Thalassemia
132
9
15
84
Key Insights
Highlights
Success Rate
86% trial completion
Published Results
28 trials with published results (21%)
Research Maturity
84 completed trials (64% of total)
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 62/100
10.6%
14 terminated out of 132 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 (132)
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)
CINC424A2X01B Rollover Protocol
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
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
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.
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
A Study on the Effect of Etavopivat on Heart Rhythm in Healthy Participants
A Research Study Looking at a Single Dose of Etavopivat in Healthy Chinese Participants
A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease
Effect of L-Glutamine on Pulmonary Artery Pressure in Patients With Non-Transfusion-Dependent Thalassemia