A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

a study on Duchenne Muscular Dystrophy

Summary

Eligibility
for males ages 4-7 (full criteria)
Location
at UCLA
Dates
study started
completion around
Principal Investigator
by Perry Shieh, MD, PhD (ucla)

Description

Summary

This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single IV infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 2 cohorts in this study, dosed sequentially. Cohort 1 will include participants 4 to <6 years of age, inclusive. Cohort 2 will only be opened after dosing and monitoring a subset of participants in Cohort 1. Cohort 2 will include participants 6 to <8 years of age, inclusive. 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

You can join if…

Open to males ages 4-7

  • Cohort 1: 4 to <6 years of age, inclusive
  • Cohort 2: 6 to <8 years of age, inclusive
  • Ambulatory as defined as "being able to walk without the use of an assistive device."
  • Negative for AAV antibodies.
  • On a stable dose of at least 0.5 mg/kg/day of oral daily prednisone or 0.75 mg/kg/day deflazacort for ≥12 weeks prior to entering the study.
  • Cohort 1: <18 kg body weight
  • Cohort 2: <30 kg body weight

You CAN'T join if...

  • Current or prior treatment with approved or investigational dystrophin modifying drugs such as eteplirsen, golodirsen, casimersen, and viltolarsen.
  • Current or prior treatment with an approved or investigational gene transfer drug.
  • Exposure to another investigational drug within 3 months prior to screening or 5 half-lives since last administration, whichever is longer.
  • Established clinical diagnosis of DMD that is associated with any deletion mutation in exons 1 to 11 or 42 to 45, inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing.

Other inclusion or exclusion criteria apply.

Locations

  • University of California, Los Angeles Medical Center accepting new patients
    Los Angeles California 90095 United States
  • Nationwide Children's Hospital accepting new patients
    Columbus Ohio 43215 United States

Lead Scientist at University of California Health

  • Perry Shieh, MD, PhD (ucla)
    HS Clinical Professor, Neurology, Medicine. Authored (or co-authored) 87 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 6 study participants
Last Updated