Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
Status:
Completed
Trial end date:
2018-04-01
Target enrollment:
Participant gender:
Summary
The main objective of this study is to evaluate the safety of a high (80mg/kg) and low
(40mg/kg) dose of NS-065/NCNP-01 delivered as an intravenous infusion in patients with
Duchenne Muscular Dystrophy (DMD) amendable to exon 53 skipping. Additional objectives
include tolerability, muscle function and strength, pharmacokinetics and pharmacodynamics.
Phase:
Phase 2
Details
Lead Sponsor:
NS Pharma, Inc.
Collaborators:
Cooperative International Neuromuscular Research Group Nippon Shinyaku Co Ltd Nippon Shinyaku Co., Ltd. Therapeutic Research in Neuromuscular Disorders Solutions