Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease
Condition: Gaucher Disease, Type 1; Cerebroside Lipidosis Syndrome; Glucocerebrosidase Deficiency Disease; Glucosylceramide Beta-Glucosidase Deficiency Disease; Gaucher Disease, Non-Neuronopathic Form
MeSH terms: Gaucher Disease; Lipidoses; Deficiency Diseases
Intervention: Cerezyme
Intervention description: Cerezyme doses of 20-60U/kg every 2 weeks (Q2 Arm) or 40-120 U/kg every 4 weeks (Q4 Arm).
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 95.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Type 1 Gaucher Disease; Glucocerebrosidase Deficiency Disease

Summary:

      This is a multicenter, randomized trial to compare the safety and efficacy of two dosing
      frequencies of Cerezyme® in patients with Gaucher disease who are currently being treated
      with Cerezyme®.

      Approximately 90 patients will be randomized in a 2:1 (q4 : q2) ratio to one of two treatment
      arms at up to 26 study centers worldwide. Patients will continue to receive the same total
      4-week dose that they were receiving prior to study enrollment, however, they will be
      randomized to receive either their total 4-week dose in two infusions, one infusion every 2
      weeks or their total 4-week dose in one infusion every 4 weeks. The randomization scheme will
      ensure a 2:1 balance between the every 4-week versus every 2-week infusion groups,
      respectively.
    

Eligibility criteria:

        Inclusion Criteria:

          -  The patient must provide written informed consent prior to undergoing any
             study-related procedures.

          -  The patient has a confirmed diagnosis of Gaucher disease with a documented deficiency
             of glucocerebrosidase by enzyme assay

          -  The patient has been genotyped or will have genotyping performed within 3 months of
             study enrollment.

          -  The patient has been treated with Cerezyme for at least 2 years prior to study
             enrollment.

          -  The patient has been on a stable dose of between 20-60 U/kg every 2 weeks for at least
             6 months prior to study enrollment.

          -  The patient is at least 18 years old.

          -  The patient has a hemoglobin value of ≥ 11.0 g/dL for women and ≥ 12.0 g/dL for men
             and a platelet count of ≥ 100,000 mm^3.

          -  The patient's liver volume is ≤ 1.8 x normal confirmed by MRI or CT within 6 months of
             randomization.

          -  The patient's spleen volume is ≤ 10 x normal confirmed by MRI or CT within 6 months of
             randomization.

          -  The patient has a serum creatinine < 2.0 mg/dL, an ASTand ALT < 2 x upper limit of
             normal and a total bilirubin < 2.0 x upper limit of normal.

          -  Female patients of childbearing potential must have a negative pregnancy test within 2
             weeks prior to randomization into the study.

        Exclusion Criteria:

          -  The patient is pregnant.

          -  The patient has evidence of neurologic or pulmonary involvement with Gaucher disease
             confirmed by medical history.

          -  The patient has evidence of current or prior bleeding varices or liver infarction
             requiring hospitalization confirmed by medical history.

          -  The patient has evidence of pathologic bone fractures, medullary infarctions, lytic
             lesions or avascular necrosis secondary to Gaucher disease confirmed by skeletal
             evaluation within 6 months of randomization.

          -  The patient has had a bone crisis (defined as pain with acute onset which requires
             immobilization of the affected area, narcotics for relief of pain and may be
             accompanied by periosteal elevation, increased white cell count, fever or debilitation
             of > 3 days) within 12 months of randomization.

          -  Patient has received an investigational drug within 30 days of the start of their
             participation in this trial. Patients may not receive any other investigational
             product throughout the course of the study.

          -  The patient has a clinically significant disease (with the exception of symptoms
             relating to Gaucher disease), including clinically significant cardiovascular,
             hepatic, immunologic, pulmonary, neurologic, or renal disease, or other medical
             condition, serious intercurrent illness, or extenuating circumstances that, in the
             opinion of the Investigator, would preclude participation in the trial or potentially
             decrease survival

          -  Patient has a medical, emotional, behavioral or psychological condition that in the
             judgment of the Investigator would interfere with the patient's compliance with the
             requirements of the study.