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

• Status: Completed
• Trial end date: 2014-08-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the safety and efficacy of ISU302 in patients with Type 1 Gaucher disease.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: ISU Abxis Co., Ltd.

Criteria

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 device within 30 days prior to study entry; such use during the study was also not permitted.

• Currently receiving red blood cell (RBC) growth factor (eg, erythropoietin) chronic systemic corticosteroids or received such treatment within the last 6 months.

• Positive for human immunodeficiency virus (HIV) and hepatitis B or C.

• Exacerbated anemia at screening (due to iron, folic acid, or vitamin B12 deficiency or infectious/immune-mediated cause).

• Significant comorbidity(ies) that could affect study data or confounded the study results (eg, malignancies, primary biliary cirrhosis, autoimmune liver disease).

• Pregnant or lactating female patients and those not willing to use highly effective barrier or medical method of contraception.