Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
Information source: Protalix
Information obtained from ClinicalTrials.gov on October 19, 2009
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Gaucher Disease
Intervention: Plant cell expressed recombinant glucocerebrosidase (prGCD) (Drug)
Phase: N/A
Status: Available
Sponsored by: Protalix Overall contact:
Raul Chertkoff, MD, Phone: +972 (4) 988-9488, Email: raul@protalix.com
Summary
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.
Clinical Details
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
Study design: N/A
Detailed description:
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.
Eligibility
Minimum age: 18 Years.
Maximum age: N/A.
Gender(s): Both.
Criteria:
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
- Presence of anti-glucocerebrosidase (GCD) antibodies
- 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.
Locations and Contacts
Raul Chertkoff, MD, Phone: +972 (4) 988-9488, Email: raul@protalix.com Additional Information
Related publications: Shaaltiel Y, Bartfeld D, Hashmueli S, Baum G, Brill-Almon E, Galili G, Dym O, Boldin-Adamsky SA, Silman I, Sussman JL, Futerman AH, Aviezer D. Production of glucocerebrosidase with terminal mannose glycans for enzyme replacement therapy of Gaucher's disease using a plant cell system. Plant Biotechnol J. 2007 Sep;5(5):579-90. Epub 2007 May 24. Aviezer D, Brill-Almon E, Shaaltiel Y, Hashmueli S, Bartfeld D, Mizrachi S, Liberman Y, Freeman A, Zimran A, Galun E. A plant-derived recombinant human glucocerebrosidase enzyme--a preclinical and phase I investigation. PLoS ONE. 2009;4(3):e4792. Epub 2009 Mar 11.
Last updated: August 18, 2009
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