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

Study Overview
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Brief Summary
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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).

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

Conditions
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Duchenne Muscular Dystrophy (DMD)

Intervention / Treatment
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* Drug: NS-089/NCNP-02
* Drug: NS-089/NCNP-02


Participation Criteria
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Eligibility Criteria
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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.
Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: NS-089/NCNP-02 40 mg/kg<br> | Drug: NS-089/NCNP-02<br>* 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).<br>|
| Experimental: NS-089/NCNP-02 80 mg/kg<br> | Drug: NS-089/NCNP-02<br>* 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).<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of adverse events |  | Up to Week 247 | 

Secondary Outcome Measures

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