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

Recruiting Phase 1 Interventional Study
Duchenne Muscular Dystrophy
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
2 and older
Sex
Male
Trial phase
Phase 1
Study type
Interventional
Purpose
Treatment
Participants needed
83 (estimated)
Sponsor
Sarepta Therapeutics, Inc. · Industry
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About This Trial

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.

Trial Locations
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Eligibility Criteria
Inclusion Criteria: * For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing. * Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening, has a performance upper limb (PUL) entry item score ≥3 at the Screening visi…
Contacts

Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4

1-888-SAREPTA (1-888-727-3782)

SareptAlly@Sarepta.com

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