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

Last updated: April 3, 2026
Sponsor: Sarepta Therapeutics, Inc.
Overall Status: Active - Recruiting

Phase

1

Condition

N/A

Treatment

delandistrogene moxeparvovec

Clinical Study ID

NCT04626674
SRP-9001-103
  • Ages > 2
  • Male

Study Summary

This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with DMD. The maximum participant duration for this study is 156 weeks.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinicalfindings and prior genetic testing.

  • 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.

  • 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 visit andhas a total PUL score of ≥20 and ≤40 at the time of Screening.

  • Ability to cooperate with motor assessment testing.

  • Cohorts 1, 2, 3, 5, 7 and 8 only: Stable dose equivalent of oral glucocorticoids forat least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the firstyear of the study.

  • Cohorts 4 and 6: Do not yet require use of chronic steroids for treatment of theirDMD, in the opinion of the Investigator, and are not receiving steroids at the timeof Screening.

  • rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.

  • Genetic mutation inclusion criteria vary by cohort.

Exclusion

Exclusion Criteria:

  • Has a concomitant illness, autoimmune disease, chronic drug treatment, and/orcognitive delay/impairment that in the opinion of the Investigator createsunnecessary risks for gene transfer.

  • Exposure to gene therapy, investigational medication, or any treatment designed toincrease dystrophin expression within protocol-specified time limits.

  • Abnormality in protocol-specified diagnostic evaluations or laboratory tests.

  • Cohort 8: Any confounding factors that would prevent the use of oral sirolimusincluding a known hypersensitivity to sirolimus or any of its excipients.

Other inclusion/exclusion criteria apply.

Study Design

Total Participants: 83
Treatment Group(s): 1
Primary Treatment: delandistrogene moxeparvovec
Phase: 1
Study Start date:
November 23, 2020
Estimated Completion Date:
February 29, 2028

Study Description

Enrollment for Cohorts 1 through 7 has been completed. Cohort 8 is currently enrolling new participants.

Connect with a study center

  • Arkansas Children's Hospital

    Little Rock, Arkansas 72202
    United States

    Active - Recruiting

  • Stanford University

    Palo Alto, California 94304
    United States

    Active - Recruiting

  • University of California, Davis

    Sacramento, California 95616
    United States

    Active - Recruiting

  • Stanford University

    Palo Alto 5380748, California 5332921 94304
    United States

    Site Not Available

  • University of California, Davis

    Sacramento 5389489, California 5332921 95616
    United States

    Site Not Available

  • Washington University in St. Louis

    Saint Louis, Missouri 21205
    United States

    Site Not Available

  • Washington University in St. Louis

    St Louis, Missouri 63110
    United States

    Active - Recruiting

  • Washington University in St. Louis

    St Louis 4407066, Missouri 4398678 21205
    United States

    Site Not Available

  • Nationwide Children's Hospital

    Columbus, Ohio 43205
    United States

    Site Not Available

  • Nationwide Children's Hospital

    Columbus 4509177, Ohio 5165418 43205
    United States

    Site Not Available

  • Neurology Rare Disease Center

    Flower Mound, Texas 75028
    United States

    Active - Recruiting

  • Children's Hospital of The King's Daughters

    Norfolk, Virginia 23507
    United States

    Active - Recruiting

  • Children's Hospital of The King's Daughters

    Norfolk 4776222, Virginia 6254928 23507
    United States

    Site Not Available

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