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

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.

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.

Trial design

500 participants in 2 patient groups

Delandistrogene Moxeparvovec

Description:

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.

Treatment:

Genetic: delandistrogene moxeparvovec

Standard of Care

Description:

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.

Treatment:

Drug: Standard of Care