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NCT06270719

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

Enrolling by Invitation
Not specifiedAges 4+Observational
Sarepta Therapeutics, Inc.
~500 participants
Updated 2026-05-04 on ClinicalTrials.gov
What's tested:Delandistrogene MoxeparvovecStandard of Care

At a glance

Recruiting sites
0 of 25 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Mean Change From Baseline in Time to Walk/Run 10 Meters (10MWR) (Calculated Velocity) at Month 12
Measured over Baseline, Month 12
+1 more outcome measured

Conditions

Duchenne Muscular Dystrophy

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Duchenne Muscular Dystrophy

NCT06270719

Where you'd take part

This study runs at 25 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • All Children's Research Institute, Inc

    St. Petersburg, Floridano site contact published

  • Ann and Robert H Lurie Childrens Hospital of Chicago

    Chicago, Illinoisno site contact published

  • Arkansas Children's Hospital

    Little Rock, Arkansasno site contact published

  • Children's Hospital Los Angeles - PIN

    Los Angeles, Californiano site contact published

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvaniano site contact published

  • Children's Hospital of the King's Daughters

    Norfolk, Virginiano site contact published

  • Children's National Medical Center

    Washington D.C., District of Columbiano site contact published

  • Childrens Hospital Medical Center of Akron

    Akron, Ohiono site contact published

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

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

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Eligibility criteria

Inclusion

Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.
Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment.
Is at least 4 years of age at the time of infusion
Will either: a) be initiating or has initiated ELEVIDYS within the last 30 days in routine clinical practice at the time of this observational study enrollment, or b) was administered ELEVIDYS in routine clinical practice and has the required minimum dataset for entry into the observational study per Sponsor approval
Is at least 4 years of age at the time of enrollment
Is unexposed to DMD gene therapy at the time of this observational study enrollment

Exclusion

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 this observational study enrollment.
Has any prior exposure to DMD gene therapy other than that described for Cohort 1c (ELEVIDYS Retrospectively Treated Cohort).
Has a medical condition or confounding circumstances (for example, prior traumatic limitation for mobility or significant behavioral comorbidity) that, in the opinion of 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.

What this trial measures

  • Mean Change From Baseline in Time to Walk/Run 10 Meters (10MWR) (Calculated Velocity) at Month 12Baseline, Month 12
  • Number of Participants Experiencing Acute Liver Injury (ALI)Baseline through Month 12