An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy (ENDURE)
Enrolling by InvitationObservationalGene Therapy
Study Overview
- Age
- 4 years and older
- Phase
- Observational
- Sponsor
- Sarepta Therapeutics
- Therapeutic Approach
- Gene Therapy
- Variant Requirement
- Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.
- Eligible Sex
- Male
- Ambulation
- Ambulatory and Non-Ambulatory
- Countries
- United States
Study Requirements and Criteria
Steroid Use
Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment.
Inclusion Criteria
- For ELEVIDYS-treated Participants (Cohorts 1a, 1b, and 1c):
- Is at least 4 years of age at the time of infusion
- Will either: a) be initiating or has initiated ELEVIDYS within the last 30 days in routine clinical practice at the time of this observational study enrollment, or b) was administered ELEVIDYS in routine clinical practice and has the required minimum dataset for entry into the observational study per Sponsor approval
- For Standard of Care Comparators (Cohort 2):
- Is at least 4 years of age at the time of enrollment
- Is unexposed to DMD gene therapy at the time of this observational study enrollment
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 this observational study enrollment.
- Has any prior exposure to DMD gene therapy other than that described for Cohort 1c (ELEVIDYS Retrospectively Treated Cohort).
- 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.
Clinical Trial Registry
NCT ID
NCT06270719This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.