NCT04129294

Exploratory Study of NS-089/NCNP-02 in DMD

Exploratory Study of NS-089/NCNP-02 in Duchenne Muscular Dystrophy

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: National Center of Neurology and Psychiatry, Japan · Academic / Other
• Sex: Male
• Age: 4 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

This study is designed to assess the safety, tolerability, efficacy and pharmacokinetics (PK) of NS-089/NCNP-02 in subjects diagnosed with Duchenne muscular dystrophy (DMD), and to determine the dosage for subsequent studies.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	NS-089/NCNP-02	NS-089/NCNP-02 for Infusion is packaged as 50 mg/mL with 3 mL per vial. Study dosages will be infused over a 1 hour period at the following dose levels. "\[Part 1\] NS-089/NCNP-02 is administered at dose levels 1 and 3 in Cohort 1 and at dose levels 2 and 4 in Cohort 2. Dose level 1: 1.62 mg/kg once weekly for 2 weeks; Dose level 2: 10 mg/kg once weekly for 2 weeks; Dose level 3: 40 mg/kg once weekly for 2 weeks; Dose level 4: 80 mg/kg once weekly for 2 weeks \[Part 2\] Based on the results from Part 1, two dosages are selected as study dosages in Part 2. Each selected dose are administered once a week for 24 weeks."

Timeline

• Start date: 2019-12-02
• Primary completion: 2022-05-31
• Completion: 2022-05-31
• First posted: 2019-10-16
• Last updated: 2022-09-29

Locations

1 site across 1 country: Japan