Summary

Eligibility
for males ages 4-7 (full criteria)
Location
at Sacramento, California and other locations
Dates
study started
estimated completion
Principal Investigator
by Craig McDonald

Description

Summary

The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.

Official Title

A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Patients With Duchenne Muscular Dystrophy (EMBARK)

Keywords

Duchenne Muscular Dystrophy Muscular Dystrophies DMD North Star Ambulatory Assessment (NSAA) Ambulatory Pediatric Gene-Delivery Muscular Dystrophy, Duchenne SRP-9001

Eligibility

You can join if…

Open to males ages 4-7

  • Is ambulatory and from 4 to under 8 years of age at time of randomization.
  • Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
  • Ability to cooperate with motor assessment testing.
  • Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
  • rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
  • A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive), with the exception of mutation fully contained within exon 45.

You CAN'T join if...

  • 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.
  • Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.

Other inclusion or exclusion criteria could apply.

Locations

  • University of California, Davis accepting new patients
    Sacramento California 95817 United States
  • Lucile Packard Children's Hospital at Stanford accepting new patients
    Palo Alto California 94304 United States

Lead Scientist at UC Davis

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Sarepta Therapeutics, Inc.
Links
Clinical Trial Website (English) Sitio Web de Estudios Clínicos (Español) Sign up for this study
ID
NCT05096221
Phase
Phase 3 Duchenne Muscular Dystrophy Research Study
Study Type
Interventional
Participants
Expecting 120 study participants
Last Updated