Trials / Completed
CompletedNCT04956289
Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)
A Phase 2 Open-label Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With Duchenne Muscular Dystrophy (DMD) Compared to Natural History Controls
- Status
- Completed
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 20 (actual)
- Sponsor
- NS Pharma, Inc. · Industry
- Sex
- Male
- Age
- 8 Years
- Healthy volunteers
- Not accepted
Summary
This is a phase II, open-label study where weekly doses of 80 mg/kg viltolarsen is administered intravenously over a 48-week treatment period to ambulant and non-ambulant DMD patients over the age of 8 years.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Viltolarsen | Received during weekly intravenous infusions |
Timeline
- Start date
- 2021-07-01
- Primary completion
- 2023-06-20
- Completion
- 2023-07-13
- First posted
- 2021-07-09
- Last updated
- 2024-08-20
- Results posted
- 2024-08-20
Locations
8 sites across 6 countries: United States, China, Italy, Russia, Spain, Turkey (Türkiye)
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04956289. Inclusion in this directory is not an endorsement.