Duchenne Muscular DystrophyPhase 3NCT04060199
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
A Phase 3 study testing Viltolarsen in people with Duchenne Muscular Dystrophy, run by NS Pharma, Inc. at 40 sites in 19 countries.
Trial summary
The main objective of this study is to evaluate the efficacy of Viltolarsen compared to placebo in Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping. Read more about this study
- Who is it for?
- Ages 4 to 7 years, 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?
- Yes, some participants may receive a placebo
- How many people
- 77 participants
- Where
- 40 sitesin 19 countries
- Study dates
- 14 Apr 2020to 19 Oct 2023
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 4 to 7 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Male ≥ 4 years and < 8 years of age
- Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin mRNA reading frame
- Able to walk independently without assistive devices
- TTSTAND < 10 seconds
- Stable dose of glucocorticoid (GC) for at least 3 months prior to study entry and is expected to remain on stable dose of GC treatment for the duration of the study
- Other inclusion criteria may apply
Who cannot join
- Current or history of chronic systemic fungal or viral infections
- Acute illness within 4 weeks prior to the first dose of study drug
- Evidence of symptomatic cardiomyopathy (Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary)
- Allergy or hypersensitivity to the study drug or to any of its constituents
- Severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator
- Previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the investigator;
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- Surgery within the 3 months prior to the first dose of study drug or surgery is planned for anytime during the duration of the study
- Participant has positive test results for hepatitis B antigen, hepatitis C antibody or human immunodeficiency virus (HIV)
- Currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of study drug or within 5 times the half-life of a medication, whichever is longer
- Previously enrolled in an interventional study of viltolarsen
- Currently taking any other exon skipping agent or has taken any other exon skipping agent within 3 months prior to the first dose of study drug
- Having taken any gene therapy
- Other exclusion criteria may apply
Trial locations and contacts
40 sites in 19 countries.
40 sites in 19 countries
China5 sites
Chinese PLA General Hospital
Beijing
The Third Medical Center of 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
Glasgow
University College London Institute of Child Health
London
Royal Manchester Children's Hospital
Manchester
Australia3 sites
Queensland Children's Hospital
Brisbane
Perth Children's Hospital
Nedlands
The Childrens Hospital at Westmead
Westmead
Canada3 sites
Alberta Children's Hospital
Calgary
CHU de Quebec Research Centre
Québec
The Hospital for Sick Children (SickKids)
Toronto, Ontario
Russia3 sites
Russian National Research Medical University n.a. N.I.Pirogov, structural branch - Research Clinical Institute of Pediatrics n.a. Academician Yu. E. Veltishchev
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
United States2 sites
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
University of California Davis Medical Center
Sacramento, California, 95817
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
Mexico2 sites
Hospital Angeles Chihuahua
Chihuahua City
Instituto Nacional de Pediatria
Mexico City
Netherlands2 sites
Leids Universitair Medisch Centrum
Leiden
Radboud Universitair Medisch Centrum
Nijmegen, Gelderland
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
Taiwan2 sites
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City
National Taiwan University Hospital
Taipei
Italy1 site
Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore
Rome
Turkey (Türkiye)1 site
Yeditepe University Kosuyolu Hospital
Istanbul
Ukraine1 site
State Institution "Ukrainian Medical rehabilitation Center for Children with organic disorders of the nervous system of the Ministry of Health of Ukraine"
Kyiv
About this study
Official title: A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)
The main objective of this study is to evaluate the efficacy of Viltolarsen compared to placebo in Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.
Read the detailed description
This is a Phase 3 randomized, double-blind, placebo-controlled, multi-center study to assess the efficacy and safety of Viltolarsen in ambulant boys with Duchenne muscular dystrophy. Eligible patients with out-of-frame deletion mutations amenable to exon 53 skipping will be randomized to receive once weekly intravenous (IV) infusions of 80 mg/kg Viltolarsen or placebo for up to 48 weeks.
The study will enroll approximately 74 patients amenable to exon 53 skipping. Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as Time to Stand Test (TTSTAND), Time to Run/Walk 10 Meters Test (TTRW), Six-minute Walk Test (6MWT), North Star Ambulatory Assessment (NSAA), Time to Climb 4 Steps Test (TTCLIMB) and Hand-held dynamometer (elbow extension, elbow flexion, knee extension and knee flexion on the dominant side only).
Safety will be assessed through the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), vital signs, and physical examinations throughout the study.
