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

• Register: ClinicalTrials.gov
• Last refreshed on: 6 November 2023
• Main ID: NCT04060199
• Date of registration: 15/08/2019
• Prospective Registration: Yes
• Primary sponsor: NS Pharma, Inc.
• Public title: Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
• Scientific title: 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)
• Date of first enrolment: April 14, 2020
• Target sample size: 77
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT04060199
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Australia, Canada, Chile, China, Greece, Hong Kong, Italy, Japan, Korea, Republic of, Mexico, Netherlands, New Zealand, Norway, Russian Federation, Spain, Sweden, Taiwan, Turkey, Ukraine, United Kingdom, United States

Key inclusion & exclusion 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



Age minimum: 4 Years
Age maximum: 7 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Placebo

Drug: Viltolarsen

Primary Outcome(s)

TTSTAND [Time Frame: baseline to 48 weeks of treatment]

Secondary Outcome(s)

Hand-held dynamometer [Time Frame: baseline to 48 weeks of treatment]

NSAA [Time Frame: baseline to 48 weeks of treatment]

6MWT [Time Frame: baseline to 48 weeks of treatment]

TTCLIMB [Time Frame: baseline to 48 weeks of treatment]

TTRW [Time Frame: baseline to 48 weeks of treatment]

Secondary ID(s)

NS-065/NCNP-01-301

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Nippon Shinyaku Co., Ltd.