Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
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ClinicalTrials.gov Identifier: NCT00962260 |
Expanded Access Status :
No longer available
First Posted : August 19, 2009
Last Update Posted : September 7, 2018
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Condition or disease | Intervention/treatment |
---|---|
Gaucher Disease | Drug: Plant cell expressed recombinant glucocerebrosidase (prGCD) |
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.
Study Type : | Expanded Access |
See clinical trials of the intervention/treatment in this expanded access record. | |
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 |

- 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 shortageOther Name: taliglucerase alfa

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00962260
United States, California | |
La Jolla Village Family Medical Group | |
La Jolla, California, United States, 92037 | |
United States, Colorado | |
University of Colorado Denver | |
Aurora, Colorado, United States, 80045 | |
United States, Florida | |
University Research Foundation for Lysosomal Storage Diseases, Inc. | |
Coral Springs, Florida, United States, 33065 | |
United States, Georgia | |
Department of Human Genetics, Emory University School of Medicine | |
Decatur, Georgia, United States, 30033 | |
United States, Illinois | |
Orchard Healthcare Research Inc. | |
Skokie, Illinois, United States, 60076 | |
United States, Kansas | |
University of Kansas Medical Center | |
Kansas City, Kansas, United States, 66160 | |
United States, Massachusetts | |
Massachusetts General Hospital Cancer Center | |
Boston, Massachusetts, United States, 02114 | |
United States, Minnesota | |
University of Minnesota | |
Minneapolis, Minnesota, United States, 55455 | |
United States, New York | |
Neurogenetics, NYU at Rivergate | |
New York, New York, United States, 10016 | |
United States, North Carolina | |
Division of Medical Genetics, Duke University Medical Center | |
Durham, North Carolina, United States, 27710 | |
United States, Pennsylvania | |
Department of Medical Genetics, Children's Hospital of Pittsburgh of UPMC | |
Pittsburgh, Pennsylvania, United States, 15213 | |
United States, Texas | |
Baylor University Medical Center at Dallas, Institute of Metabolic Disease | |
Dallas, Texas, United States, 75246 | |
United States, Virginia | |
Center for Clinical Trials | |
Springfield, Virginia, United States, 22152 | |
United States, Washington | |
University of Washington, Department of Pediatrics | |
Seattle, Washington, United States, 98195 | |
Israel | |
Sha'are Zedek Medical Center | |
Jerusalem, Israel, 91031 |
Responsible Party: | Pfizer |
ClinicalTrials.gov Identifier: | NCT00962260 |
Other Study ID Numbers: |
PB-06-004 |
First Posted: | August 19, 2009 Key Record Dates |
Last Update Posted: | September 7, 2018 |
Last Verified: | September 2018 |
glucocerebrosidase enzyme replacement therapy Gaucher disease plant cell culture |
splenomegaly hepatomegaly anemia thrombocytopenia |
Gaucher Disease Sphingolipidoses Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases |
Metabolism, Inborn Errors Genetic Diseases, Inborn Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders |