A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
Status:
Completed
Trial end date:
2009-10-01
Target enrollment:
Participant gender:
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.