Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease
Condition: Gaucher Disease
MeSH terms: Gaucher Disease
Intervention: velaglucerase alfa
Intervention description: 60 U/kg every other week intravenous infusion
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 6.0
Sex: All
Minimum age: 2 Years
Healthy volunteers: No

Summary:

      Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB)
      that leads to progressive accumulation of glucocerebroside within macrophages and subsequent
      tissue and organ damage; typically of the liver, spleen, bone marrow, and brain. The disease
      has been classified into 3 clinical subtypes based on the presence or absence of neurological
      symptoms and severity of neurological disease. Type 1 Gaucher disease affects an estimated
      30,000 persons worldwide and is the most common. Type 1 Gaucher disease does not involve the
      central nervous system. Patients with type 2 Gaucher disease present with acute neurological
      deterioration, which leads to early death. Those with type 3 disease typically display a more
      sub-acute neurological course, with later onset and slower progression.

      The primary objective of this study is to evaluate the safety of every other week dosing of
      velaglucerase alfa in Japanese patients with Gaucher disease.

      Velaglucerase alfa has been developed and approved as an enzyme replacement therapy for Type
      1 Gaucher disease.
    

Eligibility criteria:

        Inclusion Criteria:

          -  The patient has a documented diagnosis of Gaucher disease

          -  The patient is at least 2 years of age

          -  Female patients of child bearing potential must agree to use a medically acceptable
             method of contraception at all times during the study

          -  The patient, the patient's parent(s) or legal guardian(s) has provided written
             informed consent that has been approved by the Institutional Review Board/Independent
             Ethics Committee (IRB/IEC)

          -  The patient must be sufficiently cooperative to participate in this clinical study as
             judged by the Investigator

        Patients who are switched from imiglucerase ERT must meet the following additional
        criteria:

          -  Received treatment with imiglucerase for a minimum of 12 consecutive months

          -  Meet predefined limits for hemoglobin concentration and platelet counts

        Patients naïve to treatment for Gaucher disease must meet the following additional
        criteria:

          -  Not received treatment for Gaucher disease (investigational or approved products)
             within 12 months prior to study entry

          -  Have Gaucher disease related anemia and at least one of the following: moderate
             splenomegaly or, Gaucher disease-related thrombocytopenia or Gaucher disease-related
             enlarged liver

        Exclusion Criteria:

          -  Treatment with any investigational drug or device within the 30 days prior to study
             entry (time of informed consent); such use during the study is not permitted

          -  Positive for hepatitis B or hepatitis C.

          -  Non-Gaucher disease related anemia

          -  The patient, patient's parent(s), or patient's legal guardian(s) is/are unable to
             understand the nature, scope, and possible consequences of the study

          -  Significant comorbidity, as determined by the Investigator that might affect study
             data or confound the study results

          -  The patient is unable to comply with the protocol or is unlikely to complete the
             study, as determined by the Investigator

          -  The patient has experienced a severe (grade 3 or higher) infusion-related
             hypersensitivity reaction (anaphylactic or anaphylactoid reaction) to any ERT
             (approved or investigational)

          -  Currently receiving red blood cell growth factor, (eg, erythropoietin) or chronic
             systemic corticosteroids in the last 6 months

          -  Patient has had a splenectomy or the patient has an active, clinically significant
             spleen infarction within 12 months of screening

          -  Patient has worsening bone necrosis within 12 months of screening

          -  The patient is pregnant or lactating.