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

• Sponsor: Nippon Shinyaku Co., Ltd. (Industry)
• Overall Status: Active, not recruiting
• CT.gov ID: NCT05135663
• Collaborator: (none)
• Enrollment: 6
• Locations: 2
• Arms: 2
• Anticipated Duration (Months): 25.2
• Patients Per Site: 3
• Patients Per Site Per Month: 0.1

Study Details

Study Description

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).

Condition or Disease	Intervention/Treatment	Phase
Duchenne Muscular Dystrophy (DMD)	Drug: NS-089/NCNP-02; Drug: NS-089/NCNP-02	Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 6 participants
• Allocation: Non-Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase II, Open-Label, Extension Study of NS-089/NCNP-02 in Patients With Duchenne Muscular Dystrophy
• Actual Study Start Date: Jun 23, 2021
• Anticipated Primary Completion Date: Jan 31, 2023
• Anticipated Study Completion Date: Jul 31, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: NS-089/NCNP-02 40 mg/kg	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 72 weeks.
Experimental: NS-089/NCNP-02 80 mg/kg	Drug: 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 72 weeks.

Outcome Measures

Primary Outcome Measures

1. Incidence of adverse events [Up to Week 102]

Secondary Outcome Measures

1. Expression of dystrophin protein (Western blot) [Week 99]

2. Percentage of exon 44-skipped mRNA of dystrophin [Week 99]

3. North Star Ambulatory Assessment (NSAA) [Up to Week 99]

4. Time to stand test [Up to Week 99]

5. Time to run/walk 10 meters test [Up to Week 99]

6. Six-minute walk test/Two-minute walk test [Up to Week 99]

7. Timed Up & Go test [Up to Week 99]

8. Quantitative muscle strength assessment [Up to Week 99]

9. Performance of Upper Limb test [Up to Week 99]

10. Change in serum creatine kinase concentration from baseline [Up to Week 99]

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A and Older
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Kagoshima University Hospital	Kagoshima		Japan
2	National Center of Neurology and Psychiatry	Tokyo		Japan

Sponsors and Collaborators

• Nippon Shinyaku Co., Ltd.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Nippon Shinyaku Co., Ltd.
• ClinicalTrials.gov Identifier: NCT05135663
• Other Study ID Numbers: NS089/NCNP02-P2OE; jRCT2031210162
• First Posted: Nov 26, 2021
• Last Update Posted: Nov 26, 2021
• Last Verified: Nov 1, 2021

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne

Study Results

No Results Posted as of Nov 26, 2021