Extension Study of NS-089/NCNP-02 in DMD
Primary Purpose
Duchenne Muscular Dystrophy (DMD)
Status
Active
Phase
Phase 2
Locations
Japan
Study Type
Interventional
Intervention
NS-089/NCNP-02
NS-089/NCNP-02
Sponsored by
About this trial
This is an interventional treatment trial for Duchenne Muscular Dystrophy (DMD)
Eligibility Criteria
Inclusion Criteria:
- Patient completed Study NCNP/DMT02
Exclusion Criteria:
- 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.
- Patient had a treatment which was made for the purpose of dystrophin or dystrophin-related protein induction after completion of Study NCNP/DMT02.
- Patient took any other investigational drugs after completion of Study NCNP/DMT02.
- Patient was judged by the investigator and/or the Sponsor that it was not appropriate to participate in the extension study for other reasons.
Sites / Locations
- Kagoshima University Hospital
- National Center of Neurology and Psychiatry
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
NS-089/NCNP-02 40 mg/kg
NS-089/NCNP-02 80 mg/kg
Arm Description
Outcomes
Primary Outcome Measures
Incidence of adverse events
Secondary Outcome Measures
Expression of dystrophin protein (Western blot)
Percentage of exon 44-skipped mRNA of dystrophin
North Star Ambulatory Assessment (NSAA)
Time to stand test
Time to run/walk 10 meters test
Six-minute walk test/Two-minute walk test
Timed Up & Go test
Quantitative muscle strength assessment
Performance of Upper Limb test
Change in serum creatine kinase concentration from baseline
Full Information
NCT ID
NCT05135663
First Posted
October 5, 2021
Last Updated
March 1, 2023
Sponsor
Nippon Shinyaku Co., Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT05135663
Brief Title
Extension Study of NS-089/NCNP-02 in DMD
Official Title
A Phase II, Open-Label, Extension Study of NS-089/NCNP-02 in Patients With Duchenne Muscular Dystrophy
Study Type
Interventional
2. Study Status
Record Verification Date
March 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
June 23, 2021 (Actual)
Primary Completion Date
January 31, 2026 (Anticipated)
Study Completion Date
July 31, 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Nippon Shinyaku Co., Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. 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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy (DMD)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
6 (Actual)
8. Arms, Groups, and Interventions
Arm Title
NS-089/NCNP-02 40 mg/kg
Arm Type
Experimental
Arm Title
NS-089/NCNP-02 80 mg/kg
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
NS-089/NCNP-02
Intervention Description
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).
Intervention Type
Drug
Intervention Name(s)
NS-089/NCNP-02
Intervention Description
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).
Primary Outcome Measure Information:
Title
Incidence of adverse events
Time Frame
Up to Week 247
Secondary Outcome Measure Information:
Title
Expression of dystrophin protein (Western blot)
Time Frame
Week 99
Title
Percentage of exon 44-skipped mRNA of dystrophin
Time Frame
Week 99
Title
North Star Ambulatory Assessment (NSAA)
Time Frame
Up to Week 243
Title
Time to stand test
Time Frame
Up to Week 243
Title
Time to run/walk 10 meters test
Time Frame
Up to Week 243
Title
Six-minute walk test/Two-minute walk test
Time Frame
Up to Week 243
Title
Timed Up & Go test
Time Frame
Up to Week 243
Title
Quantitative muscle strength assessment
Time Frame
Up to Week 243
Title
Performance of Upper Limb test
Time Frame
Up to Week 243
Title
Change in serum creatine kinase concentration from baseline
Time Frame
Up to Week 243
10. Eligibility
Sex
Male
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Patient completed Study NCNP/DMT02
Exclusion Criteria:
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.
Patient had a treatment which was made for the purpose of dystrophin or dystrophin-related protein induction after completion of Study NCNP/DMT02.
Patient took any other investigational drugs after completion of Study NCNP/DMT02.
Patient was judged by the investigator and/or the Sponsor that it was not appropriate to participate in the extension study for other reasons.
Facility Information:
Facility Name
Kagoshima University Hospital
City
Kagoshima
Country
Japan
Facility Name
National Center of Neurology and Psychiatry
City
Tokyo
Country
Japan
12. IPD Sharing Statement
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Extension Study of NS-089/NCNP-02 in DMD
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