NCT04768062

Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)

A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)

• Status: Unknown
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 74 (actual)
• Sponsor: NS Pharma, Inc. · Industry
• Sex: Male
• Age: —
• Healthy volunteers: Not accepted

Summary

This is a Phase 3, multi-center, open-label extension study in ambulant boys with DMD who have completed the 48-week treatment period of either viltolarsen or placebo in Study NS-065/NCNP-01-301.

Detailed description

This Phase 3 study is a multi-center, open-label extension study in ambulant boys with DMD who have completed the 48-week treatment period of either viltolarsen or placebo in Study NS-065/NCNP-01-301. Patients will receive viltolarsen administered IV at weekly doses of 80 mg/kg. Study NS-065/NCNP-01-302 will be comprised of a 96-week treatment period.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Viltolarsen	Received during weekly intravenous infusions

Timeline

• Start date: 2021-04-13
• Primary completion: 2025-10-01
• Completion: 2025-11-01
• First posted: 2021-02-24
• Last updated: 2024-02-14

Locations

31 sites across 17 countries: Australia, Canada, Chile, China, Czechia, Greece, Italy, Japan, Mexico, Netherlands, New Zealand, Norway, Russia, South Korea, Spain, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study