Safety and Efficacy of Miglustat in Chinese NPC Patients
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|ClinicalTrials.gov Identifier: NCT03910621|
Recruitment Status : Not yet recruiting
First Posted : April 10, 2019
Last Update Posted : September 4, 2019
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.
|Condition or disease||Intervention/treatment||Phase|
|Niemann-Pick Disease, Type C||Drug: Miglustat||Phase 4|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||18 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Estimated Study Start Date :||December 20, 2019|
|Estimated Primary Completion Date :||July 1, 2021|
|Estimated Study Completion Date :||February 1, 2022|
Miglustat is administered three times a day as an oral capsule
capsule, oral use
- Observed change in HSEM (ms/deg) [ Time Frame: baseline to week 52 ]Change in HSEM from baseline to week 52
- Change in Pineda disability scale score [ Time Frame: baseline to week 52 ]Change in the modified Pineda disability scale score. The scale is assessing 4 key domains (manipulation, ambulation, language and swallowing). Individual scores of the domains are claculated ted into a composite score, 6 being the lowest and best score and 24 being the worst and highest score.
- Incidence of treatment-emergent AEs and SAEs [ Time Frame: Baseline to 30 days after End of Treatment (Week 52) ]Treatment-emergent adverse events (AEs) up to 30 days after EOT