Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
Last updated on July 2021Recruitment
- Recruitment Status
- Active, not recruiting
- Estimated Enrollment
- Same as current
Summary
- Conditions
- Duchenne Muscular Dystrophy
- Type
- Interventional
- Phase
- Phase 2
- Design
- Allocation: Non-RandomizedIntervention Model: Parallel AssignmentIntervention Model Description: Low Dose cohort of 40 mg/kg and High Dose cohort of 80 mg/kgMasking: None (Open Label)Primary Purpose: Treatment
Participation Requirements
- Age
- Between 4 years and 10 years
- Gender
- Only males
Description
This is a Phase II, multicenter, open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 168 weeks to boys with DMD who complete Study NS-065/NCNP-01-201. This study will evaluate the safety, tolerability, and clinical efficacy of NS-065/NCNP-01 at dose...
This is a Phase II, multicenter, open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 168 weeks to boys with DMD who complete Study NS-065/NCNP-01-201. This study will evaluate the safety, tolerability, and clinical efficacy of NS-065/NCNP-01 at dose levels of up to 80 mg/kg/week administered by weekly IV infusion over an additional treatment period of 168 weeks or until NS-065/NCNP-01 is commercially available, whichever is earlier. Patients who complete the Phase II Dose-finding Study NS-065/NCNP-01-201 are eligible to enroll.
Tracking Information
- NCT #
- NCT03167255
- Collaborators
- Nippon Shinyaku Co., Ltd.
- Cooperative International Neuromuscular Research Group
- Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS)
- Investigators
- Study Chair: Paula R. Clemens, MD University of Pittsburgh
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