Clinical Trial: Miglustat in Niemann-Pick Type C Disease

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase II Randomized Controlled Study of Miglustat in Adult and Juvenile Patients With Niemann-Pick Type C Disease

Brief 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.

Detailed Summary:
Sponsor: Actelion

Current Primary Outcome: The primary endpoints will be two parameters - Horizontal Saccadic α and Horizontal Saccadic β - which are estimated for each patient from their saccadic eye movement data [ Time Frame: Baseline to Month 12 ]

Original Primary Outcome:

Current Secondary Outcome: Highest amplitude for which a velocity measurement is obtained [ Time Frame: Baseline to Month 12 ]

Original Secondary Outcome:

Information By: Actelion

Dates:
Date Received: August 15, 2007
Date Started: January 2002
Date Completion:
Last Updated: March 24, 2010
Last Verified: March 2010