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

Sarepta Therapeutics

Status: Enrolling

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: Standard of Care; Genetic: Delandistrogene Moxeparvovec

Study type: Observational

Funder types: Industry

Identifiers

NCT06270719

SRP-9001-401

Details and patient eligibility

About

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.

Enrollment: 500 estimated patients

Sex: Male

Ages: 4+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

500 participants in 2 patient groups

Cohort 1: Delandistrogene Moxeparvovec

Description:

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.

Treatment:

Genetic: Delandistrogene Moxeparvovec

Cohort 2: Standard of Care

Description:

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.

Treatment:

Drug: Standard of Care