Ignite Creation Date:
2025-12-25 @ 2:11 AM
Ignite Modification Date:
2025-12-26 @ 12:39 AM
Study NCT ID:
NCT00962260
Status:
NO_LONGER_AVAILABLE
Last Update Posted:
2018-09-07
First Post:
2009-08-18
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D005776', 'term': 'Gaucher Disease'}, {'id': 'D013163', 'term': 'Splenomegaly'}, {'id': 'D006529', 'term': 'Hepatomegaly'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D013921', 'term': 'Thrombocytopenia'}], 'ancestors': [{'id': 'D013106', 'term': 'Sphingolipidoses'}, {'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}, {'id': 'D006984', 'term': 'Hypertrophy'}, {'id': 'D020763', 'term': 'Pathological Conditions, Anatomical'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D008107', 'term': 'Liver Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D001791', 'term': 'Blood Platelet Disorders'}, {'id': 'D000095542', 'term': 'Cytopenia'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C582473', 'term': 'taliglucerase alfa'}]}}, 'protocolSection': {'designModule': {'studyType': 'EXPANDED_ACCESS', 'nPtrsToThisExpAccNctId': 4}, 'statusModule': {'overallStatus': 'NO_LONGER_AVAILABLE', 'statusVerifiedDate': '2018-09', 'lastUpdateSubmitDate': '2018-09-05', 'studyFirstSubmitDate': '2009-08-18', 'studyFirstSubmitQcDate': '2009-08-18', 'lastUpdatePostDateStruct': {'date': '2018-09-07', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2009-08-19', 'type': 'ESTIMATED'}}, 'conditionsModule': {'keywords': ['glucocerebrosidase', 'enzyme replacement therapy', 'Gaucher disease', 'plant cell culture', 'splenomegaly', 'hepatomegaly', 'anemia', 'thrombocytopenia'], 'conditions': ['Gaucher Disease']}, 'referencesModule': {'references': [{'pmid': '17524049', 'type': 'BACKGROUND', 'citation': "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. doi: 10.1111/j.1467-7652.2007.00263.x. Epub 2007 May 24."}, {'pmid': '19277123', 'type': 'BACKGROUND', 'citation': '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. doi: 10.1371/journal.pone.0004792. Epub 2009 Mar 11.'}]}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': "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.\n\nprGCD 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.\n\nprGCD 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."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'eligibilityCriteria': "Inclusion Criteria:\n\n* Males and females, 18 years or older\n* Diagnosis of Gaucher disease treated historically with imiglucerase\n* Able to provide written informed consent\n\nExclusion Criteria:\n\n* Currently taking another experimental drug for any condition\n* History of allergy to carrots\n* Previous infusion reaction suspected to be allergic in nature to Cerezyme® or Ceredase® or receiving premedication to prevent infusion reactions\n* Allergy to beta-lactam antibiotics\n* 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."}, 'identificationModule': {'nctId': 'NCT00962260', 'briefTitle': 'Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease', 'organization': {'class': 'INDUSTRY', 'fullName': 'Pfizer'}, 'officialTitle': 'An Open-label Expanded Access Trial of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease Who Require Enzyme Replacement Therapy', 'orgStudyIdInfo': {'id': 'PB-06-004'}}, 'armsInterventionsModule': {'interventions': [{'name': 'Plant cell expressed recombinant glucocerebrosidase (prGCD)', 'type': 'DRUG', 'otherNames': ['taliglucerase alfa'], 'description': "Intravenous infusion every two weeks at the dose level equal to each patient's previous imiglucerase dose before reduction or discontinuation due to shortage"}]}, 'contactsLocationsModule': {'locations': [{'zip': '92037', 'city': 'La Jolla', 'state': 'California', 'country': 'United States', 'facility': 'La Jolla Village Family Medical Group', 'geoPoint': {'lat': 32.84727, 'lon': -117.2742}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': 'University of Colorado Denver', 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '33065', 'city': 'Coral Springs', 'state': 'Florida', 'country': 'United States', 'facility': 'University Research Foundation for Lysosomal Storage Diseases, Inc.', 'geoPoint': {'lat': 26.27119, 'lon': -80.2706}}, {'zip': '30033', 'city': 'Decatur', 'state': 'Georgia', 'country': 'United States', 'facility': 'Department of Human Genetics, Emory University School of Medicine', 'geoPoint': {'lat': 33.77483, 'lon': -84.29631}}, {'zip': '60076', 'city': 'Skokie', 'state': 'Illinois', 'country': 'United States', 'facility': 'Orchard Healthcare Research Inc.', 'geoPoint': {'lat': 42.03336, 'lon': -87.73339}}, {'zip': '66160', 'city': 'Kansas City', 'state': 'Kansas', 'country': 'United States', 'facility': 'University of Kansas Medical Center', 'geoPoint': {'lat': 39.11417, 'lon': -94.62746}}, {'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Massachusetts General Hospital Cancer Center', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'University of Minnesota', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '10016', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Neurogenetics, NYU at Rivergate', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '27710', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Division of Medical Genetics, Duke University Medical Center', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '15213', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Department of Medical Genetics, Children's Hospital of Pittsburgh of UPMC", 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '75246', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'Baylor University Medical Center at Dallas, Institute of Metabolic Disease', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '22152', 'city': 'Springfield', 'state': 'Virginia', 'country': 'United States', 'facility': 'Center for Clinical Trials', 'geoPoint': {'lat': 38.78928, 'lon': -77.1872}}, {'zip': '98195', 'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'facility': 'University of Washington, Department of Pediatrics', 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'zip': '91031', 'city': 'Jerusalem', 'country': 'Israel', 'facility': "Sha'are Zedek Medical Center", 'geoPoint': {'lat': 31.76904, 'lon': 35.21633}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Pfizer', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}