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



Status:No longer available
Conditions:Metabolic
Therapuetic Areas:Pharmacology / Toxicology
Healthy:No
Age Range:18 - Any
Updated:9/9/2018

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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.

Inclusion Criteria:

- Males and females, 18 years or older

- Diagnosis of Gaucher disease treated historically with imiglucerase

- Able to provide written informed consent

Exclusion Criteria:

- Currently taking another experimental drug for any condition

- History of allergy to carrots

- Previous infusion reaction suspected to be allergic in nature to Cerezyme® or
Ceredase® or receiving premedication to prevent infusion reactions

- Allergy to beta-lactam antibiotics

- Presence of any medical, emotional, behavioral or psychological condition that in the
judgment of the Investigator would interfere with the patient's compliance with the
requirements of the study.
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Minneapolis, Minnesota 55455
(612) 625-5000
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
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13001 E. 17th Pl
Aurora, Colorado 80045
303-724-5000
University of Colorado Denver The University of Colorado Denver | Anschutz Medical Campus provides a...
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Aurora, CO
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55 Fruit St
Boston, Massachusetts 02114
(617) 724-4000
Massachusetts General Hospital Cancer Center An integral part of one of the world
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Boston, MA
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Coral Springs, FL
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Dallas, TX
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Decatur, GA
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Durham, NC
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Jerusalem, 91031
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Jerusalem,
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3901 Rainbow Blvd
Kansas City, Kansas 66160
(913) 588-5000
University of Kansas Medical Center The University of Kansas Medical Center serves Kansas through excellence...
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La Jolla, California 92037
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New York, New York 10016
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Seattle, WA
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Skokie, Illinois 60076
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Springfield, Virginia 22152
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Springfield, VA
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