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

Completed · Phase 3 · Has a placebo group

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT04060199

Run by

NS Pharma, Inc.

People needed

77

Starts

2020-04-14

Expected to finish

2023-10-19

Last updated by the study team

2024-12-11

Who can join

Age: 4 and older, up to 7. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• University of California Davis Medical Center — Sacramento, California, United States
• Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• Queensland Children's Hospital — Brisbane, Australia
• Perth Children's Hospital — Nedlands, Australia
• The Childrens Hospital at Westmead — Westmead, Australia
• The Hospital for Sick Children (SickKids) — Toronto, Ontario, Canada
• Alberta Children's Hospital — Calgary, Canada
• CHU de Quebec Research Centre — Québec, Canada
• Hospital de Niños Roberto del Rio — Santiago, Chile
• Pontificia Universidad Católica de Chile — Santiago, Chile
• Chinese PLA General Hospital — Beijing, China
• The Third Medical Center of PLA General Hospital — Beijing, China
• Hunan Children's Hospital — Changsha, China
• Children's Hospital of Fudan University — Shanghai, China
• Shenzhen Children's Hospital — Shenzhen, China
• Agia Sofia Children's Hospital — Athens, Greece
• Hippokration General Hospital of Thessaloniki — Thessaloniki, Greece
• Hong Kong Children's Hospital — Kowloon Bay, Hong Kong
• Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore — Rome, Italy
• Hospital Angeles Chihuahua — Chihuahua City, Mexico
• Instituto Nacional de Pediatria — Mexico City, Mexico
• Radboud Universitair Medisch Centrum — Nijmegen, Gelderland, Netherlands
• Leids Universitair Medisch Centrum — Leiden, Netherlands
• New Zealand Clinical Research Ltd — Auckland, New Zealand
• Rikshospitalet — Oslo, Norway

Full record on ClinicalTrials.gov

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