INS1201 for Duchenne Muscular Dystrophy
(ASCEND Trial)
Trial Summary
Do I need to stop my current medications to join the trial?
The trial protocol does not specify if you need to stop taking your current medications. However, it excludes participants who require chronic drug treatment that might compromise safety or compliance, so it's best to discuss your specific medications with the trial team.
What is the purpose of this trial?
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 Criteria
This trial is for ambulatory male children with Duchenne Muscular Dystrophy (DMD). They must be between 2 to less than 5 years old, able to walk at least 10 meters without help, and have a specific genetic mutation in the DMD gene. Participants need to be up-to-date on vaccinations as per CDC guidelines.Inclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single intrathecal injection of INS1201
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- INS1201
Find a Clinic Near You
Who Is Running the Clinical Trial?
Insmed Gene Therapy LLC
Lead Sponsor