Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 50/100

Failure Rate

11.1%

1 terminated/withdrawn out of 9 trials

Success Rate

66.7%

-19.8% vs industry average

Late-Stage Pipeline

0 trials in Phase 3/4

Results Transparency

50%

1 of 2 completed trials have results

Key Signals

3 recruiting1 with results

Enrollment Performance

Analytics

Phase 1

3(60.0%)

Phase 2

1(20.0%)

N/A

1(20.0%)

5Total

Phase 1(3)

Phase 2(1)

N/A(1)

Activity Timeline

Global Presence

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Clinical Trials (9)

Showing 9 of 9 trials

NCT07052929Phase 1Recruiting

Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators

Role: lead

NCT03199469Phase 2Active Not Recruiting

A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)

Role: lead

NCT06150820Not ApplicableActive Not Recruiting

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

Role: lead

NCT04174105Phase 1Active Not Recruiting

Gene Transfer Study in Patients With Late Onset Pompe Disease

Role: lead

NCT06581146Recruiting

A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition

Role: lead

NCT06483802Phase 1Withdrawn

A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia

Role: lead

NCT04064307Recruiting

Myotubular and Centronuclear Myopathy Patient Registry

Role: collaborator

NCT02231697Completed

A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM)

Role: lead

NCT02704273Completed

A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects

Role: lead

All 9 trials loaded