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Gene Therapy Trial Report

Summary

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD


NCTID NCT03368742 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Duchenne Muscular Dystrophy (DMD)
Disease Ontology Term DOID:11723
Compound Name SGT-001
Compound Description AAV9.CK8.Microdystrophin
Sponsor Solid Biosciences Inc.
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 12 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant Micro-dystrophin
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV9
Dose 1 5E13 vg/kg (n=3)
Dose 2 2E14 vg/kg (n=9)

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2017-12-05
Completion Date 2026-10-15
Last Update 2026-03-03

Participation Criteria


Eligible Age 4 Years - 17 Years
Standard Ages Child
Sexes Eligible for Study MALE
Eligibility Criteria
Inclusion Criteria: * Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype * Confirmed absence of dystrophin as determined by muscle biopsy (ambulatory participants) * Anti-AAV9 antibodies below protocol-specified thresholds * Stable cardiac and pulmonary function * Adolescents: non-ambulatory by protocol-specified criteria * Children: ambulatory by protocol-specified criteria * Stable daily dose (or equivalent) of oral corticosteroids ≥ 12 weeks Exclusion Criteria: * Prior or ongoing medical condition or physical examination, ECG or laboratory findings that could adversely affect participant safety, compromise completion of treatment and follow-up, or impair assessment of study results * Abnormal liver function * Abnormal renal function * Clinically significant coagulation abnormalities * Impaired cardiovascular function based on cardiac MRI or ECHO * Impaired respiratory function based on FVC % predicted or need for daytime ventilatory support * Significant spinal deformity or presence of spinal rods * Body mass index ≥ 95th percentile for age * Exposure to another investigational drug within 3 months or 5 half-lives prior to screening * Exposure to drugs affecting dystrophin or utrophin expression within 6 months prior to screening Additional inclusion/exclusion criteria may apply.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 2
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates In September 2022, Solid Biosciences announced they would be pausing activities for SGT-001; "No longer developing" as of 2024 Annual Report

Resources/Links