2 results
Approved WMORecruiting
Primary objective· • To investigate the safety and tolerability and establish either the dose of KU-0059436 which causes inhibition of PARP in combination with an active dose of carboplatin or the maximum tolerated dose (MTD) of KU-0059436 in…
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.