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

NCT ID: NCT04768062

Last Updated: 2024-02-14

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 74 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-04-13
• Study Completion Date: 2025-11-30

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.

Detailed Description

This Phase 3 study is a 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. Patients will receive viltolarsen administered IV at weekly doses of 80 mg/kg.

Study NS-065/NCNP-01-302 will be comprised of a 96-week treatment period.

Conditions

Duchenne Muscular Dystrophy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Viltolarsen

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

Group Type: EXPERIMENTAL

Viltolarsen

Intervention Type: DRUG

Received during weekly intravenous infusions

Interventions

Viltolarsen

Received during weekly intravenous infusions

Intervention Type: DRUG

Other Intervention Names

NS-065/NCNP-01

Eligibility Criteria

Inclusion Criteria

1. Patient has completed the NS-065/NCNP-01-301 study;

2. 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;

3. Patient and parent(s)/guardian(s) are willing and able to comply with scheduled visits, investigational product (IP) administration plan, and study procedures.

Exclusion Criteria

1. 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;

2. 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;

3. Patient took any other investigational drug(s) during or after completion of Study NS-065/NCNP-01-301;

4. Patient is judged by the investigator and/or the sponsor not to be appropriate to participate in the extension study for any reason.

• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Nippon Shinyaku Co., Ltd.

INDUSTRY

Sponsor Role: collaborator

NS Pharma, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Queensland Children's Hospital

Brisbane, , Australia

The Childrens Hospital at Westmead

Westmead, , Australia

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

Hunan Children's Hospital

Changsha, , China

Children's Hospital of Fudan University

Shanghai, , China

Shenzhen Children's Hospital

Shenzhen, , China

Fakultni nemocnice Hradec Kralove

Nový Hradec Králové, , Czechia

Agia Sofia Children's Hospital

Athens, , Greece

Hippokration General Hospital of Thessaloniki

Thessaloniki, , Greece

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

Rome, , Italy

National Center of Neurology and Psychiatry

Tokyo, , Japan

Instituto Nacional de Pediatría

Mexico City, , Mexico

Leids Universitair Medisch Centrum

Leiden, , Netherlands

Radboud Universitair Medisch Centrum

Nijmegen, , Netherlands

New Zealand Clinical Research Ltd.

Auckland, , New Zealand

Rikshospitalet

Oslo, , Norway

Russian National Research Medical University

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

Yeditepe University Kosuyolu Hospital

Istanbul, , Turkey (Türkiye)

Birmingham Heartlands Hospital

Birmingham, , United Kingdom

Royal Hospital for Children

Glasgow, , United Kingdom

Royal Manchester Children's Hospital

Glasgow, , United Kingdom

University College London Institute of Child Health

London, , United Kingdom

Countries

Australia; Canada; Chile; China; Czechia; Greece; Italy; Japan; Mexico; Netherlands; New Zealand; Norway; Russia; South Korea; Spain; Turkey (Türkiye); United Kingdom

Related Links

https://clinicaltrials.gov/ct2/show/NCT04060199

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

Other Identifiers

NS-065/NCNP-01-302

Identifier Type: -

Identifier Source: org_study_id