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Application of Miglustat in Patients With Niemann-Pick Type C

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01760564
First Posted: January 4, 2013
Last Update Posted: January 4, 2013
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.
Information provided by (Responsible Party):
National Taiwan University Hospital
  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: None (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

Information from the National Library of Medicine

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, Learn About Clinical Studies.


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
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


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
First Submitted: January 2, 2013
First Posted: January 4, 2013
Last Update Posted: January 4, 2013
Last Verified: November 2012

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

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
Dementia
Aphasia
Neurobehavioral Manifestations
Neurologic Manifestations
Signs and Symptoms
TDP-43 Proteinopathies