A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
- Conditions
- Duchenne Muscular Dystrophy
- Registration Number
- NCT06138639
- Lead Sponsor
- Solid Biosciences Inc.
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- Male
- Target Recruitment
- 40
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Cohort 1: 4 to <7 years of age
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Cohort 2: 7 to <12 years of age
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Cohort 3: 0 to < 4 years of age
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Cohort 4: 12 to < 18 years of age
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Cohort 5: 10 to < 18 years of age
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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
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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.
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Negative for AAV antibodies.
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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
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Meet 10-meter walk/run time criteria
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Meet time to rise from supine criteria
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Cohort 5: Meet Performance of Upper Limb (PUL) 2.0 criteria
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Participant has body weight: ≤ 90 kg
- 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 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Primary Outcome Measures
Name Time Method Incidence of treatment-emergent adverse events (AEs) Day 360 Change from baseline in microdystrophin protein levels Day 90 Microdystrophin expression evaluation in muscle biopsies
- Secondary Outcome Measures
Name Time Method Change from Baseline of Microdystrophin Tissue Distribution by Immunofluorescence (IF) Day 90, Day 360 Change from baseline in microdystrophin protein levels Day 360 Microdystrophin expression evaluation in muscle biopsies
Change from Baseline in Time to Rise Velocity Day 360, Day 540 Change from baseline in stride velocity 95th centile (SV95C) Day 360, Day 540 Assessment of peak ambulatory performance captured by wearable activity monitoring device
Change from baseline in 10-meter walk/run velocity Day 360, Day 540 Change from baseline in 4-stair climb velocity Day 360, Day 540 Change from baseline in North Star Ambulatory Assessment (NSAA) total score Day 360, Day 540 Assessment of muscle function using a 17-item scale with each item scored from 0 to 2 and a higher score meaning a better outcome. The NSAA total score is defined as the sum of all 17 items, ranging from 0 to 34, with a higher score meaning a better outcome.
Change from baseline in 6-minute walk test (6MWT) distance Day 360, Day 540 Number of Participants with Clinically Significant Abnormalities in Laboratory Parameters Through Day 360 and Day 540 Number of Participants with Clinically Significant Abnormalities in Vital Signs Through Day 360 and Day 540 Number of Participants with Clinically Significant Abnormalities in Physical Examinations Through Day 360 and Day 540 Number of Participants with Clinically Significant Abnormalities in Electrocardiogram (ECG) or Echocardiography (ECHO) Through Day 360 and Day 540
Trial Locations
- Locations (10)
Great Ormond Street Hospital
🇬🇧London, United Kingdom
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
University of California, Los Angeles Medical Center
🇺🇸Los Angeles, California, United States
University of California, Davis
🇺🇸Sacramento, California, United States
Rare Disease Research
🇺🇸Atlanta, Georgia, United States
Washington University in St. Louis
🇺🇸Saint Louis, Missouri, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Children's Hospital of the King's Daughters
🇺🇸Norfolk, Virginia, United States
The Hospital for Sick Children
🇨🇦Toronto, Ontario, Canada
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
🇮🇹Rome, Italy
Great Ormond Street Hospital🇬🇧London, United KingdomLucinda FurtadoContact+44 (0)20 7762 6932Lucinda.furtado@gosh.nhs.ukFrancesco Muntoni, MDPrincipal Investigator