Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 45/100

Failure Rate

4.2%

1 terminated/withdrawn out of 24 trials

Success Rate

95.0%

+8.5% vs industry average

Late-Stage Pipeline

8%

2 trials in Phase 3/4

Results Transparency

37%

7 of 19 completed trials have results

Key Signals

7 with results

Enrollment Performance

Analytics

Phase 2
10(71.4%)
Phase 3
2(14.3%)
Phase 1
2(14.3%)
14Total
Phase 2(10)
Phase 3(2)
Phase 1(2)

Activity Timeline

Global Presence

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Clinical Trials (24)

Showing 20 of 24 trials
NCT02369731Completed

Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Role: collaborator

NCT03439670Phase 2Completed

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

Role: collaborator

NCT03167255Phase 2Completed

Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

Role: collaborator

NCT02740972Phase 2Completed

Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

Role: collaborator

NCT03038399Phase 2Completed

Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

Role: collaborator

NCT02760277Phase 2Completed

An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

Role: collaborator

NCT02760264Phase 2Completed

A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

Role: collaborator

NCT01126697Phase 2Completed

Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

Role: lead

NCT01539772Unknown

Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping

Role: lead

NCT01437345Completed

A Multicenter Collaborative Study on the Clinical Features, Expression Profiling, and Quality of Life of Infantile Onset FSHD

Role: lead

NCT00468832Unknown

Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD)

Role: lead

NCT02146586Unknown

Clinical Evaluator Outcomes Reliability Study

Role: lead

NCT01772043Unknown

Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping

Role: lead

NCT00033813Phase 2Completed

KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy

Role: lead

NCT00893334Completed

Evaluation of Limb-Girdle Muscular Dystrophy

Role: lead

NCT00308113Phase 3Terminated

CoQ10 and Prednisone in Non-Ambulatory DMD

Role: lead

NCT01081080Completed

Cardiac Magnetic Resonance in Children With Muscular Dystrophy

Role: lead

NCT01066455Completed

Cardiac Outcome Measures in Children With Muscular Dystrophy

Role: lead

NCT01125709Completed

Comparative Study of Clinical Endpoint in DMD: Handheld Myometry (HHM) Versus CINRG Quantitative Measurement System (CQMS)

Role: lead

NCT00110669Phase 3Completed

High-dose Prednisone in Duchenne Muscular Dystrophy

Role: lead