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ENDEAVOR

  • Status
    Accepting Candidates
  • Age
    2 Years - N/A
  • Sexes
    Male
  • Healthy Volunteers
    No
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Objective

This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with DMD. The maximum participant duration for this study is 156 weeks.

Description

Enrollment for Cohorts 1 through 7 has been completed. Cohort 8 is currently enrolling new participants.

Details

Full study title An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects with Duchenne Muscular Dystrophy (SRP-9001-103 ENDEAVOR)
Protocol number OCR49665
ClinicalTrials.gov ID NCT04626674
Phase Phase 1

Eligibility

Inclusion Criteria:

  • For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.

  • Cohort 1: Is ambulatory, and ≥4 to

Lead researcher

  • Carmen Leon Astudillo, MD
    Pediatric Neuromuscular Rehabilitation Specialist, Pediatric Pulmonologist (Child Lung Specialist)

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    Primary contact

  2. Step
    2

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  4. Step
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