A Phase 1, Multicenter, Open-label Study to Investigate the Safety and Biodistribution of a Single Intrathecal Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (The ASCEND Study)
The primary objective of this study is to evaluate the safety and tolerability of a single dose of INS1201 via IT administration in ambulatory male participants with DMD.
• Participant must be male at birth, 3 to \<5 years of age, inclusive (Part 1) and 2 to \<3 years of age (Part 2), at the time of legally authorized representative (LAR) signing and dating the informed consent form.
• Ambulatory -as defined as the ability to walk at least 10 meters unassisted (i.e., without personal assistance or use of any assistive devices) Note: children who have not yet developed the ability to walk by the time of screening (for whatever reason) will not be eligible for the study.
• Has a definitive diagnosis of DMD prior to Screening based on genetic testing. Genetic report must describe a frameshift deletion, frameshift duplication, premature stop (nonsense), canonical splice site mutation, or other pathogenic variant in the DMD gene fully contained between exons 18 to 58 (inclusive) that is expected to lead to absence of a functional dystrophin protein.
• Able to cooperate with motor assessment testing.
• Has received vaccinations recommended for the participant's age and DMD disease according to Centers for Disease Control and Prevention (CDC) recommendation on Child and Adolescent Immunization Schedule by Age, incorporating the Advisory Committee on Immunization Practices (ACIP) Vaccine Recommendations and Guidelines for Patients with Altered Immunocompetence.