PEG-Glucocerebrosidase for the Treatment of Gaucher Disease

Inclusion Criteria

• •Patients must be at least 3 years of age.
• •Must have a biochemically confirmed (enzyme) and/or genetically confirmed diagnosis of type 1 or type 3 Gaucher disease.
• •Clinical or laboratory signs suggesting need for therapy which will include at least 2 of the following: hemoglobin less than 11 gm/dl; platelets less than 90,000/mm(3); hepatomegaly and/or splenomegaly.
• •Patient/Guardian must provide written informed consent.
• •No pregnant or breast feeding women.
• •No women/men of reproductive potential unless they agree to use an effective contraceptive method.
• •No patients treated with alglucerase or imiglucerase during the 6 months prior to study entry.
• •No patients with the diagnosis of type 2 Gaucher disease.
• •No patients who have a life-threatening disease or are gravely ill.
• •No patients who have rapidly progressing fatal illness or concomitant malignancy.
• •No patients who have a chronic infectious disease including HIV or hepatitis B.
• •No patients chronically on other medications which may interfere with the drug's metabolism or activity.
• •No patients who received blood transfusion within a month prior to study entry.