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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 was to study the long-term safety of velaglucerase alfa.
Study 032 — A 12-month, randomized, double-blind, parallel-dose-group multinational safety and efficacy study in 25 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 velaglucerase alfa 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 034 — A 12-month, open-label, single-arm, multinational safety study in 40 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 velaglucerase alfa 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 velaglucerase alfa EOW dosing in patients with type 1 Gaucher disease who were previously treated with imiglucerase.
Study 039 — A 9-month, randomized, double-blind, active-controlled (imiglucerase) multinational study of velaglucerase alfa and imiglucerase in 34 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 velaglucerase alfa was aged 4 years. Patients were randomized to receive either 60 Units/kg of velaglucerase alfa (n=17) or 60 Units/kg of imiglucerase (n=17) EOW. The primary objective was to compare the effects of velaglucerase alfa and imiglucerase on hemoglobin concentrations in patients with type 1 Gaucher disease.
Study 044 — A 5-year, open-label extension study including patients from trials 032, 034, and 039. The primary objective of this study was to evaluate the long-term safety of velaglucerase alfa when given EOW. The secondary endpoints were to evaluate the effect of velaglucerase alfa on hemoglobin concentrations, platelet counts, and liver and spleen volumes.
Study 058 — An FDA-requested treatment protocol which allowed access to velaglucerase alfa for patients affected by a shortage of imiglucerase (n=211).
VPRIV® (velaglucerase alfa for injection) is a prescription medication indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.