Extension Study of NS-089/NCNP-02 in DMD

Phase 2

Active, not recruiting

• Conditions: Duchenne Muscular Dystrophy (DMD)
• Interventions: Drug: NS-089/NCNP-02

• Registration Number: NCT05135663
• Lead Sponsor: Nippon Shinyaku Co., Ltd.

Brief Summary

This is the extension study of NS-089/NCNP-02 (Study NCNP/DMT02), which is designed to assess the safety, tolerability and efficacy of NS-089/NCNP-02 in patients with Duchenne muscular dystrophy (DMD).

Detailed Description

Not available

Study Timeline

• Study Start: 2021-06-23
• Study First Submitted: 2021-10-05
• Study First Submitted QC: 2021-11-16
• Study First Posted: 2021-11-26
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-22
• Last Update Posted: 2024-11-26
• Primary Completion: 2026-01-31
• Study Completion: 2026-07-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 6

Inclusion Criteria

1. Patient completed Study NCNP/DMT02

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Exclusion Criteria

1. Patient had any serious adverse events in Study NCNP/DMT02 that, in the opinion of the Investigator and/or Sponsor, was probably or definitely related to NS-089/NCNP-02 use and precludes safe use of NS-089/NCNP-02 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 NCNP/DMT02.
3. Patient took any other investigational drugs after completion of Study NCNP/DMT02.
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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
NS-089/NCNP-02 80 mg/kg	NS-089/NCNP-02	-
NS-089/NCNP-02 40 mg/kg	NS-089/NCNP-02	-

Primary Outcome Measures

Name	Time	Method
Incidence of adverse events	Up to Week 247

Secondary Outcome Measures

Name	Time	Method
North Star Ambulatory Assessment (NSAA)	Up to Week 243
Expression of dystrophin protein (Western blot)	Week 99
Timed Up & Go test	Up to Week 243
Quantitative muscle strength assessment	Up to Week 243
Six-minute walk test/Two-minute walk test	Up to Week 243
Time to run/walk 10 meters test	Up to Week 243
Change in serum creatine kinase concentration from baseline	Up to Week 243
Time to stand test	Up to Week 243
Percentage of exon 44-skipped mRNA of dystrophin	Week 99
Performance of Upper Limb test	Up to Week 243

Trial Locations

Locations (2)

Kagoshima University Hospital; 🇯🇵 Kagoshima, Japan

National Center of Neurology and Psychiatry; 🇯🇵 Tokyo, Japan