NCT05996003

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

A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089/NCNP-02 in Boys With Duchenne Muscular Dystrophy (DMD)

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: NS Pharma, Inc. · Industry
• Sex: Male
• Age: 4 Years – 14 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Duchenne Muscular Dystrophy
• Exon 44
• DMD

Interventions

Type	Name	Description
DRUG	NS-089/NCNP-02	Cohort 1: Part 1 Dose Level 1-3: a 4-week Treatment Phase at each treatment dose level Part 2 Single Dose Level: a 24-week Treatment Phase at the MTD of Part 1 Cohort 2: Part 2 Single Dose Level: a 24-week Treatment Phase at the MTD of Part 1

Timeline

• Start date: 2024-02-22
• Primary completion: 2026-09-11
• Completion: 2026-09-11
• First posted: 2023-08-16
• Last updated: 2026-03-06

Locations

25 sites across 7 countries: United States, Australia, Canada, Japan, New Zealand, South Korea, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study