Thalassemia Major
50
5
8
28
Key Insights
Highlights
Success Rate
93% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 40/100
4.0%
2 terminated out of 50 trials
93.3%
+6.8% vs benchmark
18%
9 trials in Phase 3/4
11%
3 of 28 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 28 completed trials
Clinical Trials (50)
Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies
Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia
Effect of Incentive Respiratory Training on Pulmonary Functions and Functional Capacity in Children With B-thalassemia Major
Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia
Study to Evaluate the Long- Term Safety and Efficacy of Luspatercept in Subjects Who Received at Least One Dose of Luspatercept in the Compassionate Use Phase
EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)
To Evaluate Long- Term Safety and Efficacy of Luspatercept
Long Term Outcomes in β Thalassemia Major
In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells
Pilot Study for Patients With Poor Response to Deferasirox
Precision Exercise in Children With Malignant Hemopathies
"Iron Overload and Endocrinological Diseases"
Longitudinal Study on the QoL of Pediatric Patients After HSCT and Its Influencing Factors
Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells
Thalassemic Iron Overload Cardiomyopathy is Ameliorated by Taurine Supplementation
NAC Effect on Iron Overload and Blood Transfusion in β-thalassemia Major
Prevalence of Pulmonary Hypertension (PAH) in Patients With Thalassemia
The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study