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Gene Therapy Trial Report

Summary

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


NCTID NCT07160634 (View at clinicaltrials.gov)
Description
Development Status 🔄 Active
Indication 🔄 Duchenne Muscular Dystrophy (DMD)
Disease Ontology Term 🔄 DOID:11723
Compound Name 🔄 SGT-003
Compound Description 🔄 AAV-SLB101-CK8-microdystrophin containing R16-R17 nNOS binding domain
Sponsor Solid Biosciences Inc.
Funder Type Industry
Recruitment Status
Enrollment Count 80 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant 🔄 Micro-dystrophin
Therapy Type 🔄 Gene transfer
Therapy Route 🔄 In-vivo
Mechanism of Action 🔄 Overexpression of protective allele/gene
Route of Administration 🔄 Intravenous
Drug Product Type 🔄 Viral vector
Target Tissue/Cell 🔄 Muscle cells
Delivery System 🔄 Viral transduction
Vector Type 🔄 AAV-SLB101
Editor Type 🔄 none
Dose 1 🔄 1E14 vg/kg

Study Record Dates


Current Stage Phase3
Submit Date 2025-08-29
Completion Date 2034-01
Last Update 2026-03-03

Participation Criteria


Eligible Age 7 Years - 11 Years
Standard Ages Child
Sexes Eligible for Study MALE
Eligibility Criteria
Inclusion Criteria: * Participant is ambulatory. * Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype. * Negative for antibodies against adeno-associated virus. * On a stable daily oral regimen of at least 0.5 mg/kg/day prednisone or 0.75 milligrams per kilogram per day (mg/kg/day) deflazacort for at least 6 months prior to entering the study, allowing for weight-based dose modifications in accordance with clinical practice. * Meet 10-meter walk/run time criteria. * Meet time to rise from supine criteria. * Participant has bodyweight ≤50 kg. Exclusion Criteria: * Current or prior treatment with an approved or investigational gene transfer drug or gene editing therapy. * Exposure to vamorolone, givinostat, approved or investigational dystrophin- or disease-modifying drugs (such as eteplirsen, golodirsen, casimersen, viltolarsen, and ataluren), or another investigational drug for any indication within 6 months or 5 half-lives, whichever is longer, prior to enrollment. * Established clinical diagnosis of DMD that is associated with any deletion variant or variant predicted not to express exons 1 to 11, exons 42 to 45, or exons 57 to 69, inclusive of the DMD gene as documented by a genetic report. Other Inclusion/Exclusion criteria to be applied as per protocol.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 2
Locations Canada,Australia

Regulatory Information


Has US IND True
FDA Designations 🔄 Fast Track, Rare Pediatric Disease Designation
Recent Updates Confirmatory trial is now recruiting, drug continues to be well tolerated in 36 participants dosed in Phase 1/2 trial as of 2/9/26

Resources/Links