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Application of Miglustat in Patients With Niemann-Pick Type C

Primary Purpose

Niemann-Pick Disease Type C

Status
Completed
Phase
Phase 3
Locations
Taiwan
Study Type
Interventional
Intervention
Miglustat
Sponsored by
National Taiwan University Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Niemann-Pick Disease Type C focused on measuring Niemann-Pick disease type C, miglustat

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • National Taiwan University Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Miglustat

Arm Description

miglustat 200mg tid

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

Full Information

First Posted
January 2, 2013
Last Updated
January 3, 2013
Sponsor
National Taiwan University Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT01760564
Brief Title
Application of Miglustat in Patients With Niemann-Pick Type C
Official Title
Application of Miglustat in Patients With Niemann-Pick Type C
Study Type
Interventional

2. Study Status

Record Verification Date
November 2012
Overall Recruitment Status
Completed
Study Start Date
January 2008 (undefined)
Primary Completion Date
December 2010 (Actual)
Study Completion Date
December 2010 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
National Taiwan University Hospital

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Niemann-Pick Disease Type C
Keywords
Niemann-Pick disease type C, miglustat

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
5 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Miglustat
Arm Type
Experimental
Arm Description
miglustat 200mg tid
Intervention Type
Drug
Intervention Name(s)
Miglustat
Other Intervention Name(s)
Zavesca
Intervention Description
miglustat 200mg tid for adult. For children surface area correction will be made. A lower dose may be used initially to decrease side effect.
Primary Outcome Measure Information:
Title
Swallowing
Description
videofluoroscopic swallowing study (VFSS), or NP-C functional disability rating scale if VFSS can not be performed due to safety issue
Time Frame
12th month
Secondary Outcome Measure Information:
Title
Mental
Description
IQ test or Mini-Mental Status Examination if a full IQ test can not be performed
Time Frame
12th month

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Whu-Liang Hwu, MD, PhD
Organizational Affiliation
National Taiwan University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
National Taiwan University Hospital
City
Taipei
Country
Taiwan

12. IPD Sharing Statement

Citations:
PubMed Identifier
22476655
Citation
Chien YH, Peng SF, Yang CC, Lee NC, Tsai LK, Huang AC, Su SC, Tseng CC, Hwu WL. Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C. J Inherit Metab Dis. 2013 Jan;36(1):129-37. doi: 10.1007/s10545-012-9479-9. Epub 2012 Apr 5.
Results Reference
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Application of Miglustat in Patients With Niemann-Pick Type C

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