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

Duchenne Muscular Dystrophy (DMD) Clinical Trial

Official title:

A Phase II, Open-Label, Extension Study of NS-089/NCNP-02 in Patients With Duchenne Muscular Dystrophy

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05135663
• Other study ID #: NS089/NCNP02-P2OE
• Secondary ID: jRCT2031210162
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: June 23, 2021
• Est. completion date: July 31, 2026

Study information

• Verified date: March 2023
• Source: Nippon Shinyaku Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 6
• Est. completion date: July 31, 2026
• Est. primary completion date: January 31, 2026
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

Inclusion Criteria:

1. Patient completed Study NCNP/DMT02

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.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy (DMD)
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• NS-089/NCNP-02
The same dose administered in as Part 2 of Study NCNP/DMT02 (40 mg/kg) will be administered once weekly for 216 weeks (The total treatment period is 240 weeks including Part2 of Study NCNP/DMT02).
• NS-089/NCNP-02
The same dose administered in as Part 2 of Study NCNP/DMT02 (80 mg/kg) will be administered once weekly for 216 weeks (The total treatment period is 240 weeks including Part2 of Study NCNP/DMT02).

Locations

Country	Name	City	State
Japan	Kagoshima University Hospital	Kagoshima
Japan	National Center of Neurology and Psychiatry	Tokyo

Sponsors (1)

Lead Sponsor	Collaborator
Nippon Shinyaku Co., Ltd.

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of adverse events		Up to Week 247
Secondary	Expression of dystrophin protein (Western blot)		Week 99
Secondary	Percentage of exon 44-skipped mRNA of dystrophin		Week 99
Secondary	North Star Ambulatory Assessment (NSAA)		Up to Week 243
Secondary	Time to stand test		Up to Week 243
Secondary	Time to run/walk 10 meters test		Up to Week 243
Secondary	Six-minute walk test/Two-minute walk test		Up to Week 243
Secondary	Timed Up & Go test		Up to Week 243
Secondary	Quantitative muscle strength assessment		Up to Week 243
Secondary	Performance of Upper Limb test		Up to Week 243
Secondary	Change in serum creatine kinase concentration from baseline		Up to Week 243