Status:
ACTIVE_NOT_RECRUITING
Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)
Lead Sponsor:
NS Pharma, Inc.
Collaborating Sponsors:
Nippon Shinyaku Co., Ltd.
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
Phase:
PHASE3
Brief 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.
Detailed Description
This Phase 3 study is a 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 Criteria
Inclusion
- Patient has completed the NS-065/NCNP-01-301 study;
- 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;
- Patient and parent(s)/guardian(s) are willing and able to comply with scheduled visits, investigational product (IP) administration plan, and study procedures.
Exclusion
- 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;
- 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;
- Patient took any other investigational drug(s) during or after completion of Study NS-065/NCNP-01-301;
- Patient is judged by the investigator and/or the sponsor not to be appropriate to participate in the extension study for any reason.
Key Trial Info
Start Date :
April 13 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 1 2025
Estimated Enrollment :
74 Patients enrolled
Trial Details
Trial ID
NCT04768062
Start Date
April 13 2021
End Date
November 1 2025
Last Update
February 14 2024
Active Locations (31)
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1
Queensland Children's Hospital
Brisbane, Australia
2
The Childrens Hospital at Westmead
Westmead, Australia
3
CHU de Quebec Research Centre
Québec, Canada
4
Hospital de Niños Roberto del Rio
Santiago, Chile