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This study evaluates Velaglucerase Alfa enzyme replacement therapy in Japanese patients with Gaucher disease. It included patients aged 2 and older with a confirmed Gaucher diagnosis. It was a Phase 3 study and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
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.