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

Phase 3

Active, not recruiting

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Drug: Viltolarsen

• Registration Number: NCT04768062
• Lead Sponsor: NS Pharma, Inc.

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.

Study Timeline

• Study First Submitted: 2021-02-19
• Study First Submitted QC: 2021-02-23
• Study First Posted: 2021-02-24
• Study Start: 2021-04-13
• Status Verified: 2024-02
• Last Update Submitted: 2024-02-13
• Last Update Posted: 2024-02-14
• Primary Completion: 2025-10
• Study Completion: 2025-11

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 74

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03	baseline to up to 96 weeks of treatment

Secondary Outcome Measures

Name	Time	Method
Six-minute Walk Test (6MWT)	baseline to 96 weeks of treatment	Change in Six-minute Walk
Time to Run/Walk 10 Meters Test (TTRW)	baseline to 96 weeks of treatment	Change in Time to Run/Walk 10 meters
Time to Climb 4 Stairs Test (TTCLIMB)	baseline to 96 weeks of treatment	Change in Time to Climb 4 Stairs
Time to Stand Test (TTSTAND)	baseline to 96 weeks of treatment	Change in Time to Stand
North Star Ambulatory Assessment (NSAA)	baseline to 96 weeks of treatment	Change in North Star Ambulatory Assessment
Muscle Strength Measured by Hand-Held Dynamometer	baseline to 96 weeks of treatment	Change in Muscle Strength Measured by Hand-Held Dynamometer

Trial Locations

Locations (31)

Queensland Children's Hospital; 🇦🇺 Brisbane, Australia

Birmingham Heartlands Hospital; 🇬🇧 Birmingham, United Kingdom

Royal Hospital for Children; 🇬🇧 Glasgow, United Kingdom

Yeditepe University Kosuyolu Hospital; 🇹🇷 Istanbul, Turkey

CHU de Quebec Research Centre; 🇨🇦 Quebec City, Canada

Hunan Children's Hospital; 🇨🇳 Changsha, China

Fakultni nemocnice Hradec Kralove; 🇨🇿 Nový Hradec Králové, Czechia

Royal Manchester Children's Hospital; 🇬🇧 Glasgow, United Kingdom

University College London Institute of Child Health; 🇬🇧 London, United Kingdom

Hospital Universitario La Paz; 🇪🇸 Madrid, Spain

New Zealand Clinical Research Ltd.; 🇳🇿 Auckland, New Zealand

The Childrens Hospital at Westmead; 🇦🇺 Westmead, Australia

Pontificia Universidad Católica de Chile; 🇨🇱 Santiago, Chile

National Center of Neurology and Psychiatry; 🇯🇵 Tokyo, Japan

Hospital Sant Joan de Deu; 🇪🇸 Barcelona, Spain

Pusan National University Yangsan Hospital; 🇰🇷 Pusan, Korea, Republic of

Seoul National University Hospital; 🇰🇷 Seoul, Korea, Republic of

Chinese PLA General Hospital; 🇨🇳 Beijing, China

Shenzhen Children's Hospital; 🇨🇳 Shenzhen, China

Leids Universitair Medisch Centrum; 🇳🇱 Leiden, Netherlands

Children's Hospital of Fudan University; 🇨🇳 Shanghai, China

Instituto Nacional de Pediatría; 🇲🇽 Ciudad de mexico, Mexico

Rikshospitalet; 🇳🇴 Oslo, Norway

Hospital de Niños Roberto del Rio; 🇨🇱 Santiago, Chile

Agia Sofia Children's Hospital; 🇬🇷 Athens, Greece

Hippokration General Hospital of Thessaloniki; 🇬🇷 Thessaloníki, Greece

Radboud Universitair Medisch Centrum; 🇳🇱 Nijmegen, Netherlands

"Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre; 🇷🇺 Saint Petersburg, Russian Federation

Tomsk National Research Medical Center of Russian Academy of Sciences; 🇷🇺 Tomsk, Russian Federation

Russian National Research Medical University; 🇷🇺 Moscow, Russian Federation

Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore; 🇮🇹 Rome, Italy