Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
NCT ID: NCT04060199
Last Updated: 2024-12-11
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
77 participants
INTERVENTIONAL
2020-04-14
2023-10-19
Brief Summary
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Detailed Description
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The study will enroll approximately 74 patients amenable to exon 53 skipping. Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as Time to Stand Test (TTSTAND), Time to Run/Walk 10 Meters Test (TTRW), Six-minute Walk Test (6MWT), North Star Ambulatory Assessment (NSAA), Time to Climb 4 Steps Test (TTCLIMB) and Hand-held dynamometer (elbow extension, elbow flexion, knee extension and knee flexion on the dominant side only).
Safety will be assessed through the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), vital signs, and physical examinations throughout the study.
Blood samples will be taken periodically throughout the study to assess the pharmacokinetics of study drug.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Viltolarsen
Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks.
Viltolarsen
IV infusion
Placebo
Patients amenable to exon 53 skipping will receive placebo intravenous (IV) infusions, weekly, for up to 48 weeks.
Placebo
IV infusion
Interventions
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Viltolarsen
IV infusion
Placebo
IV infusion
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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
4 Years
7 Years
MALE
No
Sponsors
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Nippon Shinyaku Co., Ltd.
INDUSTRY
NS Pharma, Inc.
INDUSTRY
Responsible Party
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Locations
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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
Russian National Research Medical University n.a. N.I.Pirogov, structural branch - Research Clinical Institute of Pediatrics n.a. Academician Yu. E. Veltishchev
Moscow, , Russia
"Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre
Saint Petersburg, , Russia
Tomsk National Research Medical Center of Russian Academy of Sciences
Tomsk, , Russia
Pusan National University Yangsan Hospital
Pusan, , South Korea
Seoul National University Hospital
Seoul, , South Korea
Hospital Sant Joan de Deu
Barcelona, , Spain
Hospital Universitario La Paz
Madrid, , Spain
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, , Taiwan
National Taiwan University Hospital
Taipei, , Taiwan
Yeditepe University Kosuyolu Hospital
Istanbul, , Turkey (Türkiye)
State Institution "Ukrainian Medical rehabilitation Center for Children with organic disorders of the nervous system of the Ministry of Health of Ukraine"
Kyiv, , Ukraine
Birmingham Heartlands Hospital
Birmingham, , United Kingdom
Royal Hospital for Children - Glasgow
Glasgow, , United Kingdom
University College London Institute of Child Health
London, , United Kingdom
Royal Manchester Children's Hospital
Manchester, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Other Identifiers
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NS-065/NCNP-01-301
Identifier Type: -
Identifier Source: org_study_id