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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

Sarepta Therapeutics, Inc. · with Hoffmann-La Roche · NCT04626674

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 2+ · Men only
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 1
Target enrollment
83
US sites recruiting
11
Study start
November 23, 2020
Record last updated
September 11, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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.

Being studied: delandistrogene moxeparvovec

Conditions: Duchenne Muscular Dystrophy

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full 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.

Recruiting US locations

  • Arkansas Children's Hospital

    Little Rock, AR 72202

    Site contact: 501-364-1868 · KurakuVV@archildrens.org

  • University of California, San Diego

    La Jolla, CA 92037

    Site contact: Gabriella Penner · gpenner@health.ucsd.edu

  • University of California, Los Angeles

    Los Angeles, CA 90095

    Site contact: SareptAlly@Sarepta.com

  • Stanford University

    Palo Alto, CA 94304

    Site contact: neuromuscularresearch@stanford.edu

  • University of California, Davis

    Sacramento, CA 95616

    Site contact: Craig McDonald, MD

  • University of Florida

    Gainesville, FL 32610

    Site contact: ssalabarria@ufl.edu

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

    Chicago, IL 60611

    Site contact: SareptAlly@Sarepta.com

  • Washington University in St. Louis

    St Louis, MO 63110

    Site contact: 314-362-6981 · NeuromusclePediatricResearch@wustl.edu

  • Duke University Medical Center

    Durham, NC 27705

    Site contact: SareptAlly@Sarepta.com

  • Neurology Rare Disease Center

    Flower Mound, TX 75028

    Site contact: 972-999-1011 · Research@neuromdcenter.com

  • Children's Hospital of The King's Daughters

    Norfolk, VA 23507

    Site contact: Proud.Research@chkd.org

Study contacts (from the official record)

  • Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4 · 1-888-SAREPTA (1-888-727-3782) · SareptAlly@Sarepta.com
All phase 1 studies in Arkansas →
A Gene Transfer Therapy Study to Evaluate the Safety of and… (NCT04626674) | Bored Studies