Summary

Eligibility
for males ages 2 years and up (full criteria)
Location
at UC Davis
Dates
study started
completion around

Description

Summary

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.

Official 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 (ENDEAVOR)

Keywords

Muscular Dystrophy, Duchenne, Duchenne Muscular Dystrophy, Gene-Delivery, DMD, Ambulatory Non-ambulatory, Pediatric, Dystrophin, Muscular Dystrophies, delandistrogene moxeparvovec

Eligibility

You can join if…

Open to males ages 2 years and up

  • For Cohorts 1-7: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
  • Cohort 1: Is ambulatory, and ≥4 to <8 years of age at the time of Screening.
  • Cohort 2: Is ambulatory, and ≥8 to <18 years of age at the time of Screening.
  • Cohort 3: Non-ambulatory per protocol specified criteria at the time of Screening.
  • Cohort 4: Is ambulatory and ≥3 to <4 years of age at the time of Screening.
  • Cohort 5a: Is ambulatory and ≥4 to <9 years of age.
  • Cohort 5b: Non-ambulatory per protocol specified criteria at the time of Screening.
  • Cohort 6: Is ambulatory, and ≥2 to <3 years of age at the time of Screening.
  • Cohort 7: Non-ambulatory per protocol-specified criteria at the time of Screening.
  • Ability to cooperate with motor assessment testing.
  • Cohorts 1, 2, 3, 5, and 7 only: Stable dose equivalent of oral glucocorticoids for at least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the first year of the study.
  • Cohorts 4 and 6: Do not yet require use of chronic steroids for treatment of their DMD, in the opinion of the Investigator, and are not receiving steroids at the time of Screening.
  • rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
  • Genetic mutation inclusion criteria vary by cohort.

You CAN'T join if...

  • Has a concomitant illness, autoimmune disease, chronic drug treatment, and/or cognitive delay/impairment that in the opinion of the Investigator creates unnecessary risks for gene transfer.
  • Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol-specified time limits.
  • Abnormality in protocol-specified diagnostic evaluations or laboratory tests.

Other inclusion/exclusion criteria apply.

Locations

  • University of California, Davis
    Sacramento California 95616 United States
  • Stanford University
    Palo Alto California 94304 United States

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
Sarepta Therapeutics, Inc.
ID
NCT04626674
Phase
Phase 1 Duchenne Muscular Dystrophy Research Study
Study Type
Interventional
Participants
Expecting 58 study participants
Last Updated