12 Participants Needed

INS1201 for Duchenne Muscular Dystrophy

(ASCEND Trial)

Recruiting at 4 trial locations
IM
Overseen ByInsmed Medical Information
Age: < 18
Sex: Male
Trial Phase: Phase 1
Sponsor: Insmed Gene Therapy LLC
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

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

I can walk 10 meters without help or devices.
I am up to date with all vaccinations recommended for my age and condition.
I am a boy aged 3 to <5 years (Part 1) or 2 to <3 years (Part 2), at the time my legal guardian consented.
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Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single intrathecal injection of INS1201

1 day
1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • INS1201
Trial Overview The study is testing the safety of a single intrathecal injection of INS1201 in boys with DMD. The main goal is to see how well participants tolerate this dose and how the drug spreads within their bodies.
Participant Groups
4Treatment groups
Experimental Treatment
Group I: Part 2: Cohort 4Experimental Treatment1 Intervention
Participants aged 2 to \<3 years will receive a single dose level 2 of INS1201 by IT injection on Day 1.
Group II: Part 2: Cohort 3Experimental Treatment1 Intervention
Participants aged 2 to \<3 years will receive a single dose level 1 of INS1201 by IT injection on Day 1.
Group III: Part 1: Cohort 2Experimental Treatment1 Intervention
Participants aged 3 to \<5 years will receive a single dose level 2 of INS1201 by IT injection on Day 1.
Group IV: Part 1: Cohort 1Experimental Treatment1 Intervention
Participants aged 3 to \<5 years will receive a single dose level 1 of INS1201 by IT injection on Day 1.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Insmed Gene Therapy LLC

Lead Sponsor

Trials
1
Recruited
10+
Unbiased ResultsWe believe in providing patients with all the options.
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