Phase 3
N=77
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
Duchenne Muscular Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT04060199 ↗Enrolled (actual)
77
Serious AEs
6.5%
Results posted
Dec 2024
Primary outcome: Primary: Change From Baseline in Time to Stand (TTSTAND) Velocity — 0.026; 0.013; 0.027; 0.014 rise/s
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Viltolarsen (Drug); Placebo (Drug)
- Age
- Pediatric · 4+ yrs
- Sex
- Male
- Sponsor
- NS Pharma, Inc.
- Primary completion
- Oct 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Time to Stand (TTSTAND) Velocity |
0.026; 0.013; 0.027; 0.014; 0.027; 0.027 | — |
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.
Eligibility 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
Data sourced from ClinicalTrials.gov (NCT04060199). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.