An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy

Purpose

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. In addition, treatment outcomes will be collected prospectively from post-trial participants who have received delandistrogene moxeparvovec through participation in select SRP-9001 studies.

Condition

  • Duchenne Muscular Dystrophy

Eligibility

Eligible Ages
Over 4 Years
Eligible Genders
Male
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and confirmatory genetic testing. Among participants recruited from routine clinical practice: - Is aged 4 through 5 years at the time of enrollment. - Is ambulatory per protocol specified criteria. - Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment. For Delandistrogene Moxeparvovec-exposed 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

Among participants recruited from routine clinical practice: - 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

Phase
Study Type
Observational
Observational Model
Cohort
Time Perspective
Prospective

Arm Groups

ArmDescriptionAssigned Intervention
Delandistrogene Moxeparvovec Participants were either (1) prescribed delandistrogene moxeparvovec commercially as part of clinical care prior to entry into this trial or (2) previously received delandistrogene moxeparvovec through participation in select SRP-9001 studies.
  • Genetic: delandistrogene moxeparvovec
    No study medication will be provided by the sponsor during this trial.
    Other names:
    • SRP-9001
    • delandistrogene moxeparvovec-rokl
    • ELEVIDYS
Standard of Care Participants unexposed to DMD gene therapy receiving standard of care therapy (chronic glucocorticoid treatment) that does not include delandistrogene moxeparvovec or other DMD gene therapies.
  • Drug: Standard of Care
    No study medication will be provided by the sponsor during this trial.
    Other names:
    • Chronic glucocorticoid treatment

Recruiting Locations

Children's National and nearby locations

Children's National Medical Center
Washington, District of Columbia 20010

More Details

NCT ID
NCT06270719
Status
Recruiting
Sponsor
Sarepta Therapeutics, Inc.

Study Contact

Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4
1-888-SAREPTA (1-888-727-3782)
SareptAlly@sarepta.com