An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy

Last updated: April 30, 2025
Sponsor: Sarepta Therapeutics, Inc.
Overall Status: Active - Recruiting

Phase

N/A

Condition

Muscular Dystrophy

Treatment

delandistrogene moxeparvovec

Delandistrogene Moxeparvovec

Standard of Care

Clinical Study ID

NCT06270719
SRP-9001-401
  • Ages > 4
  • Male

Study Summary

This is a multicenter, prospective, observational Phase 4 study in the United States. The study is designed to collect both medical history and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at baseline in routine clinical practice.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Has a definitive diagnosis of DMD prior to Screening based on documentation ofclinical findings and confirmatory genetic testing.

  • Is currently receiving or has been prescribed to start chronic glucocorticoidtherapy at the time of this observational study enrollment.

A participant recruited to Cohorts 1a or 2:

  • Is at least 4 years of age at the time of enrollment.

  • Is ambulatory per protocol specified criteria.

A participant recruited to Cohort 1b:

  • Is non-ambulatory per protocol-specified criteria.

For Delandistrogene Moxeparvovec-treated Participants:

  • Will be initiating usual care treatment with delandistrogene moxeparvovec at thetime of study enrollment.

For Comparators:

  • Is unexposed to DMD gene therapy at the time of study enrollment.

Exclusion

Exclusion Criteria:

  • Has any deletion of exon 8 and/or exon 9 in the DMD gene.

  • Is currently participating in any DMD interventional study at the time of thisobservational study enrollment.

  • Has a medical condition or confounding circumstances (for example, prior traumaticlimitation for mobility or significant behavioral comorbidity) that, in the opinionof the Investigator, might compromise:

  • The participant's ability to comply with the protocol-required procedures,

  • The participant's wellbeing or safety, and/or

  • The clinical interpretability of the data collected from the participant.

Other inclusion/exclusion criteria may apply.

Study Design

Total Participants: 500
Treatment Group(s): 3
Primary Treatment: delandistrogene moxeparvovec
Phase:
Study Start date:
February 07, 2024
Estimated Completion Date:
December 31, 2038

Connect with a study center

  • Arkansas Children's Hospital

    Little Rock, Arkansas 72202
    United States

    Active - Recruiting

  • University of Colorado - PPDS

    Aurora, Colorado 80045
    United States

    Active - Recruiting

  • Children's National Medical Center

    Washington, District of Columbia 20010
    United States

    Active - Recruiting

  • Nicklaus Children's Hospital

    Miami, Florida 33155
    United States

    Active - Recruiting

  • Nemours Children's Hospital - Orlando

    Orlando, Florida 32827
    United States

    Active - Recruiting

  • Ann and Robert H Lurie Childrens Hospital of Chicago

    Chicago, Illinois 60611-2991
    United States

    Active - Recruiting

  • Indiana Clinical and Translational Science Institute

    Indianapolis, Indiana 46202
    United States

    Active - Recruiting

  • University of Kansas Medical Center

    Kansas City, Kansas 66160
    United States

    Active - Recruiting

  • University of Michigan

    Ann Arbor, Michigan 48109
    United States

    Active - Recruiting

  • Penn State Health Milton S. Hershey Medical Center

    Hershey, Pennsylvania 17033
    United States

    Active - Recruiting

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • Vanderbilt University Medical Center

    Nashville, Tennessee 37232
    United States

    Active - Recruiting

  • Cook Children's Hospital

    Fort Worth, Texas 76104
    United States

    Active - Recruiting

  • Children's Hospital of the King's Daughters

    Norfolk, Virginia 23507
    United States

    Active - Recruiting

  • The Medical College of Wisconsin

    Milwaukee, Wisconsin 53226-4874
    United States

    Active - Recruiting

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