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Gene Transfer Therapy for Non-Ambulatory Duchenne Muscular Dystrophy

This study is testing a gene therapy called delandistrogene moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD). Researchers want to see how safe it is and how well the body produces the dystrophin protein after a single IV (intravenous) infusion. They will measure the amount of dystrophin protein at 12 weeks. The study is also looking for any acute liver injury. You may be able to join if you have a confirmed diagnosis of DMD, are non-ambulatory (meaning you can't walk independently), and meet specific arm function scores. The study is currently enrolling new participants for the non-ambulatory group (Cohort 8).

Study design
This is an open-label study, meaning both you and the study team will know which treatment you receive. It plans to enroll 83 participants.
What's involved
You would receive a single IV infusion of delandistrogene moxeparvovec. The maximum time you would participate in the study is 156 weeks.
Compensation
Not stated in the trial record.
Follow-up
Researchers will measure dystrophin expression at 12 weeks and monitor for acute liver injury up to 72 weeks.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04626674

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
PHASE1Ages 2+InterventionalTreatment
Sarepta Therapeutics, Inc.
~83 participants
Updated 2026-09-11 on ClinicalTrials.gov
What's tested:delandistrogene moxeparvovec

At a glance

Recruiting sites
11 of 12 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Part 1 (Cohorts 1 to 5): Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot
Measured over Baseline, Week 12
+2 more outcomes measured

Conditions

Duchenne Muscular Dystrophy

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Where you'd take part

This study runs at 12 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinoisstudy coordinator listed

    Recruiting

  • Arkansas Children's Hospital

    Little Rock, Arkansasstudy coordinator listed

    Recruiting

  • Children's Hospital of The King's Daughters

    Norfolk, Virginiastudy coordinator listed

    Recruiting

  • Duke University Medical Center

    Durham, North Carolinastudy coordinator listed

    Recruiting

  • Neurology Rare Disease Center

    Flower Mound, Texasstudy coordinator listed

    Recruiting

  • Stanford University

    Palo Alto, Californiastudy coordinator listed

    Recruiting

  • University of California, Los Angeles

    Los Angeles, Californiastudy coordinator listed

    Recruiting

  • University of California, San Diego

    La Jolla, Californiastudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Medical Director · STUDY_DIRECTOR · Sarepta Therapeutics, Inc.

Who to contact

Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4
[email protected]
1-888-SAREPTA (1-888-727-3782)
Email the study team

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Eligibility criteria

Inclusion

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.
Ability to cooperate with motor assessment testing.
rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.

Exclusion

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.

What this trial measures

  • Part 1 (Cohorts 1 to 5): Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western BlotBaseline, Week 12
  • Part 1 (Cohorts 6 to 8): Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western BlotWeek 12
  • Cohort 8: Number of Participants with Acute Liver Injury (ALI)Baseline up to Week 72