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

NCT04060199 | Completed Phase 3 Results FDA Drug

• 1 other identifier: NS-065/NCNP-01-301
• Study Type: interventional
• Target: 77
• Locations: 19 countries
• Sites: 40

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.

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Time to Stand (TTSTAND) Velocity

  The change from baseline for velocity converted from TTSTAND was compared between the viltolarsen-treated patients and the placebo-treated patients. TTSTAND was assessed as the time it takes the participant to go from lying flat on the floor to standing. The time measured for TTSTAND was converted to a velocity expressed as rise per second.

  baseline, Week 13, 25, 37, 49

Study Arms (2)

Viltolarsen

EXPERIMENTAL

Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks.

Drug: Viltolarsen

Placebo

PLACEBO COMPARATOR

Patients amenable to exon 53 skipping will receive placebo intravenous (IV) infusions, weekly, for up to 48 weeks.

Drug: Placebo

Interventions

Viltolarsen DRUG

IV infusion

Also known as: NS-065/NCNP-01

Viltolarsen

Placebo DRUG

IV infusion

Placebo

Eligibility Criteria

• Age: 4 Years - 7 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17)

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

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
• 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

Sponsors & Collaborators

• NS Pharma, Inc.: lead
• Nippon Shinyaku Co., Ltd.: collaborator

Study Sites (40)

University of California Davis Medical Center

Sacramento, California, 95817, United States

Location

Ann and Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Queensland Children's Hospital

Brisbane, Australia

Location

Perth Children's Hospital

Nedlands, Australia

Location

The Childrens Hospital at Westmead

Westmead, Australia

Location

The Hospital for Sick Children (SickKids)

Toronto, Ontario, Canada

Location

Alberta Children's Hospital

Calgary, Canada

Location

CHU de Quebec Research Centre

Québec, Canada

Location

Hospital de Niños Roberto del Rio

Santiago, Chile

Location

Pontificia Universidad Católica de Chile

Santiago, Chile

Location

Chinese PLA General Hospital

Beijing, China

Location

The Third Medical Center of PLA General Hospital

Beijing, China

Location

Hunan Children's Hospital

Changsha, China

Location

Children's Hospital of Fudan University

Shanghai, China

Location

Shenzhen Children's Hospital

Shenzhen, China

Location

Agia Sofia Children's Hospital

Athens, Greece

Location

Hippokration General Hospital of Thessaloniki

Thessaloniki, Greece

Location

Hong Kong Children's Hospital

Kowloon Bay, Hong Kong

Location

Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore

Rome, Italy

Location

Hospital Angeles Chihuahua

Chihuahua City, Mexico

Location

Instituto Nacional de Pediatria

Mexico City, Mexico

Location

Radboud Universitair Medisch Centrum

Nijmegen, Gelderland, Netherlands

Location

Leids Universitair Medisch Centrum

Leiden, Netherlands

Location

New Zealand Clinical Research Ltd

Auckland, New Zealand

Location

Rikshospitalet

Oslo, Norway

Location

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

Location

"Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre

Saint Petersburg, Russia

Location

Tomsk National Research Medical Center of Russian Academy of Sciences

Tomsk, Russia

Location

Pusan National University Yangsan Hospital

Pusan, South Korea

Location

Seoul National University Hospital

Seoul, South Korea

Location

Hospital Sant Joan de Deu

Barcelona, Spain

Location

Hospital Universitario La Paz

Madrid, Spain

Location

Kaohsiung Medical University Chung-Ho Memorial Hospital

Kaohsiung City, Taiwan

Location

National Taiwan University Hospital

Taipei, Taiwan

Location

Yeditepe University Kosuyolu Hospital

Istanbul, Turkey (Türkiye)

Location

State Institution "Ukrainian Medical rehabilitation Center for Children with organic disorders of the nervous system of the Ministry of Health of Ukraine"

Kyiv, Ukraine

Location

Birmingham Heartlands Hospital

Birmingham, United Kingdom

Location

Royal Hospital for Children - Glasgow

Glasgow, United Kingdom

Location

University College London Institute of Child Health

London, United Kingdom

Location

Royal Manchester Children's Hospital

Manchester, United Kingdom

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

viltolarsen

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Medical Affairs
• Organization: NS Pharma, Inc.

trialinfo@nspharma.com

+1(201) 986-3860

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 15, 2019
• First Posted: August 19, 2019
• Study Start: April 14, 2020
• Primary Completion: October 19, 2023
• Study Completion: October 19, 2023
• Last Updated: December 11, 2024
• Results First Posted: December 11, 2024

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

HK (1); GR (2); AU (3); CL (2); ME (2); IT (1); KR (2); NL (2); ES (2); TU (1); CN (5); UA (1); US (2); RU (3); CA (3); TW (2); GB (4); NZ (1); NO (1)