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

Last updated: November 28, 2025
Sponsor: Sarepta Therapeutics, Inc.
Overall Status: Active - Enrolling

Phase

N/A

Condition

Muscular Dystrophy

Treatment

Standard of Care

delandistrogene moxeparvovec

Delandistrogene Moxeparvovec

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 the time 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: Standard of Care
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

    Site Not Available

  • Arkansas Children's Hospital

    Little Rock 4119403, Arkansas 4099753 72202
    United States

    Site Not Available

  • Children's Hospital Los Angeles - PIN

    Los Angeles 5368361, California 5332921 90027
    United States

    Site Not Available

  • University of Colorado - PPDS

    Aurora, Colorado 80045
    United States

    Site Not Available

  • University of Colorado - PPDS

    Aurora 5412347, Colorado 5417618 80045
    United States

    Site Not Available

  • Connecticut Children's Medical Center - Hartford

    Hartford 4835797, Connecticut 4831725 06106
    United States

    Site Not Available

  • Children's National Medical Center

    Washington, District of Columbia 20010
    United States

    Site Not Available

  • Children's National Medical Center

    Washington D.C. 4140963, District of Columbia 4138106 20010
    United States

    Site Not Available

  • Nicklaus Children's Hospital

    Miami, Florida 33155
    United States

    Site Not Available

  • Nemours Children's Hospital - Orlando

    Orlando, Florida 32827
    United States

    Site Not Available

  • All Children's Research Institute, Inc

    Saint Petersburg, Florida 33701
    United States

    Site Not Available

  • Nicklaus Children's Hospital

    Miami 4164138, Florida 4155751 33155
    United States

    Site Not Available

  • Nemours Children's Hospital - Orlando

    Orlando 4167147, Florida 4155751 32827
    United States

    Site Not Available

  • All Children's Research Institute, Inc

    St. Petersburg 4171563, Florida 4155751 33701
    United States

    Site Not Available

  • Ann and Robert H Lurie Childrens Hospital of Chicago

    Chicago, Illinois 60611-2991
    United States

    Site Not Available

  • Ann and Robert H Lurie Childrens Hospital of Chicago

    Chicago 4887398, Illinois 4896861 60611-2991
    United States

    Site Not Available

  • Indiana Clinical and Translational Science Institute

    Indianapolis, Indiana 46202
    United States

    Site Not Available

  • Indiana Clinical and Translational Science Institute

    Indianapolis 4259418, Indiana 4921868 46202
    United States

    Site Not Available

  • University of Kansas Medical Center

    Kansas City, Kansas 66160
    United States

    Site Not Available

  • University of Kansas Medical Center

    Kansas City 4273837, Kansas 4273857 66160
    United States

    Site Not Available

  • University of Michigan

    Ann Arbor, Michigan 48109
    United States

    Site Not Available

  • University of Michigan

    Ann Arbor 4984247, Michigan 5001836 48109
    United States

    Site Not Available

  • Duke Lenox Baker Children's Hospital

    Durham 4464368, North Carolina 4482348 27704
    United States

    Site Not Available

  • Childrens Hospital Medical Center of Akron

    Akron 5145476, Ohio 5165418 44302
    United States

    Site Not Available

  • OHSU Healthcare (Oregon Health and Science University)

    Portland, Oregon 97239
    United States

    Site Not Available

  • OHSU Healthcare (Oregon Health and Science University)

    Portland 5746545, Oregon 5744337 97239
    United States

    Site Not Available

  • Penn State Health Milton S. Hershey Medical Center

    Hershey, Pennsylvania 17033
    United States

    Site Not Available

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • Penn State Health Milton S. Hershey Medical Center

    Hershey 5193342, Pennsylvania 6254927 17033
    United States

    Site Not Available

  • Children's Hospital of Philadelphia

    Philadelphia 4560349, Pennsylvania 6254927 19104
    United States

    Site Not Available

  • Le Bonheur Children's Hospital

    Memphis, Tennessee 38103
    United States

    Site Not Available

  • Vanderbilt University Medical Center

    Nashville, Tennessee 37232
    United States

    Site Not Available

  • Le Bonheur Children's Hospital

    Memphis 4641239, Tennessee 4662168 38103
    United States

    Site Not Available

  • Vanderbilt University Medical Center

    Nashville 4644585, Tennessee 4662168 37232
    United States

    Site Not Available

  • Cook Children's Hospital

    Fort Worth, Texas 76104
    United States

    Site Not Available

  • Cook Children's Hospital

    Fort Worth 4691930, Texas 4736286 76104
    United States

    Site Not Available

  • Children's Hospital of the King's Daughters

    Norfolk, Virginia 23507
    United States

    Site Not Available

  • University of Virginia

    Charlottesville 4752031, Virginia 6254928 22903
    United States

    Site Not Available

  • Children's Hospital of the King's Daughters

    Norfolk 4776222, Virginia 6254928 23507
    United States

    Site Not Available

  • Seattle Children's Hospital

    Seattle 5809844, Washington 5815135 98105
    United States

    Site Not Available

  • The Board of Regents of the University of Wisconsin

    Madison, Wisconsin 53715-1218
    United States

    Site Not Available

  • The Medical College of Wisconsin

    Milwaukee, Wisconsin 53226-4874
    United States

    Site Not Available

  • The Board of Regents of the University of Wisconsin

    Madison 5261457, Wisconsin 5279468 53715-1218
    United States

    Site Not Available

  • The Medical College of Wisconsin

    Milwaukee 5263045, Wisconsin 5279468 53226-4874
    United States

    Site Not Available

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