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| Sponsor: | Protalix |
|---|---|
| Information provided by: | Protalix |
| ClinicalTrials.gov Identifier: | NCT00962260 |
Purpose
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.
| Condition | Intervention |
|---|---|
|
Gaucher Disease |
Drug: Plant cell expressed recombinant glucocerebrosidase (prGCD) |
| Study Type: | Expanded Access |
| 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 |
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| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| Contact: Raul Chertkoff, MD | +972 (4) 988-9488 | raul@protalix.com |
| United States, Florida | |
| University Research Foundation for Lysosomal Storage Diseases, Inc. | |
| Coral Springs, Florida, United States, 33065 | |
| Contact: Neal J Weinreb, MD 954-755-1904 boneal@winning.com | |
| Principal Investigator: Neal J Weinreb, MD | |
| United States, Georgia | |
| Department of Human Genetics, Emory University School of Medicine | |
| Decatur, Georgia, United States, 30033 | |
| Contact: Paul M Fernhoff, MD 404-778-8500 pfernhoff@genetics.emory.edu | |
| Principal Investigator: Paul M Fernhoff, MD | |
| United States, Massachusetts | |
| Massachusetts General Hospital Cancer Center | |
| Boston, Massachusetts, United States, 02114 | |
| Contact: David Kuter, MD 877-726-5130 | |
| Principal Investigator: David Kuter, MD | |
| United States, New York | |
| Neurogenetics, NYU at Rivergate | |
| New York, New York, United States, 10016 | |
| Contact: Gregory M Pastores, MD 212-263-8344 gregory.pastores@med.nyu.edu | |
| Contact: Michele Ford (212) 263-6981 michele.ford@nyumc.org | |
| Principal Investigator: Gregory M Pastores, MD | |
| United States, North Carolina | |
| Division of Medical Genetics, Duke University Medical Center | |
| Durham, North Carolina, United States, 27710 | |
| Contact: Priya Kishnani, MD 919-681-1945 | |
| Principal Investigator: Priya Kishnani, MD | |
| United States, Pennsylvania | |
| Department of Medical Genetics, Children's Hospital of Pittsburgh of UPMC | |
| Pittsburgh, Pennsylvania, United States, 15213 | |
| Contact: David Finegold, MD 412-692-5070 david.finegold@chp.edu | |
| Principal Investigator: David Finegold, MD | |
| United States, Washington | |
| University of Washington, Department of Pediatrics | |
| Seattle, Washington, United States, 98195 | |
| Contact: C. Ronald Scott, MD 206-543-3370 crscott@u.washington.edu | |
| Principal Investigator: C. Ronald Scott, MD | |
| Israel | |
| Sha'are Zedek Medical Center | |
| Jerusalem, Israel, 91031 | |
| Contact: Ari Zimran, MD +972-2-655-5673 zimran@md.huji.ac.il | |
| Principal Investigator: Ari Zimran, MD | |
More Information
| Responsible Party: | Protalix Ltd ( Einat Brill Almon, PhD ) |
| Study ID Numbers: | PB-06-004 |
| Study First Received: | August 18, 2009 |
| Last Updated: | November 24, 2009 |
| ClinicalTrials.gov Identifier: | NCT00962260 History of Changes |
| Health Authority: | United States: Food and Drug Administration; Israel: Ministry of Health |
|
glucocerebrosidase enzyme replacement therapy Gaucher disease plant cell culture |
splenomegaly hepatomegaly anemia thrombocytopenia |
|
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Reticuloendotheliosis Lysosomal Storage Diseases, Nervous System Lysosomal Storage Diseases Nervous System Diseases Central Nervous System Diseases Brain Diseases |
Lymphatic Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn Brain Diseases, Metabolic, Inborn Lipidoses Gaucher Disease Lipid Metabolism Disorders Brain Diseases, Metabolic |