Miglustat in Niemann-Pick Type C Disease

Summary

This is a phase II randomized controlled study of miglustat in adult and juvenile patients with Niemann-Pick Type C disease. Up to 42 patients will be randomised in a 2:1 ratio to either treatment with miglustat or to a non-treatment group. Both groups will follow an identical visit schedule.

Timeline

Start

2002-01

Primary completion

2006-09

Completion

2008-01

Drugs

Indications