Thalassemia
135
9
16
84
Key Insights
Highlights
Success Rate
86% trial completion
Published Results
28 trials with published results (21%)
Research Maturity
84 completed trials (62% of total)
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 62/100
10.4%
14 terminated out of 135 trials
85.7%
-0.8% vs benchmark
12%
16 trials in Phase 3/4
33%
28 of 84 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 84 completed trials
Clinical Trials (135)
Low-Dose Liposomal Amphotericin B for Invasive Fungal Infection Prophylaxis in Neutropenic Children
Effect of Exercise on Body Composition and Bone Health in Patients With Thalassemia
Dexamethasone, Intravenous Injection of Human Immunoglobulin, and Increased Infusion of Mononuclear Cells to Reduce Donor Specific Antibodies in Haploidentical Hematopoietic Stem Cell Transplantation
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
CINC424A2X01B Rollover Protocol
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
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)
A Study of SNH-119014 in Adult Participants With Non-transfusion-dependent Thalassemia (NTDT)
A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia
A Long-term Follow-up Study in Participants Who Received CTX001
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders
A Blood Stem Cell Transplant for Sickle Cell Disease
Atrial Fibrillation in Beta-Thalassemia
Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling
Multicenter Observational Study on Myocardial Iron Overload in 3 Multitransfused Populations
Evaluation of the Quality of Life in Patients With Chronic Iron Overload Due to Hemoglobinopathies in Greece.
A Study on the Effect of Etavopivat on Heart Rhythm in Healthy Participants