Trials / Recruiting
RecruitingNCT06817382
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
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)
- Status
- Recruiting
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 12 (estimated)
- Sponsor
- Insmed Gene Therapy LLC · Industry
- Sex
- Male
- Age
- 2 Years – 4 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | INS1201 | Suspension for IT injection. |
Timeline
- Start date
- 2025-07-22
- Primary completion
- 2028-01-31
- Completion
- 2028-03-31
- First posted
- 2025-02-10
- Last updated
- 2026-03-23
Locations
9 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06817382. Inclusion in this directory is not an endorsement.