Actively Recruiting
A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 Gene Therapy in Males With Duchenne Muscular Dystrophy
Led by Solid Biosciences Inc. · Updated on 2026-05-08
60
Participants Needed
15
Research Sites
208 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and effectiveness of a single intravenous infusion of SGT-003 gene therapy in males with Duchenne Muscular Dystrophy (DMD). This open-label, non-randomized, multicenter study includes five age-based cohorts ranging from newborns to 17 years old. The study aims to gather both short-term and long-term data on this genetic treatment, with participant enrollment for older cohorts depending on data from younger groups. Participants will receive one intravenous dose of SGT-003 on Day 1, with the dose and eligibility based on age and ambulatory status. Cohorts include ambulatory boys aged 4 to under 7 years, 7 to under 12 years, and 12 to under 18 years; non-ambulatory boys aged 10 to under 18 years; and children from birth to under 4 years regardless of ambulatory status. After infusion, all participants will be followed for a total of five years to monitor long-term effects. During the study, participants will undergo various assessments including muscle biopsies, physical function tests like the 10-meter walk/run and stair climb, laboratory tests, and heart monitoring through ECG and echocardiography. Researchers will track changes in microdystrophin protein levels and physical abilities over time, along with monitoring for any treatment-related side effects. The long follow-up period allows for thorough safety and efficacy evaluation of SGT-003 gene therapy.
CONDITIONS
Brief Title
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Males aged 0 to under 18 years, divided into specific age cohorts
- Ambulatory status as defined: ambulatory for cohorts 1, 2, and 4; ambulatory or non-ambulatory for cohort 3; non-ambulatory with prior ambulatory history for cohort 5
- Established clinical diagnosis of Duchenne Muscular Dystrophy confirmed by genetic testing
- Negative for AAV antibodies
- Stable daily oral steroid regimen for at least 12 weeks prior to screening for cohorts 1, 2, 4, and 5
- Meet 10-meter walk/run time and time to rise from supine criteria
- Meet Performance of Upper Limb 2.0 criteria for cohort 5
- Body weight less than or equal to 90 kg
You will not qualify if you...
- Use of dystrophin modifying drugs within 3 months prior to screening
- Previous or current treatment with approved or investigational gene transfer drugs
- Exposure to certain approved or investigational drugs within 3 months or 5 half-lives before screening
- DMD associated with deletion mutations predicted not to express specific DMD gene exons as confirmed by genetic testing
- Other unspecified exclusion criteria as detailed in the full protocol
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single day treatment with follow-up through 18 months
Participants receive a single intravenous infusion of SGT-003 gene therapy on Day 1.
1 infusion visit and multiple follow-up visits up to 18 months
Duration - Up to 18 months after treatment
Participants are monitored for safety, tolerability, and efficacy outcomes after treatment.
Regular visits for assessments through Day 540 (approximately 18 months)
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
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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