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 WMOPending
To assess the safety and tolerability ofBNT141 at different dose levels.To identify the maximum tolerated dose (MTD) or maximally administered dose (MAD) /recommended Phase IIdose (RP2D) of BNT141 based on theoccurrence of dose-limiting toxicities(…