2 results
Approved WMOWill not start
The purpose of this study is to evaluate the possible risks and efficacy (improvement of disease) with an experimental oral study drug named eliglustat in pediatric patients from 2 to 18 years with Gaucher disease.
Approved WMOCompleted
Primary objective is the delta change in whole body insulin sensitivity upon Na-PB vs. placebo treatment. Secondary objectives are muscle mitochondrial oxidative capacity, muscle and liver fat content and energy metabolism.