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Safety and Efficacy of Miglustat in Chinese NPC Patients

Niemann-Pick Disease, Type C Clinical Trial

Official title:
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

Clinical Trial 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.

Clinical Trial 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 ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03910621
Study type Interventional
Source Actelion
Contact
Status Completed
Phase Phase 4
Start date April 2, 2020
Completion date March 25, 2022
View Details
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