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

Launched by SAREPTA THERAPEUTICS, INC. · Feb 13, 2024

Keywords

ClinConnect Summary

This clinical trial is studying the effects of a new treatment called delandistrogene moxeparvovec in boys with Duchenne muscular dystrophy (DMD), a genetic condition that affects muscle strength and function. The trial is comparing the outcomes of boys receiving this new treatment with those who are currently treated with standard glucocorticoid therapy, which is a common medication used to help manage symptoms of DMD. The study aims to gather information about how well these treatments work in real-life settings.

To be eligible for the trial, boys aged 4 to 5 years old must have a confirmed diagnosis of DMD and be able to walk. They should either be starting the new treatment or already receiving glucocorticoid therapy. Participants will be recruited from various clinical settings, and those with certain genetic mutations or other health issues may not qualify. Throughout the study, participants will be monitored to see how they respond to their respective treatments. This research is important as it helps understand the best options for boys with DMD and may lead to better care in the future.

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • 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.
• 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 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.