Duchenne Muscular Dystrophy
Conditions
Brief summary
This is a Phase 3, multi-center, open-label extension study in ambulant boys with DMD who have completed the 48-week treatment period of either viltolarsen or placebo in Study NS-065/NCNP-01-301.
Detailed description
This Phase 3 study is a multi-center, open-label extension study in ambulant boys with DMD who have completed the 48-week treatment period of either viltolarsen or placebo in Study NS-065/NCNP-01-301. Patients will receive viltolarsen administered IV at weekly doses of 80 mg/kg. Study NS-065/NCNP-01-302 will be comprised of a 96-week treatment period.
Interventions
DRUGViltolarsen
Received during weekly intravenous infusions
Sponsors
Nippon Shinyaku Co., Ltd.
NS Pharma, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Healthy volunteers
No
Inclusion criteria
1. Patient has completed the NS-065/NCNP-01-301 study; 2. Patient's parent(s) or legal guardian(s) has (have) provided written informed consent and Health Insurance Portability and Accountability Act authorization, where applicable, prior to any study-related procedures; patients will be asked to give written or verbal assent according to local requirements; 3. Patient and parent(s)/guardian(s) are willing and able to comply with scheduled visits, investigational product (IP) administration plan, and study procedures.
Exclusion criteria
1. Patient had an adverse event in Study NS-065/NCNP-01-301 that, in the opinion of the investigator and/or the sponsor, precludes safe use of viltolarsen 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 NS-065/NCNP-01-301; 3. Patient took any other investigational drug(s) during or after completion of Study NS-065/NCNP-01-301; 4. Patient is judged by the investigator and/or the sponsor not to be appropriate to participate in the extension study for any reason.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03 | baseline to up to 96 weeks of treatment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Time to Run/Walk 10 Meters Test (TTRW) | baseline to 96 weeks of treatment | Change in Time to Run/Walk 10 meters |
| Six-minute Walk Test (6MWT) | baseline to 96 weeks of treatment | Change in Six-minute Walk |
| Time to Stand Test (TTSTAND) | baseline to 96 weeks of treatment | Change in Time to Stand |
| Time to Climb 4 Stairs Test (TTCLIMB) | baseline to 96 weeks of treatment | Change in Time to Climb 4 Stairs |
| Muscle Strength Measured by Hand-Held Dynamometer | baseline to 96 weeks of treatment | Change in Muscle Strength Measured by Hand-Held Dynamometer |
| North Star Ambulatory Assessment (NSAA) | baseline to 96 weeks of treatment | Change in North Star Ambulatory Assessment |
Countries
Australia, Canada, Chile, China, Czechia, Greece, Italy, Japan, Mexico, Netherlands, New Zealand, Norway, Russia, South Korea, Spain, Turkey (Türkiye), United Kingdom
Outcome results
None listed