A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice

SRP-9001-401

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.

2024-02-07

2029-12-31

2038-12-31

Recruiting

500

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.

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'COHORT', 'timePerspective': 'PROSPECTIVE'}, 'enrollmentInfo': {'count': 500, 'type': 'ESTIMATED'}}

2024-04-12