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

• Status: Recruiting
• Trial end date: 2026-06-01
• Target enrollment: 0
• Participant gender: Male

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: NS Pharma, Inc.
• Collaborator: Nippon Shinyaku Co., Ltd.

Criteria

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.