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Application of Miglustat in Patients With Niemann-Pick Type C

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ClinicalTrials.gov Identifier: NCT01760564
Recruitment Status : Completed
First Posted : January 4, 2013
Last Update Posted : January 4, 2013
Information provided by (Responsible Party):
National Taiwan University Hospital

Brief Summary:
To evaluate the changes in cognitive function after miglustat treatment in Niemann-Pick type C patients.

Condition or disease Intervention/treatment Phase
Niemann-Pick Disease Type C Drug: Miglustat Phase 3

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  (Clinical Trial)
Actual Enrollment : 5 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Application of Miglustat in Patients With Niemann-Pick Type C
Study Start Date : January 2008
Primary Completion Date : December 2010
Study Completion Date : December 2010

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 Name: Zavesca

Primary Outcome Measures :
  1. 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 :
  1. Mental [ Time Frame: 12th month ]
    IQ test or Mini-Mental Status Examination if a full IQ test can not be performed

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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

Responsible Party: National Taiwan University Hospital
ClinicalTrials.gov Identifier: NCT01760564     History of Changes
Other Study ID Numbers: 200802043M
First Posted: January 4, 2013    Key Record Dates
Last Update Posted: January 4, 2013
Last Verified: November 2012

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

Additional relevant MeSH terms:
Pick Disease of the Brain
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurocognitive Disorders
Mental Disorders
Speech Disorders
Language Disorders
Communication Disorders
Neurodegenerative Diseases
Metabolic Diseases
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Lymphatic Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Lipid Metabolism Disorders
Aphasia, Primary Progressive
Frontotemporal Dementia
Frontotemporal Lobar Degeneration
Neurobehavioral Manifestations
Neurologic Manifestations
Signs and Symptoms
TDP-43 Proteinopathies