RECRUITING Phase 1 ClinicalTrials.gov

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

Sponsor: Sarepta Therapeutics, Inc. • ClinicalTrials.gov ID: NCT04626674

Registry updated: 2026-09-11

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Description

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.

Interventions

delandistrogene moxeparvovec (Genetic)
Single IV infusion of delandistrogene moxeparvovec

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 ≥2 at the Screening visit and has a total PUL score of ≥12 and ≤40 at the time of Screening. * Cohorts 1, 2, 3, 5, 7 and 8 only: Stable dose equivalent of oral glucocorticoids for at least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the first year of the study. * Cohort 1: Is ambulatory, and ≥4 to \<8 years of age at the time of Screening. * Cohort 2: Is ambulatory, and ≥8 to \<18 years of age at the time of Screening. * Cohort 3: Non-ambulatory per protocol specified criteria at the time of Screening. * Cohort 4: Is ambulatory and ≥3 to \<4 years of age at the time of Screening. * Cohort 5a: Is ambulatory and ≥4 to \<9 years of age with time to rise from the floor ≤7 seconds at the screening visit. * Cohort 5b: Non-ambulatory per protocol specified criteria at the time of Screening. * Cohort 6: Is ambulatory, and ≥2 to \<3 years of age at the time of Screening. * Cohort 7: Non-ambulatory per protocol-specified criteria at the time of Screening. * Cohorts 4 and 6: Do not yet require use of chronic steroids for treatment of their DMD, in the opinion of the Investigator, and are not receiving steroids at the time of Screening. * Genetic mutation inclusion criteria vary by cohort. All Cohorts: * Ability to cooperate with motor assessment testing. * rAAVrh74 antibody titers are not elevated as per protocol-specified requirements. Exclusion Criteria: * Cohort 8: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients. * Has a concomitant illness, autoimmune disease, chronic drug treatment, and/or cognitive delay/impairment that in the opinion of the Investigator creates unnecessary risks for gene transfer. * Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol-specified time limits. * Abnormality in protocol-specified diagnostic evaluations or laboratory tests. Note: Other inclusion/exclusion criteria apply.

Conditions

Duchenne Muscular Dystrophy

Locations

Arkansas Children's Hospital — Little Rock, Arkansas, United States
University of California, San Diego — La Jolla, California, United States
University of California, Los Angeles — Los Angeles, California, United States
Stanford University — Palo Alto, California, United States
University of California, Davis — Sacramento, California, United States
University of Florida — Gainesville, Florida, United States
Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
Washington University in St. Louis — St Louis, Missouri, United States
Duke University Medical Center — Durham, North Carolina, United States
Nationwide Children's Hospital — Columbus, Ohio, United States
Neurology Rare Disease Center — Flower Mound, Texas, United States
Children's Hospital of The King's Daughters — Norfolk, Virginia, United States

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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

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