Key Insights

Highlights

Success Rate

93% trial completion (above average)

Clinical Risk Assessment

Based on trial outcomes

Moderate Risk

Score: 40/100

Termination Rate

4.0%

2 terminated out of 50 trials

Success Rate

93.3%

+6.8% vs benchmark

Late-Stage Pipeline

18%

9 trials in Phase 3/4

Results Transparency

11%

3 of 28 completed with results

Key Signals

3 with results93% success

Data Visualizations

Phase Distribution

35Total
Not Applicable (12)
P 1 (5)
P 2 (9)
P 3 (1)
P 4 (8)

Trial Status

Completed28
Unknown11
Recruiting5
Active Not Recruiting3
Terminated2
Suspended1

Trial Success Rate

93.3%

Benchmark: 86.5%

Based on 28 completed trials

Clinical Trials (50)

Showing 20 of 20 trials
NCT04523376Not ApplicableCompleted

Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies

NCT06931912Not ApplicableRecruiting

Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia

NCT07112703Not ApplicableRecruiting

Effect of Incentive Respiratory Training on Pulmonary Functions and Functional Capacity in Children With B-thalassemia Major

NCT06490627Phase 2Recruiting

Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia

NCT06913634Active Not RecruitingPrimary

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

NCT05444894Phase 1Active Not Recruiting

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

NCT06875219Active Not RecruitingPrimary

To Evaluate Long- Term Safety and Efficacy of Luspatercept

NCT02307786CompletedPrimary

Long Term Outcomes in β Thalassemia Major

NCT02986698Phase 1Terminated

In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)

NCT04009525Phase 4CompletedPrimary

Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study

NCT05442346Not ApplicableSuspendedPrimary

Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells

NCT00749515Phase 4Completed

Pilot Study for Patients With Poor Response to Deferasirox

NCT04090268Not ApplicableRecruiting

Precision Exercise in Children With Malignant Hemopathies

NCT06137079Unknown

"Iron Overload and Endocrinological Diseases"

NCT06026839UnknownPrimary

Longitudinal Study on the QoL of Pediatric Patients After HSCT and Its Influencing Factors

NCT06041620Not ApplicableRecruiting

Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells

NCT04291352Not ApplicableUnknownPrimary

Thalassemic Iron Overload Cardiomyopathy is Ameliorated by Taurine Supplementation

NCT05777733Phase 1UnknownPrimary

NAC Effect on Iron Overload and Blood Transfusion in β-thalassemia Major

NCT01496963CompletedPrimary

Prevalence of Pulmonary Hypertension (PAH) in Patients With Thalassemia

NCT05462548Phase 4UnknownPrimary

The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study

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