Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease
Condition: Gaucher Disease, Type 1
MeSH terms: Gaucher Disease
Intervention: ISU302
Intervention description: 60 U/kg given intravenously
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 8.0
Sex: All
Minimum age: 2 Years
Healthy volunteers: No
Keywords: Type I Gaucher; ISU302; Imiglucerase

Summary:

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

Eligibility 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.