Clinical Trial: Application of Miglustat in Patients With Niemann-Pick Type C

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Application of Miglustat in Patients With Niemann-Pick Type C

Brief Summary: To evaluate the changes in cognitive function after miglustat treatment in Niemann-Pick type C patients.

Detailed Summary: Patients with Niemann-Pick C disease will be treated with Miglutat 200mg tid. Neurologic evaluation and safety will be monitored periodically.
Sponsor: National Taiwan University Hospital

Current Primary Outcome: Swallowing [ Time Frame: 12th month ]

videofluoroscopic swallowing study (VFSS), or NP-C functional disability rating scale if VFSS can not be performed due to safety issue


Original Primary Outcome: Same as current

Current Secondary Outcome: Mental [ Time Frame: 12th month ]

IQ test or Mini-Mental Status Examination if a full IQ test can not be performed


Original Secondary Outcome: Same as current

Information By: National Taiwan University Hospital

Dates:
Date Received: January 2, 2013
Date Started: January 2008
Date Completion:
Last Updated: January 3, 2013
Last Verified: November 2012