Actively Recruiting
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Led by Solid Biosciences Inc. · Updated on 2026-05-08
60
Participants Needed
15
Research Sites
365 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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 5 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. Cohort 3 will include participants 0 to \< 4 years of age. Cohort 4 will include participants 12 to \< 18 years of age. Cohort 5 will include participants 10 to \< 18 years of age. Initiation of participant enrollment in Cohorts 4 and 5 will be subject to the accrual of safety and efficacy data from Cohorts 1-3. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.
CONDITIONS
Official Title
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male participants
- Age for Cohort 1: 4 to less than 7 years
- Age for Cohort 2: 7 to less than 12 years
- Age for Cohort 3: 0 to less than 4 years
- Age for Cohort 4: 12 to less than 18 years
- Age for Cohort 5: 10 to less than 18 years
- Ambulatory status at screening: Cohorts 1, 2, and 4 must be ambulatory
- Cohort 3 can be ambulatory or non-ambulatory
- Cohort 5 must be non-ambulatory but previously ambulatory by history
- Established clinical diagnosis of Duchenne muscular dystrophy with confirmed dystrophin gene mutation
- Negative for AAV antibodies
- Stable daily oral steroid regimen (prednisone at least 0.5 mg/kg/day or deflazacort at least 0.75 mg/kg/day) for at least 12 weeks prior to screening for Cohorts 1, 2, 4, and 5
- Meet 10-meter walk/run time criteria
- Meet time to rise from supine criteria
- Cohort 5 must meet Performance of Upper Limb (PUL) 2.0 criteria
- Body weight less than or equal to 90 kg
You will not qualify if you...
- Treatment with dystrophin modifying drugs within 3 months prior to screening
- Current or prior treatment with approved or investigational gene transfer drugs
- Exposure to certain approved or investigational drugs within 3 months prior to screening or 5 half-lives since last administration
- Diagnosis of Duchenne muscular dystrophy with specific deletion mutations predicted not to express exons 1 to 11, 42 to 45, or 57 to 69 in the DMD gene
- Other unspecified inclusion or exclusion criteria may apply
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 15 locations
1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
2
University of California, Los Angeles Medical Center
Los Angeles, California, United States, 90095
Actively Recruiting
3
University of California, Davis
Sacramento, California, United States, 95817
Actively Recruiting
4
University of California
San Diego, California, United States, 92037
Actively Recruiting
5
Rare Disease Research
Atlanta, Georgia, United States, 30329
Actively Recruiting
6
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611-2605
Actively Recruiting
7
Washington University in St. Louis
St Louis, Missouri, United States, 63110
Actively Recruiting
8
Nationwide Children's Hospital
Columbus, Ohio, United States, 43215
Actively Recruiting
9
Oregon Health and Sciences University
Portland, Oregon, United States, 97239
Actively Recruiting
10
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
11
Children's Hospital of the King's Daughters
Norfolk, Virginia, United States, 23510
Actively Recruiting
12
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Actively Recruiting
13
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 0A4
Actively Recruiting
14
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome, Italy, 00168
Actively Recruiting
15
Great Ormond Street Hospital
London, United Kingdom, WC1N 3JH
Actively Recruiting
Research Team
S
Solid Bio Clinical Trials
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
5
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