Actively Recruiting
Phase 1 Study on Safety and Distribution of a Single Intrathecal Injection of INS1201 in Boys Aged 2 to 4 With Duchenne Muscular Dystrophy
Led by Insmed Gene Therapy LLC · Updated on 2026-07-28
12
Participants Needed
10
Research Sites
8 weeks
Total Duration
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and tolerability of a single dose of INS1201 administered via intrathecal injection in ambulatory male children with Duchenne Muscular Dystrophy DMD. This phase 1, open-label study focuses on males aged 2 to less than 5 years who can walk at least 10 meters without assistance and have a confirmed genetic diagnosis of DMD involving specific mutations in the DMD gene. Participants are divided into cohorts based on age and receive a single intrathecal injection of INS1201 at one of two dose levels on Day 1. The study includes children aged 2 to less than 3 years and those aged 3 to less than 5 years, with treatment groups for each age range. The study is sequential and non-randomized, evaluating safety, biodistribution, and dosing over time. Throughout the study, participants will be monitored for treatment-emergent adverse events up to 96 weeks. Assessments include measuring micro-dystrophin DNA and protein expression at baseline, Week 16, and Week 48 to evaluate biological effects. Regular follow-ups and motor assessments are conducted to track safety, tolerability, and motor function changes during the study period lasting over 2 years.
CONDITIONS
Brief Title
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
Research Team
I
Insmed Medical Information
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