A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Study Overview
- Age
- 0–17 years
- Phase
- Phase 1/2
- Sponsor
- Solid Biosciences
- Therapeutic Approach
- Gene Therapy
- Variant Requirement
- Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype confirmed by Sponsor genetic testing. In cases where a genotype may be predictive of residual dystrophin production and/or a clear clinical diagnosis of DMD cannot be made (e.g., due to age), evaluation of dystrophin levels in baseline muscle biopsies may be required to determine eligibility under this criterion.
- Eligible Sex
- Male
- Ambulation
- Ambulatory and Non-Ambulatory
- Study Start (Actual)
- 2024-05-06
- Primary Completion (Estimated)
- 2027-05-06
- Study Completion (Estimated)
- 2031-05-06
- Enrollment (Estimated)
- 60
- Countries
- United StatesCanadaItalyUnited Kingdom
Study Requirements and Criteria
Steroid Use
- Cohorts 1, 2, 4, and 5: A stable daily oral steroid regimen of at least 0.5 mg/kg/day of prednisone or 0.75 mg/kg/day of deflazacort for ≥12 weeks prior to Screening Part A or Rescreening, allowing for weight-based modifications consistent with clinical practice.
- Cohort 3: N/A
Inclusion Criteria
- Cohort 1: 4 to <7 years of age
- Cohort 2: 7 to <12 years of age
- Cohort 3: 0 to < 4 years of age
- Cohort 4: 12 to < 18 years of age
- Cohort 5: 10 to < 18 years of age
- Participant ambulatory status at the time of Screening Part A or Rescreening, as defined by the ability to complete a 10-meter walk/run test in < 30 seconds:
- Cohorts 1, 2, and 4: Ambulatory
- Cohort 3: Either ambulatory or non-ambulatory
- Cohort 5: Non-ambulatory, but having been previously ambulatory by history
- Negative for AAV antibodies.
- Meet 10-meter walk/run time criteria
- Meet time to rise from supine criteria
- Cohort 5: Meet Performance of Upper Limb (PUL) 2.0 criteria
- Participant has body weight: ≤ 90 kg
Exclusion Criteria
- Treatment with dystrophin modifying drugs within 3 months prior to screening.
- Current or prior treatment with an approved or investigational gene transfer drug.
- Exposure to certain approved or investigational drugs 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 invariant or variant predicted to not express exons 1 to 11 or, exons 42 to 45, or exons 57 to 69, inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing.
Contacts and Locations
Contacts and Locations
Contact
- Name: Solid Bio Clinical Trials
- Phone Number: 617-337-4680
- Email: [email protected]
Locations
This study has 15 locations
United States
Arkansas Locations
Little Rock, Arkansas, United States, 72202
Actively Recruiting
Arkansas Children's Hospital
Contact: Amber Kellogg
Contact Mail: [email protected]
Contact Phone: 501-364-3389
California Locations
Los Angeles, California, United States, 90095
Actively Recruiting
University of California, Los Angeles Medical Center
Contact: Ummi Qasim
Contact Mail: [email protected]
Contact Phone: 310-825-3264
Sacramento, California, United States, 95817
Actively Recruiting
University of California, Davis
Contact: Neuromuscular Research Lab
Contact Mail: [email protected]
Contact Phone: 916-734-5057
San Diego, California, United States, 92037
Actively Recruiting
University of California
Contact: Gabriella Penner
Contact Mail: [email protected]
Contact Phone: 858-382-3061
Georgia Locations
Atlanta, Georgia, United States, 30329
Actively Recruiting
Rare Disease Research
Contact Mail: [email protected]
Contact Phone: 678-883-6897
Illinois Locations
Chicago, Illinois, United States, 60611-2605
Actively Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Contact: Alka Maheshwari
Contact Mail: [email protected]
Contact Phone: 312-227-3813
Missouri Locations
St Louis, Missouri, United States, 63110
Actively Recruiting
Washington University in St. Louis
Contact: Natalie Goedeker
Contact Mail: [email protected]
Contact Phone: 314-362-4919
Ohio Locations
Columbus, Ohio, United States, 43215
Actively Recruiting
Nationwide Children's Hospital
Contact: Destany McCain
Contact Mail: [email protected]
Contact Phone: 614-722-6961
Oregon Locations
Portland, Oregon, United States, 97239
Actively Recruiting
Oregon Health and Sciences University
Contact: Beata Dyar
Contact Mail: [email protected]
Contact Phone: 503-494-8216
Pennsylvania Locations
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
Children's Hospital of Philadelphia
Contact: Brandt Parlanti
Contact Mail: [email protected]
Contact Phone: 215-850-7769
Virginia Locations
Norfolk, Virginia, United States, 23510
Actively Recruiting
Children's Hospital of the King's Daughters
Contact: E.Ann Walker
Contact Mail: [email protected]
Contact Phone: 757-668-6543
Washington Locations
Seattle, Washington, United States, 98105
Actively Recruiting
Seattle Children's Hospital
Contact: Janaki O'Brien
Contact Mail: [email protected]
Contact Phone: 206-987-1642
Canada
Ontario Locations
Toronto, Ontario, Canada, M5G 0A4
Actively Recruiting
The Hospital for Sick Children
Contact: Ana Stosic
Contact Mail: [email protected]
Contact Phone: 416-648-6831
Italy
Rome, Italy, 00168
Actively Recruiting
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Contact: Lorenzo Stivala
Contact Mail: [email protected]
Contact Phone: +39 063-015-6330
United Kingdom
London, United Kingdom, WC1N 3JH
Actively Recruiting
Great Ormond Street Hospital
Contact: Lucinda Furtado
Contact Mail: [email protected]
Contact Phone: +44 (0)20 7762 6932
Clinical Trial Registry
NCT ID
NCT06138639This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.