Gaucher Disease Clinical Trial
Official title:
An Open-label Expanded Access Trial of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease Who Require Enzyme Replacement Therapy
This is an open-label expanded access trial of prGCD in patients with Gaucher disease who require enzyme replacement therapy (ERT) and who have been treated with imiglucerase but for whom the dose has been reduced or discontinued due to shortage of the product.
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the
human glucocerebrosidase gene (GCD), which have been mapped to chromosome 1 q21-q31, 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 accumulation leads to the visceral manifestations of hepatosplenomegaly, anemia
and thrombocytopenia, as well as to the skeletal features and less frequently also to lung
involvement.
prGCD is a plant cell expressed recombinant glucocerebrosidase enzyme for the treatment of
Gaucher disease. Expression of proteins in plant cell culture is highly efficient, does not
require post-expression modification of the protein, and is not susceptible to contamination
by agents such as viruses that are pathological to humans.
prGCD safety will be observed in this treatment protocol of patients with non-neuronopathic
Gaucher disease who require enzyme replacement therapy. Eligible patients will receive
intravenous (IV) infusions of prGCD every two weeks. The dose of prGCD will be equal to each
patient's previous imiglucerase dose before reduction or discontinuation due to shortage. The
infusions will be administered at the selected medical center.
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