Evaluating Safety and Effectiveness of GNR-097 Gene Therapy Compared to Placebo in Boys Aged 4 to 9 with Duchenne Muscular Dystrophy

What this Trial Is About

Researchers are evaluating the tolerability, safety, and effectiveness of a gene therapy called GNR-097 for boys aged 4 to 9 years with Duchenne muscular dystrophy DMD. The study includes two phases Phase I involves two dose groups receiving increasing amounts of the gene therapy, and Phase II is a randomized, single-blind, placebo-controlled study where some participants receive placebo initially with the option to receive gene therapy in the second year. Participants will receive a single intravenous infusion of GNR-097 on Day 1 during Phase I. In Phase II, participants randomized to placebo will receive a matching placebo infusion on Day 1, then have the option to receive the gene therapy infusion at the start of the second year. The study aims to determine the best dose in Phase I and assess safety and efficacy in Phase II with placebo control. Throughout the study, participants will be monitored for adverse events and safety from baseline through Week 104. Researchers will evaluate treatment-emergent adverse events, serious adverse events, and adverse events of special interest. Participants will undergo assessments of functional activity and immune response, and their health will be tracked during screening, treatment, and follow-up periods. The total study duration spans up to approximately four years from the start date in 2025 to completion in 2029.

A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy