Clinical Trial Program

The most comprehensive Gaucher disease clinical program for an ERT to date

Study 025—A 9-month, open-label study in 12 patients with type 1 Gaucher disease aged ≥18 years. Velaglucerase alfa was initially administered in a dose-escalating fashion in the first 3 patients; the remaining patients began treatment with 60 Units/kg. The primary objective was to evaluate the safety of velaglucerase alfa at a dose of 60 Units/kg every other week (EOW) for a total of 40 weeks.

Study 025 EXT—Ten of the patients who completed Study 025 enrolled in a 60-month, open-label extension study. The primary objective is to study the long-term safety of velaglucerase alfa. This study is currently ongoing.

Study I: 032—A 12-month, randomized, double-blind, parallel-dose-group multinational safety and efficacy study in 25 adult and pediatric patients. Patients were required to have Gaucher disease-related anemia and either thrombocytopenia or organomegaly. Patients were not allowed to have had disease-specific therapy for at least the previous 30 months; all but one had no prior therapy. The mean age was 26 years (range 4-62) and 40% were female. Patients were randomized to receive VPRIV at a dose of either 45 Units/kg or 60 Units/kg EOW. Primary endpoint was change from baseline in hemoglobin concentration at 53 weeks in patients receiving 60 Units/kg.

Study II: 039—A 9-month, randomized, double-blind, active-controlled (imiglucerase) multinational study of VPRIV and imiglucerase in 34 adult and pediatric patients. Patients had to have Gaucher-related anemia and either thrombocytopenia or organomegaly. Disease-specific therapy within the 12 months prior to enrollment was not allowed. The mean age was 30 years (range 3-73) and 53% were female; the youngest patient to receive VPRIV was aged 4 years. Patients were randomized to receive either 60 Units/kg of VPRIV (n=17) or 60 Units/kg of imiglucerase (n=17) EOW. The primary objective was to compare the effects of VPRIV and imiglucerase on hemoglobin concentrations in patients with type 1 Gaucher disease. 

Study III: 034—A 12-month, open-label, single-arm, multinational safety study in 40 adult and pediatric patients who had been receiving treatment with imiglucerase at doses ranging between 15 Units/kg and 60 Units/kg for a minimum of 30 consecutive months. Patients were required to have a stable biweekly dose of imiglucerase for at least 6 months prior to study enrollment. The mean age was 36 years (range 9-71) and 55% were female. Imiglucerase therapy was stopped, and treatment with VPRIV was administered EOW at the same number of units as the patient’s previous imiglucerase dose. Hemoglobin concentration and platelet counts were evaluated as changes from baseline, which was defined as the end of the patient’s treatment with imiglucerase. The primary objective was to evaluate the safety of EOW dosing with VPRIV in patients with type 1 Gaucher disease who were previously treated with imiglucerase.

Study 044—An ongoing, open-label extension study including patients from trials 032, 034, and 039. The primary objective of this study is to evaluate the long-term safety of VPRIV when given EOW. The secondary endpoints are to evaluate the effect of VPRIV on hemoglobin concentrations, platelet counts, and liver and spleen volumes.