Phase 12 Study of SGT-003 Gene Therapy Given by IV Infusion in Boys with Duchenne Muscular Dystrophy Evaluating Safety, Tolerability, and Effectiveness Across Different Age Groups

What this Trial Is About

Researchers are investigating the safety, tolerability, and potential benefits of a single intravenous infusion of SGT-003 gene therapy in males with Duchenne muscular dystrophy DMD. This multicenter, open-label, non-randomized study includes five age-based cohorts ranging from newborns to adolescents up to 17 years old. The study aims to understand how well this gene therapy works and how safe it is for different age groups affected by this genetic muscle disorder. Participants in each cohort will receive one infusion of SGT-003, a gene therapy containing the human microdystrophin gene, administered intravenously on Day 1. The cohorts are divided by age and ambulatory status, with younger and ambulatory participants in cohorts 1 to 4, and non-ambulatory participants in cohort 5. Cohorts 4 and 5 will begin enrollment only after safety and efficacy data from the earlier cohorts are reviewed. All participants will be followed for a total of 5 years to monitor long-term outcomes. During the study, participants will be monitored for treatment-emergent adverse events and changes in microdystrophin protein levels up to one year after infusion. Additional assessments will evaluate muscle function, including walking speed, stair climbing, and standardized motor scores over time. Safety will be closely tracked through laboratory tests, vital signs, physical exams, and heart monitoring. These evaluations will continue throughout the 5-year follow-up period to understand the lasting effects of the gene therapy.

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