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(s) (Cohort 1):* To demonstrate that CFZ533 600 mg and/or 300 mg bi-weekly (Q2W), subcutaneous (SC), is non-inferior to a tacrolimus-based regimen with respect to the proportion of patients who experience composite efficacy failure…