico San Carlos, Madrid, Spain |
| Feasibility Study of Unfractionated Heparin in Acute Chest Syndrome | The purpose of this study is to determine the feasibility of performing a larger multicenter phase III trial to assess the effects of unfractionated heparin (UFH) in acute chest syndrome (ACS). Prespecified feasibility criteria consists of the ability to enroll potential study participants, which includes the timely notification of hospitalized patients with ACS, the capacity to consent eligible individuals, and the ability to appropriately randomize eligible patients within 24 hours of diagnosis. Additional feasibility objectives involve ensuring appropriate eligibility criteria, proper administration of the study drug, and the ability to completely and accurately collect clinical data of interest. The final aim of our pilot study is to provide preliminary data, with respect to treatment effect and variance, to allow sample size calculation in a larger trial given the lack of data available to help guide this process. The investigators hypothesize that the use of UFH in ACS will result in a decrease in the duration of hospitalization and improve other clinical outcomes, such as the duration of hypoxemia and duration of moderate to severe pain. | Acute Chest Syndrome|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | University of Pittsburg Medical Center, Pittsburgh, Pennsylvania, 15213, United States |
| Real-time 3-Dimensional Echocardiography for Assessment of Cardiac Function and Congenital Heart Disease | The propose of this study is to generate normative data of the tree-dimensional echocardiographic(3-DE) measurements for cardiac structure and function in a large cohort of normal infants, children,and adolescents. The investigators also sought to investigate the utility of 3-DE in evaluating infants, children and adolescents with congenital and acquired heart diseases. | Congenital Heart Disease | ALL | CHILD, ADULT | Drexel University College of Medicine/St. Christopher's Hospital for Children, Philadelphia, Pennsylvania, 19140, United States |
| A Research Study of the Effect of Food on Etavopivat in Healthy Participants | The purpose of this study is to evaluate the effect of food on the amount of etavopivat in the bloodstream of healthy participants. Participants will take a single oral dose of etavopivat following a high-fat meal (i.e. fed) and on an empty stomach (i.e fasted) on two separate occasions.The study will last up to 50 days (including screening). | Healthy Volunteers Sickle Cell Disease, Thalassemia | ALL | ADULT | ICON-Salt Lake City, Salt Lake City, Utah, 84124, United States |
| Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies | The major goal of this study is to determine the risks and benefits of bone marrow transplants in patients with severe thalassemia or sickle cell disease. Participation in this project will be for two years. | Sickle Cell Anemia|Hemoglobinopathy|Thalassemia | ALL | CHILD, ADULT | Texas Children's Hospital, Houston, Texas, 77030, United States|The Methodist Hospital, Houston, Texas, 77030, United States |
| Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient With High Risk Hemoglobinopathies | The major goal of this study is to determine the risks and benefits of stem cell transplants in combination with a newer, less toxic conditioning chemotherapy treatment in patients with severe sickle cell disease (SCD) or sickle hemoglobin variants (hemoglobin SC or hemoglobin SB0/+), or homozygous b0/+ thalassemia or severe B0/+ thalassemia variants. Participation in this project will be for one year, with follow up evaluations done every 6 months thereafter for 10 years or until participants are 18 years old. | Sickle Cell Anemia|Hemoglobinopathy|Thalassemia | ALL | CHILD, ADULT, OLDER_ADULT | Texas Children's Hospital, Houston, Texas, 77030, United States|The Methodist Hospital, Houston, Texas, 77030, United States |
| Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant | This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially matched family donor. The study will assess whether immune cells, called T cells, from the family donor, that are specially grown in the laboratory and given back to the patient along with the stem cell transplant can help the immune system recover faster after transplant. As a safety measure these T cells have been programmed with a self-destruct switch so that they can be destroyed if they start to react against tissues (Graft versus host disease). | Acute Lymphoblastic Leukemia|Leukemia, Acute Myeloid (AML), Child|Lymphoma, Non-Hodgkin|Myelodysplastic Syndrome|Primary Immunodeficiency|Anemia, Aplastic|Osteopetrosis|Hemoglobinopathies|Cytopenia|Fanconi Anemia|Diamond Blackfan Anemia|Thalassemia|Anemia, Sickle Cell | ALL | CHILD, ADULT | IRCCS Ospedale Pediatrico Bambino Gesù, Roma, 00161, Italy|Royal Free London NHS Foundation Trust, London, NW3 2QG, United Kingdom|Institute of Child Health & Great Ormond Street Hospital, London, WC1N 1EH, United Kingdom|Great North Children's Hospital, Newcastle Upon Tyne, NE1 4LP, United Kingdom |
| Study of the Function and Muscle Metabolism in Drepanocytose Affected Patient During a Moderate Intensity Exercise | Drepanocytose disease is the first genetic disease in the world. It results in the synthesis of an abnormal hemoglobin (HbS), which in its deoxygenated form, polymerizes and leads to structural changes of red blood cells (RBC) which then take the shape of a sickle, become more fragile, more rigid and less deformable. The fragility of GR sickle causes their mass destruction, leading to chronic anemia (i.e. low levels of GR in the blood) associated with low tissue oxygenation. More rigid and less deformable, sickle GR tend to hang in the microvessels, leading to vaso-occlusive crises (CVO) particularly painful, can cause the failure of certain organs (spleen, kidneys, brain, lung, heart, liver , bone ...) and to life-threatening patients. Preliminary studies conducted on patients with drepanocytose disease (HbSS) have demonstrated changes of muscle tissue indicating a possible failure in the supply and use of oxygen. To date, the translation of this metabolic remodeling in the muscle work is not known. This project's main objective is to evaluate muscle function in drepanocytose attempted patients. We hypothesized that muscle remodeling associated with sickle cell disease have a functional impact on strength and muscle metabolism. The main objective is to characterize the maximal voluntary plantar flexor muscles. The criteria for these targets will be based on the comparison between healthy subjects, carriers of the sickle cell trait (HbAS) and sickle cell patients (HbSS) oxygen saturation. The strictly non-invasive approach proposed in this project will study the functional parameters of the muscle of sickle cell disease and the possible link with the clinical manifestations of the disease, including vaso-occlusive crisis, in which tissue oxygenation and pH plays a major role. | Drepanocytose | ALL | ADULT, OLDER_ADULT | Assistance Publique Hôpitaux de Marseille, Marseille, 13354, France |
| Stem Cell Transplant for Hemoglobinopathy | This study tests the clinical outcomes of one of two preparative regimens (determined by available donor source) in patients with non-malignant hemoglobinopathies. The researchers hypothesize that these regimens will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease. Regimen A2 has replaced Regimen A in this study. Two patients were treated on Regimen A but did not have evidence of initial engraftment thus triggering the stopping rule for that arm of this study. | Sickle Cell Disease|Thalassemia|Severe Congenital Neutropenia|Diamond-Blackfan Anemia|Shwachman-Diamond Syndrome | ALL | CHILD, ADULT | Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, 55455, United States |
| Effect of Plasmodium Falciparum Exposure and Sickle Cell Trait on Infection Rates and Kinetics After IV Administration of PfSPZ Challenge | The study is designed to establish infectivity of Plasmodium falciparum sporozoites (PfSPZ) via intravenous (IV) administration in three groups with different malaria immunity-status: 1. Adults with a history of lifelong malaria exposure without sickle cell trait (HbAA) 2. Adults with a history of lifelong malaria exposure with sickle cell trait (HbAS) 3. Adults without previous malaria episodes without sickle cell trait (HbAA) Initially a dose of 3,200 PfSPZ will be given and the time until thick blood smear positivity after challenge will be assessed. If in any of the groups with a history of lifelong malaria exposure, 50% or less of individuals become thick blood smear positive during the 28 days post injection of PfSPZ Challenge, the dose will be increased 4-fold to 12,800 PfSPZ in this group. | Plasmodium Falciparum Malaria|Malaria | ALL | ADULT | Centre de Recherches Médicales de Lambaréné, Lambaréné, BP 118, Gabon |
| LEARNER- Low dosE AspiRiN prEterm tRial (Angola) | This study is being conducted to evaluate the safety and effect of starting daily use of low dose (100 mg) aspirin in pregnant women with sickle cell disease, who are being followed in two county hospitals in Angola, in the first trimester versus the second trimester of the gestational period. | Sickle Cell Disease|Pregnancy Related|Pregnancy Complications|Pre-Eclampsia | FEMALE | CHILD, ADULT, OLDER_ADULT | Instituto Nacional de Investigação Em Saúde, Luanda, 00000, Angola |
| CIBMTR Research Database | The primary purpose of the Research Database is to have a comprehensive source of observational data that can be used to study HSC transplantation and cellular therapies. A secondary purpose of the Research Database is to have a comprehensive source of data to study marrow toxic injuries. Objectives: To learn more about what makes stem cell transplants and cellular therapies work well such as: * Determine how well recipients recover from their transplants or cellular therapy; * Determine how recovery after a transplant or cellular therapy can be improved; * Determine how a donor's or recipient's genetics impact recipient recovery after a transplant or cellular therapy; * Determine how access to transplant or cellular therapy for different groups of patients can be improved; * Determine how well donors recover from the collection procedures. | Autologous Stem Cell Transplantation|Allogeneic Stem Cell Transplantation|Solid Tumors|Blood Cancers|CAR-T|Gene Therapy|Non-malignant Disease | ALL | CHILD, ADULT, OLDER_ADULT | Center for International Blood and Marrow Transplant Research, Minneapolis, Minnesota, 55413, United States|Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| Investigation of the Genetics of Hematologic Diseases | The purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment of blood diseases. This is not a therapeutic or diagnostic protocol for clinical purposes. Blood, bone marrow, hair follicles, nail clippings, urine, saliva and buccal swabs, left over tissue, as well as health information will be used to study and learn about blood diseases by using genetic and/or genomic research. In general, genetic research studies specific genes of an individual; genomic research studies the complete genetic makeup of an individual. It is not known why many people have blood diseases, because not all genes causing these diseases have been found. It is also not known why some people with the same disease are sicker than others, but this may be related to their genes. By studying the genomes in individuals with blood diseases and their family members, the investigators hope to learn more about how diseases develop and respond to treatment which may provide new and better ways to diagnose and treat blood diseases. Primary Objective: * Establish a repository of DNA and cryopreserved blood cells with linked clinical information from individuals with non-malignant blood diseases and biologically-related family members, in conjunction with the existing St. Jude biorepository, to conduct genomic and functional studies to facilitate secondary objectives. Secondary Objectives: * Utilize next generation genomic sequencing technologies to Identify novel genetic alternations that associate with disease status in individuals with unexplained non-malignant blood diseases. * Use genomic approaches to identify modifier genes in individuals with defined monogenic non-malignant blood diseases. * Use genomic approaches to identify genetic variants associated with treatment outcomes and toxicities for individuals with non-malignant blood disease. * Use single cell genomics, transcriptomics, proteomics and metabolomics to investigate biomarkers for disease progression, sickle cell disease (SCD) pain events and the long-term cellular and molecular effects of hydroxyurea therapy. * Using longitudinal assessment of clinical and genetic, study the long-term outcomes and evolving genetic changes in non-malignant blood diseases. Exploratory Objectives * Determine whether analysis of select patient-derived bone marrow hematopoietic progenitor/stem (HSPC) cells or induced pluripotent stem (iPS) cells can recapitulate genotype-phenotype relationships and provide insight into disease mechanisms. * Determine whether analysis of circulating mature blood cells and their progenitors from selected patients with suspected or proven genetic hematological disorders can recapitulate genotype-phenotype relationships and provide insight into disease mechanisms. | Bone Marrow Failure Syndromes|Erythrocyte Disorder|Leukocyte Disorder|Hemostasis|Blood Coagulation Disorder|Sickle Cell Disease|Dyskeratosis Congenita|Diamond-Blackfan Anemia|Congenital Thrombocytopenia|Severe Congenital Neutropenia|Fanconi Anemia|Myelodysplastic Syndromes|Myeloproliferative Diseases | ALL | CHILD, ADULT, OLDER_ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Parvovirus B19 Infection in Hereditary Hemolytic Anemias Patients | Although many studies investigated the prevalence and manifestations of HPV-B19 infection in patients with sickle cell anemia (SCA), thalassemia, and hereditary spherocytosis (HS) separately, there is limited information about the extent to which HPV-B19 infection leads to severe complications and chronic infection. | Parvovirus B19 Infection | ALL | CHILD, ADULT, OLDER_ADULT | , Faculty of Medicine, Sohag University, Sohag, 82524, Egypt |
| ERYthrocyte Morphology Using Flow Imaging on ImageSTREAM Cytometer | Erythrocyte morphology analysis is a key step in the diagnosis flowchart of anemia. It is performed on a peripheral blood smear after May Grümwald Giemsa staining. In the context of hemolytic anemias for example, it allows the recognition of therapeutic emergencies such as sickle cell disease crisis, malaria-induced hemolysis and thrombotic microangiopathy, the latter being characterized by the presence of schistocytes and justifying an immediate clinical care. However, cytological analysis of erythrocyte morphology requires pre-analytical interventions (smear spreading + staining), the quality of which determines the accuracy of the result. Moreover, it requires a good cytological expertise and may be sometimes subjective. For several years, alternative methods for erythrocyte morphology evaluation have been developed, based on automated hematology machines or automated microscopy. Nevertheless, none of them has yet proven itself in comparison with cytology, especially in the diagnosis of thrombotic microangiopathies. By combining the advantages of flow cytometry and microscopy, flow imaging appears to be a promising technology for the diagnosis of anemias: it does not require any pre-analytical intervention, does not require any spreading and analyzes a large number of events. Moreover, it can be coupled with artificial intelligence via the generation of an apprenticeship by the constitution of a large image data base, which then allows the recognition of the different red blood cells morphologies without human eyes. The objective of this study is to build a data base containing the main red blood cell morphologies relevant in anemia, and to validate it through a comparison in anemic patients of erythrocyte morphological assessment either directly on whole blood by flow imaging or routinely by cytological analysis of peripheral blood smear after by a trained operator. | Red Blood Cells|Schistocytes|Artificial Intelligence | ALL | ADULT, OLDER_ADULT | CHU Amiens Picardie, Amiens, Picardie, 80054, France |
| Measures of Respiratory Health (MRH) | The Lung Clearance Index, measured by multiple breath washout, is a measure of lung function that is considered a research tool in Canada as the device used to measure it is not approved by Health Canada. The study will assess lung function in patients undergoing routine lung function testing for clinical indications (Cystic Fibrosis and Other Respiratory Diseases). In addition, healthy controls of different ages will be asked to perform this lung function test to gain reference data that can be used to interpret LCI in patients with lung disease. | Cystic Fibrosis|Asthma|Sickle Cell Anemia|Bronchiolitis Obliterans | ALL | CHILD, ADULT | The Hospital for Sick Children, Toronto, Ontario, M5G 1X8, Canada |
| The Effect of Pharmaceutical Grade L-glutamine (Endari) on Glycemic Control in Patients With Diabetes Mellitus Type II | The study is an initial non-blinded, non-placebo controlled trial to determine the efficacy of L-glutamine in lowering blood sugar in patients with diabetes mellitus type II without sickle cell anemia. | Diabetes Mellitus, Type 2 | ALL | ADULT, OLDER_ADULT | |
| Surveillance of Influenza Virus Shedding and Immunologic Response in Immunocompromised Children and Young Adults | Influenza virus infections are a major cause of morbidity and mortality. The limited existing knowledge about the impact of influenza in immunocompromised patients suggests that they are at increased risk of influenza virus acquisition, of developing complications and of prolonged illness and viral shedding. However, some other data about the effect of antiviral agents on the infection course, and risk of resistance in immunocompromised children are lacking. The emergence of the pandemic H1N1 swine-origin influenza A virus has generated an additional need to study the epidemiology, clinical course and outcome of influenza infections in immunocompromised children. This study proposed to conduct a prospective observational clinical study to answer these questions. | Influenza Virus | ALL | CHILD, ADULT | St . Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Autologous Testicular Tissue Transplantation | Freezing testicular tissue of prepubertal boys is a method for preserving spermatogonial stem cells (SSCs) in case of imminent gonadotoxic treatment during childhood. In case of total azoospermia in adulthood and presence of a childwish, the investigators intend to perform the first in men autologous testicular tissue transplantation to restore fertility. | Cancer|Sickle Cell Thalassemia|Hematologic Diseases | MALE | ADULT | UZ Brussel Centre for Reproductive Medicine, Brussels, 1090, Belgium |
| Study Of Efficacy,Safety of Combined Deferasirox and Deferiprone Versus Combined Deferiprone and Desferal In Conditions of Iron Overload | Interventional Allocation: Randomized Endpoint Classification: Safety/Efficacy Study of combined chelation therapy Masking: Open Label Primary Purpose: Treatment of transfusional iron overload Primary Outcome Measures: • The primary outcome measure is to assess efficacy in lowering serum ferritin level(the change in serum ferritin compared to baseline) with combining DFP and deferasirox compared to combined DFP and DFO in conditions with severe chronic iron overload; showing an up-trend of SF over previous 12 months on single chelator. Secondary Outcome Measures: • The secondary outcome measure is to determine the number of patients who will develop adverse events in order to assess safety upon administering the drugs in combination (DFP and DFX) compared to the combination of DFO and DFP. | Beta-thalassemia Major|Sickle Cell Disease|Iron Hemosiderosis | ALL | CHILD, ADULT | Pediatric Hematology clinic, Ain Shams University, Cairo, Egypt |
| Efficacy/Safety Study of ACTIQ® for Opioid-Tolerant Children and Adolescents With Breakthrough Pain (BTP) | The primary objective of the study is to evaluate the efficacy of ACTIQ treatment for the management of breakthrough pain (BTP) compared to placebo treatment in children with cancer and non-cancer pain who are receiving around-the-clock (ATC) opioid therapy and who require additional therapy for BTP episodes. This will be determined by the analysis of the pain intensity (PI), measured by the Faces Pain Scale-Revised (FPS-R) administered 15 minutes after the start of each unit of study drug with an optimal ACTIQ dosage. | Cancer|Breakthrough Pain | ALL | CHILD | Children's Hospital of Arkansas, Little Rock, Arkansas, 72202, United States|UCLA Pediatric Pain Program, Los Angeles, California, 90095, United States|Childrens Hospital of Orange, Orange, California, 92868, United States|Lucille Packard Childrens Hospital, Palo Alto, California, 94304, United States|Connecticut Childrens Medical Center, Hartford, Connecticut, 06106, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States|Nemours Childrens Clinic, Jacksonville, Florida, 32207, United States|St. Joseph's Children's Hospital, Tampa, Florida, 33607, United States|Scottish Rite Children's Medical Center, Atlanta, Georgia, 30342, United States|Kapi'olani Medical Center, Honolulu, Hawaii, 96826, United States|Children's Memorial Center, Chicago, Illinois, 60614, United States|Children's Memorial Hospital, Chicago, Illinois, 60614, United States|University Hospitals of Iowa, Iowa City, Iowa, 52242, United States|Cardinal Glennon Children's Hospital, St. Louis, Missouri, 63104, United States|Hackensack University Medical Center, Hackensack, New Jersey, 07601, United States|Cancer Institute of New Jersey, New Brunswick, New Jersey, 08901, United States|University of New Mexico, Albuquerque, New Mexico, 87131, United States|Children's Hospital at Montefiore, Bronx, New York, 10467, United States|SUNY Upstate Medical University, Syracuse, New York, 13210, United States|Duke University Hospital, Durham, North Carolina, 27710, United States|Akron Children's Hospital, Akron, Ohio, 44308, United States|Tod Children's Hospital, Youngstown, Ohio, 44501, United States|Milton S Hershey Medical Center, Hershey, Pennsylvania, 17033, United States|St. Christopher's Hospital for Children, Philadelphia, Pennsylvania, 19134, United States|Vanderbilt University Medical, Nashville, Tennessee, 37232, United States|Children's Cancer and Blood Disorders Center, San Antonio, Texas, 78207, United States|Methodist Hospital, San Antonio, Texas, 78229, United States|Sacred Heart Medical Center, Spokane, Washington, 99204, United States|West Virginia University, Morgantown, West Virginia, 99204, United States|Childrens Hospital of Wisconsin, Milwaukee, Wisconsin, 53201, United States|San Jorge Childrens Medical, San Juan, 00912, Puerto Rico |
| Uterine Irrigation Method in Infertility Patients Who Require PGD | The study will involve up to 30 pairs of male and female sexually intimate partners who are carriers for a genetic disease (e.g Sickle Cell Disease or Thalassemia) and at high risk of transmitting the gene. The female partner will be superovulated to mature multiple oocytes which can be fertilized, inseminated with her partner's sperm through intra-uterine insemination (IUI). Four to six days after IUI, the female partner will undergo a non-surgical uterine lavage procedure to recover preimplantation embryos. | Pre-Implantation Embryo Recovery|Genetic Diseases, Inborn | FEMALE | ADULT | Istanbul University School of Medicine, Istanbul, 34093, Turkey |
| Developing a Program for Transition to Adult Care for Youth With Chronic Conditions : a Mixed Method Study | Investigation in 3 steps the transition of young people with chronic diseases. FIRST STEP: qualitative survey. Interviews with carers, young people and parents. Chronic Diseases studied: epilepsy, HIV, sickle cell disease, obesity. SECOND STEP: Delphi consensus survey. Vote on internet : carers, patients' associations, young people and family. Chronic Diseases studied: all. THIRD STEP: validation survey. Vote on internet : young people. Chronic Diseases studied: all. | Transition Care | ALL | CHILD, ADULT, OLDER_ADULT | Robert Debré Hospital, Paris, 75019, France |
| Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study | This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study. | Acute Lymphoblastic Leukemia|Leukemia, Acute Myeloid (AML), Child|Lymphoma, Non-Hodgkin|Myelodysplastic Syndromes|Primary Immunodeficiency|Anemia, Aplastic|Hemoglobinopathies|Cytopenia|Fanconi Anemia|Diamond Blackfan Anemia|Thalassemia|Anemia, Sickle Cell | ALL | CHILD, ADULT | IRCCS Ospedale Pediatrico Bambino Gesù, Rome, 00161, Italy |
| Extension Study of ACTIQ Treatment for Children and Adolescents With Breakthrough Pain | The objective of the study is to monitor the safety (adverse event data) of longer-term use of ACTIQ (Oral Transmucosal Fentanyl Citrate \[OTFC\]) treatment in children with pain associated with cancer, sickle cell disease, or severe burns and breakthrough pain (BTP) who are receiving around the clock (ATC) opioid therapy. | Pain|Cancer|Sickle Cell Anemia|Severe Burns | ALL | CHILD | Children's Hospital of Arkansas, Little Rock, Arkansas, 72202, United States|Childrens Hospital of Orange, Orange, California, 92868, United States|Lucille Packard Childresns Hospital, Palo Alto, California, 94304, United States|Connecticut Childrens Medical, Hartford, Connecticut, 06106, United States|Childrens National Medical Center, Washington, District of Columbia, 20010, United States|Nemours Childrens Clinc, Jacksonville, Florida, 32207, United States|St. Joseph's Childrens Hopsital, Tampa, Florida, 33660, United States|Kapi'olani Medical Center, Honolulu, Hawaii, 96826, United States|University Hospitals of Iowa, Iowa City, Iowa, 52242, United States|David Center for Childrens Pain and Palliative Care, Hackensack, New Jersey, 07601, United States|Cancer Institute of New Jersey, New Brunswick, New Jersey, 08901, United States|University of New Mexico, Albuquerque, New Mexico, 87131, United States|Children's Hospital at Montefiore, Bronx, New York, 10467, United States|Scottish Rite Children's Medical Center, Syracuse, New York, 13210, United States|SUNY Upstate Medical University, Syracuse, New York, 13210, United States|Duke University Hospital, Durham, North Carolina, 27710, United States|Akron Children's Hospital, Akron, Ohio, 44308, United States|Tod Childrens Hospital, Youngstown, Ohio, 44501, United States|Milton S Hershey Medical Center, Hershey, Pennsylvania, 17033, United States|St. Christopher's Hospital for Children, Philadelphia, Pennsylvania, 19134, United States|Vanderbilt University Medical Center, Nashville, Tennessee, 37232, United States|Children's Cancer and Blood Disorders Center, San Antonio, Texas, 78207, United States|Methodist Hospital, San Antonio, Texas, 78229, United States|Sacred Heart Medical Center, Spokane, Washington, 99204, United States|West Virginia Univeristy, Morgantown, West Virginia, 99204, United States|Childrens Hospital of Wisconsin, Milwaukee, Wisconsin, 53201, United States|Iwk Health Center, Halifax, Nova Scotia, B3J3G9, Canada|San Jorge Childrens Medical, San Juan, 00912, Puerto Rico |
| Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant | This trial will see if extended abatacept administration (combined with a standard regimen of tacrolimus and mycophenolate mofetil) will prevent acute and chronic graft-versus-host disease (GVHD) in children and adolescents receiving unrelated donor (URD) hematopoietic stem cell transplantation (HSCT), without compromising their engraftment or reconstitution of protective immunity to infection. The study will enroll 30 pediatric patients with serious non-malignant hematologic diseases (NMHD) undergoing URD HSCT. The trial will include patients with 7/8 donors and those with 8/8 (matched) donors. All participants will receive 8 doses of abatacept. Recruitment is expected to last for about 2 years and participants will be followed for up to 3 years. | Graft Versus Host Disease | ALL | CHILD, ADULT | Children's of Alabama, Birmingham, Alabama, 35233, United States|Nemours/Alfred I. DuPont Hospital for Children, Wilmington, Delaware, 19803, United States|Childrens Healthcare of Atlanta, Atlanta, Georgia, 30322, United States|Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, 60611, United States|Dana-Farber Cancer Institute, Boston, Massachusetts, 02215, United States|University of Mississippi Medical Center, Children's Center for Cancer and Blood Disorders, Jackson, Mississippi, 39216, United States|Hackensack Meridian Health, Hackensack, New Jersey, 07601, United States|Oishei Children's Hospital, Buffalo, New York, 14203, United States|Columbia University Irving Medical Center, New York, New York, 10032, United States |
| Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants | The objective of this study is to make T-cell depleted stem cells from a family member who is a half match (haplo-identical) available on an expanded access basis to patients receiving one or two unrelated cord blood transplants who are at a higher risk of not engrafting in a safe amount of time. The purpose of the related stem cells is the give the bone marrow a "jump start" towards recovery. Ultimately, the cord blood cells will grow and permanently rescue the bone marrow. | Hematologic Malignancies|Inborn Errors of Metabolism Disorders|Immune Deficiencies | ALL | CHILD, ADULT, OLDER_ADULT | Duke University Medical Center, Durham, North Carolina, 27710, United States |
| Therapeutic Anticoagulation Strategy for Acute Chest Syndrome | Acute Chest Syndrome (ACS) is a pulmonary complication of sickle cell disease (SCD) representing the leading cause of death and the second cause of hospitalization among adult patients. Pulmonary vaso-occlusion is one of the main pathophysiologic hypotheses during ACS. Our hypothesis is that therapeutic anticoagulation may reduce the severity of ACS via the alleviation of pulmonary thrombosis. The main objective of this prospective, randomized, double-blind study is to test the efficacy and safety of a curative anticoagulation strategy during ACS. The main efficacy endpoint is time to ACS resolution. The main safety endpoint is number of major bleedings. A thoracic CT scan will be performed to check for pulmonary artery thrombosis. If the CT scan is positive (thrombosis within a large elastic artery), the patient will not be randomized and will be treated with a curative anticoagulation. If the CT scan is negative, the patient will be randomized to receive subcutaneous anticoagulation with low molecular weight heparin (tinzaparin) either at a curative dose (175 Unit International (UI)/kg/day for 7 days) or at a prophylactic dose (4500 UI/day). | Anemia|Sickle Cell|Acute Chest Syndrome|Low-Molecular-Weight Heparin | ALL | ADULT, OLDER_ADULT | Henri Mondor Hospital, Creteil, 94010, France |
| Point-of-Delivery Prenatal Test Results Through mHealth to Improve Birth Outcome | The investigators propose to adopt sustainable community networks (in this case churches) to implement an integrated community-based screening that incorporates mobile health technology (mHealth) to make prenatal test results available at the point-of-delivery to guide medical management. | HIV|Sickle Cell Disease|Hepatitis B | FEMALE | ADULT, OLDER_ADULT | Catholic Caritas Foundation, Makurdi, Benue, Nigeria |
| A Research Study Looking at a Single Dose of Etavopivat in Healthy Chinese Participants | The study tests a new medicine called etavopivat in healthy Chinese participants. During the study, the participant will be given 1 dose of the study medicine. A dose is 2 tablets which are swallowed together with a cup of water. The study doctor will measure how much of the study medicine is in the blood as time passes. After the dose is given, the study will last for 7 to 9 days. The participants will attend a screening visit 2 to 28 days before they are given the study medicine. The participant will have 6 clinic visits in total. As part of the study, participant will stay overnight at the clinic for 3 nights. In some cases the doctor may decide that participant need to stay more nights. Participant will have blood tests and other health assessments at all 6 visits. Women cannot take part if pregnant, breast-feeding or plan to get pregnant during the study period. If the participant do take part in the study, participant will need to refrain from certain activities and behaviours for up to 3 months before taking the study medicine. Participant will also need to go without food and drinks other than water in the 10 hours before and 4 hours after taking the medicine. | Healthy Volunteers|Sickle Cell Disease|Thalassemia | ALL | ADULT | Beijing Hospital, Beijing, Beijing, 100730, China |
| A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders | To investigate the safety of partially matched related human placental-derived stem cells (HPDSC) administered in conjunction with umbilical cord blood (UCB) stem cells from the same donor in subjects with various malignant or nonmalignant disorders potentially curable with stem cell transplantation and to assess potential restoration of normal hematopoiesis and immune function in subjects with these disorders | Hematologic Malignancies | ALL | CHILD, ADULT | Louisiana State University Children's Hospital, New Orleans, Louisiana, 70118, United States |
| Study of GBT021601 in Participants With Renal Impairment | Renal Impairment study of GBT021601. | Renal Impairment | ALL | ADULT, OLDER_ADULT | Orange County Research Center, Lake Forest, California, 92630, United States|Orange County Research Center, Tustin, California, 92780, United States|Advanced Pharma CR, LLC, Miami, Florida, 33147, United States|Nucleus Network, Saint Paul, Minnesota, 55114, United States |
| RNF213 Variants and Collateral Vessels in Moyamoya Disease | The purpose of this study is to detect the association between RNF213 variants and collateral vessels in patients with moyamoya disease. | Moyamoya Disease | ALL | CHILD, ADULT | Beijing Tiantan Hospital Capital Medical University, Beijing, Beijing, China |
| Exercise in Child Health | This study is a cooperative investigation funded by the NIH. The project is a collaboration among three major NIH Clinical Translational Science Awardees: 1) UCI (lead site with its affiliate CHOC), 2) Northwestern University (with its affiliate Lurie Children's Hospital), and 3) USC (with its affiliate Children's Hospital of Los Angeles). There is an increasing number of children who, through medical advances, now survive diseases and conditions that were once fatal, but which remain chronic and debilitating. A major challenge to improve both the immediate and long term care and health of such children has been the gap in our understanding of how to assess the biological effects of exercise. Like otherwise healthy children, children with chronic diseases and disabilities want to be physically active. The challenge is to determine what constitutes safe and beneficial level of physical activity when the underlying disease or condition \[e.g., cystic fibrosis (CF) or sickle cell disease (SCD)\] imposes physiological constraints on exercise that are not present in otherwise healthy children. Current exercise testing protocols were based on studies of athletes and high performing healthy individuals and were designed to test limits of performance at very high-intensity, unphysiological, maximal effort. These approaches are not optimal for children and adolescents with disease and disability. This project (REACH-Revamping Exercise Assessment in Child Health) is designed to address this gap. Cohorts of children will be identified with two major genetic diseases (CF and SCD) and measure exercise responses annually as they progress from early puberty to mid or late puberty over a 3-4year period. In addition, in the light of the pandemic, a group of children will be added who were affected by SARS-CoV-2 and investigate their responses to exercise. SARS-CoV-2 has similar long-term symptoms than CF and SCD have. Novel approaches to assessing physiological responses to exercise using advanced data analytics will be examined in relation to metrics of habitual physical activity, circulating biomarkers of inflammation and growth, leukocyte gene expression, and the impact of the underlying CF, SCD or SARS-CoV-2 condition. The data from this study will help to develop a toolkit of innovative metrics for exercise testing that will be made available to the research and clinical community. | Cystic Fibrosis|Sickle Cell Disease|SARS CoV 2 Infection | ALL | CHILD | University of California, Irvine, Irvine, California, 92697, United States |
