Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

• Status: Active, not recruiting
• Trial end date: 2022-01-01
• Target enrollment: 0
• Participant gender: Male

Summary

This is an open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 192 weeks to boys with DMD who complete Study NS-065/NCNP-01-201.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: NS Pharma, Inc.
• Collaborators: Cooperative International Neuromuscular Research Group; Nippon Shinyaku Co Ltd; Nippon Shinyaku Co., Ltd.; Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS)

Criteria

Inclusion Criteria:

1. Completed Study NS-065/NCNP-01-201 through Week 25.

2. Willing and able to comply with scheduled visits, investigational product administration plan, and study procedures.

3. Stable dose of glucocorticoid (GC), and is expected to remain on the stable dose for the duration of the study.

Exclusion Criteria:

1. Serious or severe adverse event in Study NS-065/NCNP-01-201 that precludes safe use of NS-065/NCNP-01.

2. Patient had a treatment which was made for the purpose of dystrophin or its related protein induction after completion of Study NS-065/NCNP-01-201.

3. Patient took any other investigational drugs after completion of Study NS-065/NCNP-01-201.

4. Patient was judged by the investigator and/or the Sponsor that it was not appropriate to participate in the extension study for other reasons.