Key Insights

Highlights

Success Rate

79% trial completion

Published Results

66 trials with published results (22%)

Research Maturity

143 completed trials (48% of total)

Clinical Risk Assessment

Based on trial outcomes

Moderate Risk

Score: 49/100

Termination Rate

12.9%

38 terminated out of 295 trials

Success Rate

79.0%

-7.5% vs benchmark

Late-Stage Pipeline

13%

37 trials in Phase 3/4

Results Transparency

46%

66 of 143 completed with results

Key Signals

66 with results79% success38 terminated

Data Visualizations

Phase Distribution

204Total
Not Applicable (46)
Early P 1 (2)
P 1 (58)
P 2 (61)
P 3 (28)
P 4 (9)

Trial Status

Completed143
Terminated38
Unknown38
Recruiting33
Active Not Recruiting20
Enrolling By Invitation12

Trial Success Rate

79.0%

Benchmark: 86.5%

Based on 143 completed trials

Clinical Trials (295)

Showing 20 of 20 trials
NCT07287189Phase 2RecruitingPrimary

Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

NCT06270719Enrolling By InvitationPrimary

An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy

NCT06817382Phase 1RecruitingPrimary

A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)

NCT06053814Phase 1Active Not RecruitingPrimary

NS-050/NCNP-03 in Boys With DMD (Meteor50)

NCT06713135Phase 4Active Not RecruitingPrimary

A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy

NCT03689660Not ApplicableCompleted

Feasibility of Virtual Reality in Children With Neuromuscular Disease, Effectiveness of Virtual Reality and Biofeedback

NCT07542314Phase 4Not Yet RecruitingPrimary

Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting

NCT06274983Not ApplicableCompletedPrimary

DMD- Interactive Virtual Reality Study

NCT06450639Phase 2Active Not RecruitingPrimary

A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)

NCT06280209Phase 1RecruitingPrimary

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

NCT06491927Enrolling By InvitationPrimary

Long Term Follow-up for RGX-202

NCT06114056Phase 1Active Not RecruitingPrimary

A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of JWK007 in Patients With Duchenne Muscular Dystrophy (DMD)

NCT05712447CompletedPrimary

Duchenne Muscular Dystrophy Video Assessment Registry

NCT01247207Phase 3CompletedPrimary

Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

NCT05996003Phase 2RecruitingPrimary

NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

NCT07209332Phase 2Enrolling By InvitationPrimary

Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy

NCT06692426Phase 1RecruitingPrimary

Trial of Cell Based Therapy for DMD

NCT06138639Phase 1RecruitingPrimary

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

NCT03368742Phase 1Active Not RecruitingPrimary

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

NCT07160634Phase 3RecruitingPrimary

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

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