| Bone Marrow Transplant With Abatacept for Non-Malignant Diseases | This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem cell transplant for serious non-malignant diseases as well as to assess the immunological effects of abatacept. Participants will be followed for 2 years. | Hurler Syndrome|Fanconi Anemia|Glanzmann Thrombasthenia|Wiskott-Aldrich Syndrome|Chronic Granulomatous Disease|Severe Congenital Neutropenia|Leukocyte Adhesion Deficiency|Shwachman-Diamond Syndrome|Diamond-Blackfan Anemia|Dyskeratosis-congenita|Chediak-Higashi Syndrome|Severe Aplastic Anemia|Thalassemia Major|Hemophagocytic Lymphohistiocytosis|Sickle Cell Disease | ALL | CHILD, ADULT | Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States |
| Non-Invasive Diagnosis of Pulmonary Vascular Disease Using Inhaled 129Xe Magnetic Resonance Imaging | This study seeks to deploy several forms of 129Xe MRI contrast as well as emerging conventional proton MRI technqiues for imaging lung structure and perfusion. Specifically, the 129Xe MRI scans will provide 3D images of ventilation and gas exchange, and spectroscopic indices will be evaluated to test gas exchange dynamics with high temporal resolution. The conventional 1H MRI scans will include a free-breathing ultra-short echo time (UTE) scan that provides images similar to that of a CT scan. In addition, to characterize perfusion and vascular dimensions directly, patients will undergo a gadolinium-enhanced perfusion scan. | Pulmonary Vascular Disease | ALL | ADULT, OLDER_ADULT | Duke University Medical Center, Durham, North Carolina, 27710, United States |
| Non-specific Effects of Vaccination on Mortality and Morbidity | It has long been recognized that the positive effects of vaccination on childhood mortality cannot be solely attributed to a decline in the disease targeted by the vaccine. These so-called non-specific effects of vaccination have so far mostly been linked to mortality. However, it has been suggested that non-specific effects may also effect morbidity and nutritional status. This study aims to further explore the correlation between vaccination, susceptibility to infectious diseases (particularly malaria and bacterial infections), nutritional status and immunity. With this prospective cross sectional study among healthy individuals in rural west-Africa we aim to address several research questions at the same time. This study will assess the influence of (time-point of) vaccination on morbidity, mortality and immune status among healthy individuals in a rural sub-Saharan African setting. Secondly, to explore the prevalence of subclinical malaria, iron deficiency anemia, sickle cell anemia and thallasemia among a healthy rural sub-Saharan African population. And finally to assess normal hemocytometry values among a healthy rural sub-Saharan African population. | Vaccine Reaction|Anemia|Malaria,Falciparum|Salmonella Bacteraemia | ALL | CHILD, ADULT, OLDER_ADULT | Clinical Research Unit of Nanoro, Nanoro, Boulkiemedé, Burkina Faso |
| Measurement of Brain Perfusion by a MR Perfusion Imaging Called eASL in Children's Cerebral Arteriopathies. | The MR sequence called MR-ASL is used to measure cerebral perfusion in children. This ASL sequence is used with a unique post-labeling delay (PLD) due to the technical impossibility of setting different post-labeling delays. The use of a single post-labeling, chosen by the pediatric radiology department of the Necker hospital, optimal in children without arteriopathy, may not be suitable for the lengthened arterial transit time of the spins marked in the pathological carotid network of a child with arterial disease. Recently, ASL sequences with multiple delays (multi-PLD, called eASL) have been developed to overcome this limitation in arterial disease. To date, their use in the pre- and post-treatment evaluation of a child with acute or chronic arterial disease has not been evaluated. The study hypothesis is that this eASL sequence is more efficient than single-delay ASL in measuring cerebral perfusion. The study will be performed in a population of children with acute or chronic arterial disease. | Cerebral Arteriopathy | ALL | CHILD, ADULT | Hôpital Necker-Enfants Malades, Paris, 75015, France |
| Patient-Empowered Mobile Technology in Hospitalized Patients (TRU-PAIN) | The purpose of this study is to learn more about the ways in which mobile technology can be integrated into inpatient care to help better track pain levels using mobile technology of patients with sickle cell disease, oncology patients, and bone marrow transplant patients. The study will assess whether or not daily mobile monitoring with wearable accelerometers (devices that detect movement as well as heart-rate) to monitor and manage medical treatments can have a lasting positive impact on outcomes in patients with chronic diseases. The investigator hopes to learn more about the ways in which mobile technology can be integrated into inpatient care. Specifically, the investigator is looking to help patients better track their pain, use wearable technology to track physiological measures (for example, heart rate, sleep quantity and quality), and integrate these data points into the medical care of patients by providing the information to providers. This study will first gather information regarding the feasibility and acceptability of the use of technology on the inpatient unit. This will help the study team to refine the technology of the mobile app and logistics of integration. Following this, the investigator will complete a second phase of the study, during which select patients will pilot the intervention. This will be followed by the third and final phase, during which patients will be randomly assigned to the active intervention or standard of care. This phase approach will enable the study team to refine the intervention, relying on the feedback from patients and providers, and subsequently test its utility compared to standard of care through random assignment. | Pain | ALL | CHILD, ADULT, OLDER_ADULT | Duke University, Durham, North Carolina, 27710, United States |
| Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders | The primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (\>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimens in pediatric patients \< 21 years receiving cord blood transplantation for non-malignant disorders. | Non Malignant Disorders|Immunodeficiencies|Congenital Marrow Failures|Hemoglobinopathies|Inborn Errors of Metabolism|Sickle Cell|Thalassemia|Lysosomal Storage Disease | ALL | CHILD, ADULT | Duke University Medical Center Pediatric Blood and Marrow Transplant Program, Durham, North Carolina, 27705, United States |
| Hospital-Based Program for Treatment of Severe Cardiopulmonary Disease With Inhaled Nitric Oxide | The purpose of this program is to evaluate the logistic issues and patient requirements for chronic pulsed INOmax delivery in ambulatory, home-care patients. To understand patient needs, patients with a variety of underlying diseases will be included. Safety of chronic therapy will be monitored by serial measurements of methemoglobin, platelet function assay and reported adverse events. | Pulmonary Hypertension|Lung Disease|Sickle Cell Disease|Cardiac Transplant|Lung Transplant | ALL | CHILD, ADULT, OLDER_ADULT | The Children's Hospital, Denver, Colorado, 80262, United States|University of Colorado Hospital, Denver, Colorado, 80262, United States|Rhode Island Hospital, Providence, Rhode Island, 02903, United States |
| A Retrospective Epidemiological Study on Pneumococcal Infection in Children in Ministry of Health Hospital at EHSA Region in the Eastern Province | The purpose of this study is to establish retrospectively the incidence rates of invasive pneumococcal disease in children aged less than 5 years. The investigators allocate the El Hasa region for the study because it has higher incidence of sickle cell anemia. The study will include data from 2003 to 2007(before introduction of the vaccine) and then for 2008 and 2009 (after introduction of the vaccine). | Pneumococcal Infection | ALL | CHILD | |
| Validating Pain Scales in Children and Young Adults | Background: - Assessing pain levels is important to improve treatments for different illnesses. Most pain rating scales are used to determine pain levels in adults. Pain is also a common symptom among children who have cancer. Those who have genetic conditions that may lead to cancer may also have pain symptoms. However, the pain scales used for adults have not been fully tested in children and young adults. As a result, they may not be as accurate. Researchers want to test pain rating scales in children and young adults who have cancer and genetic conditions that can lead to cancer. Objectives: - To study the effectiveness of pain rating scales given to children and adults with Sickle Cell Disease (SCD),cancer, and related genetic conditions. Eligibility: - Adults 18 and 34 years of age and older who have SCD, cancer, or other genetic conditions that can lead to cancer. Design: * Participants with SCD, cancer or related genetic conditions will fill out four questionnaires. These questionnaires will ask about pain levels and how much pain interferes with daily life. * Pain treatments will not be provided as part of this study. | Sickle Cell Disease|Solid Tumor|Leukemia|Neurofibromatosis | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions | We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research. We make it easy for patients to contribute to research for medical conditions that matter most to them, regardless of their location or ability to travel. | All Diagnosed Health Conditions|ADD/ADHD|Alopecia Areata|Ankylosing Spondylitis|Asthma|Atopic Dermatitis Eczema|Beta Thalassemia|Bipolar Disorder|Breast Cancer|Celiac Disease|Cervical Cancer|Chronic Inflammatory Demyelinating Polyneuropathy|Chronic Kidney Diseases|Chronic Obstructive Pulmonary Disease|Colon Cancer|Colorectal Cancer|Crohn's Disease|Cystic Fibrosis|Depression|Diabetes Mellitus|Duchenne Muscular Dystrophy|Endometriosis|Epilepsy|Facioscapulohumeral Muscular Dystrophy|G6PD Deficiency|General Anxiety Disorder|Hepatitis B|Hereditary Hemorrhagic Telangiectasia|HIV/AIDS|Human Papilloma Virus|Huntington's Disease|Idiopathic Thrombocytopenic Purpura|Insomnia|Kidney Cancer|Leukemia|Lung Cancer|Lupus Nephritis|Lymphoma|Melanoma|Multiple Myeloma|Multiple Sclerosis|Myositis|Myotonic Dystrophy|Ovarian Cancer|Pancreatic Cancer|Parkinson's Disease|Polycystic Kidney Diseases|Prostate Cancer|Psoriasis|Psoriatic Arthritis|Rosacea|Scleroderma|Sickle Cell Anemia|Sickle Cell Trait|Sjogren's Syndrome|Skin Cancer|Spinal Muscular Atrophy|Systemic Lupus Erythematosus|Thrombotic Thrombocytopenic Purpura|Trisomy 21|Ulcerative Colitis | ALL | ADULT, OLDER_ADULT | Sanguine Biosciences, Los Angeles, California, 91403, United States |
| Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT | The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success. | Primary Immunodeficiency (PID)|Congenital Bone Marrow Failure Syndromes|Inherited Metabolic Disorders (IMD)|Hereditary Anemias|Inflammatory Conditions|Systemic Juvenile Idiopathic Arthritis (sJIA) | ALL | CHILD, ADULT | UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, 15224, United States |
| Lidocaine 5% Plasters (Versatis® 5%) in Pediatric Neuropathic Pains and Vasoocclusive Sickle Cell Crisis Pains | Lidocaine 5% plaster (VERSATIS® 5%) showed its efficacy and safety in the post-herpetic zoster adult pains. This treatment is recommended in first intention in adult neuropathic pains with allodynia. The purpose of this study is to assess efficacy and safety of lidocaine 5% plaster (VERSATIS® 5%) in the pediatric neuropathic pains and vasoocclusive sickle cell crises pains. | Neuropathic Pains|Vasoocclusive Sickle Cell Crises Pains | ALL | CHILD, ADULT | IHOP, Lyon, 69008, France|Centre Médico-chirurgical de Réadaptation des Massues, Lyon, France |
| Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders | This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure. | Severe Sickle Cell Disease|Bone Marrow Failure Syndromes|Metabolic Disorders|Immunologic Disorders|Hemoglobinopathies|Non-malignant Disorders | ALL | CHILD, ADULT | Yale School of Medicine, New Haven, Connecticut, 06510, United States|Helen DeVos Children's Hospital, Grand Rapids, Michigan, 49503, United States|Washington University School of Medicine, Saint Louis, Missouri, 63110, United States |
| Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation | In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum effective dose required for reliable engraftment for subjects undergoing hematopoietic stem cell transplantation for non-malignant disease. | Bone Marrow Failure Syndrome|Thalassemia|Sickle Cell Disease|Diamond Blackfan Anemia|Acquired Neutropenia in Newborn|Acquired Anemia Hemolytic|Acquired Thrombocytopenia|Hemophagocytic Lymphohistiocytoses|Wiskott-Aldrich Syndrome|Chronic Granulomatous Disease|Common Variable Immunodeficiency|X-linked Lymphoproliferative Disease|Severe Combined Immunodeficiency|Hurler Syndrome|Mannosidosis|Adrenoleukodystrophy | ALL | CHILD, ADULT | UF Health Shands Children's Hospital, Gainesville, Florida, 32608, United States |
| CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant | CD34+ stem cell selection in children, adolescents and young adults receiving partially matched family donor or matched unrelated adult donor allogeneic bone marrow or peripheral blood stem cell transplant will be safe and well tolerated and be associated with a low incidence of serious (Grade III/IV) acute and chronic graft versus host disease (GVHD). | Leukemia|Lymphoma|Bone Marrow Failure|Immunodeficiencies|Histiocytosis|Sickle Cell Disease|Beta Thalassemia|Inborn Errors of Metabolism | ALL | CHILD, ADULT, OLDER_ADULT | New York Medical College, Valhalla, New York, 10595, United States |
| Fetal Cell Receptors Repertoire | The purpose of this study is to describe the transcriptomic profile of foetal cells in post-partum and more specifically to determine which chemokine receptors are overexpressed in foetal cells in post-partum women with wounds To do so, the investigators will isolate foetal cells from the peripheral blood of healthy controls post partum women as well as from post partum women with skin ulcers and then perform RNA sequencing. | Venous Ulcer|Sickle Cell Ulcer|Diabetic Ulcer|Post-partum Women|Mixed Ulcer | ALL | CHILD, ADULT, OLDER_ADULT | Dermatology unit - Cochin Hospital - APHP, Paris, Ile De France, 75014, France |
| Immunogenicity and Safety of Inactivated H1N1 Swine-origin Influenza Monovalent Vaccine in Immunocompromised Children and Young Adults | This study will evaluate the immunogenicity and safety of inactivated H1N1 swine-origin monovalent influenza vaccine in immunocompromised children and young adults. | H1N1 Pandemic Flu | ALL | CHILD, ADULT | St.Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders | Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma. Its presence in maternal plasma has allowed development of noninvasive prenatal diagnosis for single-gene disorders (SGD-NIPD). This can be performed from 9 weeks of amenorrhea and offers an early, safe and accurate definitive diagnosis without the miscarriage risk associated with invasive procedures. One of the major difficulties is distinguishing fetal genotype in the high background of maternal cfDNA, which leads to several technical and analytical challenges. Besides, unlike noninvasive prenatal testing for aneuploidy, NIPD for monogenic diseases represent a smaller market opportunity, and many cases must be provided on a bespoke, patient- or disease-specific basis. As a result, implementation of SGD-NIPD remained sparse, with most testing being delivered in a research setting. The present project aims to take advantage of the unique French collaborative network to make SGD-NIPD possible for theoretically any monogenic disorder and any family. | Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including|Sickle Cell Disease|Cystic Fibrosis|Fragile X Syndrome|Proximal Spinal Muscular Atrophy|Myotonic Dystrophy|Muscular Dystrophy, Duchenne|Muscular Dystrophy, Becker|Neurofibromatosis-Noonan Syndrome|Huntington Disease|Hemophilia a|Hemophilia B|MODY2 Diabetes|X-Linked Hydrocephalus|Autosomal Recessive Polycystic Kidney Disease | FEMALE | ADULT, OLDER_ADULT | Hôpital Cochin, Maternité Port-Royal, service de Gynécologie obstétrique, Paris, 75014, France |
| Is There Intravascular Hemolysis in Patients With Pulmonary Hypertension? | Patients with hemolytic disorders (e.g. sickle cell anemia or thalassemia) are known to develop pulmonary hypertension. Hemolysis is where red blood cells are destroyed and their contents released into the circulation. It is thought that these red-cell contents cause constriction and thrombosis of the blood vessels in the lungs. Conversely, it is possible that patients with pulmonary hypertension have hemolysis. In this study we will be drawing blood from a range of patients and normal controls for a panel of blood tests related to hemolysis. | Pulmonary Artery Hypertension | ALL | ADULT, OLDER_ADULT | Pulmonary Institute, Rabin Medical Center (Beilinson campus), Petach Tikva, 49100, Israel |
| Interferon and Ribavirin Treatment in Patients With Hemoglobinopathies | Worldwide, several studies report that 4.4% to 85.4% of thalassemia patients were positive for anti hepatitis C antibodies. Recently, three different studies reported the efficacy and the safety of combination therapy with pegylated interferon and ribavirin in thalassemic patients. This study is carried ahead to assess the impact of combination therapy with pegylated-interferon and ribavirin in a large cohort of italian patients with beta thalassemia major - transfused and not transfused, sickle cell disease and sickle/beta-thalassemia. | Hemoglobinopathies | ALL | ADULT | AOVCervello, Palermo, 90100, Italy |
| Decitabine in Treating Patients With Myelofibrosis | This phase II trial studies the side effects and how well decitabine works in treating patients with myelofibrosis, a cancer of the blood system associated with fibrosis (scar tissue) in the bone marrow that is advanced and for which there is no standard therapy. Decitabine may block the actions of some proteins that are responsible for turning certain genes off in various cancers including myelofibrosis. | Primary Myelofibrosis|Secondary Myelofibrosis | ALL | ADULT, OLDER_ADULT | University of Chicago Comprehensive Cancer Center, Chicago, Illinois, 60637, United States|Decatur Memorial Hospital, Decatur, Illinois, 62526, United States|NorthShore University HealthSystem-Evanston Hospital, Evanston, Illinois, 60201, United States|Ingalls Memorial Hospital, Harvey, Illinois, 60426, United States|Loyola University Medical Center, Maywood, Illinois, 60153, United States|Illinois CancerCare-Peoria, Peoria, Illinois, 61615, United States|Central Illinois Hematology Oncology Center, Springfield, Illinois, 62702, United States|Southern Illinois University School of Medicine, Springfield, Illinois, 62702, United States|Fort Wayne Medical Oncology and Hematology Inc-Parkview, Fort Wayne, Indiana, 46845, United States|Northern Indiana Cancer Research Consortium, South Bend, Indiana, 46628, United States|University of Maryland/Greenebaum Cancer Center, Baltimore, Maryland, 21201, United States|Oncology Care Associates PLLC, Saint Joseph, Michigan, 49085, United States|Ohio State University Comprehensive Cancer Center, Columbus, Ohio, 43210, United States|Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| Changes in Pituitary Iron and Volume With Deferasirox | Despite continuing advances in iron chelation therapy, iron toxicity of endocrine glands, particularly the pituitary gland, remains common in patients with transfusion dependent anemias. We would like to establish accurate population norms of pituitary R2 and volume and understand the progression of pituitary iron in transfused patients on Deferasirox. | Iron Overload | ALL | CHILD, ADULT | Children's Hospital Los Angeles, Los Angeles, California, 90027, United States |
| Genetic Determinants of Kidney Disease in People of African Ancestry With HIV | Black ethnicity is a major risk factor for chronic kidney disease \[CKD\] in people with HIV infection, suggesting that genetic factors are an important determinant of kidney disease progression in this population. The Gen-Africa study was established in 2018 to allow the study of genetic and clinical risk factors for CKD in people with HIV in the UK. Just over 3000 people across 15 sites were enrolled between May 2018 and January 2020. Demographic and clinical information was collected, and biological samples (buffy coats, plasma and urine) obtained. Cross-sectional analyses have revealed that participants of West-African ancestry are at higher risk of CKD and end-stage kidney disease \[ESKD\], and that genetic variants in the apolipoprotein L1 (APOL1) gene and sickle cell trait (SCT) are predictors of CKD and ESKD. The pathogenesis of APOL1- and SCT-associated CKD is incompletely understood, and additional, longitudinal data will be collected to improve understanding of the contribution of demographic, traditional CKD (diabetes, hypertension, obesity/metabolic syndrome, cardiovascular disease) and HIV (immuno-virological and hepatitis B/C co-infection status, antiretroviral medications) risk factors as well as additional genetic and epigenetic markers. | HIV Nephropathy|Kidney Injury|Kidney Diseases|Diabetes Mellitus|Obesity|Cardiovascular Diseases|Genetic Predisposition to Disease | ALL | ADULT, OLDER_ADULT | King's College Hospital NHS Foundation Trust, London, SE5 9RJ, United Kingdom |
| The Role of m6A RNA Modification in Moyamoya Disease | The purpose of this study is to detect the change of m6A RNA modification from peripheral blood of patients with moyamoya disease, and to assess the relationship between clinical characteristics. | Moyamoya Disease | ALL | CHILD, ADULT | Beijing Tiantan Hospital Capital Medical University, Beijing, Beijing, China |
| Evaluation of Women With Endocrine and Reproductive-Related Conditions | This study was designed to allow inpatient and outpatient evaluation of women with a variety of reproductive and endocrine-related disorders for purposes of research and physician education. The evaluations may include ultrasound examinations, blood, saliva, and/or urine samples. In some cases, specific laboratory or X-ray studies will be performed to confirm the diagnosis or assist in the treatment of the patient. These additional tests will be conducted within the guidelines of current gynecologic practice. In some cases, the patient will receive medical or surgical treatment for their disorder. The purpose of this study is to provide an opportunity for physicians to evaluate women with medical conditions of reproduction. These evaluations and treatments will support clinical training and research for the accredited training program in reproductive endocrinology at the National Institute of Child Health and Human Development (NICHD).\ | Endocrine Disease|Infertility|Leiomyoma|Endometriosis|Fibroids | ALL | CHILD, ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Physiology and Pathologies Linked to Human Splenic Function : Direct and Ex-vivo Perfusion Explorations | Human splenic physiology remains poorly understood due to lack of functional exploration. However, through its ability to recognize alterations or modifications in circulating cells and to trigger an innate and adaptive response in response to these anomalies, the spleen plays a central role in several diseases affecting blood cells, directly or indirectly. The analysis of the splenic clearance of abnormal cells during ex-vivo perfusions made it possible to clarify the pathogenesis of malaria and the role of the spleen in the adaptive immune response. The study's investigative team wishes to extend these explorations to other human diseases in which the spleen is involved, and to evaluate the preventive or curative potential of substances that can modify the perception of blood cells by the spleen (e.g. monoclonal antibodies directed against circulating cells, among other options). | Splenectomy | ALL | ADULT, OLDER_ADULT | Hôpital Beaujon, Clichy, 92110, France|Hôpital Saint Antoine, Paris, 75012, France|Hôpital Pitié Salpêtrière, Paris, 75013, France|Hôpital Necker-Enfants Malades, Paris, 75015, France|Institut Pasteur, Paris, 75015, France |
| Pulmonary Hypertension SOLAR | The main goal of this study is to develop a noninvasive signature for pulmonary vascular remodeling in Group 3 PH patients, using hyperpolarized 129Xe magnetic resonance imaging (129Xe MRI). Such a signature may identify Group 3 PH responders to PAH-specific therapies. PAH's unique 129Xe MRI signature has been shown in previous studies. Past studies have lacked a pathologic "ground truth" correlate of these signatures, which could be provided by comparing them with the pathology of lung explant tissue from patients who have undergone a lung transplant. This signature could be validated in a cohort of patients with Group 3 PH in future studies. | Interstitial Lung Disease|COPD|Pulmonary Arterial Hypertension | ALL | ADULT, OLDER_ADULT | Duke University Medical Center, Durham, North Carolina, 27710, United States |
| Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP) | In β-thalassaemia and Sickle Cell Disease (SCD), a significant production of fetal haemoglobin (HbF) may reduce the severity of clinical course and reactivation of γ-globin gene expression in adulthood. HbF induction is one of the best strategies to ameliorate the characteristic symptoms of these diseases. Hydroxyurea (HU) is the only medication, approved by the US Food and Drug Administration, inducing HbF. However, treatments with HU induce sufficient HbF levels in only half of the patients, and side effects including leukopenia and neutropenia are frequently reported. Therefore, novel therapeutic inducers must be identified to develop a personalized treatment in β-thalassaemia and sickle cell anaemia. The availability of new treatments depends on drugs already approved for other indications, and on pharmacokinetics and pharmacovigilance already assessed. Rapamycin (as Sirolimus) is an immunosuppressant agent, approved by the FDA for acute rejection prevention in renal transplant recipients. The ability of this drug to induce γ-globin gene expression in erythroleukemia cell line and erythroid precursors cells (ErPCs) in ß-thalassaemia patients is already known. A clinical investigation on the effects of sirolimus in ß-Thalassaemia aims to evaluate several parameters related to red blood cell status and HbF levels and is a first step for the full clinical development in this new indication. | Beta-Thalassemia | ALL | ADULT, OLDER_ADULT | University of Ferrara Department of Life Sciences and Biotechnology, Ferrara, FE, 44121, Italy|Day Hospital Thalassaemia and Haemoglobinopathies (DHTE) - Azienda Ospedaliero-Universitaria S.Anna of Ferrara, Ferrara, FE, 44124, Italy|Thalassemia and Hemoglobinopathies Center Azienda Ospedaliero Universitaria Meyer, Firenze, Fi, 50139, Italy|Pediatric oncohematology Azienda Ospedaliero Universitaria Pisana Ospedale Santa Chiara, Pisa, Pi, 56126, Italy |
| Kid Cards: Teaching Kids About Medicines | This study is being done to see if education about medicines directed toward children will improve their knowledge. The investigators also want to know if this knowledge lasts over time. Right now there are few medication instructional cards that are appropriate for children. Most of the medication cards provide information for adults. Some studies have shown that by teaching children directly, the children may take medicine at the right time for the right reason, have fewer side effects and know more about their medicine. The purpose of this research study is to see if education about medication helps children learn more about their medicine and if this knowledge lasts. | Hemophilia|Sickle Cell Disease|Neoplasms|Blood Coagulation Disorders | ALL | CHILD | Childrens Mercy Hospitals and Clinics, Kansas City, Missouri, 64108, United States |
| Single-Blind Study of STAT-205 in Mild COVID-19 | This is a randomized, single blind, study. Males and females meeting inclusion criteria who have symptoms of mild COVID-19 and in whom a positive PCR result for SARS-CoV-2 is obtained may be enrolled to the study treatment within 72 hours of the positive PCR result. Eligible patients are those considered to be at high risk for COVID-19 disease progression. This includes patients ≥ 65 years of age or with any one or more of certain medical conditions including: cancer, COPD, cardiovascular disease, immunocompromised state resulting from solid organ transplant, obesity, sickle cell disease, history of smoking, and diabetes. | COVID-19 | ALL | ADULT, OLDER_ADULT | Loma Linda University, Loma Linda, California, 92354, United States|Clinical Research Center of Florida, Pompano Beach, Florida, 33060, United States |
| Data Collection Study of Patients with Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT with RIC | This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft failure and to aid in immune reconstitution following hematopoietic stem cell transplantation. | Primary Immunodeficiency (PID)|Congenital Bone Marrow Failure Syndromes|Inherited Metabolic Disorders (IMD)|Hereditary Anemias|Inflammatory Conditions | ALL | CHILD, ADULT | UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, 15224, United States |
| ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders | In parallel with the growth of American Thrombosis and Hemostasis Network's (ATHN) clinical studies, the number of new therapies for all congenital and acquired hematologic conditions, not just those for bleeding and clotting disorders, is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have yet to demonstrate long-term safety and effectiveness beyond the pivotal trials that led to their approval. In addition, results from well-controlled, pivotal studies often cannot be replicated once a therapy has been approved for general use.(1,2,3,4) In 2019 alone, the United States Food and Drug Administration (FDA) has issued approvals for twenty-four new therapies for congenital and acquired hematologic conditions.(5) In addition, almost 10,000 new studies for hematologic diseases are currently registered on www.clinicaltrials.gov.(6) With this increase in potential new therapies on the horizon, it is imperative that clinicians and clinical researchers in the field of non-neoplastic hematology have a uniform, secure, unbiased, and enduring method to collect long-term safety and efficacy data. ATHN Transcends is a cohort study to determine the safety, effectiveness, and practice of therapies used in the treatment of participants with congenital or acquired non-neoplastic blood disorders and connective tissue disorders with bleeding tendency. The study consists of 7 cohorts with additional study "arms" and "modules" branching off from the cohorts. The overarching objective of this longitudinal, observational study is to characterize the safety, effectiveness and practice of treatments for all people with congenital and acquired hematologic disorders in the US. As emphasized in a recently published review, accurate, uniform and quality national data collection is critical in clinical research, particularly for longitudinal cohort studies covering a lifetime of biologic risk.(7) | Hematologic Disorder|Bleeding Disorder|Connective Tissue Disorder|Hemophilia|Thrombosis|Von Willebrand Diseases|Thrombophilia|Rare Bleeding Disorder|Platelet Disorder|Factor IX Deficiency|Factor VIII Deficiency|Thalassemia|Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Arizona Hemophilia and Thrombosis Treatment Center at Phoenix Children's Hospital, Phoenix, Arizona, 85016, United States|Arkansas Center for Bleeding Disorders, Little Rock, Arkansas, 72202, United States|Orthopaedic Institute for Children HTC, Los Angeles, California, 90007, United States|Childrens Hospital Los Angeles, Los Angeles, California, 90027-6016, United States|UCSF Benioff Children's Hospital Oakland, Oakland, California, 94610, United States|University of California at Davis Hemophilia Treatment Center, Sacramento, California, 95817, United States|Loma Linda Hemoglobinopathy and Inherited Bleeding Disorder Program, San Bernardino, California, 92408, United States|Hemophilia & Thrombosis Treatment Center at UC San Diego Health, San Diego, California, 92121, United States|Rady Children's Hospital San Diego, San Diego, California, 92123, United States|University of California, San Francisco Hemophilia & Thrombosis Center, San Francisco, California, 94143, United States|University of Colorado Denver Hemophilia and Thrombosis Center, Aurora, Colorado, 80045, United States|Connecticut Children's Medical Center, Hartford, Connecticut, 06106, United States|Yale Hemophilia Treatment Center, New Haven, Connecticut, 06520, United States|Delaware Hemophilia Treatment Center, Wilmington, Delaware, 19801, United States|Georgetown University, Washington, District of Columbia, 20007, United States|Children's National Hemophilia Center, Washington, District of Columbia, 20010, United States|University of Florida Hemophilia Treatment Center, Gainesville, Florida, 32610, United States|University of Miami Comprehensive Hemophilia Treatment Center, Miami, Florida, 33136, United States|Arnold Palmer Hospital for Children - The Haley Center for Children's Cancer and Blood Disorders, Orlando, Florida, 32806, United States|Johns Hopkins All Children's Hospital, Saint Petersburg, Florida, 33701, United States|St. Joseph's Hospital Center for Bleeding & Clotting Disorders, Tampa, Florida, 33607, United States|Comprehensive Bleeding Disorders Center at Emory University and Children's Healthcare of Atlanta, Atlanta, Georgia, 30308, United States|Emory/Children's Health Care of Atlanta, Atlanta, Georgia, 30322, United States|Memorial Health University Medical Center, Savannah, Georgia, 31403, United States|Rush University Medical Center, Chicago, Illinois, 60612, United States|Bleeding and Clotting Disorders Institute, Peoria, Illinois, 61664, United States|Indiana Hemophilia and Thrombosis Center, Indianapolis, Indiana, 46260, United States|Iowa Hemophilia and Thrombosis Center, Iowa City, Iowa, 52242, United States|Louisiana Center for Bleeding and Clotting Disorders, Tulane University, New Orleans, Louisiana, 70112, United States|Louisiana Center for Advanced Medicine, Slidell, Louisiana, 70461, United States|Maine Hemophilia and Thrombosis Center, Scarborough, Maine, 04074, United States|Johns Hopkins University Hemophilia Treatment Center, Baltimore, Maryland, 21287, United States|Massachusetts General Hospital Comprehensive Hemophilia and Thrombosis Treatment Center, Boston, Massachusetts, 02114, United States|Central Michigan Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|Henry Ford Health System Bleeding and Thrombosis Treatment Center, Detroit, Michigan, 48202, United States|Mayo Comprehensive Hemophilia Center, Rochester, Minnesota, 55905, United States|Mississippi Center for Advanced Medicine, Madison, Mississippi, 39110, United States|Children's Mercy Hospital - Kansas City, Kansas City, Missouri, 64108, United States|The John Bouhasin Center for Children with Bleeding Disorders, Saint Louis, Missouri, 63104, United States|Cure 4 The Kids Foundation, Las Vegas, Nevada, 89135, United States|Hemostasis and Thrombosis Center of Nevada, Reno, Nevada, 89509, United States|Newark Beth Israel Medical Center - Hemophilia Center, Newark, New Jersey, 07122, United States|University of New Mexico Ted R. Montoya Hemophilia & Thrombosis Program, Albuquerque, New Mexico, 87131, United States|Montefiore Medical Center, Bronx, New York, 10461, United States|Western New York BloodCare, Buffalo, New York, 14202, United States|Northwell Health Hemostasis and Thrombosis Center at Long Island Jewish and Cohen Children's Medical Center, Hyde Park, New York, 11040, United States|Weill Cornell Medical College - New York Presbyterian Hospital, New York, New York, 10065, United States|American Thrombosis and Hemostasis Network, Rochester, New York, 14626, United States|Comprehensive Hemophilia Treatment Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, 27517, United States|St. Jude Affiliate Clinic at Novant Health Hemby Children's Hospital, Charlotte, North Carolina, 28204, United States|East Carolina University Hemophilia Treatment Center, Greenville, North Carolina, 27834, United States|Wake Forest University Health Sciences, Winston-Salem, North Carolina, 27157, United States|Akron Children's Hospital - Showers Center for Cancer & Blood Disorders, Akron, Ohio, 44308, United States|Cincinnati Children's Hospital Medical Center, Hemophilia & Thrombosis Center, Cincinnati, Ohio, 45229, United States|University of Cincinnati Medical Center Hemophilia Treatment Center, Cincinnati, Ohio, 45267, United States|University Hospitals Health System Cleveland, Cleveland, Ohio, 44106, United States|Nationwide Children's Hospital Columbus, Columbus, Ohio, 43205, United States|Dayton Children's Hemostasis and Thrombosis Center, Dayton, Ohio, 45404, United States|Northwest Ohio Hemophilia Treatment Center at the Toledo Hospital, Toledo, Ohio, 43606, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Penn Comprehensive Hemophilia and Thrombophilia Program/Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, 19104, United States|Hemophilia Center of Western Pennsylvania, Pittsburgh, Pennsylvania, 15213, United States|Rhode Island Hospital Hemostasis and Thrombosis Center, Providence, Rhode Island, 02903, United States|St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States|Vanderbilt University Medical Center, Nashville, Tennessee, 37212, United States|Children's Blood and Cancer Center of Central Texas, Austin, Texas, 78723, United States|North Texas Hemophilia and Thrombosis Program - Pediatric Program / Center for Cancer & Blood Disorders, Dallas, Texas, 75235, United States|North Texas Comprehensive Hemophilia Treatment Center, Dallas, Texas, 75390, United States|Fort Worth Bleeding Disorders Program, Fort Worth, Texas, 76104, United States|Gulf States Hemophilia and Thrombophilia Center, Houston, Texas, 77030, United States|Texas Children's Hemophilia & Thrombosis Center/Baylor College of Medicine, Houston, Texas, 77030, United States|South Texas Comprehensive Hemophilia and Thrombophilia Treatment Center, San Antonio, Texas, 78229, United States|Washington Center for Bleeding Disorders, Seattle, Washington, 98101, United States|Hemophilia Outreach Center Green Bay, Green Bay, Wisconsin, 54311, United States|Comprehensive Center for Bleeding Disorders, Milwaukee, Wisconsin, 53226, United States |
