Duchenne Muscular DystrophyPhase 3NCT04768062
Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)
A Phase 3 study testing Viltolarsen in people with Duchenne Muscular Dystrophy, run by NS Pharma, Inc. at 31 sites in 17 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Viltolarsen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 74 participants
- Where
- 31 sitesin 17 countries
- Study dates
- 13 Apr 2021to Nov 2025
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
31 sites in 17 countries.
31 sites in 17 countries
China4 sites
Chinese PLA General Hospital
Beijing
Hunan Children's Hospital
Changsha
Children's Hospital of Fudan University
Shanghai
Shenzhen Children's Hospital
Shenzhen
United Kingdom4 sites
Birmingham Heartlands Hospital
Birmingham
Royal Hospital for Children
Glasgow
Royal Manchester Children's Hospital
Glasgow
University College London Institute of Child Health
London
Russia3 sites
Russian National Research Medical University
Moscow
"Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre
Saint Petersburg
Tomsk National Research Medical Center of Russian Academy of Sciences
Tomsk
Australia2 sites
Queensland Children's Hospital
Brisbane
The Childrens Hospital at Westmead
Westmead
Chile2 sites
Hospital de Niños Roberto del Rio
Santiago
Pontificia Universidad Católica de Chile
Santiago
Greece2 sites
Agia Sofia Children's Hospital
Athens
Hippokration General Hospital of Thessaloniki
Thessaloniki
Netherlands2 sites
Leids Universitair Medisch Centrum
Leiden
Radboud Universitair Medisch Centrum
Nijmegen
South Korea2 sites
Pusan National University Yangsan Hospital
Pusan
Seoul National University Hospital
Seoul
Spain2 sites
Hospital Sant Joan de Deu
Barcelona
Hospital Universitario La Paz
Madrid
Italy1 site
Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore
Rome
Turkey (Türkiye)1 site
Yeditepe University Kosuyolu Hospital
Istanbul
About this study
Official title: 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.
Read the 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
- Being tested
- Viltolarsen
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ViltolarsenDrug
Also called NS-065/NCNP-01
Received during weekly intravenous infusions
| Study group | Role | What they receive |
|---|---|---|
| Viltolarsen | Experimental | Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 96 weeks. Receives: Drug: Viltolarsen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03
Measured: baseline to up to 96 weeks of treatment
Secondary outcomes (6)
Time to Stand Test (TTSTAND)
Change in Time to Stand
Measured: baseline to 96 weeks of treatment
Time to Run/Walk 10 Meters Test (TTRW)
Change in Time to Run/Walk 10 meters
Measured: baseline to 96 weeks of treatment
Six-minute Walk Test (6MWT)
Change in Six-minute Walk
Measured: baseline to 96 weeks of treatment
North Star Ambulatory Assessment (NSAA)
Change in North Star Ambulatory Assessment
Measured: baseline to 96 weeks of treatment
Time to Climb 4 Stairs Test (TTCLIMB)
Change in Time to Climb 4 Stairs
Measured: baseline to 96 weeks of treatment
Muscle Strength Measured by Hand-Held Dynamometer
Change in Muscle Strength Measured by Hand-Held Dynamometer
Measured: baseline to 96 weeks of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2021
- Study start13 Apr 2021
- Primary completionOct 2025 (estimated)
- Study completionNov 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 74 participants (actual)
Sponsor and investigators
- Lead sponsor
- NS Pharma, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Nippon Shinyaku Co., Ltd.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04768062
- Sponsor's protocol ID
- NS-065/NCNP-01-302
- Status last verified
- Feb 2024
- First submitted
- 19 Feb 2021
- Last updated
- 14 Feb 2024
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT04768062
- Acronym / sponsor ID
- NS-065/NCNP-01-302
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 74 participants (actual)
- Lead sponsor
- NS Pharma, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Nippon Shinyaku Co., Ltd.
- Study start
- 13 Apr 2021 (actual)
- Primary completion
- Oct 2025 (estimated)
- Study completion
- Nov 2025 (estimated)
- First posted
- 24 Feb 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Viltolarsen | Experimental | Drug: ViltolarsenPatients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 96 weeks. |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ViltolarsenAlso: NS-065/NCNP-01Received during weekly intravenous infusions | Drug | Viltolarsen |
Primary outcome
Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03
Time frame: baseline to up to 96 weeks of treatment
6 secondary outcomes
Time to Stand Test (TTSTAND)
Time frame: baseline to 96 weeks of treatment
Time to Run/Walk 10 Meters Test (TTRW)
Time frame: baseline to 96 weeks of treatment
Six-minute Walk Test (6MWT)
Time frame: baseline to 96 weeks of treatment
North Star Ambulatory Assessment (NSAA)
Time frame: baseline to 96 weeks of treatment
Time to Climb 4 Stairs Test (TTCLIMB)
Time frame: baseline to 96 weeks of treatment
Muscle Strength Measured by Hand-Held Dynamometer
Time frame: baseline to 96 weeks of treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Feb 2024.
- Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)Link provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
31 sites in 17 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Viltolarsen in people with Duchenne Muscular Dystrophy, run by NS Pharma, Inc. at 31 sites in 17 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 14 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 31 sites in China, United Kingdom, Russia, Australia, Chile and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03 (time frame: baseline to up to 96 weeks of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Feb 2024.