drugset

drugset / Trial / NCT01760564

Application of Miglustat in Patients With Niemann-Pick Type C

NCT01760564 ↗

Phase 3 Completed 5 enrolled National Taiwan University Hospital

NaSingle-groupOpen-labelTreatment

Summary

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

Timeline

Start: 2008-01
Primary completion: 2010-12
Completion: 2010-12

Publications

Results Chien YH, Peng SF, Yang CC, Lee NC, Tsai LK, Huang AC, Su SC, Tseng CC, Hwu WL. Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C. J Inherit Metab Dis. 2013 Jan;36(1):129-37. doi: 10.1007/s10545-012-9479-9. Epub 2012 Apr 5.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Miglustat	Small molecule	200 mg	—

Indications