The double-blind, dose-ranging, multi-center, parallel group, randomized study was designed to evaluate the safety and efficacy of taliglucerase alfa in 31 treatment-naive patients suffering from Gaucher disease.
Patients were selected randomly for one of two dosing arms (60 U/kg or 30 U/kg) and received intravenous infusions of taliglucerase alfa once every two weeks for a nine month period.
The primary endpoint of the study was a mean reduction from baseline in spleen volume after nine months, as measured by MRI.
The secondary endpoints were an increase in hemoglobin, decrease in liver volume and increase in platelet count.
Shaare Zedek Medical Center Gaucher Clinic director and lead clinical investigator Ari Zimran said the efficacy and safety that they observed among Gaucher patients receiving taliglucerase alfa in this study suggests that it is a valuable long-term treatment option for patients with Gaucher disease pending regulatory approval.