NCT06138639

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

A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: Solid Biosciences Inc. · Industry
• Sex: Male
• Age: 0 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

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 5 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. Cohort 3 will include participants 0 to \< 4 years of age. Cohort 4 will include participants 12 to \< 18 years of age. Cohort 5 will include participants 10 to \< 18 years of age. Initiation of participant enrollment in Cohorts 4 and 5 will be subject to the accrual of safety and efficacy data from Cohorts 1-3. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
GENETIC	SGT-003	Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)

Timeline

• Start date: 2024-05-06
• Primary completion: 2027-05-06
• Completion: 2031-05-06
• First posted: 2023-11-18
• Last updated: 2026-03-03

Locations

15 sites across 4 countries: United States, Canada, Italy, United Kingdom

Regulatory

• FDA-regulated drug study