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Clinical Trial Report

Gene Therapy Trial Report

Summary

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


NCTID NCT06138639 (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 40 (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
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2023-11-14
Completion Date 2031-05-06
Last Update 2025-10-14

Participation Criteria


Eligible Age 0 Years - 17 Years
Standard Ages Child
Sexes Eligible for Study MALE

Locations


No.of Trial Sites 12
Locations Canada,United States,Italy,United Kingdom

Regulatory Information


Has US IND True
FDA Designations Rare Pediatric Disease Designation
Recent Updates 20 subjects expected by Q4 2025

Resources/Links