A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

Part of paid clinical trials in Little Rock, Arkansas.

Sponsor
Solid Biosciences Inc.
Study ID
NCT06138639
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
MALE
Age
0 Years - 17 Years
Healthy Volunteers
Not accepted

Interventions

  • SGT-003 — GENETIC
    Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)

Study Details

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.

Key Dates

Start date
May 6, 2024
Status verified
May 2026
Primary completion
May 6, 2027
Completion
May 6, 2031

Study Design

Enrollment
60 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: 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.
  • Experimental: 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.
  • Experimental: Cohort 3: SGT-003
    All participants from age 0 to \< 4 years will receive a single IV infusion of SGT-003 on Day 1.
  • Experimental: 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.
  • Experimental: 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.

Primary Outcome Measure

Incidence of treatment-emergent adverse events (AEs) [ Time Frame: Day 360 ]

Central Contacts

Locations (12)

FacilityCityStateZIPSite coordinators
Arkansas Children's HospitalLittle RockArkansas72202
Amber Kellogg
[email protected]
501-364-3389
Aravindhan Veerapandiyan, MD (PRINCIPAL_INVESTIGATOR)
University of California, Los Angeles Medical CenterLos AngelesCalifornia90095
Merve Kaleli
[email protected]
310-825-3264
Perry Shieh, MD, PhD (PRINCIPAL_INVESTIGATOR)
University of California, DavisSacramentoCalifornia95817
Neuromuscular Research Lab
[email protected]
916-734-5057
Craig McDonald, MD (PRINCIPAL_INVESTIGATOR)
University of CaliforniaSan DiegoCalifornia92037
Gabriella Penner
[email protected]
858-382-3061
Chamindra Laverty, MD (PRINCIPAL_INVESTIGATOR)
Rare Disease ResearchAtlantaGeorgia30329
Neona Ni
[email protected]
470-666-1884
Renata Shih, MD (PRINCIPAL_INVESTIGATOR)
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611-2605
Alka Maheshwari
[email protected]
312-227-3813
Nancy Kuntz, MD
Washington University in St. LouisSt LouisMissouri63110
Natalie Goedeker
[email protected]
314-362-4919
Craig Zaidman, MD (PRINCIPAL_INVESTIGATOR)
Nationwide Children's HospitalColumbusOhio43215
Destany McCain
[email protected]
614-722-6961
Kevin Flanigan, MD (PRINCIPAL_INVESTIGATOR)
Oregon Health and Sciences UniversityPortlandOregon97239
Beata Dyar
[email protected]
503-494-8216
Erika Finanger, MD (PRINCIPAL_INVESTIGATOR)
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
Brandt Parlanti
[email protected]
215-850-7769
John Brandsema, MD (PRINCIPAL_INVESTIGATOR)
Children's Hospital of the King's DaughtersNorfolkVirginia23510
E.Ann Walker
[email protected]
757-668-6543
Crystal Proud, MD (PRINCIPAL_INVESTIGATOR)
Seattle Children's HospitalSeattleWashington98105
Janaki O'Brien
[email protected]
206-987-1642
Alicia Henriquez, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Little Rock, AR

By condition
Duchenne muscular dystrophy
By specialty
Pediatrics
By research site
Arkansas Children's Hospital· Little Rock, ARUniversity of California, Los Angeles Medical Center· Los Angeles, CAUniversity of California, Davis· Sacramento, CAUniversity of California· San Diego, CARare Disease Research· Atlanta, GAAnn & Robert H. Lurie Children's Hospital of Chicago· Chicago, IL

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