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

NCT05996003 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2026-03-06

No results posted yet for this study

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

Interventions

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

Sponsors & Collaborators

  • Nippon Shinyaku Co., Ltd.

    collaborator INDUSTRY

  • NS Pharma, Inc.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
4 Years
Max Age
14 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-02-22
Primary Completion
2026-09-11
Completion
2026-09-11
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • Japan
  • New Zealand
  • South Korea
  • Turkey (Türkiye)

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05996003 on ClinicalTrials.gov