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Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease

Expanded access is no longer available for this treatment.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00962260
First Posted: August 19, 2009
Last Update Posted: August 14, 2013
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Protalix
August 18, 2009
August 19, 2009
August 14, 2013
 
Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
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.

Expanded Access
Drug: Plant cell expressed recombinant glucocerebrosidase (prGCD)
Intravenous infusion every two weeks at the dose level equal to each patient's previous imiglucerase dose before reduction or discontinuation due to shortage
Other Name: taliglucerase alfa
 
No longer available
Contact information is only displayed when the study is recruiting subjects
Israel,   United States
 
 
NCT00962260
Protalix
Protalix
Not Provided
Not Provided
Protalix
August 2013