Application of Miglustat in Patients With Niemann-Pick Type C

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01760564

Enrollment

Registered

2013-01-04

Start date

2008-01-31

Completion date

2010-12-31

Last updated

2013-01-04

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Niemann-Pick Disease Type C

Keywords

Niemann-Pick disease type C, miglustat

Brief summary

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

Detailed description

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

Interventions

DRUGMiglustat

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

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
Swallowing	12th month	videofluoroscopic swallowing study (VFSS), or NP-C functional disability rating scale if VFSS can not be performed due to safety issue

Secondary

Measure	Time frame	Description
Mental	12th month	IQ test or Mini-Mental Status Examination if a full IQ test can not be performed

Countries

Taiwan

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026