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:• To evaluate the effect of efgartigimod IV compared to placebo on CRESSSecondary:• To evaluate the effect of efgartigimod IV compared to placebo on the histology of the parotid gland (selected sites only)• To evaluate the safety of…