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

Expanded access is no longer available for this treatment.
Sponsor:
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT00962260
First received: August 18, 2009
Last updated: August 13, 2013
Last verified: August 2013
  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     What is 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

Resource links provided by NLM:


Further study details as provided by Protalix:

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
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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Criteria

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.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00962260

Locations
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
Sponsors and Collaborators
Protalix
  More Information

Publications:
Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT00962260     History of Changes
Other Study ID Numbers: PB-06-004
Study First Received: August 18, 2009
Last Updated: August 13, 2013
Health Authority: United States: Food and Drug Administration
Israel: Ministry of Health

Keywords provided by Protalix:
glucocerebrosidase
enzyme replacement therapy
Gaucher disease
plant cell culture
splenomegaly
hepatomegaly
anemia
thrombocytopenia

Additional relevant MeSH terms:
Gaucher Disease
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Central Nervous System Diseases
Genetic Diseases, Inborn
Lipid Metabolism Disorders
Lipid Metabolism, Inborn Errors
Lipidoses
Lysosomal Storage Diseases
Lysosomal Storage Diseases, Nervous System
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases
Sphingolipidoses

ClinicalTrials.gov processed this record on November 20, 2014