Clinical trials: Sickle Cell Disorders
Hemoglobinopathy, Sickle Cell Disease
A Long-term Follow-up Study in Patients Who Received BEAM-101
This is a Long-term Follow-up (LTFU) study in patients who received BEAM-101 in the parent study (Study BTX-AUT-001). Eligible patients who received BEAM-101 will be asked to participate in this LTFU study prior to completing…
- Trial name
- NCT07373639
- Testing
- Long-Term Follow-up Study of patients who received BEAM-101
- Where
- 17 sites in United States
- Volunteers
- 50 people
- Sponsor
- Beam Therapeutics Inc.
Anemia, Sickle Cell
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.
- Trial name
- NCT03069430
- Testing
- SELFY (Self Management for Youth with SCD)
- Where
- United States
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- Medical University of South Carolina
Iron Overload, Excessive Body Iron Burden
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.
- Trial name
- NCT01572922
- Testing
- R2*-UTE, R2*-GRE, Liver biopsy
- Where
- United States
- Volunteers
- 142 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease, Vaso-occlusive Crisis
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.
- Trial name
- NCT01891812
- Testing
- Nitrous oxide 50%
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Columbia University
Blood Disease, Hematopoietic Stem Cell Transplantation
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.
- Conditions
- Blood Disease, Hematopoietic Stem Cell Transplantation, Anemia, Sickle Cell
- Trial name
- NCT00029393
- Testing
- Hematopoietic Stem Cell Transplantation, Immunosuppression
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Chest Pain, Sickle Cell Anemia
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.
- Trial name
- NCT00001716
- Testing
- Nitric Oxide, Nitroglycerin
- Where
- United States
- Volunteers
- 58 people, healthy volunteers welcome
- Sponsor
- National Institutes of Health Clinical Center (CC)
Pyruvate Kinase Deficiency, Anemia, Hemolytic
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study
The primary objective of the study is to provide continued access to participants who are deriving clinical benefit from ongoing mitapivat treatment in an Agios-sponsored mitapivat study, in the opinion of the Investigator.
- Conditions
- Pyruvate Kinase Deficiency, Anemia, Hemolytic, Sickle Cell Disease, Thalassemia
- Trial name
- NCT05777993
- Testing
- Mitapivat
- Where
- 23 sites in Belgium, Brazil and 6 more
- Volunteers
- 55 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Sickle Cell, Sickle Cell Disease
Impact of Regional Anesthesia on Inflammatory Mechanisms During Vaso-occlusive Crisis in Sickle Cell Patients
Sickle cell disease is a chronic disease characterized by multiple vaso-occlusive complications. The basic treatment for patients with a vaso-occlusive crisis (VOC) is based on adequate hydration, oxygen therapy and pain control.
- Conditions
- Sickle Cell, Sickle Cell Disease, Vaso-Occlusive Crises
- Trial name
- ALRCVO-MP (NCT07001189)
- Where
- Guadeloupe
- Volunteers
- 34 people
- Sponsor
- Centre Hospitalier Universitaire de la Guadeloupe
Chuvash Polycythemia, Cerebrovascular Disease
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.
- Conditions
- Chuvash Polycythemia, Cerebrovascular Disease, Pulmonary Hypertension, Sickle Cell Anemia
- Trial name
- NCT00495638
- Where
- 7 sites in United States, Russia
- Volunteers
- 29 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Pain
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.
- Trial name
- NCT01393847
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- National Institute of Nursing Research (NINR)
Sickle Cell Disease, Sickle Cell Anemia
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.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Sickle Cell Disorders, Hemoglobin S Disease
- Trial name
- NCT01601340
- Testing
- HQK-1001
- Where
- 18 sites in United States, Canada and 3 more
- Volunteers
- 77 people
- Sponsor
- HemaQuest Pharmaceuticals Inc.
Sickle Cell Disease
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.
- Trial name
- NCT01848925
- Testing
- SANGUINATE™, Hydroxyurea
- Where
- 4 sites in Colombia, Panama
- Volunteers
- 24 people
- Sponsor
- Prolong Pharmaceuticals
Anemia, Sickle Cell
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…
- Trial name
- NCT00131508
- Testing
- Glutamine
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- St. Jude Children's Research Hospital
Transfusional Iron Overload, Thalassemia Major
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.
- Conditions
- Transfusional Iron Overload, Thalassemia Major, Sickle Cell Disease, Myelodysplastic Syndromes
- Trial name
- NCT01241357
- Testing
- Hepatic biomagnetic susceptibility measurement
- Where
- United States
- Volunteers
- 99 people, healthy volunteers welcome
- Sponsor
- Columbia University
Nephropathy, Sickle Cell Anemia
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.
- Trial name
- NCT01479439
- Testing
- Losartan
- Where
- 9 sites in United States
- Volunteers
- 36 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Osteonecrosis
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…
- Trial name
- NCT02458937
- Testing
- Functional Mobility Assessment, GAITRite® System, Range of Motion
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
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.
- Trial name
- TRANSFORM (NCT02757885)
- Testing
- Bone Marrow Transplant (BMT), Hydroxyurea, Thiotepa
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Emory University
Sickle Cell Disease, Pregnancy Related
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…
- Trial name
- SCOB-II (NCT05143021)
- Testing
- Multidisciplinary Sickle Cell Disease- Obstetric Team
- Where
- 3 sites in Ghana
- Volunteers
- 500 people
- Sponsor
- Vanderbilt University Medical Center
Sickle Cell Disease
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…
- Trial name
- NCT05789355
- Testing
- NUV001
- Where
- France
- Volunteers
- 12 people
- Sponsor
- LGD
Sickle Cell Disease
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…
- Trial name
- NCT05228821
- Testing
- Voxelotor Oral Tablet
- Where
- 2 sites in United States
- Sponsor
- Pfizer