Conditions: Gaucher Disease
Keywords: Gaucher Disease Enzyme replacement therapy
Allocation: NON_RANDOMIZED | Intervention model: PARALLEL
Enrollment: 45
Healthy volunteers: no

Brief summary:
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage system.

This is an extension trial to Study NCT00376168 and NCT00712348.

Detailed description:
This will be a multi-center, double-blind, parallel group, extension trial to assess the safety and efficacy of prGCD in patients completing NCT00376168. Patients will receive IV infusion of prGCD every two weeks at the selected medical center. The duration of the extension study will be fifteen months. There will be two treatment groups: 30 units/kg every 2 weeks or 60 units/kg every 2 weeks.

Arms:
arm 1: Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168) arm 2: Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168) arm 3: Continue taliglucerase alfa treatment from PB-06-002 (NCT00712348)

Interventions: intervention 1: Taliglucerase alfa
Intervention descriptions: intervention 1: Intravenous infusion every 2 weeks

Protocol document: