Summary

Eligibility
for males ages 4-11 (full criteria)
Location
at UC Davis UCLA
Dates
study started
completion around
Principal Investigator
by Perry Shieh, MD, PhD (ucla)Craig McDonald, MD (ucdavis)

Description

Summary

This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 2 cohorts in this study. Cohort 1 will include participants 4 to <7 years of age. Cohort 2 will include participants 7 to <12 years of age. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.

Official Title

A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

Keywords

Duchenne Muscular Dystrophy, DMD, Gene Therapy, Muscular Dystrophies, SGT-003

Eligibility

Locations

  • University of California, Los Angeles Medical Center accepting new patients
    Los Angeles California 90095 United States
  • University of California, Davis accepting new patients
    Sacramento California 95817 United States

Lead Scientists at University of California Health

  • Perry Shieh, MD, PhD (ucla)
    HS Clinical Professor, Neurology, Medicine. Authored (or co-authored) 90 research publications
  • Craig McDonald, MD (ucdavis)
    Professor, MED: Physical Medicine and Rehabilitation, School of Medicine. Authored (or co-authored) 250 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Solid Biosciences Inc.
ID
NCT06138639
Phase
Phase 1/2 Duchenne Muscular Dystrophy Research Study
Study Type
Interventional
Participants
Expecting 43 study participants
Last Updated