An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy
NCT ID: NCT06270719
Last Updated: 2025-12-02
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ENROLLING_BY_INVITATION
500 participants
OBSERVATIONAL
2024-02-07
2038-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Cohort 1: Delandistrogene Moxeparvovec
Participants in Cohort 1 will have a plan for prescription of delandistrogene moxeparvovec commercially as part of clinical care prior to entry into this study.
Cohort 1a includes participants who are ambulatory and aged 4 years or greater at baseline prescribed delandistrogene moxeparvovec commercially and recruited by treating physicians before infusion is administered.
Cohort 1b includes non-ambulatory DMD participants aged 4 years or greater at baseline prescribed delandistrogene moxeparvovec commercially and recruited by treating physicians before infusion is administered.
Delandistrogene Moxeparvovec
No study medication will be provided by the sponsor during this study.
Cohort 2: Standard of Care
Cohort 2 includes ambulatory DMD participants aged 4 years or greater at baseline unexposed to DMD gene therapy and receiving standard of care therapy (chronic glucocorticoid treatment) at study entry with or without baseline use of other DMD approved therapies.
Standard of Care
No study medication will be provided by the sponsor during this study.
Interventions
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Delandistrogene Moxeparvovec
No study medication will be provided by the sponsor during this study.
Standard of Care
No study medication will be provided by the sponsor during this study.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Is currently receiving or has been prescribed to start chronic glucocorticoid therapy 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 Criteria
* Is currently participating in any DMD interventional study at the time of this observational study enrollment.
* 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.
4 Years
MALE
No
Sponsors
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Sarepta Therapeutics, Inc.
INDUSTRY
Responsible Party
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Locations
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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
Countries
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Other Identifiers
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SRP-9001-401
Identifier Type: -
Identifier Source: org_study_id
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