Conditions: Gaucher Disease, Type 1 Cerebroside Lipidosis Syndrome Glucocerebrosidase Deficiency Disease Glucosylceramide Beta-Glucosidase Deficiency Disease Gaucher Disease, Non-Neuronopathic Form
Keywords: Type 1 Gaucher Disease Glucocerebrosidase Deficiency Disease
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 95
Healthy volunteers: no

Brief 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.

Detailed description:

Arms:
arm 1: Patients receiving Cerezyme one infusion every 2 weeks (Q2). arm 2: Patients receiving Cerezyme one infusion every 4 weeks (Q4).

Interventions: intervention 1: Cerezyme
Intervention descriptions: intervention 1: Cerezyme doses of 20-60U/kg every 2 weeks (Q2 Arm) or 40-120 U/kg every 4 weeks (Q4 Arm).

Protocol document: