Phase
Condition
Muscular Dystrophy
Treatment
Standard of Care
delandistrogene moxeparvovec
Delandistrogene Moxeparvovec
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 the time 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 StatesSite Not Available
Arkansas Children's Hospital
Little Rock 4119403, Arkansas 4099753 72202
United StatesSite Not Available
Children's Hospital Los Angeles - PIN
Los Angeles 5368361, California 5332921 90027
United StatesSite Not Available
University of Colorado - PPDS
Aurora, Colorado 80045
United StatesSite Not Available
University of Colorado - PPDS
Aurora 5412347, Colorado 5417618 80045
United StatesSite Not Available
Connecticut Children's Medical Center - Hartford
Hartford 4835797, Connecticut 4831725 06106
United StatesSite Not Available
Children's National Medical Center
Washington, District of Columbia 20010
United StatesSite Not Available
Children's National Medical Center
Washington D.C. 4140963, District of Columbia 4138106 20010
United StatesSite Not Available
Nicklaus Children's Hospital
Miami, Florida 33155
United StatesSite Not Available
Nemours Children's Hospital - Orlando
Orlando, Florida 32827
United StatesSite Not Available
All Children's Research Institute, Inc
Saint Petersburg, Florida 33701
United StatesSite Not Available
Nicklaus Children's Hospital
Miami 4164138, Florida 4155751 33155
United StatesSite Not Available
Nemours Children's Hospital - Orlando
Orlando 4167147, Florida 4155751 32827
United StatesSite Not Available
All Children's Research Institute, Inc
St. Petersburg 4171563, Florida 4155751 33701
United StatesSite Not Available
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois 60611-2991
United StatesSite Not Available
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago 4887398, Illinois 4896861 60611-2991
United StatesSite Not Available
Indiana Clinical and Translational Science Institute
Indianapolis, Indiana 46202
United StatesSite Not Available
Indiana Clinical and Translational Science Institute
Indianapolis 4259418, Indiana 4921868 46202
United StatesSite Not Available
University of Kansas Medical Center
Kansas City, Kansas 66160
United StatesSite Not Available
University of Kansas Medical Center
Kansas City 4273837, Kansas 4273857 66160
United StatesSite Not Available
University of Michigan
Ann Arbor, Michigan 48109
United StatesSite Not Available
University of Michigan
Ann Arbor 4984247, Michigan 5001836 48109
United StatesSite Not Available
Duke Lenox Baker Children's Hospital
Durham 4464368, North Carolina 4482348 27704
United StatesSite Not Available
Childrens Hospital Medical Center of Akron
Akron 5145476, Ohio 5165418 44302
United StatesSite Not Available
OHSU Healthcare (Oregon Health and Science University)
Portland, Oregon 97239
United StatesSite Not Available
OHSU Healthcare (Oregon Health and Science University)
Portland 5746545, Oregon 5744337 97239
United StatesSite Not Available
Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania 17033
United StatesSite Not Available
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United StatesSite Not Available
Penn State Health Milton S. Hershey Medical Center
Hershey 5193342, Pennsylvania 6254927 17033
United StatesSite Not Available
Children's Hospital of Philadelphia
Philadelphia 4560349, Pennsylvania 6254927 19104
United StatesSite Not Available
Le Bonheur Children's Hospital
Memphis, Tennessee 38103
United StatesSite Not Available
Vanderbilt University Medical Center
Nashville, Tennessee 37232
United StatesSite Not Available
Le Bonheur Children's Hospital
Memphis 4641239, Tennessee 4662168 38103
United StatesSite Not Available
Vanderbilt University Medical Center
Nashville 4644585, Tennessee 4662168 37232
United StatesSite Not Available
Cook Children's Hospital
Fort Worth, Texas 76104
United StatesSite Not Available
Cook Children's Hospital
Fort Worth 4691930, Texas 4736286 76104
United StatesSite Not Available
Children's Hospital of the King's Daughters
Norfolk, Virginia 23507
United StatesSite Not Available
University of Virginia
Charlottesville 4752031, Virginia 6254928 22903
United StatesSite Not Available
Children's Hospital of the King's Daughters
Norfolk 4776222, Virginia 6254928 23507
United StatesSite Not Available
Seattle Children's Hospital
Seattle 5809844, Washington 5815135 98105
United StatesSite Not Available
The Board of Regents of the University of Wisconsin
Madison, Wisconsin 53715-1218
United StatesSite Not Available
The Medical College of Wisconsin
Milwaukee, Wisconsin 53226-4874
United StatesSite Not Available
The Board of Regents of the University of Wisconsin
Madison 5261457, Wisconsin 5279468 53715-1218
United StatesSite Not Available
The Medical College of Wisconsin
Milwaukee 5263045, Wisconsin 5279468 53226-4874
United StatesSite Not Available

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