Blood samples will be taken periodically throughout the study to assess the pharmacokinetics of study drug.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Viltolarsen, Placebo
- 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
IV infusion
PlaceboDrug
IV infusion
| 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 48 weeks. Receives: Drug: Viltolarsen |
| Placebo | Placebo comparator | Patients amenable to exon 53 skipping will receive placebo intravenous (IV) infusions, weekly, for up to 48 weeks. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Time to Stand (TTSTAND) Velocity
The change from baseline for velocity converted from TTSTAND was compared between the viltolarsen-treated patients and the placebo-treated patients. TTSTAND was assessed as the time it takes the participant to go from lying flat on the floor to standing. The time measured for TTSTAND was converted to a velocity expressed as rise per second.
Measured: baseline, Week 13, 25, 37, 49
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2019
- Study start14 Apr 2020
- Primary completion19 Oct 2023
- Study completion19 Oct 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 77 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
- NCT04060199
- Sponsor's protocol ID
- NS-065/NCNP-01-301
- Status last verified
- Oct 2024
- First submitted
- 15 Aug 2019
- Last updated
- 11 Dec 2024
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)
- ClinicalTrials.gov ID
- NCT04060199
- Acronym / sponsor ID
- NS-065/NCNP-01-301
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 77 participants (actual)
- Lead sponsor
- NS Pharma, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Nippon Shinyaku Co., Ltd.
- Study start
- 14 Apr 2020 (actual)
- Primary completion
- 19 Oct 2023 (actual)
- Study completion
- 19 Oct 2023 (actual)
- First posted
- 19 Aug 2019 (actual)
- Results
- Posted 11 Dec 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main objective of this study is to evaluate the efficacy of Viltolarsen compared to placebo in Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.
Detailed description
This is a Phase 3 randomized, double-blind, placebo-controlled, multi-center study to assess the efficacy and safety of Viltolarsen in ambulant boys with Duchenne muscular dystrophy. Eligible patients with out-of-frame deletion mutations amenable to exon 53 skipping will be randomized to receive once weekly intravenous (IV) infusions of 80 mg/kg Viltolarsen or placebo for up to 48 weeks.
The study will enroll approximately 74 patients amenable to exon 53 skipping. Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as Time to Stand Test (TTSTAND), Time to Run/Walk 10 Meters Test (TTRW), Six-minute Walk Test (6MWT), North Star Ambulatory Assessment (NSAA), Time to Climb 4 Steps Test (TTCLIMB) and Hand-held dynamometer (elbow extension, elbow flexion, knee extension and knee flexion on the dominant side only).
Safety will be assessed through the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), vital signs, and physical examinations throughout the study.
Blood samples will be taken periodically throughout the study to assess the pharmacokinetics of study drug.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| 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 48 weeks. |
| Placebo | Placebo comparator | Drug: PlaceboPatients amenable to exon 53 skipping will receive placebo intravenous (IV) infusions, weekly, for up to 48 weeks. |
Who may qualify
- Ages
- 4 to 7 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Male ≥ 4 years and < 8 years of age
- Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin mRNA reading frame
- Able to walk independently without assistive devices
- TTSTAND < 10 seconds
- Stable dose of glucocorticoid (GC) for at least 3 months prior to study entry and is expected to remain on stable dose of GC treatment for the duration of the study
Key exclusion criteria (13 listed)
- Current or history of chronic systemic fungal or viral infections
- Acute illness within 4 weeks prior to the first dose of study drug
- Evidence of symptomatic cardiomyopathy (Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary)
- Allergy or hypersensitivity to the study drug or to any of its constituents
- Severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator
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-01IV infusion | Drug | Viltolarsen |
| PlaceboIV infusion | Drug | Placebo |
Primary outcome
Change From Baseline in Time to Stand (TTSTAND) Velocity
Time frame: baseline, Week 13, 25, 37, 49
The change from baseline for velocity converted from TTSTAND was compared between the viltolarsen-treated patients and the placebo-treated patients. TTSTAND was assessed as the time it takes the participant to go from lying flat on the floor to standing. The time measured for TTSTAND was converted to a velocity expressed as rise per second.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Dec 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
40 sites in 19 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 40 sites in 19 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 6 inclusion and 13 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: finished, as of the sponsor's last update on 11 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 40 sites in China, United Kingdom, Australia, Canada, Russia and 14 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. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 11 Dec 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change From Baseline in Time to Stand (TTSTAND) Velocity (time frame: baseline, Week 13, 25, 37, 49). 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 11 Dec 2024.