Clinical Trial

NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

Recruiting Phase 2
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Summary
This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to \<15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089/NCNP-02 administered once weekly. The study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.
Protocol Amendment History 8 amendments
This ClinicalTrials.gov record has been amended 8 times since 2023-08-10; most recent amendment 2026-03-04.
Status change: Not Yet Recruiting → Recruiting 2024-01-03
Trial Details
NCT Number NCT05996003
Lead Sponsor NS Pharma, Inc.
Collaborators: Nippon Shinyaku Co., Ltd.
Conditions Duchenne Muscular Dystrophy, Exon 44, DMD
Enrollment 20 participants
Start Date 2024-02-22
Primary Completion 2026-09-11 (estimated)
Study Completion 2026-09-11 (estimated)
Updated on ClinicalTrials.gov 2026-03-06