| Predictive Determinants of Nephrotic Syndrome Remission in Patients With At-risk Polymorphism of APOL1 | This is a multicentric retrospective observational cohort study. As primary objective, the study aims to evaluate the factors associated with nephrotic syndrome remission in patient with nephrotic syndrome, biopsy-prove minimal change disease or focal segmental glomerulosclerosis, and an at-risk variant of the APOL1 gene. As secondary objectives, this study aims: * To evaluate the benefit of corticosteroids in obtaining the remission of nephrotic syndrome * To identify the predictors of complete renal remission of nephrotic syndrome * To evaluate the benefit of corticosteroids in reducing the incidence of end-stage renal disease * To assess the adverse events of corticosteroids in patients treated with corticosteroids. | Nephrotic Syndrome|Focal Segmental Glomerulosclerosis|APOL1 Associated Kidney Disease | ALL | ADULT, OLDER_ADULT | Néphrologie & Dialyses department, Tenon Hospital, Paris, 75020, France |
| Coordinated HEalthcare for Complex Kids | The University of Illinois Health and Health Sciences System (UI Health) developed an integrated care management quality improvement model designed to provide comprehensive care coordination for Medicaid insured minority children and young adults with chronic health conditions living in Chicago. This program, called CHECK (Coordinated HEalthcare for Complex Kids), targeted children and young adults with chronic disease. | Asthma|Diabetes Mellitus|Sickle Cell Disease|Premature Birth | ALL | CHILD, ADULT | |
| Transcranial Doppler in Children With Hemoglobinopathies | Transcranial Doppler ultrasonography (TCD) is a noninvasive, portable technique for evaluating the intracranial vasculature. TCD ultrasonography is performed placing a low frequency (≤ 2 MHz) transducer on the scalp of the patient, in order to visualize the intracranial arterial vessels through specific acoustic windows, where bone is thinner, and evaluate cerebral blood flow velocity (CBFV) . Patients diagnosed to have hemoglobinopathy conditions with its most common forms thalassemia and sickle cell disease manifest both biochemical and clinical evidence of hypercoagulability conditions include deep venous thrombosis, pulmonary emboli and recurrent arterial occlusion . Cerebrovascular accidents can be identified using transcranial Doppler ultrasonography which enables evaluation of cerebral artery blood flow velocity with a sensitivity of 90% and specificity of 100 % when compared with cerebral angiography . According to the stroke prevention trial in sickle cell anemia (STOP) study ,TCDs were classified based on blood velocity in the circle of Willis, expressed as time averaged mean of the maximum velocity (TAMMV). Children with abnormal, high velocities (\>200cm/s) were at increased risk of stroke, which was reduced by 90% after starting regular blood transfusions. TAMMVs less than 170cm/s were classified as normal with annual TCD scanning recommended, whereas velocities between 170 and 200cm/s were called conditional, and followed up more closely without starting transfusion. | Hemoglobinopathies | ALL | CHILD, ADULT | |
| The Living With a Long-Term Condition Study | Psychological distress (anxiety and depression) is common in and experienced differently by people living with long-term health conditions (LTCs). Being able to measure whether psychological distress is related to living with a LTC would allow researchers and clinicians to provide interventions specifically tailored to the challenges of living with a LTC and therefore provide the most appropriate support for these patients. Such a measure would also be useful in research to identify the presence of illness-related distress in different patient groups. This project will therefore create a new measure of illness-related distress that has applications for both research and clinical practice. This will involve the psychometric validation of the new illness-related distress measure to test how valid and reliable the measure is. The aim of the project is to provide initial validation of the Illness Related Distress Scale in a community sample, recruited through online platforms. The objective of the study is to gather initial validity and reliability data for the scale. | Asthma|Atrial Fibrillation|Cancer|Cerebrovascular Disorders|Stroke|CKD|Copd|Fibromyalgia|Pain|Heart Diseases|Heart Disease Chronic|Dementia|Diabetes|Epilepsy|Heart Failure|High Blood Pressure|Hypertension|Hiv|AIDS|IBD|IBS|Liver Diseases|Long COVID|Lupus Erythematosus|Multiple Sclerosis|Obesity|Osteoarthritis|Arthritis|Rheumatoid Arthritis|Osteoporosis|Parkinson Disease|Sickle Cell Disease|Hepatitis|Endometriosis|PCOS|Neurological Disorder|POTS - Postural Orthostatic Tachycardia Syndrome|MND (Motor Neurone DIsease)|Cystic Fibrosis|Migraine|Spondylitis|Celiac Disease|Hidradenitis Suppurativa|Eczema|ME/CFS | ALL | ADULT, OLDER_ADULT | King's College London, London, SE1 9RT, United Kingdom |
| A Survey of Hospitalizations in Cardiology Units in Sub-Saharan Africa | FEVRIER study is an observatory of hospitalizations in cardiology units in sub-Saharan Africa. | Acute Coronary Syndrome|Heart Failure|Syncope|Stroke|Pericarditis|Endocarditis|Conduction Abnormalities|Rhythm; Abnormal|Pulmonary Embolism|Deep Vein Thrombosis|Other Cardiovascular Conditions | ALL | CHILD, ADULT, OLDER_ADULT | Centre national hospitalier et universitaire Koutoukou Maga, Cotonou, Benin|Centre médical Paul IV, Ouagadougou, Burkina Faso|Hopital militaire de Kamenge et polyclinique maison médicale, Bujumbura, Burundi|Hôpital Régional, Bafoussam, Cameroon|Douala General Hospital, Douala, Cameroon|Central Hospital, Yaounde, Cameroon|Hôpital Général de Yaoundé, Yaounde, Cameroon|Clinique Ngaliema, Kinshasa, Congo, The Democratic Republic of the|Clinique universitaire de Kinshasa, Kinshasa, Congo, The Democratic Republic of the|CHU, Brazzaville, Congo|Institut de Cariologie d'Abidjan, Abidjan, Côte D'Ivoire|CHU, Bouake, Côte D'Ivoire|Centre Hospitalier Universitaire (CHUL), Libreville, Gabon|CHU Ignace Deen, Conakry, Guinea|Institut du coeur, Maputo, Mozambique|Hôpital National Lamorde, Niamey, Niger|Hôpital de Fann, Dakar, Senegal|Hôpital général de Grand Yoff, Dakar, Senegal|El Hadj-Ibrahima Niass, Kaolac, Senegal|National Cardiothoracic center / Shab Teaching Hospital, Khartoum, Sudan |
| Immunization of Children Between 8 Weeks and 2 Years of Age With GSK Pneumococcal Vaccine GSK1024850A | The aim of the study is to evaluate the immunogenicity, safety and reactogenicity of GSK Biologicals' pneumococcal conjugate vaccine GSK1024850A. Children that are below 6 months at the time of enrolment will also receive the DTPw-HBV/Hib and OPV vaccines. | Infections, Streptococcal | ALL | CHILD | GSK Investigational Site, Ouagadougou, Burkina Faso |
| Massive Iron Deposit Assessment | Iron overload is a severe complication of multiple blood transfusions. As the body has no physiologic mechanism for clearing iron, repeated transfusions cause iron accumulation in organs and lead to iron toxicity. Accurate assessment of iron overload is paramount to quantify excessive iron accumulation and to monitor response to iron chelation therapy. Magnetic resonance imaging (MRI) methods have been used to noninvasively measure hepatic iron concentration (HIC). Although MRI-based measurements of transverse relaxation rates (R2 and R2\*) accurately predict biopsy-proven HICs below 15 mg Fe/g, previous studies have shown that their precision is limited for HICs above 15 mg Fe/g and inaccurate above 25 mg Fe/g. Current R2\* gradient-echo (GRE) MR techniques fail occasionally for very high iron overloads (HIC \~ 15-25 mg Fe/g) and always for massive iron overloads (HIC \> 25 mg Fe/g) because R2\* is so high that the MR signal decays before it can be measured accurately. Overall accrual: 200 patients Purpose: To determine if a new MRI (UTE) can measure the amount of iron in the liver of people with large amounts of iron and compare the results with the same patient's liver bx. Estimated patient accrual is 150. It is estimated that 41 of these patients will have clinical indication for liver biopsy. | Iron Overload|Excessive Body Iron Burden | ALL | CHILD, ADULT, OLDER_ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients | Melphalan is a chemotherapy drug used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of melphalan in their bodies and if drug levels are related to whether or not a child experiences severe side-effects during their bone marrow transplant. The hypothesis is that certain clinical and individual factors cause changes in melphalan drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects. | Hematologic Malignancies|Nonmalignant Diseases|Immunodeficiencies|Hemoglobinopathies|Genetic Inborn Errors of Metabolism|Fanconi's Anemia|Thalassemia|Sickle Cell Disease | ALL | CHILD | University of California, San Francisco, San Francisco, California, 94143, United States |
| Exercise and Diet for Pediatric Obesity | The coronavirus disease (COVID-19), is a communicable pandemic disease as stated by the world health organization (WHO), which has been affecting the world since December 2019. COVID-19 infected children develop the signs and symptoms of the disease, which can be exaggerated or life-threatening when associated with comorbidities like; obesity, sickle cell anemia, immune disorders, chromosomal abnormalities, chronic respiratory or cardiac problems, and congenital malformations.3 It is observed that children affected with COVID-19 who are physically inactive or in a sedentary lifestyle may induce and develop obesity. It is a major health concern in this pandemic situation, which can be addressed and treated with the use of appropriate physical training and proper dietary habits. | Pediatric Obesity|COVID-19 | MALE | CHILD | Gopal Nambi, Al Kharj, Riyadh, 11942, Saudi Arabia |
| Pilot Study for Patients With Poor Response to Deferasirox | This purpose of this study is to understand the differences between people who have a good response to deferasirox (exjade) compared to people who have a poor response to this medication when used for transfusion-dependent iron overload. The hypothesis is that patients with poor responses have physiologic barriers to deferasirox that may include absorption, pharmacokinetics of drug metabolism, hepatic clearance and/or genetic factors. | Transfusion-dependent Hemachromatosis|Thalassemia Major|Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital Boston, Boston, Massachusetts, 02115, United States |
| TRANSPIRE: Lung Injury in a Longitudinal Cohort of Pediatric HSCT Patients | Hematopoietic stem cell transplant (HSCT) is an effective but toxic therapy and pulmonary morbidity affects as many as 25% of children receiving transplant. Early pulmonary injury includes diffuse alveolar hemorrhage (DAH), thrombotic microangiopathy (TMA) interstitial pneumonitis (IPS) and infection, while later, bronchiolitis obliterans is a complication of chronic GVHD associated with severe morbidity and mortality. Improved diagnosis and treatment of pulmonary complications are urgently needed as survival after HSCT improves, and as HSCT is increasingly used for non-malignant disorders such as sickle cell disease. Currently, there are large and important gaps in the investigator's knowledge regarding incidence, etiology and optimal treatment of pulmonary complications. Moreover, young children unable to perform spirometry are often diagnosed late, and strategies for monitoring therapeutic response are limited. This is a prospective multi-institutional cohort study in pediatric patients undergoing allogeneic hematopoietic stem cell transplantation (alloHSCT). Assembly of a large prospective uniformly screened cohort of children receiving HSCT, together with collection of biological samples, will be an effective strategy to identify mechanisms of lung injury, test novel diagnostic strategies for earlier diagnosis, and novel treatments to reduce morbidity and mortality from lung injury after transplant. | Hematopoietic Stem Cell Transplant (HSCT)|Diffuse Alveolar Hemorrhage|Thrombotic Microangiopathies|Interstitial Pneumonitis|Bronchiolitis Obliterans | ALL | CHILD, ADULT | University of California San Francisco, San Francisco, California, 94158, United States|Dana-Farber Cancer Institute/Boston Children's Hospital, Boston, Massachusetts, 02215, United States|University of Minnesota, Minneapolis, Minnesota, 55455, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Baylor College of Medicine/Texas Children'S Hospital, Houston, Texas, 77030, United States|Seattle Children'S Hospital, Seattle, Washington, 98105, United States|Fred Hutchinson Cancer Center, Seattle, Washington, 98109, United States |
| Assessment of SARS-CoV-2 Seroprevalence in Detention | "Background France counted on January 1, 2020, 70,651 people detained, for 61,080 places. Overcrowding in detention is considered as risk factor for infectious diseases transmission, such as respiratory infections. The prison environment represents a confined environment, which could protect prisoners from possible external contamination. If one or more inmates were infected through visiting rooms, officers working in detention, or newly incarcerated people, an epidemic could spread more quickly in the prison community. Thus, few cases of COVID-19 were observed among the subjects in detention with a few weeks delay compared to the free world. However, detention conditions make it more difficult to detect suspicious cases. On the other hand, carrying out diagnostic tests is structurally more difficult to carry out there. Thus, given the plurality of clinical presentations, the non-optimal sensitivity of the SARS-CoV-2 RT-PCR, and the difficulty in carrying out diagnostic tests, it is today difficult to have a precise idea of the number of prisoners having encountered SARS-CoV-2. It is also a population that is not taken into account in the large seroprevalence studies currently conducted in the general population. In order to estimate the number of prisoners exposed to SARS-CoV-2 and in the absence of data currently available in the medical literature, a seroprevalence study in this at risk and little studied population would bring new data to the medical community. Hypothesis In adult subjects living in penal establishments in Ile de France, the seroprevalence of SARS-CoV-2 would be lower compared to the general population. Material and method Open multicenter cross-sectional study carried out in the 11 penal establishments of Ile de France. A sampling of 3,500 inmates stratified over the 16 detention areas concerned will be carried out. The inclusion criteria will be detained subjects who have expressed their consent to participate in the research, aged 18 to 80 years. Each selected detainee will be invited to the health unit to perform a venous blood test for anti-SARS-CoV-2 antibodies. The goal is to take 2,500 blood samples (30% expected refusal rate). Each sample will be analyzed in the virology laboratory at P. Brousse hospital. Expected results Obtain an assessment of the seroprevalence of SARS-CoV-2 in prisons to determine the exposure of detained persons. This assessment will make it possible to undertake public health actions and to propose the implementation of group protection measures such as vaccination if this is soon available. | COVID-19 | ALL | ADULT, OLDER_ADULT | USMP, Le Kremlin Bicêtre, 94275, France |
| Efficacy and Safety of Deferasirox in Patients With Chronic Anemia and Transfusional Hemosiderosis | The overall purpose of this trial is to further evaluate the efficacy and safety of deferasirox, dosed initially according to the transfusional iron intake, in patients with transfusion dependant anemia related to disorders other than β-thalassemia and sickle cell disease. During the study, the dose will be adjusted based on serum Ferritin.The overall purpose of the extension is to allow further treatment of patients who have already completed the core study, and to enable collection of long term efficacy and safety data. Patients will continue to receive Deferasirox at the dose they received at the end of the core study. | Chronic Anemia|Transfusional Hemosiderosis | ALL | CHILD, ADULT, OLDER_ADULT | Novartis Investigative Site, Nagoya, Aichi, 453-8511, Japan|Novartis Investigative Site, Nagoya, Aichi, 466-8560, Japan|Novartis Investigative Site, Fukuoka-city, Fukuoka, 814-0180, Japan|Novartis Investigative Site, Sapporo-city, Hokkaido, 060-8543, Japan|Novartis Investigative Site, Nishinomiya, Hyogo, 663-8501, Japan|Novartis Investigative Site, Kahoku-gun, Ishikawa, 920-0293, Japan|Novartis Investigative Site, Kanazawa, Ishikawa, 920-8641, Japan|Novartis Investigative Site, OsakaSayama, Osaka, 589-8511, Japan|Novartis Investigative Site, Suita-city, Osaka, 565-0871, Japan|Novartis Investigative Site, Shimotsuka-gun, Tochigi, 321-0293, Japan|Novartis Investigative Site, Simotsuke-city, Tochigi, 329-0498, Japan|Novartis Investigative Site, Bunkyo-ku, Tokyo, 113-8655, Japan|Novartis Investigative Site, Cyuo-ku, Tokyo, 104-8560, Japan|Novartis Investigative Site, Minato-ku, Tokyo, 108-8639, Japan|Novartis Investigative Site, Shinagawa-ku, Tokyo, 141-8625, Japan|Novartis Investigative Site, Shinjuku-ku, Tokyo, 160-0023, Japan|Novartis Investigative Site, Shinjuku-ku, Tokyo, 162-8666, Japan|Novartis Investigative Site, Hiroshima, 734-8551, Japan|Novartis Investigative Site, Kumamoto, 860-0811, Japan|Novartis Investigative Site, Kyoto, 606-8507, Japan|Novartis Investigative Site, Nagasaki, 852-8501, Japan|Novartis Investigative Site, Toyama, 930-8550, Japan|Novartis Investigative Site, Warszawa, 02-097, Poland|Novartis Investigative Site, Warszawa, 02-776, Poland|Novartis Investigative Site, Singapore, 169608, Singapore|Novartis Investigative Site, Salamanca, Castilla Y Leon, 37007, Spain|Novartis Investigative Site, Valencia, 46026, Spain|Novartis Investigative Site, Adana, 01330, Turkey|Novartis Investigative Site, Ankara, 06100, Turkey|Novartis Investigative Site, Istanbul, 34093, Turkey|Novartis Investigative Site, Izmir, 35040, Turkey |
| Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload | The purpose of this research study is to evaluate the safety of two doses of FBS0701, a new oral iron chelator, and its effectiveness in clearing iron from the liver. FBS0701 is a medication taken by mouth that causes the body to get rid of iron. Iron chelators are used in patients with β-thalassemia and other forms of anemia who experience iron overload - iron increases in the body as a result of regularly required blood transfusions. Patients who qualify will be randomized to receive one of two doses of FBS0701 for up to 24 weeks (6 months) with a total study duration of up to 33 weeks. These patients will be eligible to participate in a dosing extension for up to 72 weeks. The maximum duration of dosing will be up to 96 weeks. The safety of patients will be monitored frequently during the study by physical exams, ECGs, and blood tests. To assess the amount of iron in the liver and heart, each patient must undergo 6 MRI scans during the study. Patients will not need to stay in the hospital for this study but will need to visit the outpatient clinic up to 28 times over the 96 week period. Patients currently taking an iron chelator will be required to stop for a total of up to 26 weeks. The results of this study will help to determine if FBS0701 may be effective as an iron chelator. | Transfusional Iron Overload|Beta-thalassemia | ALL | ADULT | Children's Hospital and Research Center of Oakland, Oakland, California, 94609, United States|Children's Hospital of Boston, Boston, Massachusetts, 02115, United States|Ospedale Regionale Microcitemie, Cagliari, Italy|Centro della Microcitemia e delle Anemie Congenite, Genoa, Italy|Thalassemia Center San Luigi Hospital, Orbassano, Italy|Siriraj Hospital, Mahidol University, Bangkok, Thailand|Pediatric Hematology, Ege University Hospital, Izmir, Turkey|University College London Hospital, London, United Kingdom|Whittington Hospital, London, United Kingdom |
| Senicapoc and Dehydrated Stomatocytosis | Dehydrated stomatocytosis is a genetic disorder characterized by chronic hemolysis, variable anemia and erythrocyte dehydration. Causative mutations have been identified in either the Gardos (KCNN4) channel or the mechanosensitive channel PIEZO1. Senicapoc is a selective blocker of the Gardos channel that has been extensively studied in sickle cell disease and shown to be safe with limited side-effects. However, senicapoc did not meet the designated clinical endpoints in a pivotal phase 3 trial. The present study is an explanatory, proof-of-concept study of Senicapoc administered once daily in patients with familial dehydrated stomatocytosis caused by autosomal dominant V282 mutations in the Gardos (KCNN4) channel. | Dehydrated Hereditary Stomatocytosis | ALL | ADULT, OLDER_ADULT | Boston Children's Hospital, Boston, Massachusetts, 02115-5724, United States |
| Post-Vaccination Biological Collection | Introduction: Vaccination is a powerful weapon in the fight against infectious diseases, which has led to dramatic reduction in mortality and complications from some diseases. In this respect, vaccination is a real worldwide public health challenge (WHO). Thus, vaccine research benefits from an exponential development of knowledge in immunology and biotechnology. In particular, the advent of recent tools ("omics", new cytometric assays) and the description of new categories of immune cells (Tfh, BReg...) have revolutionized the characterization of immune responses, particularly post-vaccination. To study of the immune response following vaccination remains essential in order to define the immunological correlates to vaccine protection. This response also varies according to parameters related to the vaccine (type, adjuvant, dose, regimen...) and to the vaccinated host (genetics, age, morbidity, treatment ...). Analyzing with new generation immune assays, new data on immunological responses post-vaccination from a clinical cohort is therefore essential to better define these correlates. Objective: To develop new vaccines (HIV, emerging infectious diseases) the investigators use a "System vaccinology" method to decipher the mechanisms of immune responses set up against vaccines currently being developed or marketed, specifically in specific populations (patients with primary immune deficiency, sickle cell patients, solid organ transplanted patients, COPD). Method: Description of the genetic, molecular and cellular mechanisms of the immune response to vaccines recommended for adults, in particular influenza and pneumococcal vaccines, but also other mandatory vaccines (MMR,...) or vaccine for travelers (yellow fever, ...) as part of routine care in different population categories (healthy subjects, HIV+ subjects, COPD patients, ...), using qualitative and quantitative immunological assays: transcriptional analysis of the dynamic innate immune response, analysis of the lymphocytes B \& T responses (phenotype, repertoire analysis, functional analysis including T reg and TFH populations, antibody response), genetic analysis in the context of primary immune deficiencies) Conclusion: The data generated will allow the best possible analysis of vaccine responses according to vaccines and vaccinated populations, providing important information for the research developed within the department. | Vaccination|Immune Response | ALL | ADULT, OLDER_ADULT | Pr Gallien, Créteil, 94000, France |
| Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation | Fludarabine is a chemotherapy drug used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of fludarabine in their bodies and if drug levels are related to whether or not a child experiences severe side-effects during their bone marrow transplant. The hypothesis is that clinical and genetic factors cause changes in fludarabine drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects. | Hematologic Malignancies|Nonmalignant Diseases|Immunodeficiencies|Hemoglobinopathies|Genetic Inborn Errors of Metabolism|Fanconi Anemia|Thalassemia|Sickle Cell Disease | ALL | CHILD | University of California, San Francisco, San Francisco, California, 94143, United States |
| Collection of Blood From Persons With Hemoglobin and Erythrocyte Polymorphisms for Laboratory Malaria Research | This study will collect blood samples for use in laboratory studies of malaria. The World Health Organization reports that 40 percent of the world's population is at risk for malaria, mostly in the poorest countries. It is a serious disease caused by parasites. Each year, 300-500 million infections lead to more than a million deaths. However, there are traits in red blood cells (erythrocyte) that protect people against malaria. In this study, polymorphism refers to the various kinds of red blood cell traits. The sickle cell trait is an example of one that seems to offer a natural survival advantage in children where malaria is common. Researchers at the Laboratory of Malaria and Vector Research are investigating ways in which the blood cell traits can offer such protection, and new knowledge gained can bring about medical advances. Of particular importance is studying how the malaria organism, Plasmodium, survives inside different red blood cells. A steady, consistent, and reliable supply of fresh whole blood is necessary for testing. Patients 18 to 65, weighing more than 110 lbs. and who do not have anemia or known HIV, Hepatitis C, or Hepatitis B may be eligible for this study. Patients will undergo a medical history and general assessment including vital signs of temperature, heart rate, and blood pressure. Blood will be collected from a vein in the arm, or rarely a vein in the hand. A complete blood count, or CBC, will be done to ensure that blood levels are sufficient and that blood donation is safe for a patient to do. Patients need to have enough hemoglobin, the part of red blood cells that transports oxygen throughout the body. The blood will also be tested to confirm the type of red blood cell traits of patients. About 1 to 8 tablespoons may be collected, but most blood samples will be small, that is, 1 to 4 teaspoons. After the patients' first visit, sessions will take 5-20 minutes. Blood collection will total no more than 2 cups from a donor during any 6-week period. Although the frequency of blood donations is not known at this time, it is unlikely that a patient will be asked to donate blood more than four times a year. It may be important for patients to undergo a repeat CBC or tests for blood chemistry if results are needed for research. Risks associated with blood collection are considered minimal. They include discomfort, occasional bruising or bleeding at the puncture site, and faintness. In this study, it is possible that a small amount of blood may be stored for future research, to help the researchers to learn more about malaria. There are no plans for the results from the various research laboratory tests to be made available to patients or to their private doctors. However, patients in this study may discuss the results of routine medical tests with the study investigators. Some of the blood collected will be tested for genetic conditions. Through genetic testing, researchers can learn more about how health or illness may be passed on to people by their parents, or from people to their children. All results of tests will remain confidential. Blood samples will be labeled by code, and reference to patients' identities will be protected. Participants will receive $50 for each blood donation. This study will not have a direct benefit for participants. Future research that uses their blood samples will help researchers to learn about malaria as well as how to prevent or treat the disease. | Hemoglobin Mutations|Erythrocyte Variants | ALL | ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| Functional Outcomes Following Hip Core Decompression in Younger Participants With Osteonecrosis | Although uncommon in the general pediatric population, osteonecrosis (ON) is prevalent in children and adolescents with cancer, particularly among those that have had chronic exposure to glucocorticoids and among those having undergone allogeneic hematopoietic stem cell transplantation. Patients with hematologic disease are also at risk for developing ON. Hip Core Decompression (HCD) is a widely used surgical procedure with several studies reporting positive results; however, most are subject to criticism because of the limited sample size or the absence of appropriate functional outcome measures. The objective of this pilot study is to observe and collect information on several functional outcome measures and assess if Hip Core Decompression (HCD) potentially improves functional outcomes in children, adolescents and young adults with osteonecrosis. PRIMARY OBJECTIVE: * To describe functional outcomes of children, adolescents, and young adults with osteonecrosis of the femoral head following hip core decompression surgery. Parameters assessed will include pain, functional mobility, endurance, quality of life, and gait patterns pre-operatively and post-operatively over time. | Osteonecrosis | ALL | CHILD, ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases | FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patients participate in the study by electronic consent (eConsent) and sign a medical records release to permit data collection. Medical records are accessed from institutions directly via eFax or paper fax, online from patient electronic medical record (EMR) portals, direct from DNA/RNA sequencing and molecular profiling vendors, and via electronic health information exchanges. Patients and their treating physicians may also optionally provide medical records. Medical records are received in or converted to electronic/digitized formats (CCDA, FHIR, PDF), sorted by medical record type (clinic visit, in-patient hospital, out-patient clinic, infusion and out-patient pharmacies, etc.) and made machine-readable to support data annotation, full text searches, and natural language processing (NLP) algorithms to further facilitate feature identification. | Alpha-Thalassemia|Beta-Thalassemia|Amyloidosis|Amyotrophic Lateral Sclerosis|Creutzfeld-Jakob Disease|Cystic Fibrosis|Duchenne Muscular Dystrophy|Early-Onset Alzheimer Disease|Ehlers-Danlos Syndrome|Huntington Disease|Gaucher Disease|GM1 Gangliosidosis|Myasthenia Gravis|Pompe Disease|Sickle Cell Disease|Transthyretin Amyloid Cardiomyopathy|Rare Diseases | ALL | CHILD, ADULT, OLDER_ADULT | xCures, Los Altos, California, 94022, United States |
| Examination of Protective Factors Against Severe Malaria | This study, sponsored by the National Institutes of Health and the University of Bamako in Mali, Africa, will examine factors that may protect against progression of malaria from mild to severe disease. Infection with the malaria parasite causes disease ranging in severity from mild or no symptoms to severe. A better understanding of what factors protect against disease progression may help scientists develop improved methods of disease prevention and treatment. The objectives of this study are to: * Identify differences in protective factors for severe malaria in Malinke children residing in two Mali villages, Kela and Kangaba. Genetic variations in hemoglobin proteins called HbS and HbC appear to confer protection against severe disease in some children but not others. HbC appears to protect young Malinke children living in Kela, but not in nearby Kangaba, while HbS protects children in Kangaba but not in Kela. In addition, deficiency of an enzyme produced by red blood cells called G6PD protects males, but not females, from severe malaria. * Investigate how fetal hemoglobin (HbF) may protect against malaria in infants and determine how HbS, HbC, G6PD deficiency, and beta-thalassemia trait affect the rate of HbF decline during the first 2 years of life. Children under 11 years of age who seek medical care at Kangaba or Kela health centers for symptoms of malaria may be eligible for this study. Each will be screened with a medical history, physical examination and blood test. In addition, healthy infants born to women referred to field site clinics may be enrolled for the newborn study. Participants undergo the following procedures: Children with mild malaria are treated with artesunate and amodiaquine. Those with severe malaria are treated with quinine. Blood is collected by finger prick every day for 4 days to evaluate the response to treatment and for genetic testing. Some blood is stored for future research related to malaria. Newborns have a heel or finger prick at 1, 3 and 6 months to collect a small blood sample for genetic testing. In addition, at the time of birth, a small amount of blood is collected from one of the blood vessels of the placenta. Some infants may be followed up to 2 years, with additional drops of blood taken at 12, 18 and 24 months. Some of the blood is stored for future research related to malaria. | Malaria|Severe Malaria | ALL | CHILD | Malaria Research and Training Center, Bamako, Mali |
| Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients | Thiotepa is a chemotherapy drug used extensively in bone marrow transplantation. Thiotepa is a prodrug that undergoes metabolic conversion in the liver by CYP2B6 and CYP3A4 to its primary active metabolite, triethylene phosphoramide (TEPA). The goal of this study is to determine what causes some children to have different drug concentrations of thiotepa and TEPA in their bodies and if drug levels are related to whether or not a child experiences severe side-effects during their bone marrow transplant. The hypothesis is that certain clinical and genetic factors cause changes in thiotepa and TEPA drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects. | Hematologic Malignancies|Nonmalignant Diseases|Immune Deficiency|Hemoglobinopathies|Genetic Inborn Errors of Metabolism|Fanconi Anemia|Thalassemia|Sickle Cell Disease | ALL | CHILD | University of California, San Francisco, San Francisco, California, 94143, United States |
| Host Immune Dynamics Following Seasonal Malaria Chemoprevention in African Children | The study aims to identify immune features linked to protection against malaria, examining variations by age and sex, and measuring changes in immune responses over time following seasonal malaria chemoprevention (SMC). The primary objectives are: 1. Thorough examination of antibody immune responses in individuals before and after one or more SMC rounds. 2. Thorough examination of both innate and adaptive cellular immune responses in people residing in areas with regular SMC use. Participants will include children aged 3 to 59 months who have received at least two or three SMC rounds, as well as children aged 5 to 15 years with no prior SMC exposure. Upon identifying participants, study details will be provided, and written informed consent obtained. Demographic and clinical information will be collected using standard case record forms, including previous SMC uptake and routine malaria control interventions. Information on SMC use will be obtained from SMC cards/existing medical records, detailing the number of cycles received, adherence, and general health condition, including previous diseases and EPI vaccine uptake. Data Collection: * Face-to-face interviews by trained research assistants. * Clinical assessments, including temperature, weight, and mid-upper arm circumference (MUAC). * Sickle cell status testing at enrolment via samples analyzed at the Molecular Research Laboratory (MOLAB). * Hemoglobin measurement using a HemoCue analyzer. Malaria Diagnosis: * Blood samples collected for malaria diagnosis using rapid diagnostic tests (RDTs) and microscopy. * RDTs, such as Paracheck Pf, will be performed according to manufacturer instructions, with results available within 15 minutes. * Participants testing positive for malaria will receive a full course of antimalarial treatment and counselling to visit the nearest health facility if symptoms worsen. Thick Blood Smears: * New glass slides labelled with participant ID will be used for thick blood smears. * Blood smears will be dried and stored in a cool, dust-free environment before transportation to central laboratories. * Slides will be stained with 2% Giemsa for 30 minutes and evaluated for parasitemia and gametocytes, with densities calculated accordingly. The study will help understand the immune responses to malaria and the efficacy of SMC in different age groups. | Malaria,Falciparum | ALL | CHILD | Moroto Hospital, Moroto, Karamoja, Uganda |
| Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer | This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer. Drugs used in chemotherapy, such as fludarabine phosphate and cyclophosphamide, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays to kill cancer cells and shrink tumors. Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient?s immune cells and help destroy any remaining cancer cells. | Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive|Acute Leukemia in Remission|Acute Lymphoblastic Leukemia|Acute Myeloid Leukemia|Acute Myeloid Leukemia With FLT3/ITD Mutation|Acute Myeloid Leukemia With Gene Mutations|Aplastic Anemia|B-Cell Non-Hodgkin Lymphoma|CD40 Ligand Deficiency|Chronic Granulomatous Disease|Chronic Leukemia in Remission|Chronic Lymphocytic Leukemia|Chronic Myelogenous Leukemia, BCR-ABL1 Positive|Chronic Myelomonocytic Leukemia|Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive|Congenital Amegakaryocytic Thrombocytopenia|Congenital Neutropenia|Congenital Pure Red Cell Aplasia|Glanzmann Thrombasthenia|Immunodeficiency Syndrome|Myelodysplastic Syndrome|Myelofibrosis|Myeloproliferative Neoplasm|Paroxysmal Nocturnal Hemoglobinuria|Plasma Cell Myeloma|Polycythemia Vera|Recurrent Non-Hodgkin Lymphoma|Refractory Non-Hodgkin Lymphoma|Secondary Acute Myeloid Leukemia|Secondary Myelodysplastic Syndrome|Severe Aplastic Anemia|Shwachman-Diamond Syndrome|Sickle Cell Disease|T-Cell Non-Hodgkin Lymphoma|Thalassemia|Waldenstrom Macroglobulinemia|Wiskott-Aldrich Syndrome | ALL | CHILD, ADULT, OLDER_ADULT | Roswell Park Cancer Institute, Buffalo, New York, 14263, United States |
| National Registry of Rare Kidney Diseases | The goal of this National Registry is to is to collect information from patients with rare kidney diseases, so that it that can be used for research. The purpose of this research is to: * Develop Clinical Guidelines for specific rare kidney diseases. These are written recommendations on how to diagnose and treat a medical condition. * Audit treatments and outcomes. An audit makes checks to see if what should be done is being done and asks if it could be done better. * Further the development of future treatments. Participants will be invited to participate on clinical trials and other studies. The registry has the capacity to feedback relevant information to patients and in conjunction with Patient Knows Best (Home - Patients Know Best), allows patients to provide information themselves, including their own reported quality of life and outcome measures. | Adenine Phosphoribosyltransferase Deficiency|AH Amyloidosis|AHL Amyloidosis|AL Amyloidosis|Alport Syndrome|Atypical Hemolytic Uremic Syndrome|Autoimmune Distal Renal Tubular Acidosis|Autosomal Recessive Proximal Renal Tubular Acidosis|Autosomal Recessive Distal Renal Tubular Acidosis|Autosomal Dominant Polycystic Kidney Disease|Autosomal Recessive Polycystic Kidney Disease|Bartter Syndrome|BK Nephropathy|C3 Glomerulopathy With Monoclonal Gammopathy|C3 Glomerulopathy|Calciphylaxis|Crystalglobulinaemia|Crystal-storing Histiocytosis|Cystinosis|Cystinuria|Dense Deposit Disease|Dent Disease|Denys-Drash Syndrome|Dominant Hypophosphataemia With Nephrolithiasis and/or Osteoporosis|Drug Induced Fanconi Syndrome|Drug-Induced Hypomagnesemia|Drug-Induced Nephrogenic Diabetes Insipidus|Epilepsy, Ataxia, Sensorineural Deafness and Tubulopathy|Fabry Disease|Familial Hypomagnesemia With Hypercalciuria and Nephrocalcinosis|Familial Primary Hypomagnesemia With Hypocalcuria|Familial Primary Hypomagnesaemia With Normocalciuria|Familial Renal Glucosuria|Fanconi Renotubular Syndrome 1|Fanconi Renotubular Syndrome 2|Fanconi Renotubular Syndrome 3|Fibrillary Glomerulonephritis|Fibromuscular Dysplasia|Focal Segmental Glomerulosclerosis|Generalised Pseudohypoaldosteronism Type 1|Gitelman Syndrome|Heavy-Metal-Induced Fanconi Syndrome|Hepatocyte Nuclear Factor 1-Beta-Associated Monogenic Diabetes|Hereditary Renal Hypouricemia|Hereditary Hypophosphatemic Rickets With Hypercalciuria|Hyperuricaemic Nephropathy|IgA Nephropathy|Immunotactoid Glomerulonephritis With Organised Microtubular Mononoclonal Immunoglobulin Deposits|Inherited Renal Cancer Syndromes|Intracapillary Monoclonal IgM Without Cryoglobulin|Intraglomerular/Capillary Lymphoma/Leukaemia|Isolated Autosomal Dominant Hypomagnesaemia Glaudemans Type|Liddle Syndrome|Light Chain Cast Nephropathy|Light Chain Proximal Tubulopathy Without Crystals|Light Chain Proximal Tubulopathy With Crystals|Lowe Syndrome|Membranous Nephropathy|Membranoproliferative Glomerulonephritis|Medullary Cystic Kidney Disease|Minimal Change Nephropathy|Mitochondrial Disease Of The Kidney|Monoclonal Immunoglobulin Deposition Disease|Nail Patella Syndrome|Nephrogenic Diabetes Insipidus|Nephrogenic Syndrome of Inappropriate Antidiuresis|Nephronophthisis|Primary Hypomagnesemia With Secondary Hypocalcemia|Primary Hyperoxaluria|Proliferative Glomerulonephritis With Monoclonal IgG Deposits|Proximal Tubulopathy Without Crystals|Pseudohypoaldosteronism Type 1, 2A-2E|Pure Red Cell Aplasia|Retroperitoneal Fibrosis|Sickle Cell Nephropathy|Shiga Toxin Associated Haemolytic Uraemic Syndrome|Steroid Resistant Nephrotic Syndrome|Steroid-Sensitive Nephrotic Syndrome|Thin Basement Membrane Nephropathy|Thrombotic Microangiopathy With Monoclonal Gammopathy|Type 1 Cryoglobulinaemic Glomerulonephritis|Tuberous Sclerosis|Unclassified Monoclonal Gammopathy Of Renal Significance|Vasculitis | ALL | CHILD, ADULT, OLDER_ADULT | Zoe Plummer, Bristol, South West, BS34 7RR, United Kingdom |
| Discarded Bone Marrow for Hematology Research | The primary objective of this study is to establish a mechanism to obtain discarded bone marrow-containing bone samples from hemoglobinopathy, as well as non-hemoglobinopathy individuals. The processing of samples will help to understand how best to manipulate HSPC's from hemoglobinopathy patients with gene therapy and gene technologies in the laboratory environment. It will also allow us to establish a reservoir of samples that can be studied in the future to assess cellular function and fitness for transplant. Secondary objectives * To develop gene transfer and gene editing strategies as potentially curative therapy for hemoglobinopathies (e.g. sickle cell disease (SCD) and β-thalassemia). * To develop a drug treatment strategy which elevates the expression of fetal hemoglobin to a potentially curative level for hemoglobinopathies. * To examine the biology of bone marrow cells isolated from patients with hemoglobinopathies. | Hemoglobinopathies | ALL | CHILD, ADULT, OLDER_ADULT | St. Jude Children's Research Hospital, Memphis, Tennessee, 38105, United States |
| Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT) | Fludarabine and clofarabine are chemotherapy drugs used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of clofarabine and fludarabine in their bodies and if drug levels are related to whether or not a child experiences severe side-effects during their bone marrow transplant. The hypothesis is that clinical and individual factors cause changes in clofarabine and fludarabine drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects. | Hematologic Malignancies|Nonmalignant Diseases|Immunodeficiencies|Hemoglobinopathies|Genetic Inborn Errors of Metabolism|Fanconi's Anemia|Thalassemia|Sickle Cell Disease | ALL | CHILD | University of California, San Francisco, San Francisco, California, 94143, United States |
| A Prospective Evaluation of Colorado's New Statutory PDMP Mandates: Compliance and Patient Outcomes | The goal of this study is to test the impact of a clinical decision support (CDS) tool to improve health care provider practices in line with state law requiring review of the Colorado prescription drug monitoring program (PDMP) before prescribing an opioid analgesic (pain medications often called narcotics) or benzodiazepine (sedatives or muscle relaxants). The PDMP is a statewide database of filled controlled medication, allowing health care providers to review medications ordered by other health care providers in the state and identify high-risk factors for overdose. The CDS tool only appears when a relevant prescription is being written by a health care provider and is purely informational, not dictating care or changes in treatment. The study will track how the tool is used by health care providers, if an opioid or benzodiazepine prescription is signed, and future opioid use by patients. | Medication Abuse | ALL | CHILD, ADULT, OLDER_ADULT | University of Colorado Hospital, Aurora, Colorado, 80045, United States |
| Comorbidities and Risk Score in COVID-19 Patients | Retrospective multi-center cohort study. Consecutive patients hospitalized for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) up to October 2020 will be included. Patients are followed until discharge from hospital or death. | Covid19 | ALL | ADULT, OLDER_ADULT | ASST Grande Ospedale Metropolitano Niguarda, Milan, Italy|ASST Spedali Civili, Montichiari, Italy|ASST Monza-Ospedale San Gerardo, Monza, Italy|Humanitas Clinical and Research Hospital, Rozzano, Italy|Centre for Tropical and Infectious Diseases and Microbiology, IRCCS Sacro Cuore, Verona, Italy |
| PATient Navigator to rEduce Readmissions | Staying out of the hospital is valued by patients and their caregivers. Their interests converge with those of hospitals now that high 30-day readmission rates for some conditions place hospitals at risk for financial penalties from the Centers for Medicare and Medicaid Services. This study focuses on developing and testing a program that combines a community health worker (lay patient advocate, acting as a "Patient Navigator") and a peer-led telephone support line to improve patient experience during hospital to home transition. | Chronic Obstructive Pulmonary Disease|Heart Failure|Sickle Cell Disease|Myocardial Infarction|Pneumonia | ALL | ADULT, OLDER_ADULT | University of Illinois Hospital, Chicago, Illinois, 60612, United States |
| The Deferasirox-calcium-vitamin D3 Therapy for Postmenopausal Osteoporosis (PMOP) | In 2006, Weinberg proposed a hypothesis that iron accumulation was a risk factor for osteoporosis. Osteoporosis is a common complication in various diseases, such as hemochromatosis, African hemosiderosis, thalassemia, and sickle cell disease, which all share iron accumulation as a common denominator. Moreover, a 3-year retrospective longitudinal study has shown that iron accumulation was also associated with osteoporosis in healthy adults and especially that it can increase the risk of fractures in postmenopausal women. Based on these observations, iron chelation therapy may have a promising future in the treatment of iron accumulation-related osteoporosis by removing iron from the body. The purpose of this study is to determine whether the addition of the iron chelator, deferasirox, to standard therapy for postmenopausal osteoporosis, is safe and effective. | Postmenopausal Osteoporosis | FEMALE | ADULT, OLDER_ADULT | Second Affiliated Hospital of Soochow University, Suzhou, Jiangsu, 215004, China |
| A Study to Test How Either a Capsule or a Tablet With NDec (Decitabine and Tetrahydrouridine) Works in the Body of Healthy People | This study will look at two different oral formulations and compare them. The medicine in the study is called NDec and it is a combination of two medicines (decitabine and tetrahydrouridine). Both medicines are new for the treatment of sickle cell disease, a rare blood disease. The purpose of the study is to compare the absorption of two different NDec versions (a tablet and a capsule). Participant will either get first the tablet and then the capsule, or the other way around. The order in which participant get them is decided by chance. The study will last for about 12 to 45 days depending on the wash-out period between the two stays in the clinic and from recruitment to the first study day. | Healthy Volunteers | ALL | ADULT | ICON-Salt Lake City, Salt Lake City, Utah, 84124, United States|Profil Institut für Stoffwechselforschung GmbH, Neuss, 41460, Germany |
| Evaluation of Toxicity From Stem Cell Transplant | This study will try to determine what causes toxic side effects of stem cell transplantation, such as increased blood pressure, increased heart rate, decreased kidney function and abnormal heart rhythms. Stem cells are used to treat various diseases, including cancer, aplastic anemia and sickle cell disease. The cells may be given fresh to the patient or they may be preserved first with a chemical called DMSO and frozen for later use. Some stem cell transplant procedures include infusion of red blood cells along with the stem cells. This study will examine whether side effects of stem cell transplants are associated with the DMSO preservative in frozen cells or with hemoglobin (a protein released from defrosted red blood cells) or neither of these factors. Healthy volunteers and patients scheduled to receive a stem cell transplant may be eligible for this study. Candidates must be between 10 and 80 years of age. Transplant patients will undergo a stem cell transplant. The cells are infused through a catheter placed in a vein for the procedure. Depending on the patient s requirements, the infusion may or may not include red blood cells and may or may not contain DMSO. Healthy volunteers undergo a 4-hour saline infusion. The saline (water mixed with salt) is infused through a catheter (plastic tube) placed in a vein in the arm. In addition, all participants have the following tests and procedures: * Heart monitoring: Healthy volunteers wear a portable heart monitor, attached to the chest using four stickers, for 24 hours starting the morning of the infusion. Transplant patients wear the same device for 48 hours, starting the morning before the infusion. * Blood draws and urine collections before, during, just after and the morning after the infusion of saline or stem cells. * Heart ultrasound before, during or just after and the morning after the infusion. * Peripheral artery tonometry: A small cup is placed on one finger of each hand to measure blood flow in the finger. A blood pressure cuff is inflated around the lower arm and tight pressure is maintained for about 5 minutes. | Organ Dysfunction|Hemolysis | ALL | CHILD, ADULT, OLDER_ADULT | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States |
| Comparing CGM and OGTT in Relation to Iron Overload Detected by Pancreas T2* MRI in High-Risk Hematology Group | A prospective, observational, comparative study with no intervention.The objective of the study to compare the efficiency of detecting glycemic abnormalities using Continuous Glucose Monitoring (CGMs) versus Oral Glucose Tolerance Test (OGTT) and HbA1C (Glycated Hemoglobin) and their relation to iron overload detected by T2\* MRI of the pancreas in high-risk patients due to insulin deficiency (potential beta cell injury) and those with insulin resistance and to study the different factors that may affect the glycemic control in these patients in relation to their results like the Dose of corticosteroids and chemotherapy in ALL and Hemoglobinopathies, Liver function in ALL and Hemoglobinopathies, and Serum ferritin in Hemoglobinopathies and their transfusion status. Using Validated Tools with Permission, the participants will be selected through probability (random) sampling method with expected subjects numbers ALL/L: 30-50, Thalassemia Major: 20, Sickle cell disease: 20. | Iron Overload|Hemoglobinopathies|Lymphoma|Acute Lymphoblastic Leukemia | ALL | CHILD, ADULT, OLDER_ADULT | |
| Effects of Prebiotics on Gut Microbiome in Patients Undergoing HSCT | The purpose of this study is to see whether hematopoietic stem cell transplant (HSCT) patients can consistently eat a diet rich in prebiotics. This type of diet may be helpful in maintaining diversity in the gastrointestinal (GI) system and therefore potentially decreasing risk of other GI problems. | Multiple Myeloma|Acute Myeloid Leukemia|Acute Lymphoblastic Leukemia|Non Hodgkin Lymphoma|Acute Lymphocytic Leukemia|Hodgkin Lymphoma|Hodgkin Disease|Myelofibrosis|Myelodysplastic Syndromes|Myeloproliferative Neoplasm|Sickle Cell Disease|Mantle Cell Lymphoma | ALL | ADULT, OLDER_ADULT | University of Virginia, Charlottesville, Virginia, 22903, United States |
| High-Tc Susceptometer to Monitor Transfusional Iron Overload | The proposed research project will continue the application and development of a new method (biomagnetic susceptometry) that measures magnetic fields to determine how much iron is in the liver. The amount of iron in the liver is the best indicator of the amount of iron in the whole body. Measuring the amount of iron in the body is important because either too much (iron overload) or too little iron (iron deficiency) can be harmful. At present, the most reliable way to measure the amount of iron in the liver is to remove a sample of the liver by biopsy, either by surgery or by using a needle which pierces the skin and liver. Iron stored in the liver can be magnetized to a small degree when placed in a magnetic field. In patients with iron overload, the investigators previous studies have shown that magnetic measurements of liver iron in patients with iron overload are quantitatively equivalent to biochemical determinations on tissue obtained by biopsy. In the past the investigators have developed a device to measure the amount of magnetization, which was called a SQUID (Superconducting QUantum Interference Device) susceptometer. This device was validated and in use for over 20 years. The safety, ease, rapidity and comfort of magnetic measurements make frequent, serial studies technically feasible and practically acceptable to patients. The investigators have now developed a new susceptometer, which uses very similar technology to the SQUID, but the investigators believe is more accurate and precise. This study aims to validate this new instrument. The investigators will do prospective, serial studies of the diagnosis and management of patients with iron overload, including thalassemia major (Cooley's anemia), sickle cell disease, aplastic anemia, myelodysplasia, hereditary hemochromatosis, and other disorders. Funding Source - FDA OOPD. | Transfusional Iron Overload|Thalassemia Major|Sickle Cell Disease|Myelodysplastic Syndromes|Aplastic Anemia | ALL | CHILD, ADULT, OLDER_ADULT | Columbia University Medical Center, New York, New York, 10032, United States |
| Neonatal Hyperbilirubinaemia in the Democratic Republic of Congo | Neonatal hyperbilirubinaemia (NH) is common among healthy neonates and normally resolves within a week. Untreated pathological hyperbilirubinaemia, however, can result in long-term neurological sequelae, which compromise childhood development, or may result in perinatal death. True population-based data from middle to low-income countries are scarce and NH contribution to morbidity and mortality remains unclear. With this study the investigators aim at assessing the prevalence of neonatal hyperbilirubinaemia in a cohort of newborns in a maternity hospital in Kinshasa, the Democratic Republic of Congo, and at evaluating the possible risk factors for NH in the mother and the baby. | Neonatal Hyperbilirubinemia | ALL | CHILD | Kinshasa Medical Oxford Research Unit, Kinsasa, Congo, The Democratic Republic of the |
| Defining Immunodeficiency in Heterotaxy Syndrome: Pilot Study Data | The investigators aim with this study is to investigate the mechanisms of immune deficiency in patients with heterotaxy syndrome through the use of novel biomarkers and a prospective questionnaire survey documenting the burden of infectious sequelae following enrollment. It is known that patients with under-active spleens (functional asplenia or hyposplenia) secondary to other (non-cardiac) conditions such as Sickle Cell Disease or Inflammatory Bowel Disease have a characteristic paucity of a B cell sub-class known as IgM memory B cell. This specific sub-class of B cell normally matures in the spleen and in those with an improperly functioning spleen a significant deficiency of this B cell class is seen on flow cytometry. Similarly, these same patients are noted to have increased amounts of 'junk' DNA / nuclear remnant in their red cells. This is seen on microscopy as a dark particle inside the red cell and is termed a Howell Jolly Body (normally less than 2% of red cells have these dark particles present). Part of a functioning spleen's normal task is to rid the blood of red cells that contain nuclear remnants and an under-active spleen gets behind on this task with a build-up of Howell Jolly Bodies in red cells present in the bloodstream. Flow cytometry can very quickly and accurately quantify Howell Jolly Bodies as well as IgM memory B cells from a small (\~1.5cc) sample of blood. Normal IgM memory B cell ranges are known for healthy children from infancy onwards allowing interpretation of results against normative data ranges. The investigators aim to enroll 10 patients in this pilot study who have a diagnosis of heterotaxy syndrome (both asplenia and polysplenia) and to prospectively follow them after obtaining the initial biomarker sample. The family will be contacted once every two weeks for a period of 12 weeks and asked a series of simple questions taking approximately 5 minutes on any recent infectious sequelae or symptoms. The questions will elucidate history of minor illness such as low-grade fever or cough to more significant events such as admission for in-patient antibiotic therapy of bacterial sepsis. Ultimately, with this pilot study, the investigators hope to obtain sufficient data to support funding applications for a larger, multi-center trial that will allow us to develop biomarker thresholds for future risk of sepsis. | Heterotaxy Syndrome | ALL | CHILD | Boston Children's Hospital, Boston, Massachusetts, 02115, United States |
| A Nationwide Italian Survey on Asplenia | Spleen is involved in several functions, such as the production of protective antibodies, the removal of unwanted particulate matter from the blood (eg bacteria) and also the storing of blood cells, especially white cells and platelets. Asplenia is a status due to spleen absence or dysfunction, which results from several rare diseases. Congenital Asplenia is a condition with absent or dysfunctional spleen, associated with other congenital abnormalities; functional asplenia is a status with present but dysfunctional spleen, related to many rare diseases, such as sickle cell disease, thalassemia, essential thrombocythaemia, lymphoproliferative diseases and splenectomy is the surgical removal of the spleen in order to treat a huge number of rare hematological and oncological diseases. So, asplenia is the final result of a numerous variety of rare disorders, and it leads to a high risk of infections and thrombotic events with significant mortality and morbidity. Antibiotic prophylaxis and specific vaccinations are recommended in this high risk population but adherence was shown to be very poor. A national register was demonstrated to improve population outcomes and reduce health care costs and facilitate research and public health purposes in this target population. In Europe very little experience exists in comprehensive national program for management of asplenia, and only in a restricted part of England and in Ireland surveys of post-splenectomized patients have been performed. In Italy no common policy of patient care has yet been developed, and management of asplenia is mainly case or locally directed. | Splenectomy; Status | ALL | CHILD, ADULT, OLDER_ADULT | |
| Utility of the Cardiac Electrical BiomarkerDisease | This project is aiming to identify the diagnostic utility CEB (Cardiac Electrical Biomarker) in patients who are undergoing cardiac investigations. | Acute Chest Syndrome | ALL | ADULT, OLDER_ADULT | Milton Keynes University Hospital NHS Foundation Trust, Milton Keynes, Bucks, MK6 5LD, United Kingdom |
| Hematogenous Osteomyelitis in Childhood Can Relapse Dozens of Years in Adulthood | To our knowledge, few cases of relapse in adulthood are described in the literature except in patients with sickle cell disease and the epidemiological, clinical, laboratory, radiological features and the management of osteomyelitis relapsing in adulthood are not described. The aim of this retrospective multicentric cohort study : in France is to describe the epidemiological, clinical, laboratory, and radiological features and the management of adult patients who experienced a relapse between 2003-2015 of an acute hematogenous osteomyelitis acquired in the childhood (description of characterization of the period between the first episode of osteomyelitis and the second episode, description of signs of relapse and description of treatments used in the relapse). The data are analysed with non-comparative descriptive statistics. | Osteomyelitis|Hematogenous Osteomyelitis Relapse | ALL | ADULT, OLDER_ADULT | Hospices Civils de Lyon - Hopital de la Croix Rousse, Lyon, 69004, France |
| Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload | The purpose of this research study is to study the safety of increasing doses of FBS0701, and to see how quickly the study medication is absorbed and how quickly it disappears from the bloodstream. FBS0701 is a new, oral iron chelator - a medication taken by mouth that increases the body's elimination of iron. Iron chelators are used in patients who develop iron overload from their transfusions. Four increasing doses of FBS0701 will be tested during this study. The study will start with the lowest dose given to 4 patients (3 mg/kg/day. The next group of 4 patients will receive the next high dose (8mg/kg/day only after the results of the first 4 patients are examined and it is determined safe to continue. Participating patients will take the study medication for 7 days and be followed for 28 days after their last dose to determine if they have any reactions to the study medication - therefore a total of 35 days on study. Patients will need to give up to 17 blood samples over the screening period and first 15 days of the study (a total of about 9 tablespoons). Patients will not need to stay overnight in the clinic but will need to visit the clinic 10 times for screening and on-study visits over the 35 days. Patients currently taking an iron chelator will need to stop that treatment for up to 22 days (up to 5 days before they start the study and for 15 days during the study). The results of this study will be helpful in determining the safety of the drug and the best doses of FBS0701 to be used in the next study which will assess the effectiveness of this new iron chelator. | Transfusional Iron Overload|Beta-thalassemia | ALL | ADULT, OLDER_ADULT | Children's Hospital of Oakland, Oakland, California, 94609, United States|Children's Hospital of Boston, Boston, Massachusetts, 02115, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Royal Adelaide Hospital, Adelaide, Australia|Siriraj Hospital, Mahidol University, Bangkok, 10700, Thailand |
| Evaluation of a Mobile Direct Observation Therapy (DOT) Approach in Children and Young People With Asthma | Mobilte Direct Observation Therapy (MDOT) is a technology has the potential to be a cost effective approach to direct observation of therapy administration, the latter being one of the most accurate methods of evaluating adherence. Use to date, as confirmed by the rapid systematic review, has been limited mainly to TB and sickle cell disease and there have been no published reports on the use of MDOT to monitor inhaled therapy. Due to the increasing incidence of childhood asthma worldwide, there is a need for new innovative approaches to support children and their parents with asthma management, especially since national and international guidelines have advised healthcare providers to periodically assess inhaler use as part of asthma management. | Asthma in Children | ALL | CHILD | |
| Risk Factors and Etiologies of Epilepsy in Urban and Rural Rwanda | Epilepsy is one of the most common chronic brain disorders. Up to 85% of persons living with epilepsy (PwE) live in the developing world. In sub-Saharan Africa (SSA), Rwanda has one of the highest prevalence rates (±5%). Higher prevalence in low-and middle-income countries (LMICs) can partly be attributed to differences in risk factors for epilepsy of which a great number are preventable. Expanding knowledge on risk factors and etiologies of epilepsy in Rwanda can lower the portion of preventable epilepsies and decrease the high number of Rwandan PwE. This project will focus on the investigation of risk factors and etiologies of epilepsy in urban and rural Rwanda using a nationwide approach. | Epilepsy | ALL | CHILD, ADULT, OLDER_ADULT | King Faisal Hospital, Kigali, Rwanda |
| New Tools for Predicting Capillary Leak Shock During Dengue Fever | Evaluate the prognostic value of different methods (Osmometry / clinical-biological score) compared to the occurrence of capillary leak shock during dengue fever. | Dengue Fever|Children|Adults | ALL | CHILD, ADULT, OLDER_ADULT | General Hospital of Cayenne, Cayenne, French Guiana, 97306, France |
| Distracting Through Procedural Pain and Distress | Children with acute and chronic illness undergo frequent, painful, and distressing procedures. This randomized control trial was used to evaluate the effectiveness of guided imagery (GI) vs virtual reality (VR) on the procedural pain and state anxiety of children and young adults undergoing un-sedated procedures. We explored the role of trait anxiety and pain catastrophizing in intervention response. | Chronic Illness|Hematologic Malignancy|Bone Marrow Transplant Infection|Oncology|Sickle Cell Disease | ALL | CHILD, ADULT | Children's Wisconsin, Milwaukee, Wisconsin, 53226, United States|Medical College of Wisconsin, Wauwatosa, Wisconsin, 53226, United States |
| Assessment of Immediate Postoperative Delirium (IPD) in Adult Patients: Incidence and Etiologic Factors | Delirium is considered to be acute failure of central nervous system. It is acute confusional state characterized by decline from baseline mental level, attention deficit and disorganized thinking. Postoperative delirium is known to prolong length of stay in hospital, cause functional decline and dementia, increase all-cause mortality and increase the medical cost. It is also associated with other outcomes like cardiac arrest, ventricular tachycardia or fibrillation, myocardial infarction, pulmonary edema, pulmonary embolism, bacterial pneumonia, respiratory failure requiring intubation, renal failure requiring dialysis and stroke. There are well known predisposing and precipitating factors related to its etiology. However, the effect of type of anesthesia is not very clear. There have been no major clinical trials in this part of the world to delineate the incidence of immediate postoperative delirium (IPD). The investigators have undertaken this prospective observational study to determine the incidence of IPD and its etiological factors in adult patients during their stay in the Post-Anesthesia Care Unit (PACU) following surgery under different types of anesthesia (general anesthesia, regional anesthesia and monitored anesthesia care). The study was done over a period of about three months. Assessment for delirium was done using Confusion Assessment Method-Intensive Care Unit (CAM-ICU score, English/Arabic version). Sedation and Agitation were assessed using Richmond Agitation Sedation Score (RASS). Pain was assessed using Numeric Pain Score (NPS). Assessment was done within 24 hours prior to surgery and was repeated at three different intervals in PACU. Details of perioperative management were recorded and analyzed. The incidence of IPD and its etiologic factors were identified thereby leading to corrective action. | Delirium|Anesthesia; Adverse Effect | ALL | ADULT, OLDER_ADULT | Sultan Qaboos University Hospital,, Muscat, 123, Oman |
| A Health System Wide Evaluation of Clinical Decision Support Tools to Improve PDMP Utilization and Patient Outcomes | This is a study comparing three clinical decision support (CDS) tools to enhance care by easing health care provider review of the Colorado prescription drug monitoring program (PDMP) prior to prescribing opioids (pain medications often called narcotics) or benzodiazepines (sedatives or muscle relaxants). The tools screen information from the PDMP (a statewide database of filled controlled medication) and a patient's medical record to identify high-risk factors for overdose. The tools only appear when relevant, are purely informational to facilitate an evidence-based practice (PDMP review) and do not dictate care or suggest changes in treatment. The study will track how each of the tools are used and if providers use the PDMP. Secondary outcomes include if a controlled medication prescription was written and future opioid use by patients. | Medication Abuse | ALL | CHILD, ADULT, OLDER_ADULT | University of Colorado Hospital, Aurora, Colorado, 80045, United States |
| Combination of Hydroxyurea and Verapamil for Refractory Meningiomas | Meningiomas account for 20% of primary adult brain tumors, occurring at an annual incidence of 6 per 100,000 (Louis, Scheithauer et al. 2000). Complete surgical resection is the treatment of choice but may not possible when the tumor invades critical structures (e.g., skull base, sagittal sinus) (Mirimanoff, Dosoretz et al. 1985; al-Rodhan and Laws 1990; Al-Rodhan and Laws 1991; Newman 1994; De Monte 1995; Levine, Buchanan et al. 1999; Barnett, Suh et al. 2000; Ragel and Jensen 2003). Up to 20% of meningiomas exhibit a more aggressive phenotype that does not respond to standard therapies (Kyritsis 1996). Adjuvant therapies are critical for patients with this subset of meningiomas. Radiation therapy and stereotactic radiosurgery are good adjuvant therapies but are limited by radiation neurotoxicity, tumor size constraints, and injury to adjacent vascular structures or cranial nerves (Goldsmith, Wara et al. 1994; Barnett, Suh et al. 2000; Goldsmith and Larson 2000). Standard chemotherapeutic treatments have been disappointing (Kyritsis 1996). Even drugs like temozolomide that have shown efficacy against malignant brain tumors have failed to inhibit the growth of refractory meningiomas in a phase II study (Chamberlain, Tsao-Wei et al. 2004). | Cancer|Brain Cancer|Meningioma | ALL | ADULT, OLDER_ADULT | Huntsman Cancer Institute, Salt Lake City, Utah, 84112, United States |
| Using Social Media to Decrease Healthcare Utilization for Pediatric Asthma | The purpose of this study is to evaluate if social media enhanced education (SME) will reduce total number of treatment days defined as summation of days of subsequent hospital admission, emergency room visits, and clinic visits, reduce missed school days, reduce total costs from a health system perspective, have increased effect with increased social media engagement, have increased effect in participants with both caregiver and patient (combined) with social media accounts, compared to participants where only the patient or only the caregiver uses social media (single),have increased patient satisfaction in the asthma education received and to obtain the experience and data needed to refine SME to be able to expand this platform for other chronic medical conditions with high healthcare utilization including pediatric diabetes, epilepsy, and sickle cell disease in children with asthma. | Asthma in Children | ALL | CHILD | The University of Texas Health Science Center at Houston, Houston, Texas, 77030, United States |
