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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
  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: 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 ]
    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 ]
    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

Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

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
Pick Disease of the Brain
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Lymphatic Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Neurocognitive Disorders
Mental Disorders
Speech Disorders
Language Disorders
Communication Disorders
Neurodegenerative Diseases
Aphasia, Primary Progressive
Frontotemporal Dementia
Sphingolipidoses
Histiocytosis, Non-Langerhans-Cell
Histiocytosis
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Frontotemporal Lobar Degeneration

ClinicalTrials.gov processed this record on June 26, 2017