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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 πŸ”„ Active
Indication πŸ”„ Duchenne Muscular Dystrophy (DMD)
Disease Ontology Term πŸ”„ DOID:11723
Drug Product Name πŸ”„ SGT-003
Drug Product Description πŸ”„ 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 πŸ”„ Micro-dystrophin
Therapeutic Modality πŸ”„ Gene transfer
Therapy Route πŸ”„ in vivo
Mechanism of Action πŸ”„ Overexpression of protective allele/gene
Route of Administration πŸ”„ Intravenous
Drug Product Type πŸ”„ Viral vector
Target Tissue/Cell πŸ”„ Muscle cells
Gene Delivery System Type πŸ”„ Viral transduction
Vector Type πŸ”„ Adeno-associated virus
Viral Vector Subtype AAV-SLB101
Dose 1 πŸ”„ 1E14 vg/kg

Study Record Dates


Current Phase Phase3
Submit Date 2025-08-29
Completion Date 2034-01
Last Update πŸ”„ 2026-07-16

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 πŸ”„ 6
Locations πŸ”„ Canada,United States,Australia

Regulatory Information


Has US IND True
FDA Designations πŸ”„ Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates Drug continues to be well tolerated in 47 participants dosed as of 5/11/26, dosing is ongoing in Phase 3 trial

Resources/Links