Miglustat in Niemann-Pick Type C Disease

Niemann-Pick Type C Disease Clinical Trial

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

Status Completed

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Niemann-Pick Disease, Type C
• Niemann-Pick Diseases
• Niemann-Pick Type C Disease

• NCT number: NCT00517153
• Study type: Interventional
• Source: Actelion
• Status: Completed
• Phase: Phase 2
• Start date: January 2002
• Completion date: January 2008