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A Phase 3, Double-Blind, Randomized Trial Evaluating the Efficacy and Safety of Elritercept (TAK-226) Compared to Placebo in Participants With Myelofibrosis and Anemia on Concurrent Ruxolitinib Therapy
The main aim of this study is to find out how well elritercept works to improve anemia in participants with myelofibrosis (MF) who are taking ruxolitinib when …
A Phase 1 Study to Evaluate the Safety and Tolerability of Escalating Doses of VAS-101 in Subjects With Stable 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. Research has shown t…
Supplemental Choline to Prevent and Treat Learning and Memory Deficits of Early Iron Deficiency: the SupCHO Study
BACKGROUND: Iron deficiency limits the neurodevelopmental potential of more than 200 million children each year. Iron therapy is typically started when iron d…
RALLY-IBD: A Phase 2 Randomized, Double-Blind Study to Evaluate the Safety, Tolerability, and Efficacy of DISC-0974 in Participants With Inflammatory Bowel Disease and Anemia of Inflammation
This is a Phase 2, multicenter, randomized, double-blind placebo-controlled study of DISC-0974 to evaluate safety, tolerability, and efficacy in participants w…
A Phase 1b/2a, Open-label Study of Velinotamig for the Treatment of Adults With Relapsing and Refractory Immune Thrombocytopenia and Warm Autoimmune Hemolytic Anemia
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…
A Multicenter Phase 2 Study of Luspatercept in Patients With Anemia Due to Chronic Kidney Disease
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…
Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
Background: Fanconi anemia (FA) is an inherited disorder. People with FA are more likely to get certain cancers, especially squamous cell carcinoma (SCC). The…
A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Dibotatug in Adults With Bone Marrow Failure Syndromes
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.
A Phase 3, Multicenter, Open-Label, Randomized Trial to Compare the Efficacy and Safety of Elritercept Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes in ESA-naïve Adult Participants Who Require Red Blood Cell Transfusions
The main aim of this study is to assess how elritercept works in lowering the need for RBC (red blood cell) transfusions and how safe elritercept is when compa…
A Phase 1/2 Dose Evaluation Trial of the Safety and Preliminary Efficacy of Anti CD19 Allogeneic CRISPR-Cas9-Engineered T Cells (CTX112) in Adult Participants With Relapsed/Refractory Hematologic Autoimmune Disease
This is a single-arm, open-label, multicenter, ascending dose Phase 1/2 trial evaluating the safety and preliminary efficacy of CTX112 or Zugocabtagene geleuce…
Dissecting the Role of Acetaldehyde in Oral Carcinogenesis
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…
A Phase 1, Open-label, Single-arm Study of APR 2020 in Transfusion Dependent, Steroid Resistant Pediatric and Adolescent Subjects With RPS19 Deficient Diamond Blackfan Anemia by Transplantation of Autologous CD34+ Stem Cells Transduced With CLIN LV EFS coRPS19 PRE* (APR-2020)
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…