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

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
74

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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 Católica de Chile
      • Santiago, Chile
        • Hospital de Niños Roberto del Río
  • 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
        • Fakultni nemocnice Hradec Kralove
  • 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 Pediatría
  • 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 Déu
      • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
NS Pharma, Inc.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 13, 2021

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 1, 2025

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 1, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 19, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 23, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 24, 2021

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
February 14, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 13, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
February 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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