A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

Part of paid clinical trials in Little Rock, Arkansas.

Sponsor
Solid Biosciences Inc.
Study ID
NCT07160634
Phase
PHASE3
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
MALE
Age
7 Years - 11 Years
Healthy Volunteers
Not accepted

Interventions

  • SGT-003 — DRUG
    Adeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5)
  • Placebo — DRUG
    IV infusion

Study Details

This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ambulant male participants with DMD. The secondary objectives include the evaluation of additional efficacy and safety outcomes. The study will be divided into 2 parts. Participants will be randomized 1:1 to either SGT-003 in Part 1 followed by placebo in Part 2 or to placebo in Part 1 followed by SGT-003 in Part 2. Participants will continue to be monitored in long term follow up (LTFU) for at least 5 years from their SGT-003 dosing date.

Key Dates

Start date
Oct 22, 2025
Status verified
Jun 2026
Primary completion
Jan 31, 2029
Completion
Jan 31, 2034

Study Design

Enrollment
80 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: SGT-003 followed by Placebo
    Enrolled participants will receive a single intravenous (IV) infusion of SGT-003 in Part 1 and a single IV infusion of matching Placebo in Part 2.
  • Experimental: Placebo followed by SGT-003
    Enrolled participants will receive a single intravenous (IV) infusion of matching Placebo in Part 1 and a single IV infusion of SGT-003 in Part 2.

Primary Outcome Measure

Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540 [ Time Frame: Baseline, Day 540 ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
Arkansas Children's HospitalLittle RockArkansas72202
Amber Kellogg, RN
[email protected]
Neurology Rare Disease CenterFlower MoundTexas75082
Jennifer Avelar, CRC
[email protected]
Children's Hospital of the King's DaughtersNorfolkVirginia23510
Jennifer Beachum, BSHS
[email protected]

Find similar trials in Little Rock, AR

By condition
Duchenne muscular dystrophy
By specialty
Pediatrics
By research site
Arkansas Children's Hospital· Little Rock, ARNeurology Rare Disease Center· Flower Mound, TXChildren's Hospital of the King's Daughters· Norfolk, VA

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