| Malaria Surveillance in Rakai, Uganda | Background: - Malaria is a leading cause of morbidity and mortality in Uganda, accounting for more than a quarter of all outpatient visits at health facilities, 20 percent of hospital admissions, and about 10 percent of inpatient deaths. Children under 10 years of age, pregnant women, and HIV-infected individuals bear the greatest burden of disease. To provide baseline information for future malaria vaccine research, development, and testing, researchers are interested in collecting malaria infection data from the Rakai district in southern Uganda. Objectives: - To assess the epidemiology of malaria infection among children aged 6 months to less than 10 years and adults living in same households with children in Rakai district, Uganda. Eligibility: - Children between 6 months and 10 years of age, as well as their primary caregiver and an additional randomly selected adolescent or adult resident of the household, from the Rakai district of Uganda. Design: * Participants will have monthly household visits for a 1-year surveillance period. * Each visit will include a structured interview/questionnaire of the primary caregiver or legal guardian of the child and clinical and laboratory assessments of each child, the primary caregiver, and the additional adolescent or adult resident of the household. The questionnaire will ask about malaria treatment and prevention measures. * Children will provide a blood sample for testing. Individuals (children or adults) who are diagnosed with malaria or anemia during the course of the study will be recommended for treatment. * Researchers will also track usage of the district health clinic and hospital services to link medical records for study participants. | Malaria | ALL | CHILD, ADULT, OLDER_ADULT | Rakai Health Sciences Program, Rakai, Uganda |
| Improving Quality by Maintaining Accurate Problems in the EHR | The overall goal of the IQ-MAPLE project is to improve the quality of care provided to patients with several heart, lung and blood conditions by facilitating more accurate and complete problem list documentation. In the first aim, the investigators will design and validate a series of problem inference algorithms, using rule-based techniques on structured data in the electronic health record (EHR) and natural language processing on unstructured data. Both of these techniques will yield candidate problems that the patient is likely to have, and the results will be integrated. In Aim 2, the investigators will design clinical decision support interventions in the EHRs of the four study sites to alert physicians when a candidate problem is detected that is missing from the patient's problem list - the clinician will then be able to accept the alert and add the problem, override the alert, or ignore it entirely. In Aim 3, the investigators will conduct a randomized trial and evaluate the effect of the problem list alert on three endpoints: alert acceptance, problem list addition rate and clinical quality. | Asthma|Atrial Fibrillation|Chronic Obstructive Pulmonary Disease|Coronary Artery Disease|Congestive Heart Failure|Hyperlipidemia|Hypertension|Myocardial Infarction|Sickle Cell Disease|Sleep Apnea|Smoking|Stroke|Tuberculosis | ALL | ADULT, OLDER_ADULT | Brigham and Women's Hospital, Boston, Massachusetts, 02115, United States|Oregon Health and Science University, Portland, Oregon, 97239, United States|Holy Spirit Hospital, Camp Hill, Pennsylvania, 17011, United States|Vanderbilt University Medical Center, Nashville, Tennessee, 37235, United States |
| Evaluation of the Attenuation by Aes-103 of Hypoxia Mediated Decrements in Endurance Exercise Performance | In low oxygen environments, such as high-altitude, some adults may become ill and suffer from acute mountain sickness. Further, all adults will find that exercising becomes much more difficult when compared with exercise at lower altitudes (e.g. sea-level). The purpose of this investigation is to study the effects of a new medicine called Aes-103. A company called AesRx, LLC makes this medicine. The active ingredient in the medicine is 5-Hydroxymethyl-2-Furfural (5HMF), a naturally occurring substance that can be found in coffee, honey, dried fruits, fruit juices, malt, barley, Balsamic vinegar and caramel.The investigators believe that Aes-103 may help people adjust to high-altitude quickly and prevent them from becoming ill. The purpose of the study is to determine if Aes-103 will promote endurance performance in low oxygen environments in healthy adult humans. Aes-103 is currently being investigated by AesRx, LLC (Newton, MA) in collaboration with the National Heart, Lung and Blood Institute of the NIH (Bethesda, MD) as a potential anti-sickling agent in sickle cell disease. Sickle-cell disease is characterized by problems in blood that prevent blood cells from carrying oxygen. Aes-103 might be able to help blood cells carry more oxygen. It is for this reason that the investigators in this study believe Aes-103 might help people adjust to high-altitude quickly. There are no known special safety considerations with the active ingredient in Aes-103 (5-HMF). In recent, placebo controlled, clinical safety tests, Aes-103 was given in single doses of 300 mg, 1000 mg,2000 mg and 4000 mg to healthy normal volunteers. Additionally, the toxicological effects of Aes-103 have been studied when given acutely, sub-acutely, and chronically in rodents, and for up to 28 days in dogs. Based on these safety studies, single doses of Aes-103 are expected to have no significant negative/toxicological effect at the doses being evaluated in this study. | Hypoxia | MALE | ADULT | Colorado State University, Dept. of Health and Exercise Science, Fort Collins, Colorado, 80523-1582, United States |
| Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders | The main purpose of this study is to examine the outcome of a combined bone marrow and kidney transplant from a partially matched related (haploidentical or "haplo") donor. This is a pilot study, you are being asked to participate because you have a blood disorder and kidney disease. The aim of the combined transplant is to treat both your underlying blood disorder and kidney disease. We expect to have about 10 people participate in this study. Additionally, because the same person who is donating the kidney will also be donating the bone marrow, there may be a smaller chance of kidney rejection and less need for long-term use of anti-rejection drugs. Traditionally, very strong cancer treatment drugs (chemotherapy) and radiation are used to prepare a subject's body for bone marrow transplant. This is associated with a high risk for serious complications, even in subjects without kidney disease. This therapy can be toxic to the liver, lungs, mucous membranes, and intestines. Additionally, it is believed that standard therapy may be associated with a higher risk of a complication called graft versus host disease (GVHD) where the new donor cells attack the recipient's normal body. Recently, less intense chemotherapy and radiation regimens have been employed (these are called reduced intensity regimens) which cause less injury and GVHD to patients, and thus, have allowed older and less healthy patients to undergo bone marrow transplant. In this study, a reduced intensity regimen of chemotherapy and radiation will be used with the intent of producing fewer toxicities than standard therapy. Typical therapy following a standard kidney transplant includes multiple lifelong medications that aim to prevent the recipient's body from attacking or rejecting the donated kidney. These are called immunosuppressant drugs and they work by "quieting" the recipient's immune system to allow the donated kidney to function properly. One goal in our study is to decrease the duration you will need to be on immunosuppressant drugs following your kidney transplant as the bone marrow transplant will provide you with the donor's immune system which should not attack the donor kidney. | Chronic Kidney Disease|Acute Myeloid Leukemia (AML)|Acute Lymphoblastic Leukemia (ALL)|Chronic Myelogenous Leukemia (CML)|Chronic Lymphocytic Leukemia (CLL)|Non-Hodgkin's Lymphoma (NHL)|Hodgkin Disease|Multiple Myeloma|Myelodysplastic Syndrome (MDS)|Aplastic Anemia|AL Amyloidosis|Diamond Blackfan Anemia|Myelofibrosis|Myeloproliferative Disease|Sickle Cell Anemia|Autoimmune Diseases|Thalassemia | ALL | ADULT, OLDER_ADULT | Massachusetts General Hospital, Boston, Massachusetts, 02114, United States |
| ORCHARD- Optimising Home Assessment of Rural Patients | This project assesses feasibility of providing medically vulnerable rural patients with Medical-Self-Assessment-Boxes containing equipment to use at home during telephone and video consultations (telemedicine) with GPs and other healthcare professionals. COVID-19 has caused an upsurge in primary care telemedicine which the investigators believe can be sustained and optimized to make things better for medically vulnerable rural patients beyond the pandemic. The investigators will achieve this by equipping the participants to self-measure and report key clinical measurements (e.g. blood pressure, temperature, oxygen levels) during telemedicine consultations. Before conducting a major evaluation of the Medical-Self-Assessment-Box for medically vulnerable rural patients the investigators must establish three things: First, to show the investigators can issue a Medical-Self-Assessment-Box to medically vulnerable rural patients and enable them to use it properly. Second, to determine that patients can use the Medical-Self-Assessment-Box effectively during telemedicine consultations. Third, to show that it is possible to measure how well the Medical-Self-Assessment-Box is working by counting how often the boxes are being used and whether use is appropriate and helpful. The knowledge gained will provide the investigators with the information needed to develop a funding proposal to evaluate Medical-Self-Assessment-Boxes for medically vulnerable rural patients in the whole of the UK. | Organ Transplant|Cancer|Inborn Errors of Metabolism|Immunosuppression|COPD Asthma|Sickle Cell Disease | ALL | ADULT, OLDER_ADULT | |
| Sildenafil To Prevent Clot | The advent of continuous flow (CF) pumps for patients with severe heart failure has led to marked improvements in survival; however, pump operation remains fraught with adverse thrombotic events. This climbing rate of thrombosis and stroke during CF pump support has led to a recent warning by the US Food and Drug Administration. Despite a rising incidence of pump thrombosis and its downstream complications of stroke, the hematologic mechanisms behind these devastating adverse events remain uncertain. Recently, it has been recognized that CF pump induced hemolysis precedes and is associated with thrombosis. In-vitro studies show increased platelet function with exposure to products of hemolysis, which is also known to occur in diseases of intravascular hemolysis such as sickle cell anemia. This proposal will investigate if hemolysis associated increased platelet function can be reduced by a potentiation of nitric oxide signaling by an oral phosphodiesterase-5 inhibitor, sildenafil. Elucidating mechanisms of hemolysis induced thrombosis may inform best strategies for prevention of end organ damage and maintaining optimal CF pump operation. | Thrombosis|Hemolysis | ALL | ADULT, OLDER_ADULT | Montefiore Medical Center, Bronx, New York, 10467, United States |
| Covid-19 Vaccine Cohort in Specific Populations | Multicentre national cohort study with prospective data collection and biological specimen collection. Ancillary study in this cohort : pediatric cohort with participants from 5 to 17 years old. Enrollment complete for adult cohort. Active recruting for ancillary pediatric cohort. | Immune Deficiency | ALL | ADULT, OLDER_ADULT | Cmg-Ec U1219, Bordeaux, France|Nîmes CHU, Nîmes, France|Paris Cochin APHP, Paris, France |
| Peer i-Coaching for Activated Self-Management Optimization in Adolescents and Young Adults With Chronic Conditions | The purpose of this study is to test the efficacy of a peer support coaching intervention to improve activated chronic illness self-management versus an attention control group in 225 adolescents and young adults with childhood onset chronic conditions. | Sickle Cell Disease|Chronic Kidney Diseases|Systemic Lupus Erythematosus|Childhood Cancer|Inflammatory Bowel Diseases|Stem Cell Transplant|Organ Transplant|Diabetes Mellitus, Type 1|Cystic Fibrosis | ALL | CHILD, ADULT | Duke University, Durham, North Carolina, 27710, United States |
| Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives | To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and preventative methods for specific and complex conditions. | Age-Related Macular Degeneration|Allergies|Alpha-Gal Syndrome|Alzheimer Disease|Amyloidosis|Ankylosing Spondylitis|Arthritis|Alopecia Areata|Asthma|Atopic Dermatitis|Autism|Autoimmune Hepatitis|Behcet's Disease|Beta-Thalassemia|Cancer|Celiac Disease|Kidney Diseases|COPD|Crohn Disease|Cystic Fibrosis|Diabetes|Dravet Syndrome|DMD|Fibromyalgia|Graves Disease|Thyroid Diseases|Hepatitis|Hidradenitis Suppurativa|ITP|Leukemia|ALS|Lupus or SLE|Lymphoma|Multiple Sclerosis|Myasthenia Gravis|Heart Diseases|Parkinson Disease|Pemphigus Vulgaris|Cirrhosis|Psoriasis|Schizophrenia|Scleroderma|Sickle Cell Disease|Stroke|Ulcerative Colitis|Vasculitis|Vitiligo | ALL | ADULT, OLDER_ADULT | Sanguine Biosciences, Waltham, Massachusetts, 02451, United States |
| Hyperglycaemia In Childhood Hematologic Malignancies | BACKGROUND/AIM: Secondary forms of diabetes are often understudied and underdiagnosed in children and adolescents with cancer. The objectives of this cohort study were to study the incidence and risk factors for hyperglycaemia in leukaemia and lymphoma patients. METHODS: The investigators retrospectively collected 15 years of data from paediatric patients treated for acute lymphoblastic leukaemia (ALL), Hodgkin's lymphoma (HL), and non-Hodgkin's lymphoma (NHL) immediately at cancer diagnosis. They studied risk factors for hyperglycaemia in univariate and multivariate analyses. | Diabetes Mellitus Risk | ALL | CHILD, ADULT | Cliniques Universitaires Saint-Luc - UCLouvain, Brussel, 1200, Belgium |
| Central Venous Catheter-Related Thrombosis in Critically Ill Patients | This project aims to identify catheter-related thrombosis (CRT) and determine its incidence, associated factors, and outcomes. The eligible patients will undergo daily ultrasound just distal to the central venous catheter insertion site for CRT. A staff radiologist will review all positive cases. These cases will be monitored by daily ultrasound examination till three days after the removal of the catheter or till the patient stays in the intensive care unit (ICU). The patient demographics, ICU treatment details, and outcomes will be noted. The data will be then analyzed. A preventive strategy will be prepared and disseminated to the department personnel to improve the quality of care. | Catheter Related Complication | ALL | ADULT, OLDER_ADULT | Sultan Qaboos University Hospital, Muscat, 123, Oman |
| Incomplete Follow Up After Positive FIT or Stool DNA Testing: A Multimethod Approach | This study seeks to determine whether a patient navigator can help improve follow up care after a stool test shows the presence of blood or other abnormal markers. | Positive FIT or Stool DNA Testing Follow up | ALL | ADULT, OLDER_ADULT | University Hospitals Cleveland Medical Center, Case Comprehensive Cancer Center, Cleveland, Ohio, 44106, United States |
| A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT | Research has suggested that children with sufficient vitamin D levels undergoing hematopoietic stem cell transplant (HSCT) have improved outcomes, including lower incidences of infection and graft-versus-host disease (GVHD), as well as overall improved survival. However, supplementation in children undergoing HSCT has shown to be a challenge using standard or aggressive supplementation strategies. The primary objective of this study is to determine the safety and efficacy of a single, high dose oral vitamin D (Stoss Therapy) at the start of transplant followed by maintenance supplementation in children undergoing HSCT. | Vitamin D Deficiency|Stem Cell Transplant Complications|Pediatric Cancer|Blood Disorder|Pediatric Acute Myeloid Leukemia|Pediatric Acute Lymphoid Leukemia|Myelodysplastic Syndromes|Sickle Cell Anemia in Children|Aplastic Anemia|Thalassemia in Children | ALL | CHILD, ADULT | Phoenix Children's Hospital, Phoenix, Arizona, 85016, United States |
| Can we Transfuse Blood Over Shorter Period ? | Blood transfusion is very lengthy procedure and consumes a substantial time from patients and health care providers. On average, it may take most if not all the working day which leads to significant constrains on hospital bed utilization. It starts from pre-transfusion testing, clinical assessment, actual administration of blood and post-transfusion care. The main bulk of this procedure is usually related to administration of the blood which typically given over 3 hours (5ml/kg/hour), although there is no strong evidence to support that. Indeed, it has been accepted as standard of care to transfuse blood over short time as in emergency situations. OBJECTIVE: To determine the maximum tolerated blood transfusion rate that can be safely delivered in patient who required blood transfusion i.e. transfusing blood over short time. METHOD: This is a phase I, open label, nonrandomized, prospective and rate-finding study. A well-known dose escalation design called 3+3 design will be used to identify the maximum tolerated rate. To assure the safety of such procedure, blood transfusion rate will be escalated very slowly by 1 ml/kg/hour for each cohort until rate-limiting toxicities or maximum of 10ml/kg/hour. | Blood Transfusion Complication | ALL | CHILD | |
| COVID-19 Testing in Underserved and Vulnerable Populations | As part of National Institutes of Health Rapid Acceleration of Diagnostics-Underserved Populations (RADx-UP) program, the goal of the RADxUP study is to develop, test, and evaluate a rapid, scalable capacity building project to enhance COVID-19 testing in three regional community health centers (CHCs) in San Diego County, California. In collaboration with CHC partners, their consortium organization, Health Quality Partners (HQP), investigators are pursuing the following Specific Aims: 1) Compare the effectiveness of automated calls vs text messaging for uptake of COVID-19 testing among asymptomatic adult patients with select medical conditions and those 65 years of age and older receiving care at participating CHCs. Secondarily, investigators will invite all study participants to receive flu vaccination and will assess feasibility and acceptability of study participants to refer adult family household members who are essential workers for COVID-19 testing. 2) Gather patient, provider, CHC leadership, and community stakeholder insights to establish best practices for future scale-up of COVID-19 testing sustainability and vaccination. | Heart Failure|Coronary Artery Disease|Cancer|Chronic Kidney Diseases|COPD|Obesity|Sickle Cell Disease|Diabetes Mellitus, Type 2 | ALL | ADULT, OLDER_ADULT | University of California, San Diego, La Jolla, California, 92093, United States |
| Building TrUst and UNiting Teams Through DouLa partnErship - BUNDLE | The BUNDLE study is a prospective mixed-methods study focused on the early integration of community doula into prenatal care. The study will have three phases: Phase 1 is the qualitative phase of conducting focus groups with Black/African American (AA) birthing people and with medical and community healthcare providers to elicit feedback on how best to integrate community-based doulas and obstetricians into one united model of prenatal care to promote trust and improved maternal health outcomes. Phase 2 tests the effectiveness of the newly developed model on healthcare engagement, trust, and adverse maternal outcomes using randomized control trial of 412 Black/AA pregnant participants. Phase 3 is dissemination of BUNDLE findings in scholarly and community-based forums, including with healthcare leaders and policy makers in Wisconsin, advocating for doula coverage and health system sustainability of the integrated model. | Pregnancy Related | FEMALE | ADULT, OLDER_ADULT | |
| Hydroxyurea in Pulmonary Arterial Hypertension | Pulmonary arterial hypertension (PAH) is a serious and eventually fatal disease damaging the lungs and the heart. It results from narrowing and eventual blockage of small blood vessels in the lung, due to abnormal proliferation of cells in the blood vessel (arterial). Patients with PAH suffer from fatigue, shortness of breath, low oxygen levels, blood clots and heart failure. No therapies reverse the disease process in the lung arteries, however there are three approved drugs that can temporarily dilate the vessels and improve symptoms. However, all three drugs have significant side effects and toxicities, they do not work effectively in many patients, survival remains on average only 2 to 3 years once symptoms begin, and none of these drugs prevent the underlying disease process in the small arteries of the lung. PAH is known to develop in patients with a pre-existing class of bone marrow diseases called myeloproliferative disorders (MPDs). We and others have recently shown that patients with PAH have bone marrow changes similar to those seen in patients with MPDs, even without other signs and symptoms of those bone marrow diseases such as anemia or high platelet and white blood cell counts. Compared to healthy volunteers, patients with PAH have a higher frequency of immature stem and progenitor cells able to produce blood cells and vascular wall cells in their bone marrow. They also have higher circulating numbers of these cells in the blood, and increased localization of these cells in the lung blood vessels. When immature bone marrow cells from PAH patients and normal volunteers were infused into mice, the mice receiving PAH marrow cells developed similar lung and heart problems to PAH patients, suggesting that the bone marrow problem is a primary cause of the lung problems, and that the increased numbers of immature bone marrow cells in the bone marrow and blood of PAH patients causes the lung blood vessel disease. The drug hydroxyurea is used to inhibit the abnormally high level of bone marrow cell proliferation in patients with MPDs. It has been shown to reduce the numbers of circulating immature bone marrow cells in patients with MPDs. Hydroxyurea has been available for almost fifty years, and has been used to treat patients with MPDs, sickle cell anemia, and congenital heart disease for very prolonged periods of time, up to twenty or more years in individual patients. It has an excellent long-term safety profile and few side effects and is generally well tolerated. It does not appear to result in an increased rate of leukemia even with many years of treatment. In the current protocol, we hypothesize that treating patients with PAH with hydroxyurea will decrease the level of circulating immature bone marrow cells and interrupt the abnormal narrowing and occlusion of lung arteries. We will treat patients with moderately severe primary (no known underlying cause) PAH with 6 months of hydroxyurea, carefully monitoring side effects and adjusting dosage as necessary, and measure the effect on circulating immature cells, lung blood vessel pressures, other blood markers of active PAH, and exercise tolerance. | Pulmonary Hypertension | ALL | ADULT, OLDER_ADULT | |
| Clinical Transplant-Related Long-term Outcomes of Alternative Donor Allogeneic Transplantation (BMT CTN 1702) | The purpose of this study is to determine if a search strategy of searching for an HLA-matched unrelated donor for allogeneic transplantation if possible then an alternative donor if an HLA-matched unrelated donor is not available versus proceeding directly to an alternative donor transplant will result in better survival for allogeneic transplant recipients within 2 years after study enrollment. | Acute Myeloid Leukemia|Acute Lymphoblastic Leukemia|Myelodysplastic Syndromes|Non-hodgkin Lymphoma|Hodgkin Lymphoma|Acquired Aplastic Anemia|Sickle Cell Disease | ALL | CHILD, ADULT, OLDER_ADULT | City of Hope, Duarte, California, 91010, United States|University of California, San Diego Medical Center, La Jolla, California, 92093, United States|Children's Hospital Los Angeles, Los Angeles, California, 90027, United States|Stanford Hospitals and Clinics, Stanford, California, 94305, United States|Children's National Medical Center, Washington, District of Columbia, 20010, United States|University of Florida, Gainesville, Florida, 32610, United States|University of Miami, Miami, Florida, 33136, United States|Memorial Healthcare System, Pembroke Pines, Florida, 33028, United States|H. Lee Moffitt Cancer Center, Tampa, Florida, 33612, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, 30322, United States|Emory University, Atlanta, Georgia, 30322, United States|Northside Hospital, Atlanta, Georgia, 30342, United States|Loyola University, Maywood, Illinois, 60153, United States|Indiana University, Indianapolis, Indiana, 46202, United States|University of Maryland, Baltimore, Maryland, 21201, United States|Dana Farber Cancer Institute, Boston, Massachusetts, 02215, United States|University of Michigan, Ann Arbor, Michigan, 48109, United States|Karmanos Cancer Institute, Detroit, Michigan, 48201, United States|University of Minnesota, Minneapolis, Minnesota, 55414, United States|Mayo Clinic Rochester, Rochester, Minnesota, 55905, United States|University of Mississippi, Jackson, Mississippi, 39216, United States|Children's Mercy Hospital, Kansas City, Missouri, 64108, United States|St. Louis Children's Hospital, Saint Louis, Missouri, 63110, United States|Washington University in St. Louis, Saint Louis, Missouri, 63110, United States|University of Nebraska Medical Center - Adults, Omaha, Nebraska, 68105, United States|University of Nebraska Medical Center - Pediatrics, Omaha, Nebraska, 68105, United States|Rosewell Park Cancer Institute, Buffalo, New York, 14263, United States|Mount Sinai Medical Center, New York, New York, 10029, United States|Memorial Sloan Kettering Cancer Center, New York, New York, 10065, United States|University of North Carolina, Chapel Hill, North Carolina, 27599, United States|Levine Cancer Institute, Charlotte, North Carolina, 28204, United States|Duke University Medical Center, Durham, North Carolina, 27710, United States|Wake Forest Baptist Health, Winston-Salem, North Carolina, 27157, United States|University Hospitals Cleveland Medical Center, Cleveland, Ohio, 44106, United States|Cleveland Clinic, Cleveland, Ohio, 44195, United States|Nationwide Children's Hospital, Columbus, Ohio, 43205, United States|The Ohio State University, Columbus, Ohio, 43210, United States|University of Oklahoma, Oklahoma City, Oklahoma, 73104, United States|Oregon Health and Science University, Portland, Oregon, 97239, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|University of Pennsylvania, Philadelphia, Pennsylvania, 19104, United States|Medical University of South Carolina, Charleston, South Carolina, 29425, United States|Children's Medical Center Dallas, Dallas, Texas, 75235, United States|Cook Children's Medical Center, Fort Worth, Texas, 76104, United States|Baylor College of Medicine, Houston, Texas, 77030, United States|M.D. Anderson Cancer Center, Houston, Texas, 77030, United States|University of Utah, Salt Lake City, Utah, 84113, United States|University of Virginia, Charlottesville, Virginia, 22903, United States|Virginia Commonwealth University, Richmond, Virginia, 23298, United States|Fred Hutchinson Cancer Research Center, Seattle, Washington, 98109, United States|University of Wisconsin, Madison, Wisconsin, 53792, United States|Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, United States |
| Improving Quality of Life in Men and Women With Overweight or Obesity | The purpose of this study is to evaluate the effect of the Noom Healthy Weight Program, a digital behavior change weight loss intervention, on quality of life as measured both by self-report and objective measures, compared to a waitlist control condition. | Obesity|Overweight and Obesity|Quality of Life|Health Behavior | ALL | ADULT, OLDER_ADULT | Noom, New York, New York, 10001, United States |
| Palatability and Tolerability of Deferasirox Taken With Meals, With Different Liquids or Crushed and Added to Food | This single-arm, open-label, multi-center study enrolled 65 patients from approximately 20 centers. All patients who met the study criteria and were taking, beginning or resuming treatment with Deferasirox were allowed. The study will began with a one month run-in phase, where all patients were instructed to take Deferasirox according to their physician's prescribing information. | Transfusional Hemosiderosis | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital and Research Center, Oakland, California, 94609, United States|Stanford University, Palo Alto, California, 94304-1812, United States|Bay Area Cancer Research Group, Pleasant Hill, California, 94523, United States|University of Colorado Denver, Colorado Sickle Cell Treatment and Research Center, Aurora, Colorado, 80045, United States|Yale University School of Medicine, New Haven, Connecticut, 06520, United States|Medical College of Georgia, Augusta, Georgia, 30912, United States|Children's Memorial, Chicago, Illinois, 60614, United States|Tulane University Health Sciences Center, New Orleans, Louisiana, 70118, United States|University of Maryland Greenebaum Cancer Center, Baltimore, Maryland, 21201, United States|Children's Hospital of Boston, Boston, Massachusetts, 02115, United States|Boston Medical Center, Boston, Massachusetts, 02118, United States|Washington University School of Medicine, Saint Louis, Missouri, 63110, United States|The Cancer Center at Hackensack University Medical Center, Hackensack, New Jersey, 07601, United States|Cancer Institute of New Jersey, New Brunswick, New Jersey, 08901, United States|St Joseph Children's Hospital, Paterson, New Jersey, 07503, United States|Schneider Children's Hospital, New Hyde Park, New York, 11040, United States|New York Presbyterian Hospital, New York, New York, 10065, United States|New York Medical College, Valhalla, New York, 10595, United States|Wake Forest University Health Sciences, Winston-Salem, North Carolina, 27157, United States|University of Oklahoma, Oklahoma City, Oklahoma, 73104, United States|Penn State Children's Hospital, Hershey, Pennsylvania, 17033, United States|St Christopher's Hospital for Children, Philadelphia, Pennsylvania, 19134, United States|Texas Children's Cancer Center and Hematology Services, Houston, Texas, 77030, United States |
| Neonatal Hearing Screening at Neonatal Intensive Care Unit | Hearing loss is one of the most common congenital anomalies . It has been shown to be greater than that of most other diseases and syndromes (eg, phenylketonuria, sickle cell disease) screened at birth. Data from the newborn hearing-screening programs in Rhode Island, Colorado, and Texas showed that 2-4 of every 1000 neonates have hearing loss. Early Intervention at or before 6 months of age allows a child with impaired hearing to develop normal speech and language, alongside his or her hearing peers and can prevent severe psychosocial, educational, and language impairment. One of the most high risk population are neonates who spend time in the newborn intensive care unit , exposed to high frequency ventilation, hyperbilirubinemia, low birth-weight, and exposed to ototoxic medications. Auditory brainstem response , otoacoustic emissions , and automated Auditory brainstem response testing have all been used in newborn hearing-screening programs. otoacoustic emissions are fast objective, efficient, and frequency-specific measurements of peripheral auditory sensitivity are used to assess response of the outer hair cells to acoustic stimuli. To measure otoacoustic emissions, a probe assembly is placed in the ear canal, tonal or click stimuli are delivered, and the otoacoustic emissions generated by the cochlea is measured with a microphone . | Hearing Loss | ALL | CHILD | Women Health Hospital - Assiut university, Assiut, 71111, Egypt |
| Teduglutide for Enterocutaneous Fistula (ECF) | The purpose of this study is to determine whether Teduglutide is safe and feasible to be given for the treatment of enterocutaneous fistula (ECF). The hypothesis is that the drug will be well tolerated and will improve the volume of daily ECF output as well as improve the functional quality of life. | Postoperative Fistula | ALL | ADULT, OLDER_ADULT | University of Miami, Miami, Florida, 33136, United States |
| Compassionate Use of Concizumab if You Have Haemophilia | The compassionate use programme will give participants concizumab for free, even though it is not yet approved by health authorities. This is because participants need this medicine to treat their haemophilia properly. The programme will check that participants are safe and that the medicine works for them. The programme may last for years. Participants will take one injection under their skin every day. Participants will have 4-5 visits with the study doctor for the first half year. After that they will have 1 visit every half year. At all clinic visits participants will have blood samples taken. Participants will fill in a diary between the visits. A patient is considered to have completed the programme when any of the following criteria occurred first: 1) when the patient is included in a clinical trial with concizumab or 2) up to 6 months after concizumab is commercially available in the patient's country and approved for the patient (The time span of 6 months should provide ample time for the patient to obtain concizumab commercially) or 3) the sponsor decides to discontinue concizumab clinical development for the patient's population. | Congenital Haemophilia | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital Los Angeles - Endocrinology, Los Angeles, California, 90027, United States|Connecticut Children's Medical Center, Hartford, Connecticut, 06106, United States|Georgetown University Medical Center, Washington, District of Columbia, 20007, United States|Memorial Health University Medical Center, Savannah, Georgia, 31404, United States|Childrens Hospital of Chicago, Chicago, Illinois, 60611, United States|Indiana Hemophilia-Thromb Ctr, Indianapolis, Indiana, 46260, United States|Children's Hospital of Michigan, Detroit, Michigan, 48201, United States|Southern Specialty Clinic, Flowood, Mississippi, 39232, United States|Louisiana Ctr for Adv Med-LCAM, Madison, Mississippi, 39110, United States|The Children's Mercy Hospital, Kansas City, Missouri, 64108, United States|Children's Nebraska, Omaha, Nebraska, 68114, United States|ECU Sickle Cell Comp Clinic, Greenville, North Carolina, 27834, United States|Nationwide Children's Hospital, Columbus, Ohio, 43205, United States|Penn State Hershey Medical Center, Hershey, Pennsylvania, 17033, United States|BI-LO Chrt Childn's Cancer Ctr, Greenville, South Carolina, 29605, United States|Cook Children's Hospital-Hematology-Oncology, Fort Worth, Texas, 76104, United States|Texas Children's Hospital_Houston, Houston, Texas, 77030, United States|Univ TX Hlth Sci Ctr Houston, Houston, Texas, 77030, United States|Virginia Commonwealth University_Richmond_1, Richmond, Virginia, 23298-0461, United States|UMHAT "Sveti Georgi" Clinica of Pediatric, Plovdiv, 4002, Bulgaria|Koagulationsmottagningen, Solna, 171 64, Sweden |
| Acetaminophen for the Reduction of Oxidative Injury in Severe Sepsis | Cell-free hemoglobin can be measured in the plasma of patients with sickle cell anemia, hemodialysis, after red blood cell transfusion, and in patients with sepsis. Cell-free hemoglobin in these patient population has been associated with poor outcomes, including an association with an increased risk of death. Acetaminophen may have a protective effect in these patient populations by inhibiting hemoprotein-mediated lipid peroxidation. The purpose of the present trial is to study the effect of acetaminophen on lipid peroxidation in adults with severe sepsis and detectable cell-free hemoglobin. The primary hypothesis is that systemic markers of oxidative stress and lipid peroxidation, as measured by F2-isoprostanes, will be significantly lower in patients with severe sepsis and detectable cell-free hemoglobin who receive acetaminophen compared to placebo. The secondary hypothesis is that patients with severe sepsis and detectable cell-free hemoglobin treated with acetaminophen will have better clinical outcomes, including decreased incidence of acute kidney injury and lower rates of hospital mortality, compared to those who receive placebo. | Severe Sepsis | ALL | ADULT, OLDER_ADULT | Vanderbilt University Medical Center, Nashville, Tennessee, 37232, United States |
