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Application of Miglustat in Patients With Niemann-Pick Type C

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 participants
Allocation: N/A
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
Actual Primary Completion Date : December 2010
Actual 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, Older Adult
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

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National Taiwan University Hospital
Taipei, Taiwan
Sponsors and Collaborators
National Taiwan University Hospital
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Principal Investigator: Whu-Liang Hwu, MD, PhD National Taiwan University Hospital
Publications of Results:
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Responsible Party: National Taiwan University Hospital
ClinicalTrials.gov Identifier: NCT01760564    
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:
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Pick Disease of the Brain
Aphasia, Primary Progressive
Frontotemporal Dementia
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Pathologic Processes
Frontotemporal Lobar Degeneration
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurocognitive Disorders
Mental Disorders
Speech Disorders
Language Disorders
Communication Disorders
Neurobehavioral Manifestations
Neurologic Manifestations
TDP-43 Proteinopathies
Neurodegenerative Diseases
Proteostasis Deficiencies
Metabolic Diseases
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Histiocytosis, Non-Langerhans-Cell