Trials / Active Not Recruiting
Active Not RecruitingNCT06128564
A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)
A Two-Part, Open-Label Systemic Gene Delivery Study to Evaluate the Safety and Expression of RO7494222 (SRP-9001) in Subjects Under the Age of Four With Duchenne Muscular Dystrophy
- Status
- Active Not Recruiting
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 13 (actual)
- Sponsor
- Hoffmann-La Roche · Industry
- Sex
- Male
- Age
- 2 Years – 3 Years
- Healthy volunteers
- Not accepted
Summary
This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | delandistrogene moxeparvovec | Single IV infusion of delandistrogene moxeparvovec |
Timeline
- Start date
- 2023-11-29
- Primary completion
- 2030-02-28
- Completion
- 2034-01-30
- First posted
- 2023-11-13
- Last updated
- 2026-02-10
Locations
7 sites across 6 countries: Belgium, France, Germany, Italy, Spain, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06128564. Inclusion in this directory is not an endorsement.