Beta-Thalassemia
84
13
17
35
Key Insights
Highlights
Success Rate
90% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 40/100
4.8%
4 terminated out of 84 trials
89.7%
+3.2% vs benchmark
15%
13 trials in Phase 3/4
26%
9 of 35 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 35 completed trials
Clinical Trials (84)
ALS20-101 Lentiviral Gene Therapy for Beta 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
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia
CS-101 in Patients With β-thalassemia
A Follow-up Study to Evaluate the Long-term Safety and Efficacy in Participants Who Received CS-101 Injection
A Long-term Follow-up Study in Participants Who Received CS-101
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia
A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
A Long-term Follow-up Study in Participants Who Received CS-101
A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Long Term Beta Thalassemia Treatment: Findings From The Extension Period
Early Check: Expanded Screening in Newborns
ERN-EuroBloodNet Registry on Patients With Rare Red Blood Cell Defects and COVID-19