Phase
Condition
Muscular Dystrophy
Treatment
delandistrogene moxeparvovec
Delandistrogene Moxeparvovec
Standard of Care
Clinical Study ID
Ages > 4 Male
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Has a definitive diagnosis of DMD prior to Screening based on documentation ofclinical findings and confirmatory genetic testing.
Is currently receiving or has been prescribed to start chronic glucocorticoidtherapy 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 thetime of study enrollment.
For Comparators:
- Is unexposed to DMD gene therapy at the time of study enrollment.
Exclusion
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 thisobservational study enrollment.
Has a medical condition or confounding circumstances (for example, prior traumaticlimitation for mobility or significant behavioral comorbidity) that, in the opinionof 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.
Study Design
Connect with a study center
Arkansas Children's Hospital
Little Rock, Arkansas 72202
United StatesActive - Recruiting
University of Colorado - PPDS
Aurora, Colorado 80045
United StatesActive - Recruiting
Children's National Medical Center
Washington, District of Columbia 20010
United StatesActive - Recruiting
Nicklaus Children's Hospital
Miami, Florida 33155
United StatesActive - Recruiting
Nemours Children's Hospital - Orlando
Orlando, Florida 32827
United StatesActive - Recruiting
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois 60611-2991
United StatesActive - Recruiting
Indiana Clinical and Translational Science Institute
Indianapolis, Indiana 46202
United StatesActive - Recruiting
University of Kansas Medical Center
Kansas City, Kansas 66160
United StatesActive - Recruiting
University of Michigan
Ann Arbor, Michigan 48109
United StatesActive - Recruiting
Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania 17033
United StatesActive - Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United StatesActive - Recruiting
Vanderbilt University Medical Center
Nashville, Tennessee 37232
United StatesActive - Recruiting
Cook Children's Hospital
Fort Worth, Texas 76104
United StatesActive - Recruiting
Children's Hospital of the King's Daughters
Norfolk, Virginia 23507
United StatesActive - Recruiting
The Medical College of Wisconsin
Milwaukee, Wisconsin 53226-4874
United StatesActive - Recruiting
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