Transfusion-dependent Beta-Thalassemia
12
5
8
1
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 15/100
0.0%
0 terminated out of 12 trials
100.0%
+13.4% vs benchmark
25%
3 trials in Phase 3/4
100%
1 of 1 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 1 completed trials
Clinical Trials (12)
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia
Use of the Hemanext One® Hypoxic Red Blood Cell Storage System for Transfusion in Thalassemia Patients
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients
Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
Β-Thalassemia Treatment with KL003 Cell Injection
Plasticizer Exposure and Its Consequences on Health
Deciphering Effects of Thalidomide on Red Blood Cells in Transfusion Dependents Beta Thalassemia Patients
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy