Key Insights

Highlights

Success Rate

90% trial completion (above average)

Clinical Risk Assessment

Based on trial outcomes

Moderate Risk

Score: 40/100

Termination Rate

4.8%

4 terminated out of 84 trials

Success Rate

89.7%

+3.2% vs benchmark

Late-Stage Pipeline

15%

13 trials in Phase 3/4

Results Transparency

26%

9 of 35 completed with results

Key Signals

9 with results90% success

Data Visualizations

Phase Distribution

64Total
Not Applicable (12)
Early P 1 (3)
P 1 (11)
P 2 (25)
P 3 (9)
P 4 (4)

Trial Status

Completed35
Unknown16
Recruiting13
Enrolling By Invitation6
Active Not Recruiting4
Terminated4

Trial Success Rate

89.7%

Benchmark: 86.5%

Based on 35 completed trials

Clinical Trials (84)

Showing 20 of 20 trials
NCT06364774Phase 1RecruitingPrimary

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

NCT04208529Phase 3Enrolling By InvitationPrimary

A Long-term Follow-up Study in Participants Who Received CTX001

NCT05477563Phase 3RecruitingPrimary

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

NCT05356195Phase 3Active Not RecruitingPrimary

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

NCT06024876Early Phase 1CompletedPrimary

A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia

NCT06328764Early Phase 1Enrolling By InvitationPrimary

CS-101 in Patients With β-thalassemia

NCT06717932Enrolling By InvitationPrimary

A Follow-up Study to Evaluate the Long-term Safety and Efficacy in Participants Who Received CS-101 Injection

NCT06685536Enrolling By InvitationPrimary

A Long-term Follow-up Study in Participants Who Received CS-101

NCT06647979Phase 1Recruiting

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

NCT05577312Phase 1Enrolling By InvitationPrimary

Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia

NCT04143724Phase 2RecruitingPrimary

Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia

NCT03655678Phase 2CompletedPrimary

A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

NCT06308159Phase 1RecruitingPrimary

An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia

NCT04064060Phase 3Recruiting

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

NCT06479616Not ApplicableRecruitingPrimary

A Long-term Follow-up Study in Participants Who Received CS-101

NCT05567458Phase 2Active Not RecruitingPrimary

A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.

NCT04099966Phase 2Recruiting

AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

NCT06490601Phase 2Active Not Recruiting

Long Term Beta Thalassemia Treatment: Findings From The Extension Period

NCT03655223Enrolling By Invitation

Early Check: Expanded Screening in Newborns

NCT06831799Completed

ERN-EuroBloodNet Registry on Patients With Rare Red Blood Cell Defects and COVID-19

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