Tests SGT-003 gene therapy in boys with Duchenne muscular dystrophy
Tests a single infusion of SGT-003 gene therapy in boys with Duchenne muscular dystrophy.
Plain-language title generated from the registry record
Registry title: A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Key facts
- Where
- Atlanta, Chicago, Columbus, Little Rock, Los Angeles, Norfolk, Philadelphia, Portland, Sacramento, San Diego, Seattle, and St Louis, United StatesSee the 12 sites
- Phase
- Phase 1/2
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 0 and 17 years old?
Source: NCT06138639, who can take part
Where in United States
- Rare Disease ResearchAtlanta, United StatesTaking people
- Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, United StatesTaking people
- Nationwide Children's HospitalColumbus, United StatesTaking people
- Arkansas Children's HospitalLittle Rock, United StatesTaking people
- University of California Los Angeles Medical CenterLos Angeles, United StatesTaking people
- Children's Hospital of The King's DaughtersNorfolk, United StatesTaking people
- Children's Hospital of PhiladelphiaPhiladelphia, United StatesTaking people
- Oregon Health and Sciences UniversityPortland, United StatesTaking people
- University of California - DavisSacramento, United StatesTaking people
- University of CaliforniaSan Diego, United StatesTaking people
- Seattle Children's HospitalSeattle, United StatesTaking people
- Washington University in St. LouisSt Louis, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Solid Biosciences Inc.
- Trial code
- NCT06138639EU 2024-514501-57-00
- Record updated
- 8 July 2026
- Original description
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.Read full original descriptionShow less
A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
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.
Inclusion: 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; 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; Negative for AAV antibodies; Steroid regimen:; 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; 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: 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; Other inclusion or exclusion criteria apply.