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Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00962260
Expanded Access Status : No longer available
First Posted : August 19, 2009
Last Update Posted : September 7, 2018
Information provided by (Responsible Party):

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.

Condition or disease Intervention/treatment
Gaucher Disease Drug: Plant cell expressed recombinant glucocerebrosidase (prGCD)

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.

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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

Intervention Details:
  • 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 shortage
    Other Name: taliglucerase alfa

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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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.

Information from the National Library of Medicine

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 identifier (NCT number): NCT00962260

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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
Sha'are Zedek Medical Center
Jerusalem, Israel, 91031
Sponsors and Collaborators
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Responsible Party: Pfizer 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
Keywords provided by Pfizer:
enzyme replacement therapy
Gaucher disease
plant cell culture
Additional relevant MeSH terms:
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Gaucher Disease
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
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders