An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Genetic: Delandistrogene MoxeparvovecDrug: Standard of Care
- Registration Number
- NCT06270719
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Brief Summary
This is a multicenter, prospective, observational Phase 4 study in the United States. The study is designed to collect both medical history and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at baseline in routine clinical practice.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- Male
- Target Recruitment
- 500
- Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and confirmatory genetic testing.
- 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.
-
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 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.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Cohort 1: Delandistrogene Moxeparvovec 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. Cohort 2: Standard of Care 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.
- Primary Outcome Measures
Name Time Method Mean Change From Baseline in Time to Walk/Run 10 Meters (10MWR) (Calculated Velocity) at Month 12 Baseline, Month 12
- Secondary Outcome Measures
Name Time Method Time to Rise From Floor (Supine to Stand) Up to 10 years Loss of Ambulation (LOA) Up to 10 years Performance of Upper Limb (PUL) Version 2.0 Entry Item A Score Up to 10 years Patient-reported Outcomes Measurement Information (PROMIS) Domain Scores of Mobility, Upper Extremity and Fatigue Up to 10 years Pulmonary Function, as Measured by Forced Vital Capacity (FVC) (% Predicted) Up to 10 years Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO) Up to 10 years Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) Up to 10 years Time to Walk/Run 10 Meters (Calculated Velocity) Up to 10 years
Trial Locations
- Locations (15)
Ann and Robert H Lurie Childrens Hospital of Chicago
🇺🇸Chicago, Illinois, United States
The Medical College of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
University of Colorado - PPDS
🇺🇸Aurora, Colorado, United States
Children's National Medical Center
🇺🇸Washington, District of Columbia, United States
Nicklaus Children's Hospital
🇺🇸Miami, Florida, United States
Nemours Children's Hospital - Orlando
🇺🇸Orlando, Florida, 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
Penn State Health Milton S. Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
Cook Children's Hospital
🇺🇸Fort Worth, Texas, United States
Children's Hospital of the King's Daughters
🇺🇸Norfolk, Virginia, United States