NCT06270719
An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy
Enrolling by Invitation
Not specifiedAges 4+ObservationalSarepta 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
Where it's being run
25 sites across 19 statesFlorida3
Pennsylvania2
Tennessee2
Virginia2
Wisconsin2
Arkansas1
California1
Colorado1
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
Do you actually qualify for this trial?
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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