A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
NCT ID: NCT06138639
Last Updated: 2025-12-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
60 participants
INTERVENTIONAL
2024-05-06
2031-05-06
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Cohort 1: SGT-003
All ambulatory participants from age 4 to \< 7 years will receive a single IV infusion of SGT-003 on Day 1.
SGT-003
Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)
Cohort 2: SGT-003
All ambulatory participants from age 7 to \< 12 years will receive a single IV infusion of SGT-003 on Day 1.
SGT-003
Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)
Cohort 3: SGT-003
All participants from age 0 to \< 4 years will receive a single IV infusion of SGT-003 on Day 1.
SGT-003
Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)
Cohort 4: SGT-003
All ambulatory participants from age 12 to \< 18 years will receive a single IV infusion of SGT-003 on Day 1.
SGT-003
Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)
Cohort 5: SGT-003
All non-ambulatory participants from age 10 to \< 18 years will receive a single IV infusion of SGT-003 on Day 1.
SGT-003
Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)
Interventions
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SGT-003
Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)
Eligibility Criteria
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Inclusion Criteria
* 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 Criteria
* 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.
0 Years
17 Years
MALE
No
Sponsors
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Solid Biosciences Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Solid Bio Clinical Trials
Role: STUDY_DIRECTOR
Solid Biosciences
Locations
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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
University of California
San Diego, California, United States
Rare Disease Research
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Washington University in St. Louis
St Louis, Missouri, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Oregon Health and Sciences University
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, United States
Seattle Children's Hospital
Seattle, Washington, United States
The Hospital for Sick Children
Toronto, Ontario, Canada
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome, , Italy
Great Ormond Street Hospital
London, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Amber Kellogg
Role: primary
Merve Kaleli
Role: primary
Neuromuscular Research Lab
Role: primary
Gabriella Penner
Role: primary
Neona Ni
Role: primary
Alka Maheshwari
Role: primary
Nancy Kuntz, MD
Role: backup
Natalie Goedeker
Role: primary
Destany McCain
Role: primary
Beata Dyar
Role: primary
Brandt Parlanti
Role: primary
E.Ann Walker
Role: primary
Janaki O'Brien
Role: primary
Ana Stosic
Role: primary
Lorenzo Stivala
Role: primary
Lucinda Furtado
Role: primary
Other Identifiers
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2024-514501-57-00
Identifier Type: CTIS
Identifier Source: secondary_id
1010251
Identifier Type: OTHER
Identifier Source: secondary_id
SGT-003-101
Identifier Type: -
Identifier Source: org_study_id