Ireland
--:--IST
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 UK tracker
Phase
Phase 3
Started
2021-04-13
Last updated
2024-02-14

Condition(s) studied

Duchenne Muscular Dystrophy

Investigational drug(s) / intervention(s)

Viltolarsen →

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

Sex
MALE
Min age
—
Max age
—
Healthy volunteers
No
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)

FacilityCityRegionStatus
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
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 the UK