3 results
Approved WMOPending
The primary objective of this study is to assess the efficacy and safety of Genz-112638 compared with Cerezyme after 52 weeks of treatment in patients with Gaucher disease type 1 who have been stabilized with Cerezyme. The secondary objective of…
Approved WMOCompleted
Primary* To evaluate the effect of EDP1066 in multiple formulations on the systemic immune system.Secondary* To evaluate the safety and tolerability of EDP1066 in multiple formulations.
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.