| Radial Shockwave Therapy for Erectile Dysfunction | Objective: To evaluate the efficacy and safety of radial shock waves for the treatment of erectile dysfunction \[ED\]. Patients and methods: Randomized, double-blind clinical trial. The study will include patients of legal age with diagnosis of ED and score on the International Index of Erectile Function \[IIEF-EF\] scale between 11 and 21 points, who voluntarily decide to participate and sign the informed consent. Patients with bladder cancer, prostate cancer or active colon, ED of psychological origin, any psychiatric disorder, spinal cord injury, clinical suspicion of hypogonadism (score on the Aging Males' Symptoms scale greater than 36), infections or active lesions of the penis or pubic area, ED secondary to treatment with medications (antiandrogenic therapy, use of corticosteroids, anti-Parkinson's, antipsychotics), radical prostatectomy or other radical pelvic surgery, history of pelvic radiotherapy, penile implantation, or endocrine diseases that occur with ED (acromegaly, gigantism, Addison's disease, hyperprolactinemia, androgenic deficiency), sickle cell anemia, and anticoagulated patients will be excluded. Patients will be randomly assigned to one of the following treatment arms: * Arm 1 (Standard treatment (oral sildenafil) + Radial wave therapy) * Arm 2 (Standard treatment (oral sildenafil) + Placebo therapy) Measurements will be made of the Erection Hardness Score \[EHS\] and IIEF-EF scale scores, of the use of medication and of the possible adverse events of the therapy, at the beginning and end of the treatment, and one month after the therapies are finished. | Erectile Dysfunction | MALE | ADULT, OLDER_ADULT | Boston Medical Group Colombia, Bogotá, Cundinamarca, 11022, Colombia |
| Role of Ajwa Derived Polyphenols in Dyslipidaemias | World Health Organization report notifies of the escalating global burden of cardiovascular diseases (CVD), projecting that it will become the major worldwide cause of death and disability by 2020. The South Asian countries have the highest rates of CVD globally. It is widely acknowledged that South Asians have 40-60% higher risk of CVD linked to mortality, compared with other populations. Multiple human population studies have established the concentration of high density lipoprotein (HDL) cholesterol as an independent, inverse predictor of the risk of having a cardiovascular event. Furthermore, HDLs have several well-documented functions with the potential to protect against cardiovascular disease. This study trial is designed to find out the role of alternative medicine such as functional food to improve the dyslipidemia and particularly increase the levels of HDL in general population. We expect that the use of Ajwa dates will significantly enhance the level of HDL and reduce cardiovascular events in general population. | Dyslipidemias|Hyperglycemia|Liver Dysfunction|Oxidative Stress|Hemoglobin SC|Anemia | ALL | ADULT, OLDER_ADULT | Aga Khan University, Karachi, Sindh, 74800, Pakistan |
| Lactoferrin With Ferrous Gluconate Versus Ferrous Gluconate in Treatment of Iron Deficiency Anemia During Pregnancy | Study Procedures: All patients will undergo the following: Informed consent will be obtained from all the participants in this study before enrolling in this study and all participants will be subjected to a detailed clinical assessment including: a detailed history, general, abdominal examinations, Investigations. 1. History taking: * Personal history: name, age, occupation and address. * Menstrual and obstetric history: Date of LMP, expected date of delivery which will be calculated according to Naegle's rule and gestational age. In addition to history of presence of any menstrual irregularities, duration. * Past History: of Anemia in previous pregnancy, other diseases like Thalassemia, sickle cell anemia, liver or renal diseases or any other condition that may affect hemoglobin. 2. Medical examination: * General: Assessment of complexion and vital data (blood pressure, pulse, capillary refill) * Abdominal examination to assess fundal height. 3. Investigations to perform will include: Laboratory: Complete blood count (microcytic hypochromic anemia) Imaging: Ultrasound to assess biometry to exclude fetal growth restriction. Women will be divided in two groups with 20 in each group, the first group will receive one tab of ferrous gluconate 300mg administered orally twice per day for 4 weeks and the second group will receive lactoferrin sachets 100mg with ferrous gluconate 300mg twice per day for 4 weeks Patients were assigned to take the medication orally; once daily before breakfast, and Pravotin (100 sachets were be dissolved each in ¼ glass of water and taken before breakfast). Patients were advised to avoid the intake of tea, coffee, milk, milk products, antacids and calcium preparation within 2 hours before or after iron capsules. Women will be told to record side effects as nausea, vomiting, abdominal discomfort and constipation. Women will have a blood sample (CBC) withdrawn after 2 to 4 weeks to assess rise in pregnant anemia. | Lactoferrin With Ferrous Gluconate More Superior Than Ferrous Gluconate Alone in Treatment of Iron Deficiency Anemia | FEMALE | ADULT | Ain shams university maternity hospital, Cairo, 6825344, Egypt |
| Lifestyle Changes Through Exercise and Nutrition | The goal of The LEAN project intervention is to promote weight loss, healthy dietary habits, increased physical activity, improved blood glucose and cholesterol, and weight related quality of life in obese African American adults through a sustainable, technology enhanced, church-based program. | Obesity|Diabetes | ALL | ADULT, OLDER_ADULT | Pennington Biomedical Research Center, Baton Rouge, Louisiana, 70808, United States |
| A Prospective Trial of Virtual Home Rehabilitation After Burn Injury | The overarching goal for this prospective randomized controlled trial (PRCT) is to determine whether a virtual-environment, home-rehabilitation program improves functional outcomes for individuals after a burn injury. Specifically, this study will test the efficacy of a technology-assisted rehabilitation program against current standard of home therapy. | Burns|Burn Scar|Contracture | ALL | ADULT, OLDER_ADULT | Harborview Medical Center, Seattle, Washington, 98104, United States |
| Cohort of Patients Presenting Unexplained Recurrent Miscarriages and Identification of Early Miscarriage Recidivism Factors | About 1 to 3% of women of childbearing age have repeated early spontaneous miscarriages (RCF) defined by at least 3 fetal losses before 14 weeks of gestation. RCFs may be related to parental chromosomal abnormalities, congenital or acquired uterine abnormalities, hormonal causes (e.g. type 1 and 2 diabetes, ovarian failure), infectious etiology, constitutional or acquired thrombophilia or sickle cell disease. The presence of antiphospholipid antibodies, antithyroid and anti-transglutaminase antibodies in approximately 10% of cases suggests an autoimmune origin for these fetal losses. The role of other antibodies, in particular unconventional antiphospholipid antibodies, remains to be established. Indeed half of RCF cases would be due to an immunological dysregulation of the mother leading to a decrease in tolerance to the fetus. Several studies have shown immune abnormalities, such as an imbalance of pro and anti-inflammatory cytokines, an increase in cytotoxic cells and a defect in regulatory cells in the blood of patients. The assessment of these immune abnormalities is not currently carried out routinely in France in women with recurrent early miscarriages. When one of these known causes is excluded, it is unexplained RCF which represents 50% of RCF. In these women with unexplained RCF, slightly more than half could be linked to aneuploidies and primary recurrent spontaneous abortions. The evaluation of the degree of aneuploidy and the genetic origin of fetal losses remains difficult, the examination of the sample of tissue from the miscarriage being rarely available, due to the spontaneous nature of the loss. The constitution of a prospective cohort of patients with RCF is an essential step in exploring the factors associated with the success of treatment. | Miscarriage | FEMALE | ADULT | Internal medicine department, hospital Saint Antoine, Paris, 75012, France |
| A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload | This study uses a single arm, multi-center, open-label trial design. The study will assess the efficacy and safety of 52 weeks of treatment with deferasirox (ICL670) in patients with evidence of transfusion induced iron overload. | Transfusion-dependent Iron Overload | ALL | CHILD, ADULT, OLDER_ADULT | Novartis Investigative Site, Adelaide, Australia|Novartis Investigative Site, Camperdown, Australia|Novartis Investigative Site, Clayton, Australia|Novartis Investigative Site, Melbourne, Australia|Novartis Investigative Site, Perth, Australia|Novartis Investigative Site, South Brisbane, Australia|Novartis Investigative Site, Westmead, Australia|Novartis Investigative Site, Graz, Austria|Novartis Investigative Site, Linz, Austria|Novartis Investigative Site, Wien, Austria|Novartis Investigative Site, Brussels, Belgium|Novartis Investigative Site, Gent, Belgium|Novartis Investigative Site, Godinne, Belgium|Novartis Investigative Site, La Louviere, Belgium|Novartis Investigative Site, Leuven, Belgium|Novartis Investigative Site, GuangZhou, China|Novartis Investigative Site, Nanjing, China|Novartis Investigative Site, Shanghai, China|Novartis Investigative Site, Arhus, Denmark|Novartis Investigative Site, Copenhagen, Denmark|Novartis Investigative Site, Herlev, Denmark|Novartis Investigative Site, Hillerod, Denmark|Novartis Investigative Site, Cairo, Egypt|Novartis Investigative Site, Angers, France|Novartis Investigative Site, Avignon, France|Novartis Investigative Site, Bobigny, France|Novartis Investigative Site, Creteil, France|Novartis Investigative Site, Lille Cedex, France|Novartis Investigative Site, Lyon, France|Novartis Investigative Site, Nice, France|Novartis Investigative Site, Paris Cedex 14, France|Novartis Investigative Site, Paris, France|Novartis Investigative Site, Pessac Cedex, France|Novartis Investigative Site, Rennes, France|Novartis Investigative Site, Toulouse Cedex, France|Novartis Investigative Site, Vandoeuvre Les Nancy, France|Novartis Investigative Site, Augsburg, Germany|Novartis Investigative Site, Braunschweig, Germany|Novartis Investigative Site, Dresden, Germany|Novartis Investigative Site, Dusseldorf, Germany|Novartis Investigative Site, Frankfurt/Main, Germany|Novartis Investigative Site, Frankfurt, Germany|Novartis Investigative Site, Frieburg, Germany|Novartis Investigative Site, Gottingen, Germany|Novartis Investigative Site, Greifswald, Germany|Novartis Investigative Site, Hannover, Germany|Novartis Investigative Site, Mainz, Germany|Novartis Investigative Site, Muenchen, Germany|Novartis Investigative Site, Ulm, Germany|Novartis Investigative Site, Athens, Greece|Novartis Investigative Site, Larissa, Greece|Novartis Investigative Site, Patras, Greece|Novartis Investigative Site, Thessaloniki, Greece|Novartis Investigative Site, Hong Kong, Hong Kong|Novartis Investigative Site, Afula, Israel|Novartis Investigative Site, Jerusalem, Israel|Novartis Investigative Site, Petach-Tikva, Israel|Novartis Investigative Site, Bologna, Italy|Novartis Investigative Site, Brindisi, Italy|Novartis Investigative Site, Cagliari, Italy|Novartis Investigative Site, Cona, Italy|Novartis Investigative Site, Genova, Italy|Novartis Investigative Site, Milano, Italy|Novartis Investigative Site, Napoli, Italy|Novartis Investigative Site, Orbassano, Italy|Novartis Investigative Site, Palermo, Italy|Novartis Investigative Site, Pavia, Italy|Novartis Investigative Site, Pisa, Italy|Novartis Investigative Site, Reggio Calabria, Italy|Novartis Investigative Site, Roma, Italy|Novartis Investigative Site, Sassari, Italy|Novartis Investigative Site, Seoul, Korea, Republic of|Novartis Investigative Site, Hazmiyeh, Lebanon|Novartis Investigative Site, Kota Bahru, Malaysia|Novartis Investigative Site, Kuala Lumpur, Malaysia|Novartis Investigative Site, Nijmegen, Netherlands|Novartis Investigative Site, Johannesburg, South Africa|Novartis Investigative Site, Parktown, South Africa|Novartis Investigative Site, Baracaldo, Spain|Novartis Investigative Site, Barcelona, Spain|Novartis Investigative Site, Madrid, Spain|Novartis Investigative Site, Sevilla, Spain|Novartis Investigative Site, Valencia, Spain|Novartis Investigative Site, Geneve, Switzerland|Novartis Investigative Site, Zurich, Switzerland|Novartis Investigative Site, Taichung, Taiwan|Novartis Investigative Site, Taipei, Taiwan|Novartis Investigative Site, Bangkok, Thailand|Novartis Investigative Site, Chaingmai, Thailand|Novartis Investigative Site, Adana, Turkey|Novartis Investigative Site, Ankara, Turkey|Novartis Investigative Site, Istanbul, Turkey|Novartis Investigative Site, Izmir, Turkey|Novartis Investigative Site, Leeds, United Kingdom|Novartis Investigative Site, Leicester, United Kingdom|Novartis Investigative Site, London, United Kingdom|Novartis Investigative Site, Manchester, United Kingdom|Novartis Investigative Site, Sheffield, United Kingdom |
| PET Study in Patients With Non-Hodgkin Lymphoma | RATIONALE: Diagnostic procedures, such as fluorine 18-fludeoxyglucose positron emission tomography (PET) scans, may help doctors predict a patient's response to treatment and help plan the best treatment. PURPOSE: This phase I trial is studying fluorine 18-fludeoxyglucose PET scan to see how well it predicts outcomes in patients who have undergone high-dose chemotherapy and autologous stem cell transplant for non-Hodgkin lymphoma. | Lymphoma | ALL | CHILD, ADULT, OLDER_ADULT | Vanderbilt-Ingram Cancer Center - Cool Springs, Nashville, Tennessee, 37064, United States|Vanderbilt-Ingram Cancer Center at Franklin, Nashville, Tennessee, 37064, United States|Vanderbilt-Ingram Cancer Center, Nashville, Tennessee, 37232-6838, United States |
| SELF-BREATHE RCT for Chronic Breathlessness | A feasibility RCT comprising two groups: 1. Intervention (SELF-BREATHE in addition to standard NHS care) 2. Control group (standard / currently available NHS care) | Cancer|COPD|Asthma|Bronchiectasis Adult|Interstitial Lung Disease|Cystic Fibrosis|Chronic Heart Failure|Sickle Cell Disease|Renal Failure|Liver Failure|Post COVID-19|Dyspnea | ALL | ADULT, OLDER_ADULT | King's College Hospital NHS Foundation TRUST, London, SE5 9RS, United Kingdom |
| Effectiveness, Safety, and Tolerability Study of Oxymorphone Immediate Release (IR) Oral Liquid in Opioid Tolerant Pediatric Subjects | The purpose of this study is to evaluate the effectiveness, tolerability, and safety of oxymorphone immediate release (IR) oral liquid as an analgesic in pediatric subjects having severe to moderate chronic pain. | Chronic Pain | ALL | CHILD | |
| A Pragmatic Randomized Controlled Trial to Predict Postpartum Hemorrhage | This research project aims to enhance the safety of childbirth by using advanced computer models to predict the risk of postpartum hemorrhage (PPH). PPH is a significant concern for mothers during and after delivery. Current risk assessment tools are basic and do not adapt to changing conditions. This study will investigate whether a new and recently validated model for predicting PPH, combined with a provider-facing Best Practice Advisory (BPA) regarding currently recommended strategies triggered by an increased predicted risk, can improve perinatal outcomes. This study will compare the current category based risk assessment tool with a new, enhanced prediction model which calculates risk based on 21 factors, automatically updates as new information becomes available during labor and, if elevated, provides a provider-facing Best Practice Advisory (BPA) recommending consideration of strategies that are institutionally agreed to represent high-quality practice. Investigators hypothesize that the enhanced care approach will result in improved perinatal outcomes. The goal of the study is to improve the wellbeing of mothers during childbirth by harnessing the power of modern technology and data analysis. | Post Partum Hemorrhage | FEMALE | CHILD, ADULT, OLDER_ADULT | Vanderbilt University Medical Center, Nashville, Tennessee, 37212, United States |
| Epidemiology of Burkitt Lymphoma in East Africa Children or Minors (EMBLEM) | Burkitt lymphoma (BL) is an aggressive monoclonal B-cell malignancy that is rare (sporadic) worldwide, but is 100-fold more common (endemic) in equatorial Africa, particularly among children. Epstein-Barr virus (EBV) and malaria are epidemiologically linked to endemic BL in epidemiologic studies, but questions remain about role of EBV variants and the evidence for association with malaria is weak. EBV is ubiquitous, yet only few children develop BL, possibly because only a few EBV variants are pathogenically relevant. The association of BL with malaria is based on ecologic and non-comparative clinical studies. Two case-control studies have reported significant association of high anti-malarial antibodies with BL (OR=5_ among children in Uganda and in Malawi, but selection bias (cases and controls came from dissimilar geographical areas) and reverse causality bias were limitations. Three studies were conducted in the 1960s and 70s to test association of carriage of malaria-resistance gene with BL, two of which reported a significant or marginal inverse association. These pioneering studies were small (240 cases all together) and looked at one polymorphism in one gene (sickle cell gene). Improvements in technologies to characterize genetic variation allow the EBV and malaria hypotheses to be examined with greater power by looking at genetic variation across multiple genes. Epidemiology of Burkitt lymphoma in East African children and minors (EMBLEM) is a case-control study of 1500 BL cases and 3000 age-, sex- and residence-frequency matched controls we are proposing to conduct in East Africa. The study will enroll cases at four hospitals in four regions in East Africa, where malaria transmission is holoendemic and year round. The controls will be enrolled from general population attendees at Health Center II (HC-II) units where the cases originated. The primary study objectives are: 1) to test the hypothesis that genetic resistance to malaria is associated with a lower risk of BL, and 2) to use genome-wide association methods to discover genetic variation that may be associated with decreased or increased risk of BL. Because genetic variation conveys no information on actual exposure to malaria or EBV, in secondary analyses, we will use empiric epidemiological questionnaire and laboratory methods: a) to measure exposure to malaria and its association with BL, and b) to measure EBV variants and their association with BL. To examine issues related to bias and to obtain data to correct for deviations, we will also enroll 2250 population controls from 5% of the villages to obtain population distribution of key exposures variables. This data will be used to reweight the distribution in HC-II controls back to the general population. ... | Lymphoma, Non-Hodgkin|Malaria|Herpesvirus 4, Human | ALL | CHILD | Bugando Medical Center, Mwanza, Tanzania|Shirati Health, Educational, and Development Foundation, Shirati, Tanzania|St.Mary's Hospital Lacor, Gulu, Uganda|Kuluva Hospital (Arua), Kampala, Uganda|Homabay District Hospital, Nyanza, Uganda|Webuye District Hospital, Webuye, Uganda |
| Screening for Alpha Thalassemia in Healthy Volunteers | Background: Alpha thalassemia is a blood disorder. It is caused by genetic deletions. Part of the DNA is missing from a group of genes called alpha globin. Alpha thalassemias are some of the most common genetic deletions. We are testing for alpha thalassemia trait. Alpha thalassemia trait is when someone has only two out of the normal four alpha globin genes. In some people, they lead to no symptoms. Others have changes that lead to disease, including mild anemia. Researchers want to learn more about alpha thalassemia and blood vessels. This may allow them to develop new treatments for blood diseases such as sickle cell disease. Objective: To better understand how alpha globin deletions in healthy people affect blood vessels. Eligibility: Healthy volunteers ages 18-39 who self-report African ancestry. Design: Participants will provide a one-time saliva sample. This can be by mail, in-person at a study event, or at NIH. Participants will get a small kit to collect their saliva sample. The kit has easy instructions. The sample does not need to be put in the refrigerator. Participants will spit a small amount of saliva (less than half a teaspoon) into a collection tube. Participants will close the funnel lid tightly, and then unscrew the funnel lid from the tube. They will then close the tube tightly with the small cap provided and shake the tube for 5 seconds. Participants will place the tube in the provided envelope and mail it to NIH. The specimen will be stored and processed in the lab. Participants may be invited to participate in more research studies, whether or not researchers find that they have alpha thalassemia trait. | Alpha Thalassemia | ALL | ADULT | National Institutes of Health Clinical Center, Bethesda, Maryland, 20892, United States |
| Graft Maturity After Blood Flow Restriction Training in ACL Reconstruction | Rationale Graft maturity is correlated with strength and biomechanical properties of the reconstructed ACL. There are concerns that heavy-load resistance training (HLRT) may have detrimental effects on ACL graft maturation. Therefore, low-load blood flow restriction training (LL-BFRT) has been suggested as an alternative to HLRT. As LL-BFRT is an increasingly popular method for the rehabilitation after an ACL reconstruction, it is important to evaluate the value of this treatment. Objectives The main objective is to evaluate the effect of LL-BFRT on MRI-based graft maturity after ACL reconstruction compared to HLRT. The secondary objectives are the effect of LL-BFRT on donor-site morbidity, range of motion, knee stability, patient reported outcome measurements, muscle strength, safe return to pre-injury level of sport and patient satisfaction. Furthermore, feasibility and safety of rehabilitation will be assessed. Study design Randomized controlled trial . Study population Patients who will undergo primary bone-patellar tendon-bone ACL reconstruction and rehabilitation at Knie-Heup centrum Plus will be assessed for eligibility. The exclusion criteria are: venous thromboembolism, sickle cell anemia, severe hypertension, contra-indication for accelerated rehabilitation, contra-indication for MRI scan or patients who are not willing/able to participate. Intervention LL-BFRT includes 12 weeks of biweekly strength training and starts two weeks after surgery. Comparison HLRT includes 12 weeks of biweekly strength training and starts two weeks after surgery. Main study endpoints The main endpoints are MRI-based graft maturity defined as signal-to-noise quotient three and nine months after surgery. | Anterior Cruciate Ligament Reconstruction|Bone-Patellar Tendon-Bone Grafting | ALL | ADULT, OLDER_ADULT | Zuyderland Medical Center, Sittard, Limburg, 6162 BG, Netherlands |
| Pharmacokinetic (PK) and Pharmacodynamics (PD) Study of Ilera Specific Products | This is an observational study of medical marijuana manufactured and dispensed by Ilera and given as standard treatment for a variety of approved serious medical conditions as defined by individual state law. All patients who are receiving one of the four formulations (Dream, Soothe, Shine and Ease) of medical marijuana will be provided a study flyer and asked to contact the study team via phone or email. Once the study team confirms eligibility, the study team will meet the subject face-to-face most likely at their dispensary (or other mutually agreeable location) and obtain informed consent, and assent when appropriate. Initial baseline demographic information, medical history and medication inventory will be completed. Also, since it is possible that the Investigators will enroll subjects across the region, Investigators anticipate the need to seek consent over the phone for many patients. This will be done via Skype, Go to Meeting, Facetime or similar platforms so that the Investigators can have a face to face interaction with the potential subjects. Regardless of where this discussion takes place (i.e., in person or via the web), all reasonable safeguards to ensure patient privacy will be taken. Patients or their legally authorized representative (LAR) will be given sufficient (i.e., up to several hours/days) to make a decision to participate in this study. Study staff will fax or email the consent form for their signature and no study procedures will begin until the signed consent form is received by the study team. The subjects or their LARs will be instructed on obtaining the blood samples. Blood draws will be completed in the subjects' home after one of their standard doses is taken. | ALS|Autism Spectrum Disorder|Cancer|Spasticity, Muscle|Dyskinetic Syndrome|Epilepsy|Glaucoma|Huntington Disease|Inflammatory Bowel Disease (IBD)|Multiple Sclerosis|Neuropathy|Opioid Use|Parkinson Disease|HIV/AIDS|Ptsd|Intractable Pain|Sickle Cell Disease|Terminal Illness | ALL | CHILD, ADULT, OLDER_ADULT | Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States |
| Introducing Fetal Scalp Stimulation as an Adjunct to Intermittent Auscultation in Low-Resource Settings. | This study is a pilot study taking place in Moshi, Tanzania at the Kilimanjaro Christian Medical Centre (KCMC). The study aims to introduce fetal scalp stimulation into the intermittent auscultation protocols at KCMC, and to validate whether or not a handheld Doppler device can perform the fetal scalp stimulation test accurately. | Fetal Distress|Stillbirth|Birth Asphyxia|Acidosis | FEMALE | CHILD, ADULT | Kilimanjaro Christian Medical Center, Moshi, Kilimanjaro, Tanzania |
| Reducing Racial Disparities in Severe Maternal Morbidity | There is a paucity of research examining the intersection of race, ethnicity, maternal safety bundles, doulas, and maternal outcomes in Black women at increased risk of severe maternal morbidity and mortality. The proposed mixed-methods study is the first systematic investigation of pregnancy complications and outcomes among Black women with whom maternal safety bundles are being implemented including racial disparities, hemorrhage, and hypertension. Additionally, through the analysis of secondary state level data, this study will examine perinatal care, maternal outcomes, and healthcare utilization of Black women at increased risk of severe maternal morbidity and mortality compared with non-Latino white women. Finally, through individual interviews with Black women and focus groups with obstetric health providers and doulas, the study will examine disparities and improve care by creating and disseminating a set of practice recommendations for maternity care for Black women at increased risk of morbidity and mortality. Research has not yet examined the intersection of race/ethnicity, doulas, and quality improvement (QI) interventions, such as maternal safety bundles, on reducing SMM and mortality among non-Hispanic Black (NHB) women. The overall goal of this mixed-methods study is to use analysis of existing big data and the evaluation of two interventions to ultimately develop targeted recommendations for addressing these inequities. Our approach leverages multiple data sources to study maternal outcomes and access to care during the prenatal, birth, and postpartum periods in order to identify commonalities among women who experienced SMM and use those findings to create a risk profile of women who are more likely to experience SMM; examine the implementation of maternal safety bundles on SMM and MM outcomes for women up to 1 year postpartum (Intervention 1); gather in-depth data from obstetric care providers on factors that support or hinder safety bundle implementation (Intervention 1); and gather in-depth data from individual women and doulas on facilitators of barriers to the use of doulas to improve care and address inequities (Intervention 2). | Maternal Death | FEMALE | CHILD, ADULT, OLDER_ADULT | Tufts University, Boston, Massachusetts, 02128, United States |
| Using Advanced Data Systems to Improve Health in Early Life in Rural Nepal | The goal of this cluster randomized controlled trial is to study the effect of a mobile-phone based application used by pregnant women on maternal and newborn health indicators. The main objective is to compare the rates of institutional deliveries in the intervention and control arms. Ancillary objectives are to compare the birth-preparedness and complication readiness parameters, severe maternal morbidity rates and neonatal adverse outcomes rates in the two arms. The participants are pregnant women. In the intervention arm pregnant women will be given a smart mobile phone with an application that they will use to input information related to their health. This information can be shared with their healthcare workers. The healthcare workers will also be able to access all the health-related details of the pregnant women and mothers under their care by accessing this app in their mobile phones and be in touch with their patients through the mobile phone application. The control arm will adhere to existing practices of pregnant woman and health worker communication without the use of a smart mobile phone with an existing application. Records related to the pregnant woman will be kept in paper-based forms as is the usual norm. The investigators will compare the intervention arm and the control arm to see if there are differences in the rates of the outcomes. | Maternal Health|Infant Health|Institutional Delivery|Birth Preparedness and Complication Readiness|Severe Maternal Morbidity|Neonatal Adverse Outcome | FEMALE | CHILD, ADULT, OLDER_ADULT | Patan Academy of Health Sciences, Lalitpur, Bagmati, Nepal |
| A Trial of Hydroxyurea in Spinal Muscular Atrophy | Spinal muscular atrophy (SMA) is an autosomal recessive disorder in humans which results in the loss of motor neurons. It is caused by reduced levels of the survival motor neuron (SMN) protein as a result of loss or mutation of the SMN1 gene. SMN protein is encoded by two genes, SMN1 and SMN2, which essentially differ by an single nucleotide in exon 7. As a result, the majority of the transcript from SMN2 lacks exon 7. According to clinical severity, SMA was classified to three types, including type I, type II, and type III. Drugs capable of modifying the transcription pattern of SMN2 to increase the full-length of SMN mRNA expression and the amount of SMN protein may have therapeutic effects for SMA patients. In order to test this hypothesis, we used EBV-transformed lymphoblastoid cell lines derived from the different types of SMA patients to screen the effect of various drugs on SMN2 gene expression. Hydroxyurea (HU) was found to be effective among the drugs we tested. HU is an effective therapeutic agent for patients with thalassemia and sickle cell disease which the toxicity is minimal and is well-tolerated and safely used in children. We had undergone a small-scaled 33 SMA patients randomized pilot trial (HU treatment for 8 weeks and then follow up drug-free 8 weeks) to evaluate the effect of HU in SMA patients and we got a promising preliminary data. We found that HU could significantly increase in the manual muscle testing scores at 4 weeks, and full-length SMN mRNA level in the 30mg/kg/day subgroup at 8 weeks relative to baseline, and it is safe under the dose 30mg/kg/day. In this study, we plan to enroll 60 type II and III SMA patients and conduct a single-center, randomized, double-blind, placebo-controlled, prospective trial of two-year duration to evaluate the efficacy and safety of HU.The primary end points are the changes in full-length SMN expression, SMN protein, motor function and lung function in SMA patients. We also design a safety monitoring system to investigate the adverse effects and to assure the patients' safety. We hope we can find and prove the efficacy and safety of HU in SMA patients and set up a evaluating model for multi-center trials in the future. | Spinal Muscular Atrophy | ALL | CHILD, ADULT, OLDER_ADULT | Kaohsiung Medical University Hospital, Kaohsiung, 807, Taiwan |
| Iron and Infection: Neonatal Nutritional Immunity | The motivation for this study was produced from our preliminary data, which showed that during the first 96 hours of life a full-term neonate will actively reduce the overall serum iron concentration of their blood and the transferrin saturation decreases rapidly from 45% in cord blood to \~20% by six hours post-delivery. The Investigators hypothesise that this active sequestration of iron, which results in hypoferremia, is done in an effort to limit susceptibility to infection, a process referred to as nutritional immunity. Currently, little is known about iron regulation and iron homeostasis during the first week of life and even less is known about the comparisons of nutritional immunity between full term, preterm and low birth weight neonates. Additionally, limited research has been conducted on the impact of these processes on bacterial pathogens. In an effort to study the neonatal nutritional immunity and its role in neonatal susceptibility to infection, The investigator will conduct an observational study in full-term, preterm and low birth weight vaginally-delivered neonates born at Serrekunda General Hospital, The Gambia. The investigators will fully characterise and quantify nutritional immunity during the early neonatal period and the investogators will assess how this impacts bacterial growth. Study sensitisation will occur at the antenatal clinic, during the mother's second trimester of pregnancy. Mothers will be consented and enrolled at delivery. Blood samples will be collected once from the umbilical cord and at serial time points from the neonates over the first week of life. | Neonatal Infection|Nutritional Anemia | ALL | CHILD | Serrekunda General Hospital, Kanifing, Near Banjul, Gambia |
| ST Elevation in Acute Chest Pain; Could Measurement of Lipoprotein-associated Phospholipase A2 (Lp-PLA2) be Helpful to the Clinician? | A blood test (2-3 cc peripheral venous blood) drawn /used from already available required lab tests to distinguish between pericarditis accompanied with electrocardiogram (ECG) signs mimicking infarction. A test of clinical potential if proven to be able to support either origin of acute chest pain etiology. | Acute Chest Syndrome | ALL | ADULT, OLDER_ADULT | The E.Wolfson MC, Holon, 52100, Israel |
| Computerized PAINRelieveIt Protocol for Cancer Pain Control in Hospice | The study purpose is to compare usual hospice care and PAINRelieveIt groups for effects on: (1) patient outcomes (analgesic adherence; worst pain intensity, satisfaction, and misconceptions) and lay caregiver outcome (pain misconceptions) in a diverse sample of 250 cancer patient-caregiver dyads receiving hospice care; and (2) nurse outcomes (obtained appropriate analgesics for patient) in a sample of hospice nurses. The investigators hypothesize that at posttest, controlling for pretest data and compared to the usual care group, the PAINRelieveIt group will: a) report decreased scores for worst pain intensity and pain misconceptions; b) have increased analgesic adherence (primary outcome); and c) have a larger proportion who report satisfaction with pain intensity and whose nurses obtained appropriate analgesics for the patients' pain. | Cancer|Pain | ALL | ADULT, OLDER_ADULT | |
| Is Fetal Hemoglobin a Key for Improvement of Hypoxia and Saving Last Breath in COVID-19 Patient?. A Pilot Study. | In December 2019, a sudden public health incident (the corona virus disease \[COVID-19\] epidemic) occurred in Wuhan, China. Clinical features of those with pneumonia include fever and cough, and in many cases a sudden and accelerating respiratory distress originated from interstitial pneumonia . Many hypotheses have explained hypoxemia in COVID-19 patients, such as hyperimmune reaction to viral infection and cytokine storm that leads to serious lung tissue and alveolar damage or even direct viral insult . Mortality are as high as 15% in critically ill patients requiring intensive care unit admission and oxygen therapy , suggesting an urgent need to try therapeutic interventions in addition to supportive treatment. There is more than one type of hemoglobin. In adults, Hb A or Adult hemoglobin which is the main hemoglobin in the blood. But there is another type of hemoglobin called fetal hemoglobin. Fetal hemoglobin (hemoglobin F, Hb F, or α2γ2) is the main oxygen carrier protein in the human fetus. and the levels remain high after birth until the baby is roughly 2-4 months old . Hemoglobin F has a different composition from hemoglobin A and higher affinity to oxygen . At birth, hemoglobin F accounts for 50-95% of the infant's hemoglobin and at around 6 months after birth, hemoglobin A becomes the predominant type.The key feature that allows hemoglobin F to bind more strongly to oxygen is by having γ subunits (instead of β, in Hb A for example). 2,3-BPG interacts much more with hemoglobin A than hemoglobin F . A hypothesis for the low incidence of the COVID-19 infection in pediatric is the presence of fetal hemoglobin (HbF) . In a preliminary study about the prevalence of hemoglobinopathies in different countries and the mortality rate of COVID-19, it appears that the mortality is lower in countries with a higher prevalence of hemoglobinopathies . Mice treated with GBT1118 (a compound that enhances the oxygen affinity of hemoglobin) showed a sustained significant increase in SpO2 over 4 h of hypoxia exposure. People with haemoglobinopathies like sickle cell anemia or beta-thalassemia attributed with high amount of fetal hemoglobin, become mostly asymptomatic or have mild symptoms . The volume of umbilical cord blood varies from 50 ml to 140 ml with a mean of 85 ml rich in fetal hemoglobin . Mesenchymal stem cells (MSCs) have been widely used in the clinical setting, not only for autoimmune diseases but also for infectious diseases , and their safety and effectiveness have been well elucidated . As a noninvasive treatment, hUC-MSC therapy is a very effective and promising method for clinical application and promotion to treat severe COVID-19 the investigators offer a solution by increasing fetal hemoglobin by cord blood containing fetal blood transfusion in the critical patients as a trial to combat the course of the disease and minimize the morbidity especially in sever cases who suffer from desaturation until suppression of the immune dysregulation and avoidance of the impending death. | COVID-19 Acute Respiratory Distress Syndrome | ALL | ADULT, OLDER_ADULT | |
