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

February 13, 2024 updated by: NS Pharma, Inc.

A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

74

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Brisbane, Australia
        • Queensland Children's Hospital
      • Westmead, Australia
        • The Childrens Hospital at Westmead
  • Canada
      • Quebec City, Canada
        • CHU de Quebec Research Centre
  • Chile
      • Santiago, Chile
        • Pontificia Universidad Catolica de Chile
      • Santiago, Chile
        • Hospital de Niños Roberto del Rio
  • China
      • Beijing, China
        • Chinese PLA General hospital
      • Changsha, China
        • Hunan Children's Hospital
      • Shanghai, China
        • Children's Hospital of Fudan University
      • Shenzhen, China
        • Shenzhen Children's Hospital
  • Czechia
      • Nový Hradec Králové, Czechia
        • Fakultní nemocnice Hradec Králové
  • Greece
      • Athens, Greece
        • AGIA SOFIA Children's Hospital
      • Thessaloníki, Greece
        • Hippokration General Hospital of Thessaloniki
  • Italy
      • Rome, Italy
        • Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore
  • Japan
      • Tokyo, Japan
        • National Center of Neurology and Psychiatry
  • Korea, Republic of
      • Pusan, Korea, Republic of
        • Pusan National University Yangsan Hospital
      • Seoul, Korea, Republic of
        • Seoul National University Hospital
  • Mexico
      • Ciudad de mexico, Mexico
        • Instituto Nacional de Pediatria
  • Netherlands
      • Leiden, Netherlands
        • Leids Universitair Medisch Centrum
      • Nijmegen, Netherlands
        • Radboud Universitair Medisch Centrum
  • New Zealand
      • Auckland, New Zealand
        • New Zealand Clinical Research Ltd.
  • Norway
      • Oslo, Norway
        • Rikshospitalet
  • Russian Federation
      • Moscow, Russian Federation
        • Russian National Research Medical University
      • Saint Petersburg, Russian Federation
        • "Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre
      • Tomsk, Russian Federation
        • Tomsk National Research Medical Center of Russian Academy of Sciences
  • Spain
      • Barcelona, Spain
        • Hospital Sant Joan de Deu
      • Madrid, Spain
        • Hospital Universitario La Paz
  • Turkey
      • Istanbul, Turkey
        • Yeditepe University Kosuyolu Hospital
  • United Kingdom
      • Birmingham, United Kingdom
        • Birmingham Heartlands Hospital
      • Glasgow, United Kingdom
        • Royal Hospital for Children
      • Glasgow, United Kingdom
        • Royal Manchester Children's Hospital
      • London, United Kingdom
        • University College London Institute of Child Health

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Viltolarsen
Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 96 weeks.
Drug: Viltolarsen
Received during weekly intravenous infusions
Other Names:
  • NS-065/NCNP-01

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03
Time Frame: baseline to up to 96 weeks of treatment
baseline to up to 96 weeks of treatment

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

NS Pharma, Inc.

Collaborators

Nippon Shinyaku Co., Ltd.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 13, 2021

Primary Completion (Estimated)

October 1, 2025

Study Completion (Estimated)

November 1, 2025

Study Registration Dates

First Submitted

February 19, 2021

First Submitted That Met QC Criteria

February 23, 2021

First Posted (Actual)

February 24, 2021

Study Record Updates

Last Update Posted (Estimated)

February 14, 2024

Last Update Submitted That Met QC Criteria

February 13, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NS-065/NCNP-01-302

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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