Safety and Efficacy of Miglustat in Chinese NPC Patients

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.

Timeline

Start

2020-04-02

Primary completion

2022-03-25

Completion

2022-03-25

Publications

• Zhang H, Xiong H, Wei C, Yi M, Che Y, Zhuo J, Li X. Evaluation of the safety and efficacy of miglustat for the treatment of Chinese patients with Niemann-Pick disease type C: A prospective, open-label, single-arm, phase IV trial. Intractable Rare Dis Res. 2024 Nov 30;13(4):227-235. doi: 10.5582/irdr.2024.01056.

Drugs

Indications