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Microdystrophin Gene Transfer Study in Adolescents and Children With DMD | Clinical Trials | Clareo Health

ACTIVE_NOT_RECRUITINGPHASE1/PHASE2

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

A Randomized, Controlled, Open-label, Single-ascending Dose, Phase I/II Study to Investigate the Safety and Tolerability, and Efficacy of Intravenous SGT-001 in Male Adolescents and Children With Duchenne Muscular Dystrophy

NCT03368742•Solid Biosciences Inc.

View on ClinicalTrials.gov

Summary

This is a controlled, open-label, single-ascending dose study to evaluate the safety and tolerability of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Participants will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years. The protocol was amended to drop the control arm after 4 participants were dosed.

Eligibility

Age

4 - 17 years

Sex

MALE

Healthy Volunteers

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

Locations (2)

David Geffen School of Medicine at UCLA

Los Angeles, California, United States

University of Florida

Gainesville, Florida, United States

Key Dates

Start Date

December 6, 2017

Primary Completion Date

October 15, 2026

Completion Date

October 15, 2026

Last Updated

March 3, 2026

Enrollment

ACTUAL participants

Conditions

Duchenne Muscular Dystrophy

Interventions

SGT-001

GENETIC

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44

NCT07037862

Duchenne Muscular Dystrophy (DMD)

View study

RECRUITINGPHASE3

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

NCT07160634

Duchenne Muscular Dystrophy

View study

RECRUITINGPHASE2

A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 3, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

Inclusion Criteria

Exclusion Criteria

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44

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

A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)

Fear of Falling in Muscular Dystrophy

Vasodilator and Exercise Study for DMD (VASO-REx)

DMD Voice: Qualitative Interviews With Patients and Caregivers