Application of Miglustat in Patients With Niemann-Pick Type C
To evaluate the changes in cognitive function after miglustat treatment in Niemann-Pick type C patients.
|Study Design:||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Application of Miglustat in Patients With Niemann-Pick Type C|
- Swallowing [ Time Frame: 12th month ] [ Designated as safety issue: No ]videofluoroscopic swallowing study (VFSS), or NP-C functional disability rating scale if VFSS can not be performed due to safety issue
- Mental [ Time Frame: 12th month ] [ Designated as safety issue: No ]IQ test or Mini-Mental Status Examination if a full IQ test can not be performed
|Study Start Date:||January 2008|
|Study Completion Date:||December 2010|
|Primary Completion Date:||December 2010 (Final data collection date for primary outcome measure)|
miglustat 200mg tid
miglustat 200mg tid for adult. For children surface area correction will be made. A lower dose may be used initially to decrease side effect.
Other Name: Zavesca
Patients with Niemann-Pick C disease will be treated with Miglutat 200mg tid. Neurologic evaluation and safety will be monitored periodically.
|National Taiwan University Hospital|
|Principal Investigator:||Whu-Liang Hwu, MD, PhD||National Taiwan University Hospital|