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

NCT04768062 | Active Not Recruiting Phase 3 FDA Drug

• 1 other identifier: NS-065/NCNP-01-302
• Study Type: interventional
• Target: 74
• Locations: 17 countries
• Sites: 31

Brief Summary

This is a Phase 3, multi-center, open-label extension study in ambulant boys with DMD who have completed the 48-week treatment period of either viltolarsen or placebo in Study NS-065/NCNP-01-301.

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

• Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03

  baseline to up to 96 weeks of treatment

Secondary Outcomes (6)

• Time to Stand Test (TTSTAND)

  baseline to 96 weeks of treatment

• Time to Run/Walk 10 Meters Test (TTRW)

  baseline to 96 weeks of treatment

• Six-minute Walk Test (6MWT)

  baseline to 96 weeks of treatment

• North Star Ambulatory Assessment (NSAA)

  baseline to 96 weeks of treatment

• Time to Climb 4 Stairs Test (TTCLIMB)

  baseline to 96 weeks of treatment

Study Arms (1)

Viltolarsen

EXPERIMENTAL

Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 96 weeks.

Drug: Viltolarsen

Interventions

Viltolarsen DRUG

Received during weekly intravenous infusions

Also known as: NS-065/NCNP-01

Viltolarsen

Eligibility Criteria

• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient has completed the NS-065/NCNP-01-301 study;
• Patient's parent(s) or legal guardian(s) has (have) provided written informed consent and Health Insurance Portability and Accountability Act authorization, where applicable, prior to any study-related procedures; patients will be asked to give written or verbal assent according to local requirements;
• Patient and parent(s)/guardian(s) are willing and able to comply with scheduled visits, investigational product (IP) administration plan, and study procedures.

You may not qualify if:

• Patient had an adverse event in Study NS-065/NCNP-01-301 that, in the opinion of the investigator and/or the sponsor, precludes safe use of viltolarsen for the patient in this study;
• Patient had a treatment which was made for the purpose of dystrophin or dystrophin-related protein induction after completion of Study NS-065/NCNP-01-301;
• Patient took any other investigational drug(s) during or after completion of Study NS-065/NCNP-01-301;
• Patient is judged by the investigator and/or the sponsor not to be appropriate to participate in the extension study for any reason.

Sponsors & Collaborators

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

Study Sites (31)

Queensland Children's Hospital

Brisbane, Australia

Location

The Childrens Hospital at Westmead

Westmead, Australia

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

Hunan Children's Hospital

Changsha, China

Location

Children's Hospital of Fudan University

Shanghai, China

Location

Shenzhen Children's Hospital

Shenzhen, China

Location

Fakultni nemocnice Hradec Kralove

Nový Hradec Králové, Czechia

Location

Agia Sofia Children's Hospital

Athens, Greece

Location

Hippokration General Hospital of Thessaloniki

Thessaloniki, Greece

Location

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

Rome, Italy

Location

National Center of Neurology and Psychiatry

Tokyo, Japan

Location

Instituto Nacional de Pediatría

Mexico City, Mexico

Location

Leids Universitair Medisch Centrum

Leiden, Netherlands

Location

Radboud Universitair Medisch Centrum

Nijmegen, Netherlands

Location

New Zealand Clinical Research Ltd.

Auckland, New Zealand

Location

Rikshospitalet

Oslo, Norway

Location

Russian National Research Medical University

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

Yeditepe University Kosuyolu Hospital

Istanbul, Turkey (Türkiye)

Location

Birmingham Heartlands Hospital

Birmingham, United Kingdom

Location

Royal Hospital for Children

Glasgow, United Kingdom

Location

Royal Manchester Children's Hospital

Glasgow, United Kingdom

Location

University College London Institute of Child Health

London, United Kingdom

Location

Related Links

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

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 19, 2021
• First Posted: February 24, 2021
• Study Start: April 13, 2021
• Primary Completion: October 1, 2025
• Study Completion: November 1, 2025
• Last Updated: February 14, 2024

Record last verified: 2024-02

Locations

RU (3); ES (2); TU (1); CL (2); KR (2); CA (1); CN (4); IT (1); ME (1); GB (4); JP (1); NZ (1); NL (2); AU (2); CZ (1); GR (2); NO (1)