An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy (ENDURE)
Study Overview
- Age
- 4 years and older
- Phase
- Observational
- Sponsor
- Sarepta Therapeutics
- Therapeutic Approach
- Gene Therapy
- Variant Requirement
- Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.
- Eligible Sex
- Male
- Ambulation
- Ambulatory and Non-Ambulatory
- Study Start (Actual)
- 2024-02-07
- Primary Completion (Estimated)
- 2029-12-31
- Study Completion (Estimated)
- 2038-12-31
- Enrollment (Estimated)
- 500
- Countries
- United States
Study Requirements and Criteria
Steroid Use
Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment.
Inclusion Criteria
- 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
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 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.
Contacts and Locations
Contacts and Locations
Locations
This study has 25 locations
United States
Arkansas Locations
Little Rock, Arkansas, United States, 72202
Arkansas Children's Hospital
California Locations
Los Angeles, California, United States, 90027
Children's Hospital Los Angeles - PIN
Colorado Locations
Aurora, Colorado, United States, 80045
University of Colorado - PPDS
Connecticut Locations
Hartford, Connecticut, United States, 06106
Connecticut Children's Medical Center - Hartford
District of Columbia Locations
Washington D.C., District of Columbia, United States, 20010
Children's National Medical Center
Florida Locations
Miami, Florida, United States, 33155
Nicklaus Children's Hospital
Orlando, Florida, United States, 32827
Nemours Children's Hospital - Orlando
St. Petersburg, Florida, United States, 33701
All Children's Research Institute, Inc
Illinois Locations
Chicago, Illinois, United States, 60611-2991
Ann and Robert H Lurie Childrens Hospital of Chicago
Indiana Locations
Indianapolis, Indiana, United States, 46202
Indiana Clinical and Translational Science Institute
Kansas Locations
Kansas City, Kansas, United States, 66160
University of Kansas Medical Center
Michigan Locations
Ann Arbor, Michigan, United States, 48109
University of Michigan
North Carolina Locations
Durham, North Carolina, United States, 27704
Duke Lenox Baker Children's Hospital
Ohio Locations
Akron, Ohio, United States, 44302
Childrens Hospital Medical Center of Akron
Oregon Locations
Portland, Oregon, United States, 97239
OHSU Healthcare (Oregon Health and Science University)
Pennsylvania Locations
Hershey, Pennsylvania, United States, 17033
Penn State Health Milton S. Hershey Medical Center
Philadelphia, Pennsylvania, United States, 19104
Children's Hospital of Philadelphia
Tennessee Locations
Memphis, Tennessee, United States, 38103
Le Bonheur Children's Hospital
Nashville, Tennessee, United States, 37232
Vanderbilt University Medical Center
Texas Locations
Fort Worth, Texas, United States, 76104
Cook Children's Hospital
Virginia Locations
Charlottesville, Virginia, United States, 22903
University of Virginia
Norfolk, Virginia, United States, 23507
Children's Hospital of the King's Daughters
Washington Locations
Seattle, Washington, United States, 98105
Seattle Children's Hospital
Wisconsin Locations
Madison, Wisconsin, United States, 53715-1218
The Board of Regents of the University of Wisconsin
Milwaukee, Wisconsin, United States, 53226-4874
The Medical College of Wisconsin
Clinical Trial Registry
NCT ID
NCT06270719This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.