Safety and Efficacy of Miglustat in Chinese NPC Patients
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|ClinicalTrials.gov Identifier: NCT03910621|
Recruitment Status : Recruiting
First Posted : April 10, 2019
Last Update Posted : April 22, 2020
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|
|Actual Study Start Date :||April 2, 2020|
|Estimated Primary Completion Date :||January 1, 2022|
|Estimated Study Completion Date :||January 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03910621
|Contact: Study Contact||844-434-4210||JNJ.CT@sylogent.com|
|Peking University first Hospital||Not yet recruiting|
|Beijing, China, 100034|
|Xin Hua Hospital, Shanghai Jiao Tong University||Recruiting|
|Shanghai, China, 200092|
|Study Director:||Yue Wu||Janssen China R&D|