Application of Miglustat in Patients With Niemann-Pick Type C

January 3, 2013 updated by: National Taiwan University Hospital
To evaluate the changes in cognitive function after miglustat treatment in Niemann-Pick type C patients.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

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

Study Type

Interventional

Enrollment (Actual)

5

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Taiwan
      • Taipei, Taiwan
        • National Taiwan University Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Miglustat
miglustat 200mg tid
Drug: Miglustat
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 Names:
  • Zavesca

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
12th month

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Mental
Time Frame: 12th month
IQ test or Mini-Mental Status Examination if a full IQ test can not be performed
12th month

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Taiwan University Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2008

Primary Completion (Actual)

December 1, 2010

Study Completion (Actual)

December 1, 2010

Study Registration Dates

First Submitted

January 2, 2013

First Submitted That Met QC Criteria

January 3, 2013

First Posted (Estimate)

January 4, 2013

Study Record Updates

Last Update Posted (Estimate)

January 4, 2013

Last Update Submitted That Met QC Criteria

January 3, 2013

Last Verified

November 1, 2012

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 200802043M

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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