clinical trial · NCT00517153
Miglustat in Niemann-Pick Type C Disease
Actelion·phase2·completed·n = 29
Niemann-Pick Type C Diseasemiglustat
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.
started
Jan 1, 2002
primary completion
Sep 1, 2006
completion
Jan 1, 2008
last updated
Feb 4, 2025
official title
A Phase II Randomized Controlled Study of Miglustat in Adult and Juvenile Patients With Niemann-Pick Type C Disease
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol