NCT04060199

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

A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 77 (actual)
• Sponsor: NS Pharma, Inc. · Industry
• Sex: Male
• Age: 4 Years – 7 Years
• Healthy volunteers: Not accepted

Summary

The main objective of this study is to evaluate the efficacy of Viltolarsen compared to placebo in Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.

Detailed description

This is a Phase 3 randomized, double-blind, placebo-controlled, multi-center study to assess the efficacy and safety of Viltolarsen in ambulant boys with Duchenne muscular dystrophy. Eligible patients with out-of-frame deletion mutations amenable to exon 53 skipping will be randomized to receive once weekly intravenous (IV) infusions of 80 mg/kg Viltolarsen or placebo for up to 48 weeks. The study will enroll approximately 74 patients amenable to exon 53 skipping. Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as Time to Stand Test (TTSTAND), Time to Run/Walk 10 Meters Test (TTRW), Six-minute Walk Test (6MWT), North Star Ambulatory Assessment (NSAA), Time to Climb 4 Steps Test (TTCLIMB) and Hand-held dynamometer (elbow extension, elbow flexion, knee extension and knee flexion on the dominant side only). Safety will be assessed through the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), vital signs, and physical examinations throughout the study. Blood samples will be taken periodically throughout the study to assess the pharmacokinetics of study drug.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Viltolarsen	IV infusion
DRUG	Placebo	IV infusion

Timeline

• Start date: 2020-04-14
• Primary completion: 2023-10-19
• Completion: 2023-10-19
• First posted: 2019-08-19
• Last updated: 2024-12-11
• Results posted: 2024-12-11

Locations

40 sites across 19 countries: United States, Australia, Canada, Chile, China, Greece, Hong Kong, Italy, Mexico, Netherlands, New Zealand, Norway, Russia, South Korea, Spain, Taiwan, Turkey (Türkiye), Ukraine, United Kingdom

Regulatory

• FDA-regulated drug study