Clinical Trials in Japan / NCT04768062
Active, not recruiting
Phase 3
Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)
NCT04768062 · tracked via the Priya Life Science Japan tracker
Condition(s) studied
Duchenne Muscular Dystrophy
Investigational drug(s) / intervention(s)
Viltolarsen: Received during weekly intravenous infusions
Study 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.
Eligibility
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.
Primary outcome measure(s)
- Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03 — baseline to up to 96 weeks of treatment
Trial sites (31)
| Facility | City | Region | Status |
|---|---|---|---|
| Queensland Children's Hospital | Brisbane | Australia | |
| The Childrens Hospital at Westmead | Westmead | Australia | |
| CHU de Quebec Research Centre | Québec | Canada | |
| Hospital de Niños Roberto del Rio | Santiago | Chile | |
| Pontificia Universidad Católica de Chile | Santiago | Chile | |
| Chinese PLA General Hospital | Beijing | China | |
| Hunan Children's Hospital | Changsha | China | |
| Children's Hospital of Fudan University | Shanghai | China | |
| Shenzhen Children's Hospital | Shenzhen | China | |
| Fakultni nemocnice Hradec Kralove | Nový Hradec Králové | Czechia | |
| Agia Sofia Children's Hospital | Athens | Greece | |
| Hippokration General Hospital of Thessaloniki | Thessaloniki | Greece | |
| Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore | Rome | Italy | |
| National Center of Neurology and Psychiatry | Tokyo | Japan | |
| Instituto Nacional de Pediatría | Mexico City | Mexico | |
| Leids Universitair Medisch Centrum | Leiden | Netherlands | |
| Radboud Universitair Medisch Centrum | Nijmegen | Netherlands | |
| New Zealand Clinical Research Ltd. | Auckland | New Zealand | |
| Rikshospitalet | Oslo | Norway | |
| Russian National Research Medical University | Moscow | Russia | |
| "Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre | Saint Petersburg | Russia | |
| Tomsk National Research Medical Center of Russian Academy of Sciences | Tomsk | Russia | |
| Pusan National University Yangsan Hospital | Pusan | South Korea | |
| Seoul National University Hospital | Seoul | South Korea | |
| Hospital Sant Joan de Deu | Barcelona | Spain | |
| Hospital Universitario La Paz | Madrid | Spain | |
| Yeditepe University Kosuyolu Hospital | Istanbul | Turkey (Türkiye) | |
| Birmingham Heartlands Hospital | Birmingham | United Kingdom | |
| Royal Hospital for Children | Glasgow | United Kingdom | |
| Royal Manchester Children's Hospital | Glasgow | United Kingdom | |
| University College London Institute of Child Health | London | United Kingdom |
More NS Pharma, Inc. trials in Japan
Trial of Efficacy and Safety of NS-229 Versus Placebo in Patients With Eosinophilic Granulomatosis With Polyangiitis
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NS-050/NCNP-03 in Boys With DMD (Meteor50)
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NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
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Other trials for the same condition
Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
Active, not recruiting
NS-050/NCNP-03 in Boys With DMD (Meteor50)
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A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
Enrolling by invitation
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
Active, not recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT04768062 on ClinicalTrials.gov ↗ ← All trials in Japan