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Application of Miglustat in Patients With Niemann-Pick Type C

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
National Taiwan University Hospital
ClinicalTrials.gov Identifier:
NCT01760564
First received: January 2, 2013
Last updated: January 3, 2013
Last verified: November 2012
History of Changes
  Purpose

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:

Drug Information available for: Miglustat
U.S. FDA Resources

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.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01760564

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

Publications:

Responsible Party: National Taiwan University Hospital
ClinicalTrials.gov 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
miglustat

Additional relevant MeSH terms:
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Aphasia, Primary Progressive
Pick Disease of the Brain
Frontotemporal Dementia
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Histiocytosis, Non-Langerhans-Cell
Histiocytosis
 Lymphatic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Dementia
Aphasia
Speech Disorders
Language Disorders
Communication Disorders
Neurobehavioral Manifestations
Neurologic Manifestations

ClinicalTrials.gov processed this record on May 16, 2013