A Phase I and II Study of PEG-Glucocerebrosidase in Patients With Type 1 or Type 3 Gaucher Disease

Date de début de l'étude : 1 octobre 1993

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.