| Fibroblast Growth Factor 23 in Chronic Respiratory Failure | Fibroblast growth factor 23 (FGF23) is a key hormone of the mineral metabolism produced in bone and acting on the kidney to lower phosphatemia. FGF23 is subject to inactivating proteolytic cleavage which results in the presence of C-terminal and N-terminal fragments heretofore described as inactive. We recently showed an increase in FGF23Ct in sickle cell patients, its association with left ventricular mass as well as a direct, pro-hypertrophic effect of FGF23Ct on rat cardiomyocytes. Data from the literature suggest that hypoxia (linked or not to anemia) is responsible for an increase in the production and cleavage of FGF23, either via the hypoxia inducible factor (HIF1α) or via the increase in erythropoietin (EPO). We hypothesize that the FGF23Ct / FGF23i ratio is increased in response to chronic tissue hypoxia, in the absence of anemia, in patients with chronic respiratory failure (CRF) either due to a direct response to hypoxia via the stimulation of HIF1α, or indirectly via the increase in the circulating concentration of EPO. This elevation, if proven, could contribute to the increased risk of heart disease seen in some populations of CRF. We propose to test this hypothesis by assaying FGF23Ct and FGF23i in a cohort of adult CRF patients before and after initiation of oxygen therapy. The object of the present study is to study the FGF23Ct / FGF23i ratio in incident patients presenting with a non treated CRF as well as the modifications of this ratio under oxygen therapy and to study the correlations between FGF23 Ct and FGF23 and i) oxygen saturation and PaO2 ii) echocardiographic parameters and iii) EPO concentrations. Three visits are planned: Baseline (before initiation of oxygen therapy), and two visits after initiation of oxygen therapy, at 3 months (M3) and at 12 months (M12). For each visit, anthropometric and clinical data, treatment and biological results will be collected. FGF23 intact , FGF23 C-terminal and Erythropoietin will be measured. A cardiac ultrasound will be performed at baseline and at M12. | Chronic Respiratory Failure | ALL | ADULT, OLDER_ADULT | |
| Early Check: Expanded Screening in Newborns | Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and families who screen positive, and 3) evaluate the Early Check program. The Early Check screening will lead to earlier identification of newborns with rare health conditions in addition to providing important data on the implementation of this model program. Early diagnosis may result in health and development benefits for the newborns. Infants who have newborn screening in North Carolina will be eligible to participate, equating to over 120,000 eligible infants a year. Over 95% of participants are expected to screen negative. Newborns who screen positive and their parents are invited to additional research activities and services. Parents can enroll eligible newborns on the Early Check electronic Research Portal. Screening tests are conducted on residual blood from existing newborn screening dried blood spots. Confirmatory testing is provided free-of-charge for infants who screen positive, and carrier testing is provided to mothers of infants with fragile X. Affected newborns have a physical and developmental evaluation. Their parents have genetic counseling and are invited to participate in surveys and interviews. Ongoing evaluation of the program includes additional parent interviews. | Spinal Muscular Atrophy|Fragile X Syndrome|Fragile X - Premutation|Duchenne Muscular Dystrophy|Hyperinsulinemic Hypoglycemia, Familial 1|Diabetes Mellitus|Adrenoleukodystrophy, Neonatal|Medium-chain Acyl-CoA Dehydrogenase Deficiency|Very Long Chain Acyl Coa Dehydrogenase Deficiency|Beta-ketothiolase Deficiency|Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency|Primary Hyperoxaluria Type 1|Congenital Bile Acid Synthesis Defect Type 2|Pyridoxine-Dependent Epilepsy|Hereditary Fructose Intolerance|Hypophosphatasia|Hyperargininemia|Mucopolysaccharidosis Type 6|Argininosuccinic Aciduria|Citrullinemia, Type I|Wilson Disease|Maple Syrup Urine Disease, Type 1A|Maple Syrup Urine Disease, Type 1B|Biotinidase Deficiency|Neonatal Severe Primary Hyperparathyroidism|Intrinsic Factor Deficiency|Usher Syndrome Type 1D/F Digenic (Diagnosis)|Cystic Fibrosis|Stickler Syndrome Type 2|Stickler Syndrome Type 1|Alport Syndrome, Autosomal Recessive|Alport Syndrome, X-Linked|Carbamoyl Phosphate Synthetase I Deficiency Disease|Carnitine Palmitoyl Transferase 1A Deficiency|Carnitine Palmitoyltransferase II Deficiency|Cystinosis|Chronic Granulomatous Disease|Cerebrotendinous Xanthomatoses|Maple Syrup Urine Disease, Type 2|Severe Combined Immunodeficiency Due to DCLRE1C Deficiency|Thyroid Dyshormonogenesis 6|Thyroid Dyshormonogenesis 5|Supravalvar Aortic Stenosis|Factor X Deficiency|Hemophilia A|Hemophilia B|Tyrosinemia, Type I|Fructose 1,6 Bisphosphatase Deficiency|Glycogen Storage Disease Type I|G6PD Deficiency|Glycogen Storage Disease II|Galactokinase Deficiency|Mucopolysaccharidosis Type IV A|Galactosemias|Guanidinoacetate Methyltransferase Deficiency|Agat Deficiency|Glutaryl-CoA Dehydrogenase Deficiency|Gtp Cyclohydrolase I Deficiency|Hyperinsulinism-Hyperammonemia Syndrome|Primary Hyperoxaluria Type 2|3-Hydroxyacyl-CoA Dehydrogenase Deficiency|Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency|Mitochondrial Trifunctional Protein Deficiency|Sickle Cell Disease|Beta-Thalassemia|Holocarboxylase Synthetase Deficiency|3-Hydroxy-3-Methylglutaric Aciduria|Primary Hyperoxaluria Type 3|Hermansky-Pudlak Syndrome 1|Hermansky-Pudlak Syndrome 4|Apparent Mineralocorticoid Excess|HSDB|CBAS1|Mucopolysaccharidosis Type 2|Mucopolysaccharidosis Type 1|Severe Combined Immunodeficiency, X Linked|Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency|Diabetes Mellitus, Permanent Neonatal|Isovaleric Acidemia|Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder)|Jervell and Lange-Nielsen Syndrome 2|Hyperinsulinemic Hypoglycemia, Familial, 2|Diabetes Mellitus, Permanent Neonatal, With Neurologic Features|Jervell and Lange-Nielsen Syndrome 1|Lysosomal Acid Lipase Deficiency|CblF|3-Methylcrotonyl CoA Carboxylase 1 Deficiency|3-Methylcrotonyl CoA Carboxylase 2 Deficiency|Waardenburg Syndrome Type 2A|Methylmalonic Aciduria cblA Type|Methylmalonic Aciduria cblB Type|Methylmalonic Aciduria and Homocystinuria Type cblC|MAHCD|Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency|Congenital Disorder of Glycosylation Type 1B|Mthfr Deficiency|Methylcobalamin Deficiency Type Cbl G (Disorder)|Methylcobalamin Deficiency Type cblE|Usher Syndrome, Type 1B|N-acetylglutamate Synthase Deficiency|Ornithine Transcarbamylase Deficiency|Phenylketonurias|Waardenburg Syndrome Type 1|Congenital Hypothyroidism|Propionic Acidemia|Usher Syndrome, Type 1F|Pancreatic Agenesis 1|Hereditary Hypophosphatemic Rickets|Glycogen Storage Disease IXB|Glycogen Storage Disease IXC|MOWS|Epilepsy, Early-Onset, Vitamin B6-Dependent|Pyridoxal Phosphate-Responsive Seizures|Pituitary Hormone Deficiency, Combined, 1|Ptsd|Dihydropteridine Reductase Deficiency|Severe Combined Immunodeficiency Due to RAG1 Deficiency|Severe Combined Immunodeficiency Due to RAG2 Deficiency|Retinoblastoma|Multiple Endocrine Neoplasia Type 2B|Pseudohypoaldosteronism, Type I|Liddle Syndrome|Biotin-Responsive Basal Ganglia Disease|SCD|DIAR1|GSD1C|Acrodermatitis Enteropathica|Thyroid Dyshormonogenesis 1|Riboflavin Transporter Deficiency|Waardenburg Syndrome, Type 2E|SRD|Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency|Barth Syndrome|Adrenocorticotropic Hormone Deficiency|Transcobalamin II Deficiency|Thyroid Dyshormonogenesis 3|Segawa Syndrome, Autosomal Recessive|Autosomal Recessive Nonsyndromic Hearing Loss|Thyroid Dyshormonogenesis 2A|Congenital Isolated Thyroid Stimulating Hormone Deficiency|Hypothyroidism Due to TSH Receptor Mutations|Usher Syndrome Type 1C|Usher Syndrome Type 1G (Diagnosis)|Von Willebrand Disease, Type 3|Combined Immunodeficiency Due to ZAP70 Deficiency|Adenine Phosphoribosyltransferase Deficiency|Metachromatic Leukodystrophy|Canavan Disease|Menkes Disease|Carbonic Anhydrase VA Deficiency|Developmental and Epileptic Encephalopathy 2|17 Alpha-Hydroxylase Deficiency|Smith-Lemli-Opitz Syndrome|Krabbe Disease|Glutathione Synthetase Deficiency|Mucopolysaccharidosis Type 7|Rett Syndrome|Molybdenum Cofactor Deficiency, Type A|Niemann-Pick Disease, Type C1|Niemann-Pick Disease Type C2|Ornithine Aminotransferase Deficiency|3-Phosphoglycerate Dehydrogenase Deficiency|Leber Congenital Amaurosis 2|Dravet Syndrome|Mucopolysaccharidosis Type 3 A|Ornithine Translocase Deficiency|Carnitine-acylcarnitine Translocase Deficiency|Glucose Transporter Type 1 Deficiency Syndrome|Creatine Transporter Deficiency|Niemann-Pick Disease Type A|Pitt Hopkins Syndrome|Tuberous Sclerosis 1|Tuberous Sclerosis 2|Ataxia With Isolated Vitamin E Deficiency|Angelman Syndrome|Prader-Willi Syndrome|Homocystinuria|Permanent Neonatal Diabetes Mellitus|Transient Neonatal Diabetes Mellitus|Factor VII Deficiency|Glycogen Storage Disease Type IXA1|Glycogen Storage Disease, Type IXA2|Glycogen Storage Disease IC|Glycogen Storage Disease Type IB|Central Hypoventilation Syndrome With or Without Hirschsprung Disease | ALL | CHILD | RTI International, Research Triangle Park, North Carolina, 27709, United States |
| Study of an Intervention to Improve Problem List Accuracy and Use | The aim of this study is to identify patients with problem list gaps and intervene to correct these gaps by creating clinical decision support interventions that alert providers to likely problem list gaps and offer clinicians the opportunity to correct them. The investigators will randomize the clinics that will receive the intervention and formally evaluate the study after a period of 6 months for improved problem list completeness to determine the effectiveness of our intervention. | Attention Deficit Disorder With Hyperactivity|Asthma|COPD|Breast Cancer|Coronary Artery Disease|Congestive Heart Failure|Diabetes|Glaucoma|Hemophilia|Hypertension|Hyperthyroidism|Hypothyroidism|Myasthenia Gravis|Osteoporosis|Osteopenia|Renal Failure|Renal Insufficiency|Sickle Cell Disease|Stroke | ALL | ADULT, OLDER_ADULT | Brigham and Women's Hospital, Boston, Massachusetts, 02115, United States |
| Perinatal Attentional Retraining Intervention for Smoking for Minority Women | The proposed research intends to randomize 50 abstinent pregnant Black or Hispanic smokers to receive either the attentional retraining (AR) or control VP task. Participants will be asked to carry around a smartphone as they go about their daily lives for 2 weeks in their last month of pregnancy (Phase 1). The smartphone will sound an alert randomly during the day, at which time participants will be asked to respond to a short set of questions assessing subjective states; this will be followed by a request to complete the AR (or control) procedures. This same procedure will be repeated for 2 weeks immediately after delivery (Phase 2). Women will undergo a follow-up visit 3 months after the end of Phase 2, and complete an unmodified VP and follow-up assessments. | Postpartum Smoking Relapse | FEMALE | ADULT | Yale University, New Haven, Connecticut, 06510, United States |
| Molecular Immunohematology in Ethiopian Sub-Populations | Alloimmunization to blood products in transfused patients is a recognized management challenge in the clinical setting. In particular the ethnic and racial specificity of RBC antigens and the limited availability of matched healthy volunteer blood donors have intensified the dilemma. The presence of low prevalence clinically significant RBC antigens among minorities account for the higher rate of alloimmunization observed in patients from this group. This is partly due to the racial and ethnic differences between the blood donor and recipient populations in the U.S. The increasing number of new Ethiopian-American immigrants in the US presenting to the health care system with blood transfusion requirements makes understanding the unique transfusion needs of this minority population imperative. Although the majority of African Americans claim origin to West Africa, Ethiopians, being from East Africa, represent a rapidly growing population in the United States. Furthermore, identifying genetic similarities and disparities to their West African counterparts will certainly have clinical implications in terms of transfusion support and disease modifiers. This additional information would help in understanding the natural history and transfusion requirements of certain debilitating diseases, such as Sickle Cell Disease (SCD), which are known to occur more commonly in African Americans. Identifying ethnically and racially similar individuals could assist in recruiting healthy volunteer donors with similar RBC antigen profiles potentially supplementing the rare donor pool. Although extensive archeological and sporadic serologic RBC antigen studies have been conducted in Ethiopia, there are no population wide RBC antigen molecular studies. Our study population is selected by altitude and migration history. Ethiopia, being in close proximity to the Red Sea in the northeast, Indian Ocean in the southeast and the rest of Africa in the south/west has diverse population. The study is a population based analysis of genetic variation of blood group antigens in three distinct and conserved Ethiopian sub-populations. Statistical analysis using Chi-square tests will be performed for each blood group antigen to detect differences in the distribution between the three sub-populations. The The Lead Associate Investigator (LAI), and, as appropriate, additional Investigators, will travel to Ethiopia to collect blood samples, which will be analyzed in the Department of Transfusion Medicine (DTM) at the National Institutes of Health (NIH) using the standard serologic methods and currently available molecular genotyping systems. Samples will also be stored for future high throughput sequencing analysis and other studies. The study will be a systematic analysis of the distribution of blood group antigens in Ethiopian sub-populations. Furthermore, new variants could be detected allowing insight into the correlation of particular genotypes and phenotypes. | Immunohematology | ALL | ADULT, OLDER_ADULT | Addis Ababa University, Addis Ababa, Ethiopia |
| Baby Detect : Genomic Newborn Screening | Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple blood test, rare genetic conditions can be easily detected, and the early start of transformative treatment will help avoid severe disabilities and increase the quality of life. Baby Detect Project is an innovative NBS program using a panel of target sequencing that aims to identify 126 treatable severe early onset genetic diseases at birth caused by 361 genes. The list of diseases has been established in close collaboration with the Paediatricians of the University Hospital in Liege. The investigators use dedicated dried blood spots collected between the first day and 28 days of life of babies, after a consent sign by parents. | Congenital Adrenal Hyperplasia|Familial Hyperinsulinemic Hypoglycemia 1|Phosphoglucomutase 1 Deficiency|Maturity Onset Diabetes of the Young|Cystic Fibrosis|Hypophosphatasia, Infantile|Congenital Hypothyroidism|Deficit in Anterior Pituitary Function and Variable Immunodeficiency|Pituitary Hormone Deficiency, Combined|Diamond Blackfan Anemia|Wiskott-Aldrich Syndrome|Fanconi Anemia|Hemophilia A|Hemophilia B|Glucose 6 Phosphate Dehydrogenase Deficiency|Alpha-Thalassemia|Sickle Cell Disease|Shwachman-Diamond Syndrome|Alpha 1-Antitrypsin Deficiency|Inflammatory Bowel Disease 25, Autosomal Recessive|Wilson Disease|Progressive Familial Intrahepatic Cholestasis|Crigler-Najjar Syndrome|Familial Chylomicronemia|Lysosomal Acid Lipase Deficiency|Familial Hemophagocytic Lymphocytosis|Griscelli Syndrome|Chediak-Higashi Syndrome|Severe Congenital Neutropenia|Severe Combined Immune Deficiency|Chronic Granulomatous Disease|Menkes Disease|Adrenoleukodystrophy|Smith-Lemli-Opitz Syndrome|Ataxia With Vitamin E Deficiency|Thiamine Metabolism Dysfunction Syndrome 5 (Episodic Encephalopathy Type)|Thiamine Metabolism Dysfunction Syndrome 4 (Bilateral Striatal Degeneration and Progressive Polyneuropathy Type)|Thiamine-Responsive Megaloblastic Anemia|Thiamine Metabolism Dysfunction Syndrome 2|Deficiency of GOT2|Cerebral Folate Transport Deficiency|Segawa Syndrome, Autosomal Recessive|Congenital Myasthenic Syndrome|Metachromatic Leukodystrophy|Sepiapterin Reductase Deficiency|Dopamine Beta Hydroxylase Deficiency|Glut1 Deficiency Syndrome|Late-Infantile Neuronal Ceroid Lipofuscinosis|Aromatic L-amino Acid Decarboxylase Deficiency|Charcot-Marie-Tooth Disease, Type 6C|Hereditary Hyperekplexia|Brain Dopamine-Serotonin Vesicular Transport Disease|Very Long Chain Hydroxy Acyl Dehydrogenase Deficiency|Tyrosinemia, Type I|Disaccharide Intolerance I|Beta Ketothiolase Deficiency|Phosphoglycerate Dehydrogenase Deficiency|Succinyl-Coa:3-Ketoacid Coa-Transferase Deficiency|Pyridoxine-5'-Phosphate Oxidase Deficiency|Pyridoxine-Dependent Epilepsy|Propionic Acidemia|Pompe Disease|Phenylalanine Hydroxylase Deficiency|Ornithine Transcarbamylase Deficiency|N Acetyl Glutamate Synthetase Deficiency|Riboflavin Deficiency|Maple Syrup Urine Disease|Medium Chain Acyl CoA Dehydrogenase Deficiency|Malonic Acidemia|Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency|Isovaleric Acidemia|Phosphoserine Aminotransferase Deficiency|Phosphoserine Phosphatase Deficiency|Hyperornithinemia-Hyperammonemia-Homocitrullinuria|S-Adenosylhomocysteine Hydrolase Deficiency|Mucopolysaccharidosis VII|Mucopolysaccharidosis VI|Mucopolysaccharidosis IV A|Mucopolysaccharidosis II|Mucopolysaccharidosis I|Transcobalamin Deficiency|Isolated Methylmalonic Acidemia|Cobalamin Deficiency|Homocystinuria|Holocarboxylase Synthetase Deficiency|Fanconi Bickel Syndrome|Glycogen Storage Disease|Glycine Encephalopathy|Glutaric Acidemia I|Glucose Galactose Malabsorption|Gaucher Disease, Type 1|Galactosemias|Fructosemia|Fructose-1,6-Diphosphatase Deficiency|Carbamoyl Phosphate Synthase 1 Deficiency|Citrullinemia Type II|Citrullinemia 1|Creatine Deficiency Syndrome|Systemic Primary Carnitine Deficiency|Carnitine Palmitoyltransferase Deficiency 2|Carnitine Palmitoyltransferase Deficiency 1|Carnitine Acylcarnitine Translocase Deficiency|Riboflavin Transporter Deficiency|Branched-Chain Keto Acid Dehydrogenase Kinase Deficiency|Andersen Tawil Syndrome|Timothy Syndrome|Jervell-Lange Nielsen Syndrome|Catecholaminergic Polymorphic Ventricular Tachycardia|Familial Hypertrophic Cardiomyopathy Type 4|Pseudohypoaldosteronism, Type II|Pseudohypoaldosteronism Type 1|Primary Hyperoxaluria|X Linked Hypophosphatemia|Hereditary Nephrogenic Diabetes Insipidus|Cystinosis|Congenital Nephrotic Syndrome, Finnish Type|Alport Syndrome|Hereditary Retinoblastoma|Biotinidase Deficiency|Aciduria, Argininosuccinic|Argininemia|Acyl-CoA Dehydrogenase Family, Member 9, Deficiency of|3-Hydroxy 3-Methyl Glutaric Aciduria|3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency | ALL | CHILD | CRMN, Hôpital La Citadelle, Liege, Wallonia, 4000, Belgium |
| Genetics of Type 2 Diabetes in West Africans | Background: * Type 2 diabetes (T2D) and associated complications are major contributors to the global disease burden. T2D is already a major health threat in populations in developed countries and is rapidly taking hold in the developing world. * It is believed that understanding the complex interplay between genetic and lifestyle characteristics in the etiology of T2D and related complications will lead to the development of better preventive and therapeutic strategies. In Addition, the results of this project will facilitate our understanding of causes of diabetes in African Americans, other US and world populations Objectives: * To conduct a genome-wide association study (GWAS) to identify susceptibility genetic variants for diabetes among the Yoruba people in Ibadan, Nigeria. * To enroll and examine 300 unrelated cases of T2D and 300 ethnicity-matched Yoruba controls. * To conduct resequencing of positional candidate gene/loci to identify likely functional variants in a subset of the cohort. * To conduct replication studies of the top-100 scoring variants in three independent African and European ancestry samples. * To investigate whether diabetes-associated variants discovered in European populations increase diabetes risk in West Africans. Eligibility: * Patients 18 years of age with confirmed T2D who are newly diagnosed or on treatment of Yoruba ethnicity in Ibadan, Nigeria. Control subjects are nondiabetics ethnically matched to patients. Design: * The study design for both patients and controls consists of the following steps: * Discuss informed consent process and obtain signed informed consent form. Informed consent will be administered by trained clinic staff. * Assign study ID (barcode) * Administer questionnaires * Obtain spot urine sample * Measure blood pressure * Obtain anthropometric measurements including body composition * Perform finger prick for blood glucose level * Obtain venous blood samples * Perform eye examination * On the following day, perform confirmatory blood glucose for the small subset of participants requiring confirmation of previous test result DNA extraction of stored samples will be done at either the National Institutes of Health or the laboratory in Nigeria. * GWAS will be conducted using publicly available software packages. | Hypertension|Diabetes | ALL | ADULT, OLDER_ADULT | University of Ghana, Accra, Ghana|University of Science and Tech, Kumasi, Ghana|University of Nigeria, Enugu, Nigeria|University of Ibadan, Ibadan, Nigeria|University of Lagos, Lagos, Nigeria |
| A Study to Learn How Different Preparations of Osivelotor Taste and Enter the Blood With Food or Liquids or With an Antacid in Healthy Adults | A study to learn how different preparations of Osivelotor taste and enter the blood with food or liquids, or with an antacid in healthy adults. | Healthy | ALL | ADULT, OLDER_ADULT | |
| Asthma Academy: Use of Simulation and Telehealth to Empower Family Caregivers | The goal of this project is to implement and evaluate a novel intervention for low-income families to reduce the burden of caregivers of a child with asthma. | Caregiver Burnout | ALL | ADULT, OLDER_ADULT | University of Miami, Coral Gables, Florida, 33146, United States |
| Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload | This is an open-label study to assess the pharmacokinetics, safety, efficacy and tolerability of SSP-004184AQ. The study consists of two phases: the pharmacokinetic phase, using a single 16 mg/kg dose of SSP-004184AQ; and the chronic dosing phase, during which patients will receive an additional 48 weeks of SSP-004184AQ dosing. Two age groups will be studied: 6-\<12, and 12-\<18 years old. The study is designed to initially assess the pharmacokinetics and safety of SSP-004184AQ in older children (adolescents, 12-\<18 years old) and then if deemed safe, in younger children (6-\<12 years old). | Transfusional Iron Overload|Beta-Thalassemia | ALL | CHILD | Children's Hospital Boston, Boston, Massachusetts, 02115, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Toronto Sick Kids Hospital, Toronto, Ontario, Canada|Ospedale Regionale Mecrocitemie, Cagliari, 09121, Italy|Centro della Microcitemia e delle Anemie Congenite, Genoa, Italy|Thalassemia Center San Luigi Hospital, Orbassano, Italy|American University of Beirut Medical Center, Beirut, Lebanon|Chronic Care Center, Beirut, Lebanon|Ege University Hospital, Izmir, 35100, Turkey |
| Outcomes Mandate National Integration With Cannabis as Medicine | This will be a multistate, multicenter clinical study to determine the efficacy and safety of medical cannabis for a wide variety of chronic medical conditions. | Chronic Pain|Chronic Pain Syndrome|Chronic Pain Due to Injury|Chronic Pain Due to Trauma|Fibromyalgia|Seizures|Hepatitis C|Cancer|Crohn Disease|HIV/AIDS|Multiple Sclerosis|Traumatic Brain Injury|Sickle Cell Disease|Post Traumatic Stress Disorder|Tourette Syndrome|Ulcerative Colitis|Glaucoma|Epilepsy|Inflammatory Bowel Diseases|Parkinson Disease|Amyotrophic Lateral Sclerosis|Chronic Traumatic Encephalopathy|Anxiety|Depression|Insomnia|Autism|Opioid-use Disorder|Bipolar Disorder|Covid19|SARS-CoV Infection|COVID-19|Corona Virus Infection|Coronavirus | ALL | CHILD, ADULT, OLDER_ADULT | OMNI Medical Services, Boca Raton, Florida, 33433, United States|OMNI Medical Services, Bradenton, Florida, 34209, United States|OMNI Medical Services, Fort Lauderdale, Florida, 33308, United States|OMNI Medical Services, Fort Myers, Florida, 34135, United States|OMNI Medical Services, Gainesville, Florida, 36201, United States|OMNI Medical Services, Merritt Island, Florida, 32952, United States|OMNI Medical Services, Miami, Florida, 33137, United States|OMNI Medical Services, Ocoee, Florida, 34761, United States|OMNI Medical Services, Pensacola, Florida, 32505, United States|OMNI Medical Services, Pompano Beach, Florida, 33060, United States|OMNI Medical Services, Tampa, Florida, 33614, United States|OMNI Medical Services, Wesley Chapel, Florida, 33544, United States|OMNI Medical Services, Beechwood, Ohio, 44122, United States|OMNI Medical Services, Bowling Green, Ohio, 43402, United States|OMNI Medical Services, Sandusky, Ohio, 44870, United States|OMNI Medical Services, Toledo, Ohio, 43604, United States|OMNI Medical Services, Toledo, Ohio, 43611, United States |
| Effect of Platelet Inhibition and / or Lipid Lowering in Non-ACS-patients With Positive Troponin | The study evaluates the effect of platelet inhibition and / or lipid lowering in non-ACS-patients with symptoms suggestive for ACS, and elevated high-sensitivity troponin values | Acute Chest Syndrome | ALL | ADULT, OLDER_ADULT | University of Berlin, Campus Benjamin-Franklin, Berlin, 12203, Germany|Asklepios Harz-Hospital Goslar, Goslar, 38642, Germany|University Heart Center Hamburg, Hamburg, 20246, Germany|University of Heidelberg, Heidelberg, 69120, Germany|University of Leipzig, Leipzig, 04103, Germany|Robert-Bosch-Hospital Stuttgart, Stuttgart, 70376, Germany|University of Ulm, Ulm, 89081, Germany |
| A Study to Find a Suitable Dose of ASP8731 and Check for Medical Problems at Each Dose in Healthy Adults | ASP8731 is a potential new treatment for people with sickle cell disease. Before ASP8731 is available as a treatment, the researchers need to understand how it is processed by and acts upon the body. They do this to find a suitable dose and to check for potential medical problems from the treatment. This type of study usually includes healthy adults but can include people with the relevant condition. The main aim of this study is to learn if healthy adults have any medical problems after taking different doses of ASP8731. This study will be in 2 parts. In Part 1, different small groups of people will take lower to higher doses of ASP8731 or a placebo. In this study a placebo looks like ASP8731 but will not have any medicine in it. This is done to find suitable doses of ASP8731 and work out how often it should be taken. Some of this information will be used in Part 2 of the study. The first group will take the lowest dose of ASP8731 or the placebo. A medical expert panel will check the results from this group and decide which ASP8731 dose the next group can take. The panel will do this for each group until all groups have taken ASP8731 or until a suitable dose of ASP8731 has been reached. In Part 1, people will visit the study clinic 4 times. The first visit is to check if they can take part. People will be asked about their medical history, have a physical exam, and their vital signs checked (pulse rate, temperature, and blood pressure). Also, they will have an ECG to check their heart rhythm and have some blood samples taken for laboratory tests. For women this will include a pregnancy test. At the second visit, people will stay in the study clinic for a few days. In most groups, people will be picked to take either ASP8731 or the placebo by chance alone. They will fast before taking ASP8731 or the placebo. 1 group will take ASP8731 with food. No-one in that group will take the placebo. After taking ASP8731 or the placebo (or just ASP8731 with food), people in all groups will give blood and urine samples during the next 3 days. Also, they will be checked for any medical problems during their clinic stay. People will return to the study clinic for a 2 follow-up visits - at about 8 and 30 days after they last took ASP8731 or the placebo. At these visits people will be asked if they have any medical problems. They will have a physical exam, have their vital signs checked (pulse rate, temperature, and blood pressure), and have some blood samples taken for laboratory tests. For women, this will include another pregnancy test. In Part 2, other different small groups of people will take lower to higher doses of ASP8731 or a placebo. How often it is taken will be worked out from Part 1.The first group will take the lowest dose of ASP8731 or the placebo each day over several days. A medical expert panel will check the results from this group and decide which ASP8731 dose the next group can take. The panel will do this for each group until all groups have taken ASP8731. In Part 2, people will visit the study clinic 5 times. The first visit is to check if they can take part. People will be asked about their medical history, have a physical exam, and their vital signs checked. Also, they will have an ECG and have some blood samples taken for laboratory tests. For women this will include a pregnancy test. At the second visit, people will stay in the study clinic for several days. In all groups, people will be picked to take either ASP8731 or the placebo by chance alone. After taking ASP8731 or the placebo, people in all groups will give blood samples throughout their stay. They will give urine samples during the last few days of their stay. Also, they will be checked for any medical problems during their clinic stay. After discharge, people will return to the clinic 4 days later for a third visit. At this visit, they will have some blood samples taken for laboratory tests. Also, they will have their vital signs checked and will be asked if they have any medical problems. People will return to the study clinic for a 2 follow-up visits - at about 15 and 30 days after they last took ASP8731 or the placebo. At these visits people will be asked if they have any medical problems. They will have a physical exam and have their vital signs checked. They will also have an ECG and have some blood samples taken for laboratory tests. For women, this will include another pregnancy test. No other visits are planned during this study. | Healthy Participants | ALL | ADULT | California Clinical Trials Medical Group, Glendale, California, 91206, United States |
