Application of Miglustat in Patients With Niemann-Pick Type C

National Taiwan University Hospital5 enrolled

Overview

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

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Niemann-Pick Disease Type C

Interventions

MiglustatDRUG

miglustat 200mg tid for adult. For children surface area correction will be made. A lower dose may be used initially to decrease side effect.

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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

Locations (1)

National Taiwan University Hospital

Taipei, Taiwan

Outcomes

Primary Outcomes

Swallowing

videofluoroscopic swallowing study (VFSS), or NP-C functional disability rating scale if VFSS can not be performed due to safety issue

Time frame: 12th month

Secondary Outcomes

Mental

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

Time frame: 12th month

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.