At a glance
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A Phase III, Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
In Brief
A Phase 3 clinical trial evaluating Plant cell expressed recombinant glucocerebrosidase (prGCD) for Gaucher Disease. Completed, enrolled 32 participants across 11 sites in 8 countries.
Detailed 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 the second trial to utilize a recombinant active form of lysosomal enzyme, glucocerebrosidase, (human prGCD) which is expressed and purified in a bioreactor system from transformed carrot plant root cell line.
Study Details
Timeline
Interventions
Intravenous infusion every two weeks for 9 months
Intravenous infusion every 2 weeks for 9 months