Clinical trials

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Search and review clinical trials. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort

Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed. This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular Dystrophy (DMD). The maximum participant duration for this study is 156 weeks.

Participants needed: 83
Trial details
Phase: Phase 1Age: 2+Biological sex: MaleType: InterventionalSponsor: Sarepta Therapeutics, Inc.Updated: Jun 24, 2026Locations: 7
Eligibility criteria

For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical... [+14]

Cohort 8: Any confounding factors that would prevent the use of oral sirolimus i... [+3]

Status: Recruiting

A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease

This is a first-in-human, multi-center trial studying the effects of SRP-1005 in participants with Huntington's disease (HD).

Participants needed: 32
Trial details
Phase: Phase 1Age: 21-70Biological sex: AllType: InterventionalSponsor: Sarepta Therapeutics, Inc.Updated: Jun 4, 2026Locations: 2
Eligibility criteria

Genetically confirmed diagnosis of huntingtin (HTT) cytosine-adenine-guanine rep... [+2]

Any condition that would compromise the safety or feasibility of lumbar puncture... [+5]

Status: Not yet recruiting

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

The primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.

Participants needed: 20
Trial details
Phase: Phase 4Age: 4+Biological sex: MaleType: InterventionalSponsor: Sarepta Therapeutics, Inc.Updated: May 26, 2026
Eligibility criteria

Cohort 1 only: Is male at birth, ambulatory, and ≥ 4 years of age at the time of... [+5]

Cohort 1 only: Contraindicated to receive ELEVIDYS per the United States Package... [+8]