| Study to Assess the Safety and Efficacy of Ribociclib (LEE011) in Combination With Letrozole for the Treatment of Men and Pre/Postmenopausal Women With HR+ HER2- aBC | The purpose of this study is to collect additional safety and efficacy data for the combination of ribociclib + letrozole in men and pre/postmenopausal women with HR+HER2- advanced breast cancer and no prior hormonal treatment for advanced disease.. | Breast Cancer | ALL | ADULT, OLDER_ADULT | Alaska Cancer Research and Education Center, Anchorage, Alaska, 99508, United States|Ironwood Cancer and Research Centers, Chandler, Arizona, 85224, United States|Arizona Oncology Associates, Phoenix, Arizona, 85016, United States|Arizona Oncology Associates Arizona Oncology Assoc. (2), Tucson, Arizona, 85745, United States|Highlands Oncology Group, Fayetteville, Arkansas, 72703, United States|Beverly Hills Cancer Center, Beverly Hills, California, 90211, United States|Pacific Shores Medical Group SC, Long Beach, California, 90813, United States|USC Norris Cancer Center, Los Angeles, California, 90033, United States|University of California Irvine UC Irvine (11), Orange, California, 92868, United States|Ventura County Hematology and Oncology, Oxnard, California, 93030, United States|PCR Oncology, Pismo Beach, California, 93449, United States|California Pacific Medical Center Onc Dept, San Francisco, California, 94120-7999, United States|Centura Health Research Center Centura Health Research Center, Denver, Colorado, 80210, United States|Poudre Valley Hospital Poudre Valley Health System, Fort Collins, Colorado, 80528, United States|Valley View Hospital Cancer Center, Glenwood Springs, Colorado, 81601, United States|Florida Cancer Research Institute Dept of Oncology, Davie, Florida, 33328, United States|Foundation for Sickle Cell Disease Research, Hollywood, Florida, 33021, United States|Watson Clinic Center for Research 1730 Location, Lakeland, Florida, 33805, United States|Mid Florida Hematology and Onc Ctr, Orange, Florida, 32763, United States|Summit Cancer Care Summit Cancer Care (SC), Savannah, Georgia, 31405, United States|John D Archbold Memorial Hospital John D. Archbold Mem Hosp (4), Thomasville, Georgia, 31792, United States|Saint Alphonsus Regional Medical Center, Boise, Idaho, 83706, United States|Stroger Cook County Hospital Division of Hematology & Onc, Chicago, Illinois, 60612, United States|Joliet Oncology-Hematology Associates Presence Cancer Center, Joliet, Illinois, 60435, United States|Mid Illinois Hematology Oncology Mid Illinois Hema/Onc (3), Normal, Illinois, 61761, United States|Alpha Med Physician Group, LLC, Tinley Park, Illinois, 60487, United States|Indian Univ Health Goshen Center forCancer SC, Goshen, Indiana, 46526, United States|Northwest Oncology, Munster, Indiana, 46321, United States|June E. Nylan Cancer Center, Sioux City, Iowa, 51101, United States|University of Kansas Medical Center University of Kansas Med Ctr 9, Kansas City, Kansas, 66160-7330, United States|Sarah Cannon at Overland Park Regional Medical Center, Overland Park, Kansas, 66209, United States|John Ochsner Heart and Vascular Institute Clinical Trials, New Orleans, Louisiana, 70121, United States|Northern Light Mercy Hospital SC, Portland, Maine, 04102, United States|Greater Baltimore Medical Center Cancer Center Greater Baltimore Medical Ctr, Baltimore, Maryland, 21204-6831, United States|Maryland Oncology Hematology P A Columbia, Rockville, Maryland, 20850, United States|Kaiser Permanente, Rockville, Maryland, 20879, United States|Medical Faculty Assc Inc Medical Faculty Assc., Inc. (2, Washington DC, Maryland, 20037, United States|Jackson Oncology Associates, Jackson, Mississippi, 39202, United States|Nebraska Hematology-Oncology, P.C., Lincoln, Nebraska, 68506, United States|Nebraska Cancer Specialists Oncology Hematology West, Omaha, Nebraska, 68154, United States|Comprehensive Cancer Centers of Nevada CCC of Nevada Henderson (4), Henderson, Nevada, 89052, United States|Trinitas Comprehensive Cancer Center, Elizabeth, New Jersey, 07207, United States|Englewood Health, Englewood, New Jersey, 07631, United States|The Valley Hospital / Luckow Pavillion, Paramus, New Jersey, 07652, United States|Somerset Hematology Oncology Associates Somerset Hematolgy Onc -MI, Somerset, New Jersey, 08873, United States|New Mexico Cancer Care Alliance ., Albuquerque, New Mexico, 87106, United States|San Juan Oncology Associates, Farmington, New Mexico, 87401, United States|Eastchester Center for Cancer Care, Bronx, New York, 10469, United States|Clinical Research Alliance, Lake Success, New York, 11042, United States|Oncology Speciialists of Charlotte, Charlotte, North Carolina, 28207, United States|Aultman Cancer Center Main Centre, Canton, Ohio, 44710, United States|The Christ Hospital Cancer Center Research Program Linder Research Center, Cincinnati, Ohio, 45219, United States|University Hospitals of Cleveland Seidman Cancer Center Cleveland Medical Center, Cleveland, Ohio, 44106, United States|Cleveland Clinic Foundation Cleveland Clinic (5), Cleveland, Ohio, 44195, United States|Dayton Physicians, Kettering, Ohio, 45409, United States|Oklahoma Cancer Specialists and Research Institute SC-2, Tulsa, Oklahoma, 74136, United States|Oregon Health Sciences University SC-5, Portland, Oregon, 97239, United States|McLeod Center for Cancer Treatment and Research, Florence, South Carolina, 29506, United States|Carolina Blood and Cancer Care of South Carolina, Rock Hill, South Carolina, 29732, United States|Millennium Research Clin Develop Millennium Oncology - FL, Houston, Texas, 77090, United States|Mays Cancer Ctr Uthsa Mdacc, San Antonio, Texas, 78229, United States|Hope Cancer Center of East Texas, Tyler, Texas, 75701, United States|Virginia Oncology Associates, Norfolk, Virginia, 23502, United States|PeaceHealth St Joseph Medical Center, Bellingham, Washington, 98225, United States|Providence Regional Medical Centre of Everett, Everett, Washington, 98201, United States|Kadlec Clinic Hematology and Onco, Kennewick, Washington, 99336, United States|Valley Medical Center Research Valley Medical Center, Renton, Washington, 98055, United States|Virginia Mason Medical Center-Oncology SC, Seattle, Washington, 98101, United States|Northwest Medical Specialties Dept.ofNW Med. Specialties, Tacoma, Washington, 98405, United States|Columbia St Mary s Hospital of Milwaukee St. Mary's Hospital Ozaukee, Milwaukee, Wisconsin, 53211, United States|Cheyenne Regional Medical Center Cheyenne Regional Med Ctr (3), Cheyenne, Wyoming, 82001, United States|Novartis Investigative Site, Caba, Buenos Aires, C1125ABD, Argentina|Novartis Investigative Site, Caba, Buenos Aires, C1426ANZ, Argentina|Novartis Investigative Site, Rosario, Santa Fe, S2000KZE, Argentina|Novartis Investigative Site, San Miguel De Tucuman, Tucuman, T4000IAK, Argentina|Novartis Investigative Site, La Rioja, 5300, Argentina|Novartis Investigative Site, Innsbruck, Tyrol, 6020, Austria|Novartis Investigative Site, Graz, 8036, Austria|Novartis Investigative Site, Leoben, A 8700, Austria|Novartis Investigative Site, Rankweil, A-6830, Austria|Novartis Investigative Site, Salzburg, 5020, Austria|Novartis Investigative Site, Vienna, 1090, Austria|Novartis Investigative Site, Vienna, A 1090, Austria|Novartis Investigative Site, Wien, A-1130, Austria|Novartis Investigative Site, Sint Niklaas, Oost Vlaanderen, 9100, Belgium|Novartis Investigative Site, Antwerp, 2020, Belgium|Novartis Investigative Site, Bonheiden, 2820, Belgium|Novartis Investigative Site, Brugge, 8000, Belgium|Novartis Investigative Site, Brussels, BE-B-1200, Belgium|Novartis Investigative Site, Brussel, 1090, Belgium|Novartis Investigative Site, Bruxelles, 1000, Belgium|Novartis Investigative Site, Charleroi, 6000, Belgium|Novartis Investigative Site, Genk, 3600, Belgium|Novartis Investigative Site, Gent, 9000, Belgium|Novartis Investigative Site, Hasselt, 3500, Belgium|Novartis Investigative Site, Kortrijk, 8500, Belgium|Novartis Investigative Site, Leuven, 3000, Belgium|Novartis Investigative Site, Liege, 4000, Belgium|Novartis Investigative Site, Luxembourg, 1210, Belgium|Novartis Investigative Site, Mons, 7000, Belgium|Novartis Investigative Site, Namur, 5000, Belgium|Novartis Investigative Site, Roeselare, 8800, Belgium|Novartis Investigative Site, Verviers, 4800, Belgium|Novartis Investigative Site, Wilrijk, 2610, Belgium|Novartis Investigative Site, Gabrovo, 5300, Bulgaria|Novartis Investigative Site, Plovdiv, 4004, Bulgaria|Novartis Investigative Site, Sofia, 1303, Bulgaria|Novartis Investigative Site, Sofia, 1527, Bulgaria|Novartis Investigative Site, Sofia, 1606, Bulgaria|Novartis Investigative Site, Sofia, 1756, Bulgaria|Novartis Investigative Site, Calgary, Alberta, T2N 4N2, Canada|Novartis Investigative Site, Edmonton, Alberta, T6G 1Z2, Canada|Novartis Investigative Site, Kelowna, British Columbia, V1Y 5L3, Canada|Novartis Investigative Site, Surrey, British Columbia, V3V 1Z2, Canada|Novartis Investigative Site, Vancouver, British Columbia, V5Z 4E6, Canada|Novartis Investigative Site, Winnipeg, Manitoba, R3E 0V9, Canada|Novartis Investigative Site, Moncton, New Brunswick, E1C 6Z8, Canada|Novartis Investigative Site, Halifax, Nova Scotia, B3H 2Y9, Canada|Novartis Investigative Site, Barrie, Ontario, L4M 6M2, Canada|Novartis Investigative Site, Cambridge, Ontario, N1R 3G2, Canada|Novartis Investigative Site, Hamilton, Ontario, L8V 5C2, Canada|Novartis Investigative Site, Kingston, Ontario, K7L 5P9, Canada|Novartis Investigative Site, Kitchener, Ontario, N2G 1G3, Canada|Novartis Investigative Site, London, Ontario, N6A 4G4, Canada|Novartis Investigative Site, Newmarket, Ontario, L3Y 2P9, Canada|Novartis Investigative Site, North York, Ontario, M2K1E1, Canada|Novartis Investigative Site, Oshawa, Ontario, L1G 2B9, Canada|Novartis Investigative Site, Ottawa, Ontario, KIH 7W9, Canada|Novartis Investigative Site, Sault Ste Marie, Ontario, P6B 0A8, Canada|Novartis Investigative Site, Sudbury, Ontario, P3E 5J1, Canada|Novartis Investigative Site, Toronto, Ontario, M3M 0B2, Canada|Novartis Investigative Site, Toronto, Ontario, M4N 3M5, Canada|Novartis Investigative Site, Toronto, Ontario, M5B 1W8, Canada|Novartis Investigative Site, Greenfield Park, Quebec, J4V 2H1, Canada|Novartis Investigative Site, Laval, Quebec, H7M 3L9, Canada|Novartis Investigative Site, Montreal, Quebec, H3T 1E2, Canada|Novartis Investigative Site, Montreal, Quebec, H3T 1M5, Canada|Novartis Investigative Site, Montreal, Quebec, H4J 1C5, Canada|Novartis Investigative Site, Sherbrooke, Quebec, J1H 5N4, Canada|Novartis Investigative Site, Regina, Saskatchewan, S4T 7T1, Canada|Novartis Investigative Site, Saskatoon, Saskatchewan, S7N 4H4, Canada|Novartis Investigative Site, Quebec, G1S 4L8, Canada|Novartis Investigative Site, Santiago, 8420383, Chile|Novartis Investigative Site, Brno, Czech Republic, 656 53, Czechia|Novartis Investigative Site, Liberec, Czech Republic, 46063, Czechia|Novartis Investigative Site, Prague 8, Czech Republic, 180 00, Czechia|Novartis Investigative Site, Zlin, Czech Republic, 762 75, Czechia|Novartis Investigative Site, Hradec Kralove, CZE, 500 05, Czechia|Novartis Investigative Site, Olomouc, CZE, 779 00, Czechia|Novartis Investigative Site, Brno, 625 00, Czechia|Novartis Investigative Site, Ceske Budejovice, 370 87, Czechia|Novartis Investigative Site, Pardubice, 532 03, Czechia|Novartis Investigative Site, Prague 5, 150 00, Czechia|Novartis Investigative Site, Praha 10, 100 34, Czechia|Novartis Investigative Site, Praha 4, 140 59, Czechia|Novartis Investigative Site, Praha, 12808, Czechia|Novartis Investigative Site, Copenhagen, DK-2100, Denmark|Novartis Investigative Site, Vejle, 7100, Denmark|Novartis Investigative Site, Helsinki, 00029, Finland|Novartis Investigative Site, Oulu, FIN-90220, Finland|Novartis Investigative Site, Tampere, FIN-33521, Finland|Novartis Investigative Site, Nice Cedex 2, Alpes Maritimes, 06189, France|Novartis Investigative Site, Dijon, Cote D Or, 21034, France|Novartis Investigative Site, Limoges, Haute Vienne, 87000, France|Novartis Investigative Site, Saint-Cloud, Hauts De Seine, 92210, France|Novartis Investigative Site, Tours 9, Indre Et Loire, 37044, France|Novartis Investigative Site, Reims, Marne, 51056, France|Novartis Investigative Site, Albi, 81000, France|Novartis Investigative Site, Angers Cedex 02, 49055, France|Novartis Investigative Site, Avignon, 84082, France|Novartis Investigative Site, Besancon Cedex, 25030, France|Novartis Investigative Site, Bobigny Cedex, 93009, France|Novartis Investigative Site, Bordeaux Cedex, 33000, France|Novartis Investigative Site, Bordeaux, 33076, France|Novartis Investigative Site, Caen, 14021, France|Novartis Investigative Site, Clermont-Ferrand, 63011, France|Novartis Investigative Site, Colmar Cedex, 68024, France|Novartis Investigative Site, Corbeil Essonnes, 91100, France|Novartis Investigative Site, Creteil, 94010, France|Novartis Investigative Site, Le Mans Cedex, 72015, France|Novartis Investigative Site, Lille Cedex, 59020, France|Novartis Investigative Site, Limoges, 87000, France|Novartis Investigative Site, Lyon Cedex 08, 69373, France|Novartis Investigative Site, Lyon, 69373, France|Novartis Investigative Site, Marseille, 13273, France|Novartis Investigative Site, Mont de Marsan cedex, 40024, France|Novartis Investigative Site, Montpellier, 34070, France|Novartis Investigative Site, Montpellier, 34298, France|Novartis Investigative Site, Nancy, 54000, France|Novartis Investigative Site, Nantes Cedex, 44277, France|Novartis Investigative Site, Paris 10, 75475, France|Novartis Investigative Site, Paris 13, 75651, France|Novartis Investigative Site, Paris, 75012, France|Novartis Investigative Site, Paris, 75231, France|Novartis Investigative Site, Paris, 75970, France|Novartis Investigative Site, Perigueux Cedex, 24004, France|Novartis Investigative Site, Pierre Benite, 69495, France|Novartis Investigative Site, Plerin Sur Mer, 22190, France|Novartis Investigative Site, Reims, 51100, France|Novartis Investigative Site, Rouen, 76038, France|Novartis Investigative Site, Saint Herblain, 44805, France|Novartis Investigative Site, Strasbourg Cedex, 67091, France|Novartis Investigative Site, Strasbourg, 67010, France|Novartis Investigative Site, Toulon La Seyne Sur Mer, 83056, France|Novartis Investigative Site, Troyes, 10003, France|Novartis Investigative Site, Vandoeuvre-les-Nancy, 54519, France|Novartis Investigative Site, Ioannina, GR, 455 00, Greece|Novartis Investigative Site, Thessaloniki, GR, 54645, Greece|Novartis Investigative Site, Athens, 115 28, Greece|Novartis Investigative Site, Athens, GR 115 22, Greece|Novartis Investigative Site, Athens, GR14564, Greece|Novartis Investigative Site, Patras, 265 00, Greece|Novartis Investigative Site, Thessaloniki, 540 07, Greece|Novartis Investigative Site, Hong Kong, Hong Kong|Novartis Investigative Site, Kowloon, Hong Kong|Novartis Investigative Site, Pokfulam, Hong Kong|Novartis Investigative Site, Budapest, 1134, Hungary|Novartis Investigative Site, Budapest, H 1122, Hungary|Novartis Investigative Site, Budapest, H-1032, Hungary|Novartis Investigative Site, Debrecen, 4032, Hungary|Novartis Investigative Site, Szeged, 6725, Hungary|Novartis Investigative Site, Hyderabad, Andhra Pradesh, 500 034, India|Novartis Investigative Site, Tamil Nadu, Chennai, 600035, India|Novartis Investigative Site, New Delhi, Delhi, 110092, India|Novartis Investigative Site, Admedabad, Gujarat, 380060, India|Novartis Investigative Site, Bangalore, Karnataka, 560027, India|Novartis Investigative Site, Nashik, Maharashtra, 422 004, India|Novartis Investigative Site, Pune, Maharashtra, 411013, India|Novartis Investigative Site, Jaipur, Rajasthan, 302017, India|Novartis Investigative Site, Vellore, Tamil Nadu, 632 004, India|Novartis Investigative Site, Kolkata, West Bengal, 700160, India|Novartis Investigative Site, Delhi, 110 085, India|Novartis Investigative Site, Beer-Sheva, 8457108, Israel|Novartis Investigative Site, Haifa, 3109601, Israel|Novartis Investigative Site, Jerusalem, 9112001, Israel|Novartis Investigative Site, Petach Tikva, 4941492, Israel|Novartis Investigative Site, Ramat Gan, 52621, Israel|Novartis Investigative Site, Tel Aviv, 6423906, Israel|Novartis Investigative Site, Alessandria, AL, 15100, Italy|Novartis Investigative Site, Ancona, AN, 60126, Italy|Novartis Investigative Site, L'Aquila, AQ, 67100, Italy|Novartis Investigative Site, Arezzo, AR, 52100, Italy|Novartis Investigative Site, Asti, AT, 14100, Italy|Novartis Investigative Site, Avellino, AV, 83100, Italy|Novartis Investigative Site, Bari, BA, 70124, Italy|Novartis Investigative Site, Bergamo, BG, 24127, Italy|Novartis Investigative Site, Treviglio, BG, 24047, Italy|Novartis Investigative Site, Benevento, BN, 82100, Italy|Novartis Investigative Site, Bologna, BO, 40138, Italy|Novartis Investigative Site, Bologna, BO, 40139, Italy|Novartis Investigative Site, Brindisi, BR, 72100, Italy|Novartis Investigative Site, Brescia, BS, 25123, Italy|Novartis Investigative Site, Brescia, BS, 25124, Italy|Novartis Investigative Site, Monserrato, CA, 09042, Italy|Novartis Investigative Site, Cuneo, CN, 12100, Italy|Novartis Investigative Site, Cremona, CR, 26100, Italy|Novartis Investigative Site, Catania, CT, 95124, Italy|Novartis Investigative Site, Cona, FE, 44100, Italy|Novartis Investigative Site, San Giovanni Rotondo, FG, 71013, Italy|Novartis Investigative Site, Firenze, FI, 50134, Italy|Novartis Investigative Site, Genova, GE, 16132, Italy|Novartis Investigative Site, Grosseto, GR, 58100, Italy|Novartis Investigative Site, Lecco, LC, 23900, Italy|Novartis Investigative Site, Livorno, LI, 57124, Italy|Novartis Investigative Site, Lucca, LU, 55100, Italy|Novartis Investigative Site, Monza, MB, 20900, Italy|Novartis Investigative Site, Macerata, MC, 62100, Italy|Novartis Investigative Site, Messina, ME, 98158, Italy|Novartis Investigative Site, Taormina, ME, 98039, Italy|Novartis Investigative Site, Milano, MI, 20121, Italy|Novartis Investigative Site, Milano, MI, 20132, Italy|Novartis Investigative Site, Milano, MI, 20133, Italy|Novartis Investigative Site, Milano, MI, 20141, Italy|Novartis Investigative Site, Milano, MI, 20162, Italy|Novartis Investigative Site, Rozzano, MI, 20089, Italy|Novartis Investigative Site, Modena, MO, 41124, Italy|Novartis Investigative Site, Nuoro, NU, 08100, Italy|Novartis Investigative Site, Palermo, PA, 90127, Italy|Novartis Investigative Site, Palermo, PA, 90146, Italy|Novartis Investigative Site, Padova, PD, 35100, Italy|Novartis Investigative Site, Pisa, PI, 56126, Italy|Novartis Investigative Site, Aviano, PN, 33081, Italy|Novartis Investigative Site, Prato, PO, 59100, Italy|Novartis Investigative Site, Parma, PR, 43100, Italy|Novartis Investigative Site, Fano, PU, 61032, Italy|Novartis Investigative Site, Pavia, PV, 27100, Italy|Novartis Investigative Site, Reggio Calabria, RC, 89124, Italy|Novartis Investigative Site, Reggio Emilia, RE, 42123, Italy|Novartis Investigative Site, Roma, RM, 00128, Italy|Novartis Investigative Site, Roma, RM, 00152, Italy|Novartis Investigative Site, Roma, RM, 00161, Italy|Novartis Investigative Site, Roma, RM, 00168, Italy|Novartis Investigative Site, Roma, RM, 00189, Italy|Novartis Investigative Site, Salerno, SA, 84131, Italy|Novartis Investigative Site, Siena, SI, 53100, Italy|Novartis Investigative Site, Sassari, SS, 07100, Italy|Novartis Investigative Site, Savona, SV, 17100, Italy|Novartis Investigative Site, Trento, TN, 38100, Italy|Novartis Investigative Site, Candiolo, TO, 10060, Italy|Novartis Investigative Site, Torino, TO, 10126, Italy|Novartis Investigative Site, Terni, TR, 05100, Italy|Novartis Investigative Site, Udine, UD, 33100, Italy|Novartis Investigative Site, Saronno, VA, 21047, Italy|Novartis Investigative Site, Mirano, VE, 30035, Italy|Novartis Investigative Site, Negrar, VR, 37024, Italy|Novartis Investigative Site, Verona, VR, 37126, Italy|Novartis Investigative Site, Viterbo, VT, 01100, Italy|Novartis Investigative Site, Napoli, 80131, Italy|Novartis Investigative Site, Napoli, 80138, Italy|Novartis Investigative Site, Novara, 28100, Italy|Novartis Investigative Site, Amman, 11941, Jordan|Novartis Investigative Site, Ashrafieh, 166830, Lebanon|Novartis Investigative Site, Beirut, 10999, Lebanon|Novartis Investigative Site, Saida, 652, Lebanon|Novartis Investigative Site, Tanjong Bungah, Penang, 11200, Malaysia|Novartis Investigative Site, Petaling Jaya, Selangor, 46050, Malaysia|Novartis Investigative Site, Kuala Lumpur, Wilayah Persekutuan, 50586, Malaysia|Novartis Investigative Site, Putrajaya, Wilayah Persekutuan, 62250, Malaysia|Novartis Investigative Site, Kuala Lumpur, 59100, Malaysia|Novartis Investigative Site, Monterrey, Nuevo Leon, 64710, Mexico|Novartis Investigative Site, Ciudad de Mexico, 04700, Mexico|Novartis Investigative Site, Mexico City, 01120, Mexico|Novartis Investigative Site, Maastricht, AZ, 5800, Netherlands|Novartis Investigative Site, Venray, CE, 5801, Netherlands|Novartis Investigative Site, Apeldoorn, DZ, 7334, Netherlands|Novartis Investigative Site, Arnhem, Gelderland, 6815 AD, Netherlands|Novartis Investigative Site, Beverwijk, 1942 LE, Netherlands|Novartis Investigative Site, Breda, 4818 CK, Netherlands|Novartis Investigative Site, Delft, 2625 AD, Netherlands|Novartis Investigative Site, Den Bosch, 5223 GZ, Netherlands|Novartis Investigative Site, Den Haag, 2545 CH, Netherlands|Novartis Investigative Site, Dordrecht, 3318AT, Netherlands|Novartis Investigative Site, Ede, 6716 RP, Netherlands|Novartis Investigative Site, Eindhoven, 5623EJ, Netherlands|Novartis Investigative Site, Enschede, 7513 ER, Netherlands|Novartis Investigative Site, Goes, 4462 RA, Netherlands|Novartis Investigative Site, Groningen, 9728 NZ, Netherlands|Novartis Investigative Site, Hilversum, XZ1213, Netherlands|Novartis Investigative Site, Hoofddorp, 2134 TM, Netherlands|Novartis Investigative Site, Leeuwarden, 8934 AD, Netherlands|Novartis Investigative Site, Roermond, 6043 CV, Netherlands|Novartis Investigative Site, Rotterdam, 3079 DZ, Netherlands|Novartis Investigative Site, Sittard-Geleen, 6162 BG, Netherlands|Novartis Investigative Site, Utrecht, 3543 AZ, Netherlands|Novartis Investigative Site, Zutphen, 7207 AE, Netherlands|Novartis Investigative Site, Zwolle, 8025 AB, Netherlands|Novartis Investigative Site, Gralum, 1714, Norway|Novartis Investigative Site, Oslo, 0379, Norway|Novartis Investigative Site, Oslo, NO 0450, Norway|Novartis Investigative Site, Stavanger, NO-4068, Norway|Novartis Investigative Site, Muscat, 123, Oman|Novartis Investigative Site, Panama City, 0801, Panama|Novartis Investigative Site, Taguig City, Metro Manila, 1634, Philippines|Novartis Investigative Site, Las Pinas, 1740, Philippines|Novartis Investigative Site, San Juan City, 1500, Philippines|Novartis Investigative Site, Zory, Slaskie, 44-240, Poland|Novartis Investigative Site, Bydgoszcz, 85 796, Poland|Novartis Investigative Site, Bytom, 41-902, Poland|Novartis Investigative Site, Gdansk, 80 952, Poland|Novartis Investigative Site, Krakow, 31-501, Poland|Novartis Investigative Site, Lublin, 20 090, Poland|Novartis Investigative Site, Opole, 45-061, Poland|Novartis Investigative Site, Rzeszow, 35-021, Poland|Novartis Investigative Site, Warszawa, 02 781, Poland|Novartis Investigative Site, Wroclaw, 53 413, Poland|Novartis Investigative Site, Braga, 4710243, Portugal|Novartis Investigative Site, Guimaraes, 4835-044, Portugal|Novartis Investigative Site, Lisboa, 1400-038, Portugal|Novartis Investigative Site, Lisboa, 1500 650, Portugal|Novartis Investigative Site, Lisboa, 1649-035, Portugal|Novartis Investigative Site, Lisboa, 1998-018, Portugal|Novartis Investigative Site, Porto, 4099-001, Portugal|Novartis Investigative Site, Porto, 4200-072, Portugal|Novartis Investigative Site, Porto, 4200-319, Portugal|Novartis Investigative Site, Leningrad Region, Russia, 188663, Russian Federation|Novartis Investigative Site, Kazan, Tatarstan Republic, 420029, Russian Federation|Novartis Investigative Site, Chelyabinsk, 454087, Russian Federation|Novartis Investigative Site, Irkutsk, 664035, Russian Federation|Novartis Investigative Site, Kaluga, 248007, Russian Federation|Novartis Investigative Site, Krasnoyarsk, 660022, Russian Federation|Novartis Investigative Site, Moscow Region Istra Village, 143423, Russian Federation|Novartis Investigative Site, Moscow Rerion Balashiha, 143900, Russian Federation|Novartis Investigative Site, Moscow, 115478, Russian Federation|Novartis Investigative Site, Moscow, 125284, Russian Federation|Novartis Investigative Site, Rostov-na-Donu, 344037, Russian Federation|Novartis Investigative Site, Samara, 443031, Russian Federation|Novartis Investigative Site, St Petersburg, 197022, Russian Federation|Novartis Investigative Site, St Petersburg, 197758, Russian Federation|Novartis Investigative Site, Ufa, 450054, Russian Federation|Novartis Investigative Site, Yaroslavl, 150054, Russian Federation|Novartis Investigative Site, Dammam, 15215, Saudi Arabia|Novartis Investigative Site, Makkah, 57657, Saudi Arabia|Novartis Investigative Site, Riyadh, 11211, Saudi Arabia|Novartis Investigative Site, Riyadh, 11426, Saudi Arabia|Novartis Investigative Site, Singapore, 217562, Singapore|Novartis Investigative Site, Singapore, 258500, Singapore|Novartis Investigative Site, Singapore, 308433, Singapore|Novartis Investigative Site, Banska Bystrica, Slovak Republic, 97401, Slovakia|Novartis Investigative Site, Kosice, 04191, Slovakia|Novartis Investigative Site, Poprad, 058 01, Slovakia|Novartis Investigative Site, Ljubljana, 1000, Slovenia|Novartis Investigative Site, Maribor, 2000, Slovenia|Novartis Investigative Site, Elche, Alicante, 03203, Spain|Novartis Investigative Site, Almeria, Andalucia, 04009, Spain|Novartis Investigative Site, Cordoba, Andalucia, 14004, Spain|Novartis Investigative Site, Granada, Andalucia, 18014, Spain|Novartis Investigative Site, Huelva, Andalucia, 21005, Spain|Novartis Investigative Site, Jaen, Andalucia, 23007, Spain|Novartis Investigative Site, Malaga, Andalucia, 29010, Spain|Novartis Investigative Site, Sevilla, Andalucia, 41009, Spain|Novartis Investigative Site, Sevilla, Andalucia, 41013, Spain|Novartis Investigative Site, Sevilla, Andalucia, 41014, Spain|Novartis Investigative Site, Sabadell, Barcelona, 08208, Spain|Novartis Investigative Site, Jerez, Cadiz, 11407, Spain|Novartis Investigative Site, Toledo, Castilla La Mancha, 45071, Spain|Novartis Investigative Site, Salamanca, Castilla Y Leon, 37007, Spain|Novartis Investigative Site, Barcelona, Cataluna, 08024, Spain|Novartis Investigative Site, Lerida, Cataluna, 25198, Spain|Novartis Investigative Site, Badalona, Catalunya, 08916, Spain|Novartis Investigative Site, Barcelona, Catalunya, 08035, Spain|Novartis Investigative Site, Barcelona, Catalunya, 08036, Spain|Novartis Investigative Site, Girona, Catalunya, 17007, Spain|Novartis Investigative Site, Hospitalet de LLobregat, Catalunya, 08907, Spain|Novartis Investigative Site, Alicante, Comunidad Valenciana, 03010, Spain|Novartis Investigative Site, Alicante, Comunidad Valenciana, 03550, Spain|Novartis Investigative Site, Castellon, Comunidad Valenciana, 12002, Spain|Novartis Investigative Site, Valencia, Comunidad Valenciana, 46009, Spain|Novartis Investigative Site, Valencia, Comunidad Valenciana, 46010, Spain|Novartis Investigative Site, Valencia, Comunidad Valenciana, 46014, Spain|Novartis Investigative Site, Valencia, Comunidad Valenciana, 46015, Spain|Novartis Investigative Site, Badajoz, Extremadura, 06080, Spain|Novartis Investigative Site, Caceres, Extremadura, 10003, Spain|Novartis Investigative Site, La Coruna, Galicia, 15006, Spain|Novartis Investigative Site, Santiago de Compostela, Galicia, 15706, Spain|Novartis Investigative Site, Palma De Mallorca, Islas Baleares, 07120, Spain|Novartis Investigative Site, Las Palmas De Gran Canarias, Las Palmas De Gran Canaria, 35016, Spain|Novartis Investigative Site, El Palmar, Murcia, 30120, Spain|Novartis Investigative Site, Pamplona, Navarra, 31008, Spain|Novartis Investigative Site, Bilbao, Pais Vasco, 48013, Spain|Novartis Investigative Site, San Sebastian, Pais Vasco, 20080, Spain|Novartis Investigative Site, Vigo, Pontevedra, 36212, Spain|Novartis Investigative Site, La Laguna, Santa Cruz De Tenerife, 38320, Spain|Novartis Investigative Site, Reus, Tarragona, 43201, Spain|Novartis Investigative Site, Barcelona, 08041, Spain|Novartis Investigative Site, Burgos, 09006, Spain|Novartis Investigative Site, Granollers, 08402, Spain|Novartis Investigative Site, Madrid, 28006, Spain|Novartis Investigative Site, Madrid, 28009, Spain|Novartis Investigative Site, Madrid, 28034, Spain|Novartis Investigative Site, Madrid, 28040, Spain|Novartis Investigative Site, Madrid, 28041, Spain|Novartis Investigative Site, Madrid, 28046, Spain|Novartis Investigative Site, Madrid, 28222, Spain|Novartis Investigative Site, Murcia, 30008, Spain|Novartis Investigative Site, Valencia, 46026, Spain|Novartis Investigative Site, Zaragoza, 50009, Spain|Novartis Investigative Site, Gothenburg, SE-431 45, Sweden|Novartis Investigative Site, Orebro, 701 85, Sweden|Novartis Investigative Site, Stockholm, SE-118 83, Sweden|Novartis Investigative Site, Uppsala, 751 85, Sweden|Novartis Investigative Site, Vaxjo, SE-351 85, Sweden|Novartis Investigative Site, Taichung, 40447, Taiwan|Novartis Investigative Site, Tainan, 70403, Taiwan|Novartis Investigative Site, Taipei, 114, Taiwan|Novartis Investigative Site, Songkhla, Hat Yai, 90110, Thailand|Novartis Investigative Site, Khon Kaen, THA, 40002, Thailand|Novartis Investigative Site, Bangkok, 10400, Thailand|Novartis Investigative Site, Chiang Mai, 50200, Thailand|Novartis Investigative Site, Peterborough, Cambridgeshire, PE3 9GZ, United Kingdom|Novartis Investigative Site, Truro, Cornwall, TR1 3LJ, United Kingdom|Novartis Investigative Site, Portsmouth, Hants, PO6 3LY, United Kingdom|Novartis Investigative Site, Maidstone, Kent, ME16 9QQ, United Kingdom|Novartis Investigative Site, York, North Yorkshire, YO31 8HE, United Kingdom|Novartis Investigative Site, Aberdeen, Scotland, AB25 2ZN, United Kingdom|Novartis Investigative Site, Ipswich, Suffolk, IP4 5PD, United Kingdom|Novartis Investigative Site, Guildford, Surrey, GU2 7XX, United Kingdom|Novartis Investigative Site, Sutton, Surrey, SM2 5PT, United Kingdom|Novartis Investigative Site, Bristol, BS2 8ED, United Kingdom|Novartis Investigative Site, Cardiff, CF14 2TL, United Kingdom|Novartis Investigative Site, East Sussex, BN2 5BE, United Kingdom|Novartis Investigative Site, Edinburgh, EH4 2XU, United Kingdom|Novartis Investigative Site, Exeter, EX2 5DW, United Kingdom|Novartis Investigative Site, Glasgow, G12 0YN, United Kingdom|Novartis Investigative Site, Leeds, LS9 7TF, United Kingdom|Novartis Investigative Site, Leicester, LE1 5WW, United Kingdom|Novartis Investigative Site, London, NW1 2PJ, United Kingdom|Novartis Investigative Site, London, SW3 6JJ, United Kingdom|Novartis Investigative Site, London, W8 6RF, United Kingdom|Novartis Investigative Site, Manchester, M20 4BX, United Kingdom|Novartis Investigative Site, Newcastle upon Tyne, NE7 7DN, United Kingdom|Novartis Investigative Site, Nottingham, NG5 1PB, United Kingdom|Novartis Investigative Site, Oxford, OX3 7LJ, United Kingdom|Novartis Investigative Site, Plymouth, PL6 8DH, United Kingdom|Novartis Investigative Site, Preston, PR2 9HT, United Kingdom|Novartis Investigative Site, Sheffield, S10 2SJ, United Kingdom|Novartis Investigative Site, Stoke-on-Trent, ST4 6QG, United Kingdom |
| A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors | This study will test how well a new medicine called concizumab works for participants who have haemophilia A or B with or without inhibitors. The purpose is to show that concizumab can prevent bleeds and is safe to use. Participants will have to inject the study medicine every day under the skin with a pen-injector. The study will last for at least 2 years and up to about 4 years. The length of time the participant will be in the study depends on if the study medicine will be available for purchase in their country. | Haemophilia A and B With and Without Inhibitors | ALL | CHILD, ADULT, OLDER_ADULT | Rady Childrens Hosp San Diego, San Diego, California, 92123, United States|Arnold Palmer Children's Hospital, Orlando, Florida, 32806, United States|Nemours Child Orlando Hem/Onc., Orlando, Florida, 32827, United States|Augusta Univ/Childrens Hosp-GA, Augusta, Georgia, 30912, United States|Memorial Health University Medical Center, Savannah, Georgia, 31404, United States|Childrens Hospital of Chicago, Chicago, Illinois, 60611, United States|Indiana Hemophilia-Thromb Ctr, Indianapolis, Indiana, 46260, United States|Children's Hosp-New Orleans, New Orleans, Louisiana, 70118, United States|The Children's Mercy Hospital, Kansas City, Missouri, 64108, United States|Children's Nebraska, Omaha, Nebraska, 68114, United States|ECU Sickle Cell Comp Clinic, Greenville, North Carolina, 27834, United States|Nationwide Children's Hospital, Columbus, Ohio, 43205, United States|St Christopher Hosp for Child, Philadelphia, Pennsylvania, 19134, United States|Vanderbilt Hemostasis Treatment Clinic, Nashville, Tennessee, 37212, United States|Cook Children's Hospital-Hematology-Oncology, Fort Worth, Texas, 76104, United States|Pediatrics Hematology/Oncology Clinic Battle Building, Charlottesville, Virginia, 22908, United States|Haematology and Blood Bank Department, Algiers, 16000, Algeria|CHU Constantine BEN BADIS/ Hematology department, Constantine, 25000, Algeria|University Clinical Center of Republic Srpska (545), Banja Luka, 78000, Bosnia and Herzegovina|University Clinical Centre Tuzla, Tuzla, 75000, Bosnia and Herzegovina|UMHAT "Sveti Georgi" Clinica of Pediatric, Plovdiv, 4002, Bulgaria|UMHAT "Tsaritsa Yoanna-ISUL", Sofia, 1527, Bulgaria|UMHAT "Sveta Marina" EAD, Varna, 9010, Bulgaria|BC Children's Hospital, Vancouver, British Columbia, V6H 3V4, Canada|McMaster Children's Hospital, Hamilton, Ontario, L8N 3Z5, Canada|Tallinn Children's Hospital, Tallinn, 13419, Estonia|Centre Hospitalier Metropole Savoie, Chambery, 73000, France|Ap-Hp-Hopital de Bicetre-1, Le Kremlin Bicetre Cedex, 94275, France|Ap-Hp-Hopital Necker-1, Paris, 75015, France|Aghia Sophia Childrens' Hospital, Athens, GR-11527, Greece|'Ippokrateio' General Hospital of Thessaloniki, Thessaloniki, GR 54642, Greece|'Ippokrateio' General Hospital of Thessaloniki, Thessaloniki, GR-54642, Greece|Guwahati Medical College, Guwahati, Assam, 781032, India|Nirmal Hospital Pvt. Ltd., Surat, Gujarat, 395002, India|Seth GS Medical College & KEM Hospital, Mumbai, Maharashtra, 400012, India|K.J Somaiya Hospital and Research Centre, Mumbai, Maharashtra, 400022, India|Sahyadri Super Speciality Hospital, Pune, Maharashtra, 411004, India|MCGM - Comprehensive Thalassemia Care, Mumbai, Maharastra, 400066, India|S.C.B. Medical College, Cuttack, Orissa, 753007, India|J K Lon Hospital, Jaipur, Rajasthan, 302004, India|Post Graduate Institute of Child Health, Noida, Uttar Pradesh, 201303, India|SGPGI, Lucknow, Uttart Pradesh, 226014, India|A.O.U policlinico "G. Rodolico-San Marco", Catania, 95123, Italy|Dipartimento di Ematologia Univ. Firenze, Firenze, 50134, Italy|Azienda Ospedaliera di Padova, Padova, 35128, Italy|Azienda Ospedaliera-Universitaria Parma, Parma, 43126, Italy|St. Marianna University School of Medicine Hospital_Pediatrics, Kanagawa, 216-8511, Japan|Saitama Children's Med Centre_Hematology-Oncology, Saitama, 330-8777, Japan|Saint George Hospital University Medical Center, Beirut, 961, Lebanon|Hospital Nini, Tripoli, 1434, Lebanon|Centre of Oncology and Hematology, Vilnius University, Vilnius, LT-08406, Lithuania|Hospital Tunku Azizah, Kampung Baru, Kuala Lumpur, 50300, Malaysia|Hospital Pulau Pinang, George Town, Penang, 10450, Malaysia|Sarawak General Hospital, Kuching, Sarawak, 93586, Malaysia|Hospital Sultanah Nur Zahirah, Kuala Terengganu, Terengganu, 20400, Malaysia|PHI University Clinic for Children's Diseases Skopje, Skopje, 1000, North Macedonia|Klinisk forskningspost, Oslo, 0372, Norway|Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego, Wroclaw, Dolnoslaskie, 50-556, Poland|Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego, Wroclaw, Dolnoslaskie, 50-556, Poland|Uniwersyteckie Centrum Kliniczne, Gdansk, 80-952, Poland|Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa, Lublin, 20-093, Poland|Uniwersyteckie Centrum Kliniczne WUM, Warszawa, 02-091, Poland|Clinic of Haematology, Fundeni Clinical Institute, Bucharest, 022328, Romania|Spitalul Clinic de Urgenta pentru Copii Cluj Napoca, Cluj-Napoca, 400177, Romania|Spitalul Clinic Judetean De Urgenta Bihor, Oradea, 410469, Romania|Spitalul Clinic Judetean De Urgenta Bihor, Oradea, 410469, Romania|Children Regional Clinical Hospital, Krasnodar, 350007, Russian Federation|Morozovskaya municipal children hospital, Moscow, 119049, Russian Federation|City out-patient clinic 37, City Hemophilia Centre, Saint-Petersburg, 191186, Russian Federation|Charlotte Maxeke Johannesburg Academic Hospital, Parktown, Johannesburg, Gauteng, 2193, South Africa|Hospital Virgen de la Arrixaca - Hematología, El Palmar, Murcia, 30120, Spain|Hospital Vall d'Hebron, Barcelona, 08035, Spain|Hospital Universitario La Paz, Madrid, 28046, Spain|Hospital Universitario Regional de Málaga, Málaga, 29009, Spain|Koagulationscentrum, Göteborg, 413 46, Sweden|Sunpasitthiprasong Hospital, Ubon Ratchathani, Mueang Distirct,, 34000, Thailand|King Chulalongkorn Memorial Hospital_Bangkok_0, Bangkok, 10330, Thailand|Ramathibodi Hospital, Bangkok, 10400, Thailand|Siriraj Hospital - Hematology and Oncology, Bangkok, 10700, Thailand|Gazi University, Ankara, Beşevler/Ankara, 06500, Turkey|Acibadem Adana Hastanesi, Adana, 01130, Turkey|Ege Universitesi Tip Fakultesi, Izmir, 35100, Turkey|Ondokuz Mayis University Medical Faculty Ped. Haematology, Samsun, 55139, Turkey|Birmingham Children's Hospital, Birmingham, B4 6NH, United Kingdom|University Hospitals Bristol & Weston NHS Foundation Trust, Bristol, BS2 8BJ, United Kingdom|Evelina London Children's Hospital, London, SE1 7EH, United Kingdom|Great Ormond Street Hospital for Children, London, WC1N 3HR, United Kingdom |
| AIM's Writing for Healing: A Workshop for Individuals Living With Paralysis | The UAB Institute for Arts In Medicine (AIM) is currently implementing an expressive emotional writing pilot project for adults with paralysis caused by neurological conditions such as traumatic head or spinal cord injury. | Spinal Cord Injuries|Multiple Sclerosis|Transverse Myelitis|Amyotrophic Lateral Sclerosis | ALL | ADULT, OLDER_ADULT | 354 School of Health Professions Building, Birmingham, Alabama, 35294, United States |
| Trial to Describe the Safety, Tolerability, and Immunogenicity of Trumenba When Administered to Immunocompromised Participants ≥10 Years of Age | The aim of this study is to evaluate the safety, tolerability, and immunogenicity of 2 doses of Trumenba® (on a 0- and 6-month schedule) in immunocompromised participants by functionally assessing antibody production in asplenic and complement-deficient individuals ≥10 years of age. | Meningococcal Vaccine | ALL | CHILD, ADULT, OLDER_ADULT | Fakultni Nemocnice Brno - Detska Nemocnice - Klinika Detskych Infekcnich Nemoci Center 3, Brno 2, 61300, Czechia|Fakultni Nemocnice Brno - Detska Nemocnice - Klinika Detskych Infekcnich Nemoci Center 3, Brno, 613 00, Czechia|Fakultni nemocnice v Motole, Praha 5, 150 06, Czechia|IN-VIVO Sp. z o.o., Bydgoszcz, 85-048, Poland|Centrum Badań Klinicznych Jagiellońskie Centrum Innowacji sp. z o.o., Krakow, 30-348, Poland|WIP Warsaw IBD Point Profesor Kierkus, Warszawa, 00-728, Poland|Szpital Bielański im. Ks. Jerzego Popiełuszki SPZOZ, Warszawa, 01-809, Poland|Baskent Universitesi Dr. Turgut Noyan Adana Uygulama ve Arastirma Merkezi, Adana, Yüreği̇r, 01120, Turkey|Baskent Universitesi Dr. Turgut Noyan Adana Uygulama ve Arastirma Merkezi, Adana, Yüreği̇r, Turkey|Baskent Universitesi Dr. Turgut Noyan Adana Uygulama ve Arastirma Merkezi, Adana, 01120, Turkey|Acibadem Adana Hastanesi, Adana, 01130, Turkey|Baskent Universitesi Dr. Turgut Noyan Adana Uygulama ve Arastirma Merkezi, Adana, 01240, Turkey|Hacettepe Universitesi Tip Fakultesi, Ankara, 06230, Turkey|Mersin Universitesi Tip Fakultesi Hastanesi, Mersin, 33343, Turkey |
| The Impact of Expressive Emotional Writing on Facilitating Grief Resolution in Adults With Spinal Cord Injury | The aim of this study is to evaluate the therapeutic benefits of a 10-week online coach-guided EEWP on psychosocial health among adults with SCI. | Spinal Cord Injuries|Transverse Myelitis | ALL | ADULT, OLDER_ADULT | University of Alabama at Birmingham, Birmingham, Alabama, 35294, United States |
| Real World Evidence of the Effectiveness of Paritaprevir/r - Ombitasvir, ± Dasabuvir, ± Ribavirin in Patients With Chronic Hepatitis C in Colombia | This is a prospective, multi-center observational study in adult participants chronically infected with hepatitis C virus (HCV) receiving the interferon-free ABBVIE REGIMEN (ombitasvir/paritaprevir/ritonavir with or without dasabuvir) with or without ribavirin (RBV). The prescription of a treatment regimen was at the discretion of the physician in accordance with local clinical practice and label. This study focused on collecting real world data. Follow-up visits, treatment, procedures and diagnostic methods followed physicians' routine clinical practice using a 12-week treatment regimen (four visits plus two interim data collection windows) or a 24-week treatment regimen (four visits plus three interim data collection windows) and is based on the anticipated regular follow-up for patients undergoing treatment for chronic hepatitis C (CHC). Participants are observed for the duration of the ABBVIE REGIMEN therapy and for up to 24 weeks after treatment completion. | Chronic Hepatitis C | ALL | ADULT, OLDER_ADULT | Fundacion Cardioinfantil, Bogotá, Colombia|Cic Cali, Cali, 760001, Colombia|Centro Medico lmbanaco de Cali I, Cali, Colombia|Pharos Centro de Estudios Clin, Cartagena, 130013, Colombia|IPS Medicos Internistas Del Ca I, Manizales, 170004, Colombia|Fundacion Hospitalaria San Vin, Medellín, 050010, Colombia |