Application of Miglustat in Patients With Niemann-Pick Type C

This study has been completed.
Information provided by (Responsible Party):
National Taiwan University Hospital Identifier:
First received: January 2, 2013
Last updated: January 3, 2013
Last verified: November 2012
To evaluate the changes in cognitive function after miglustat treatment in Niemann-Pick type C patients.

Condition Intervention Phase
Niemann-Pick Disease Type C
Drug: Miglustat
Phase 3

Study Type: Interventional
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

Resource links provided by NLM:

Further study details as provided by National Taiwan University Hospital:

Primary Outcome Measures:
  • 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

Secondary Outcome Measures:
  • 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

Enrollment: 5
Study Start Date: January 2008
Study Completion Date: December 2010
Primary Completion Date: December 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 Name: Zavesca

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

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
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Please refer to this study by its identifier: NCT01760564

National Taiwan University Hospital
Taipei, Taiwan
Sponsors and Collaborators
National Taiwan University Hospital
Principal Investigator: Whu-Liang Hwu, MD, PhD National Taiwan University Hospital
  More Information

Responsible Party: National Taiwan University Hospital Identifier: NCT01760564     History of Changes
Other Study ID Numbers: 200802043M 
Study First Received: January 2, 2013
Last Updated: January 3, 2013
Health Authority: Taiwan: Department of Health

Keywords provided by National Taiwan University Hospital:
Niemann-Pick disease type C

Additional relevant MeSH terms:
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Niemann-Pick Diseases
Pick Disease of the Brain
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Central Nervous System Diseases
Communication Disorders
Genetic Diseases, Inborn
Language Disorders
Lipid Metabolism Disorders
Lymphatic Diseases
Lysosomal Storage Diseases
Lysosomal Storage Diseases, Nervous System
Mental Disorders
Metabolic Diseases
Nervous System Diseases
Neurocognitive Disorders
Neurodegenerative Diseases
Speech Disorders
Aphasia, Primary Progressive
Frontotemporal Dementia
Frontotemporal Lobar Degeneration
Histiocytosis, Non-Langerhans-Cell
Lipid Metabolism, Inborn Errors
Lipidoses processed this record on May 25, 2016