Application of Miglustat in Patients With Niemann-Pick Type C

National Taiwan University

Status and phase: Completed; Phase 3

Conditions: Niemann-Pick Disease Type C

Treatments: Drug: Miglustat

Study type: Interventional

Funder types: Other

Identifiers

NCT01760564

200802043M

Details and patient eligibility

About

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

Full description

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

Enrollment: 5 patients

Sex: All

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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5 participants in 1 patient group

Miglustat

Experimental group

Description:

miglustat 200mg tid

Treatment:

Drug: Miglustat