Application of Miglustat in Patients With Niemann-Pick Type C

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

Completed

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Key information

Conditions

Niemann-Pick Disease Type C Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hemic and Lymphatic Diseases Histiocytosis Histiocytosis, Non-Langerhans-Cell Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lymphatic Diseases Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Niemann-Pick Disease, Type C Niemann-Pick Diseases Nutritional and Metabolic Diseases Sphingolipidoses

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

National Taiwan University Hospital

Taipei, Taiwan

About this study

Patients with Niemann-Pick C disease will be treated with Miglutat 200mg tid. Neurologic evaluation and safety will be monitored periodically.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

miglustat

Drug

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 names: Zavesca

Primary outcomes

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 outcomes

Mental

Time frame: 12th month

IQ test or Mini-Mental Status Examination if a full IQ test can not be performed

Sponsors and collaborators

Lead sponsor

National Taiwan University Hospital

Other

Registry information

Important dates

Study start: 2008
Primary completion: 2010
Study completion: 2010
First posted: Jan 4, 2013
Registry last updated: Jan 4, 2013

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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Completed