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

Solid Biosciences

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Duchenne Muscular Dystrophy

Treatments: Genetic: SGT-003

Study type: Interventional

Funder types: Industry

Identifiers

NCT06138639

SGT-003-101

Details and patient eligibility

About

This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 2 cohorts in this study. Cohort 1 will include participants 4 to <7 years of age. Cohort 2 will include participants 7 to <12 years of age. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.

Enrollment: 43 estimated patients

Sex: Male

Ages: 4 to 11 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Cohort 1: 4 to <7 years of age
• Cohort 2: 7 to <12 years of age
• Participants who are ambulatory. Ambulatory as defined as "being able to walk without the use of an assistive device."
• Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype confirmed by Sponsor genetic testing.
• Negative for AAV antibodies.
• On a stable dose of at least 0.5 mg/kg/day of oral daily prednisone or 0.75 mg/kg/day deflazacort for ≥12 weeks prior to entering the study.
• Meet 10-meter walk/run time criteria
• Meet time to rise from supine criteria
• Participant has body weight: ≤50 kg

Exclusion criteria

• Treatment with dystrophin modifying drugs within 3 months prior to screening.
• Current or prior treatment with an approved or investigational gene transfer drug.
• Exposure to certain approved or investigational drugs within 3 months prior to screening or 5 half-lives since last administration, whichever is longer.
• Established clinical diagnosis of DMD that is associated with any deletion mutation in exons 1 to 11 or 42 to 45, inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing.

Other inclusion or exclusion criteria apply.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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43 participants in 2 patient groups

Cohort 1: SGT-003

Experimental group

Description:

All participants will receive a single IV infusion of SGT-003 on Day 1.

Treatment:

Genetic: SGT-003

Cohort 2: SGT-003

Experimental group

Description:

All participants will receive a single IV infusion of SGT-003 on Day 1.

Treatment:

Genetic: SGT-003