Application of Miglustat in Patients With Niemann-Pick Type C

Phase 3

Completed

• Conditions: Niemann-Pick Disease Type C
• Interventions: Drug: Miglustat

• Registration Number: NCT01760564
• Lead Sponsor: National Taiwan University Hospital

Brief Summary

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

Detailed Description

Patients with Niemann-Pick C disease will be treated with Miglutat 200mg tid. Neurologic evaluation and safety will be monitored periodically.

Study Timeline

• Study Start: 2008-01
• Primary Completion: 2010-12
• Study Completion: 2010-12
• Status Verified: 2012-11
• Study First Submitted: 2013-01-02
• Study First Submitted QC: 2013-01-03
• Last Update Submitted: 2013-01-03
• Study First Posted: 2013-01-04
• Last Update Posted: 2013-01-04

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

• Confirm diagnosis of Niemann-Pick C disease
• Symptomatic including motor or mental symptoms

Exclusion Criteria

• Unknown severe diarrhea for more than 7 day
• Allergy to miglustat

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Miglustat	Miglustat	miglustat 200mg tid

Primary Outcome Measures

Name	Time	Method
Swallowing	12th month	videofluoroscopic swallowing study (VFSS), or NP-C functional disability rating scale if VFSS can not be performed due to safety issue

Secondary Outcome Measures

Name	Time	Method
Mental	12th month	IQ test or Mini-Mental Status Examination if a full IQ test can not be performed

Trial Locations

Locations (1)

National Taiwan University Hospital; 🇨🇳 Taipei, Taiwan