Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)

PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

74

Participants

Timeline

Start Date

April 13, 2021

Primary Completion Date

October 31, 2025

Study Completion Date

November 30, 2025

Conditions
Duchenne Muscular Dystrophy
Interventions
DRUG

Viltolarsen

Received during weekly intravenous infusions

Trial Locations (31)

Unknown

Queensland Children's Hospital, Brisbane

The Childrens Hospital at Westmead, Westmead

CHU de Quebec Research Centre, Québec

Hospital de Niños Roberto del Rio, Santiago

Pontificia Universidad Católica de Chile, Santiago

Chinese PLA General Hospital, Beijing

Hunan Children's Hospital, Changsha

Children's Hospital of Fudan University, Shanghai

Shenzhen Children's Hospital, Shenzhen

Fakultni nemocnice Hradec Kralove, Nový Hradec Králové

Agia Sofia Children's Hospital, Athens

Hippokration General Hospital of Thessaloniki, Thessaloniki

Fondazione Policlinico Universitario A. Gemelli - Universita Cattolica del Sacro Cuore, Rome

National Center of Neurology and Psychiatry, Tokyo

Instituto Nacional de Pediatría, Mexico City

Leids Universitair Medisch Centrum, Leiden

Radboud Universitair Medisch Centrum, Nijmegen

New Zealand Clinical Research Ltd., Auckland

Rikshospitalet, Oslo

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

Pusan National University Yangsan Hospital, Pusan

Seoul National University Hospital, Seoul

Hospital Sant Joan de Deu, Barcelona

Hospital Universitario La Paz, Madrid

Yeditepe University Kosuyolu Hospital, Istanbul

Birmingham Heartlands Hospital, Birmingham

Royal Hospital for Children, Glasgow

Royal Manchester Children's Hospital, Glasgow

University College London Institute of Child Health, London

Sponsors

Lead Sponsor

Collaborators (1)

All Listed Sponsors
collaborator

Nippon Shinyaku Co., Ltd.

INDUSTRY

lead

NS Pharma, Inc.

INDUSTRY

NCT04768062 - Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X) | Biotech Hunter | Biotech Hunter