NCT03167255

Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

A Phase II, Open-Label, Extension Study to Assess the Safety and Efficacy of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: NS Pharma, Inc. · Industry
• Sex: Male
• Age: 4 Years – 10 Years
• Healthy volunteers: Not accepted

Summary

This is an open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 192 weeks to boys with DMD who complete Study NS-065/NCNP-01-201.

Detailed description

This is a Phase II, multicenter, open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 192 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 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier. Patients who complete the Phase II Dose-finding Study NS-065/NCNP-01-201 are eligible to enroll.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	NS-065/NCNP-01	Received during weekly intravenous infusions

Timeline

• Start date: 2017-07-06
• Primary completion: 2021-10-20
• Completion: 2021-11-15
• First posted: 2017-05-25
• Last updated: 2022-12-28
• Results posted: 2022-11-18

Locations

6 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study