2 results
Approved WMOPending
Primary:- To assess the safety and tolerability of MK2140- To evaluate objective response rate (ORR) of MK2140 as assessed by BICR per RECIST 1.1Secondary:-To evaluate the duration of response (DOR) of MK2140 as assessed by BICR per RECIST 1.1
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.