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

Enrolling by invitation

Conditions studied: Duchenne Muscular Dystrophy

In brief

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

Key facts

Study ID

NCT06270719

Run by

Sarepta Therapeutics, Inc.

People needed

500

Starts

2024-02-07

Expected to finish

2038-12-31

Last updated by the study team

2026-05-04

Who can join

Age: 4 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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.
• For ELEVIDYS-treated Participants (Cohorts 1a, 1b, and 1c):
• 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
• For Standard of Care Comparators (Cohort 2):
• 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

You may not qualify if…

• 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.
• Other inclusion/exclusion criteria may apply.

Where it is running

• Arkansas Children's Hospital — Little Rock, Arkansas, United States
• Children's Hospital Los Angeles - PIN — Los Angeles, California, United States
• University of Colorado - PPDS — Aurora, Colorado, United States
• Connecticut Children's Medical Center - Hartford — Hartford, Connecticut, United States
• Children's National Medical Center — Washington D.C., District of Columbia, United States
• Nicklaus Children's Hospital — Miami, Florida, United States
• Nemours Children's Hospital - Orlando — Orlando, Florida, United States
• All Children's Research Institute, Inc — St. Petersburg, Florida, United States
• Ann and Robert H Lurie Childrens Hospital of Chicago — Chicago, Illinois, United States
• Indiana Clinical and Translational Science Institute — Indianapolis, Indiana, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• University of Michigan — Ann Arbor, Michigan, United States
• Duke Lenox Baker Children's Hospital — Durham, North Carolina, United States
• Childrens Hospital Medical Center of Akron — Akron, Ohio, United States
• OHSU Healthcare (Oregon Health and Science University) — Portland, Oregon, United States
• Penn State Health Milton S. Hershey Medical Center — Hershey, Pennsylvania, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Le Bonheur Children's Hospital — Memphis, Tennessee, United States
• Vanderbilt University Medical Center — Nashville, Tennessee, United States
• Cook Children's Hospital — Fort Worth, Texas, United States
• University of Virginia — Charlottesville, Virginia, United States
• Children's Hospital of the King's Daughters — Norfolk, Virginia, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• The Board of Regents of the University of Wisconsin — Madison, Wisconsin, United States
• The Medical College of Wisconsin — Milwaukee, Wisconsin, United States

Full record on ClinicalTrials.gov

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