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This study evaluates gene-activated human glucocerebrosidase (GA-GCB, VPRIV) enzyme replacement therapy in patients with Type 1 Gaucher disease. It included patients over 2 with confirmed enzyme deficiency and findings such as anemia and enlarged spleen/liver. 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: * Patient has a documented diagnosis of type 1 Gaucher disease, as determined by deficient glucocerebrosidase (GCB) activity relative to normal as measured in leukocytes or by genotype analysis and is willing and able to provide written informed consent prior to initiating any study-related procedures * Patient is at least 2 years of age * Patient has Gaucher disease-related anemia and * Patient has at least moderate splenomegaly or * Patient has Gaucher disease-related thrombocytopenia or * Patient has a readily palpable enlarged liver * Patient has not received treatment for Gaucher disease within 30 months prior to study entry * Female patients of child-bearing potential agree to use a medically acceptable method of contraception. Male patients must agree to use a medically acceptable method of birth control. * Patient must be sufficiently cooperative to participate in the study as judged by the Investigator. Exclusion Criteria: Includes: * Patient has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease * Patient is antibody-positive to imiglucerase during screening or has experienced an anaphylactic reaction to imiglucerase * Patient has received treatment with any investigational drug or device within the 30 days prior to study entry * Patient is Human immunodeficiency virus (HIV) positive * Patient is hepatitis positive * Patient presents with iron, folic acid and/or vitamin B12 deficiency sustained anemia during screening * 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 * Patient has a significant comorbidity(ies)that might affect study data or confound the study results * Patient is a pregnant and/or lactating female * Patient is unable to comply with the protocol or is unlikely to complete the study, as determined by the Investigator