Duchenne Muscular Dystrophy (DMD)
Conditions
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).
Interventions
DRUGNS-089/NCNP-02
The same dose administered in as Part 2 of Study NCNP/DMT02 (40 mg/kg) will be administered once weekly for 288 weeks (The total treatment period is 312 weeks including Part2 of Study NCNP/DMT02).
Sponsors
Nippon Shinyaku Co., Ltd.
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of adverse events | Up to Week 319 |
Secondary
| Measure | Time frame |
|---|---|
| Percentage of exon 44-skipped mRNA of dystrophin | Week 99 |
| North Star Ambulatory Assessment (NSAA) | Up to Week 315 |
| Time to stand test | Up to Week 315 |
| Time to run/walk 10 meters test | Up to Week 315 |
| Expression of dystrophin protein (Western blot) | Week 99 |
| Timed Up & Go test | Up to Week 315 |
| Quantitative muscle strength assessment | Up to Week 315 |
| Performance of Upper Limb test | Up to Week 315 |
| Change in serum creatine kinase concentration from baseline | Up to Week 315 |
| Six-minute walk test/Two-minute walk test | Up to Week 315 |
Countries
Japan
Outcome results
None listed