Duchenne muscular dystrophy
Conditions
Interventions
The same dose as Part 2 of Study NCNP/DMT02 (40 mg/kg or 80 mg/kg) will be administered once weekly for 288 weeks (The total treatment period is 312 weeks including Part2 of Study NCNP/DMT02).
Sponsors
Higashioka Masaya
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Patient completed Study NCNP/DMT02
Exclusion criteria
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 |
|---|---|
| Adverse event and adverse drug reaction (Safety and Tolerability) | — |
Secondary
| Measure | Time frame |
|---|---|
| 1) Expression of dystrophin protein (Western blot) 2) Exon 44 skipping efficiency 3) Motor function assessment North Star Ambulatory Assessment Time to stand Time to run/walk 10 meters Six-minute walk test and Two-minute walk test Timed Up & Go test Quantitative muscle strength assessment Grip strength Pinch force Performance of Upper Limb (PUL) 4) Change in serum creatine kinase concentration | — |
Contacts
Public ContactClinical Development Operations
Nippon Shinyaku Co., Ltd.
Outcome results
None listed