Status:
COMPLETED
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
Lead Sponsor:
NS Pharma, Inc.
Collaborating Sponsors:
Nippon Shinyaku Co., Ltd.
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
4-7 years
Phase:
PHASE3
Brief 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...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
April 14 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 19 2023
Estimated Enrollment :
77 Patients enrolled
Trial Details
Trial ID
NCT04060199
Start Date
April 14 2020
End Date
October 19 2023
Last Update
December 11 2024
Active Locations (40)
Enter a location and click search to find clinical trials sorted by distance.
1
University of California Davis Medical Center
Sacramento, California, United States, 95817
2
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
3
Queensland Children's Hospital
Brisbane, Australia
4
Perth Children's Hospital
Nedlands, Australia