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)
Insmed Gene Therapy LLC
Summary
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.
Eligibility
- Age range
- 2–4 years
- Sex
- Male
- Healthy volunteers
- No
Inclusion Criteria * 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 (ie, 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 or as part of Screen…
Interventions
- GeneticINS1201
Suspension for IT injection.
Locations (10)
- USA012Little Rock, Arkansas
- USA010Davis, California
- USA009Los Angeles, California
- USA002Palo Alto, California
- USA005San Diego, California
- Rare Disease Research (USA004)Atlanta, Georgia