Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
An Open-label Expanded Access Trial of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease Who Require Enzyme Replacement Therapy
1 other identifier
expanded_access
N/A
2 countries
15
Brief 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.
Trial Health
Trial Health Score
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15 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 18, 2009
CompletedFirst Posted
Study publicly available on registry
August 19, 2009
CompletedSeptember 7, 2018
September 1, 2018
August 18, 2009
September 5, 2018
Conditions
Keywords
Interventions
Intravenous infusion every two weeks at the dose level equal to each patient's previous imiglucerase dose before reduction or discontinuation due to shortage
Eligibility Criteria
You may qualify if:
- Males and females, 18 years or older
- Diagnosis of Gaucher disease treated historically with imiglucerase
- Able to provide written informed consent
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Pfizerlead
Study Sites (15)
La Jolla Village Family Medical Group
La Jolla, California, 92037, United States
University of Colorado Denver
Aurora, Colorado, 80045, United States
University Research Foundation for Lysosomal Storage Diseases, Inc.
Coral Springs, Florida, 33065, United States
Department of Human Genetics, Emory University School of Medicine
Decatur, Georgia, 30033, United States
Orchard Healthcare Research Inc.
Skokie, Illinois, 60076, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
Massachusetts General Hospital Cancer Center
Boston, Massachusetts, 02114, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Neurogenetics, NYU at Rivergate
New York, New York, 10016, United States
Division of Medical Genetics, Duke University Medical Center
Durham, North Carolina, 27710, United States
Department of Medical Genetics, Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15213, United States
Baylor University Medical Center at Dallas, Institute of Metabolic Disease
Dallas, Texas, 75246, United States
Center for Clinical Trials
Springfield, Virginia, 22152, United States
University of Washington, Department of Pediatrics
Seattle, Washington, 98195, United States
Sha'are Zedek Medical Center
Jerusalem, 91031, Israel
Related Publications (2)
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: 17524049BACKGROUNDAviezer 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.
PMID: 19277123BACKGROUND