NCT03910621

Safety and Efficacy of Miglustat in Chinese NPC Patients

A Single Arm Uncontrolled 12 Months Clinical Study to Evaluate the Safety and Efficacy of Miglustat (Zavesca) for the Treatment of Niemann Pick Type C Disease (NPC) in Chinese Subjects

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: Actelion · Industry
• Sex: All
• Age: 4 Years
• Healthy volunteers: Not accepted

Summary

This is a prospective, multi-center, open-label, non-randomized, single-arm Phase IV confirmatory study. Approximately 19 subjects with Niemann Pick Type C disease (NPC) will be enrolled in this study. The study will be conducted at 2 sites in China.

Detailed description

This is a prospective, multi-center, open-label, non-randomized, single-arm Phase IV confirmatory study.The study is conducted in Chinese subjects aged 4 years and older with Niemann Pick Type C disease (NPC). Approximately 19 subjects will be enrolled in this study. The study will be conducted at 2 sites in China. Patients will be treated with miglustat for 12 months, efficacy and safety outcomes will be measured

Conditions

• Niemann-Pick Disease, Type C

Interventions

Type	Name	Description
DRUG	Miglustat	capsule, oral use

Timeline

• Start date: 2020-04-02
• Primary completion: 2022-03-25
• Completion: 2022-03-25
• First posted: 2019-04-10
• Last updated: 2025-03-30

Locations

2 sites across 1 country: China