NCT02081625

Exploratory Study of NS-065/NCNP-01 in DMD

Exploratory Study of NS-065/NCNP-01 in Duchenne Muscular Dystrophy

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: National Center of Neurology and Psychiatry, Japan · Academic / Other
• Sex: Male
• Age: 5 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

This study is designed to assess the safety, tolerability, efficacy and pharmacokinetics (PK) of NS-065/NCNP-01 in subjects diagnosed with Duchenne muscular dystrophy (DMD).

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	NS-065/NCNP-01	NS-065/NCNP-01 for Infusion is packaged as 25 mg/mL in phosphate buffered saline with 1 mL per vial. Study dosages will be infused over a 1 hour period with Normal saline as follows: Cohort 1: 1.25mg/kg once weekly for 12 weeks; Cohort 2: 5.0mg/kg once weekly for 12 weeks; Cohort 3: 20.0mg/kg once weekly for 12 weeks

Timeline

• Start date: 2013-06-01
• Primary completion: 2014-11-01
• Completion: 2015-08-01
• First posted: 2014-03-07
• Last updated: 2020-02-26

Locations

1 site across 1 country: Japan