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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
95
Cerezyme doses of 20-60U/kg every 2 weeks (Q2 Arm) or 40-120 U/kg every 4 weeks (Q4 Arm).
Children's National Medical Center
Washington D.C., District of Columbia, United States
University Research Foundation for Lysosomal Storage Disease, Inc.
Coral Springs, Florida, United States
Emory University
Atlanta, Georgia, United States
Children's Memorial Hospital
Chicago, Illinois, United States
Midwest Cancer Research Group, Inc.
Skokie, Illinois, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Institute for Genetics Medicine Saint Peter's University Hospital
New Brunswick, New Jersey, United States
Holy Name Hospital
Teaneck, New Jersey, United States
Hemophilia Center of Western New York
Buffalo, New York, United States
New York Oncology/Hematology PC
Latham, New York, United States
...and 16 more locations
Number of Participants With Clinical Success at Month 24/Discontinuation
Patients are considered to be a clinical success if ALL of the following are met: The patient's hemoglobin does not fall more than 1.25g/dL for women or 1.5 g/dL for men below the patient's baseline value, platelet count does not fall more than 25% below the patient's baseline value or does not fall below 80,000 mm3, liver and spleen volumes are not greater than 20% above the patient's baseline value, no evidence of bone disease progression, including no incidence of pathologic fractures, medullary infarctions, lytic lesions or avascular necrosis and has had no bone crises during the study.
Time frame: Month 24 (or at time of discontinuation)
Mean Composite Scores of the SF-36 Health Survey at Baseline
The mean composite scores (0 being worst and 100 being best) for both treatment groups at Baseline. Composite scores for both treatment groups approximated those of the general population at baseline and at Month 24.
Time frame: Baseline
Mean Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation.
The mean composite scores (0 being worst and 100 being best) for both treatment groups at Month 24/Discontinuation. The mean composite scores for both treatment groups approximated those of the general population at baseline and at Month 24.
Time frame: Month 24 (or at time of discontinuation)
Mean Change From Baseline in Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation
The mean composite scores (0 being worst and 100 being best) for both treatment groups approximated those of the general population at baseline. Composite score - The overall composite scores were comprised of a standardized physical and mental component score.
Time frame: Baseline and Month 24 (or at time of discontinuation)
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