Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease

Inclusion Criteria: * Diagnosis of Type 1 GD. * Documented glucocerebrosidase deficiency. * GD-related anemia, defined as hemoglobin levels of at least 1 g/dL below the lower limit of normal for age and gender and one or more of the following 3 criteria: * At least moderate splenomegaly (2 to 3 cm below the left costal margin) by palpation, * GD-related thrombocytopenia, defined as a platelet count \<90 x 109 platelets/L, * GD-related readily palpable enlarged liver. * Not received treatment for GD (investigational products, miglustat, velaglucerase alfa, or imiglucerase) within 12 months prior to study entry. * Ability to comprehend and willing to sign the ICF. * Legal guardian (and patient if age appropriate) understood the nature of the procedure, was willing to comply with associated follow-up evaluations, and provided written informed consent and assent prior to the procedure. * Female patients of childbearing potential must had agreed to use a medically acceptable method of contraception at all the times during the study. Male patients must have used a medically acceptable method of birth control throughout their participation in the study and were required to report the pregnancy of a partner. Exclusion Criteria: * Type 2 or 3 GD. * Splenectomy. * Antibody positive to ISU302 or imiglucerase during screening or the patient had experienced an anaphylactic reaction to ISU302 or imiglucerase. - Treatment with any non-GD-related investigational drug or medical dev…