103 recruiting studies matching your filters
The purpose of this study is to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of BM…
The overall objective of this study is to evaluate the effectiveness and safety of transfusing hypoxic red blood cells manufactured with the Hemanext ONE syste…
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…
BACKGROUND: Iron deficiency limits the neurodevelopmental potential of more than 200 million children each year. Iron therapy is typically started when iron d…
A Phase 1b/2a, open-label dose escalation and dose expansion study of subcutaneously (SC) administered velinotamig for the treatment of adults with relapsing a…
The researchers are doing this study to test whether luspatercept works to improve hemoglobin levels in people with anemia caused by chronic kidney disease (CK…
This is a multicenter, open-label, Phase 1/2 basket study to evaluate the safety and efficacy of Dibotatug (DR-01) in adults with Bone Marrow Failure syndromes.
This is a minimal risk intervention study where healthy volunteers and individuals with Fanconi anemia will consume a single dose of alcohol and provide primar…
Brief summary The goal of this clinical trial is to learn if APR-2020 is safe and can help treat Diamond-Blackfan Anemia (DBA) in adolescents and children. Th…
A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell disease and osteonecrosis
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 materia…
The purpose of this study is to evaluate the safety and efficacy of Zola-cel (BMS-986353), in participants with chronic immune thrombocytopenia (cITP) and auto…