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

• Status: Recruiting
• Trial end date: 2024-12-01
• Target enrollment: 0
• Participant gender: Male

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: NS Pharma, Inc.
• Collaborator: Nippon Shinyaku Co., Ltd.

Criteria

Inclusion Criteria:

• Male ≥ 4 years and < 8 years of age

• Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin mRNA reading frame

• Able to walk independently without assistive devices

• TTSTAND < 10 seconds

• Stable dose of glucocorticoid (GC) for at least 3 months prior to study entry and is expected to remain on stable dose of GC treatment for the duration of the study

• Other inclusion criteria may apply

Exclusion Criteria:

• Current or history of chronic systemic fungal or viral infections

• Acute illness within 4 weeks prior to the first dose of study drug

• Evidence of symptomatic cardiomyopathy (Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary)

• Allergy or hypersensitivity to the study drug or to any of its constituents

• Severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator

• Previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the investigator;

• Surgery within the 3 months prior to the first dose of study drug or surgery is planned for anytime during the duration of the study

• Participant has positive test results for hepatitis B antigen, hepatitis C antibody or human immunodeficiency virus (HIV)

• Currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of study drug or within 5 times the half-life of a medication, whichever is longer

• Previously enrolled in an interventional study of viltolarsen

• Currently taking any other exon skipping agent or has taken any other exon skipping agent within 3 months prior to the first dose of study drug

• Having taken any gene therapy

• Other exclusion criteria may apply