RACER-53X
Research type
Research Study
Full 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)
IRAS ID
1004329
Contact name
Amin Boateng
Contact email
Sponsor organisation
NS Pharma, Inc.
Eudract number
2021-000122-10
Clinicaltrials.gov Identifier
Research summary
DMD is a disorder of progressive weakness leading to severe disability and ultimately death caused by a deficiency of the dystrophin protein. The symptoms of DMD are often first noted at about 3 to 5 years of age.
Boys who completed NS-065/NCNP-01-301 study will receive infusions of viltolarsen injection or intravenously (IV) once weekly for 96 weeks. Participants will be dosed at 80 mg/kg/ week.
This is an open-label study this means that study participants and researchers both know which treatment the patient is receiving. it is anticipated that approximately 74 participants will will enter this study whom have previously completed the NS-065/NCNP-01-301 study.
This study is sponsored by by NS Pharma and this study will be conducted across Europe, Asia, and North and south America.REC name
East of England - Cambridge South Research Ethics Committee
REC reference
21/EE/0259
Date of REC Opinion
19 Jan 2022
REC opinion
Further Information Favourable Opinion