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Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
Expanded access is currently available for this treatment.
Verified by Protalix, November 2009
First Received: August 18, 2009   Last Updated: November 24, 2009   History of Changes
Sponsor: Protalix
Information provided by: Protalix
ClinicalTrials.gov Identifier: NCT00962260
  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
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
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
  • Presence of anti-glucocerebrosidase (GCD) antibodies
  • 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00962260

Contacts
Contact: Raul Chertkoff, MD +972 (4) 988-9488 raul@protalix.com

Locations
United States, Florida
University Research Foundation for Lysosomal Storage Diseases, Inc.
Coral Springs, Florida, United States, 33065
Contact: Neal J Weinreb, MD     954-755-1904     boneal@winning.com    
Principal Investigator: Neal J Weinreb, MD            
United States, Georgia
Department of Human Genetics, Emory University School of Medicine
Decatur, Georgia, United States, 30033
Contact: Paul M Fernhoff, MD     404-778-8500     pfernhoff@genetics.emory.edu    
Principal Investigator: Paul M Fernhoff, MD            
United States, Massachusetts
Massachusetts General Hospital Cancer Center
Boston, Massachusetts, United States, 02114
Contact: David Kuter, MD     877-726-5130        
Principal Investigator: David Kuter, MD            
United States, New York
Neurogenetics, NYU at Rivergate
New York, New York, United States, 10016
Contact: Gregory M Pastores, MD     212-263-8344     gregory.pastores@med.nyu.edu    
Contact: Michele Ford     (212) 263-6981     michele.ford@nyumc.org    
Principal Investigator: Gregory M Pastores, MD            
United States, North Carolina
Division of Medical Genetics, Duke University Medical Center
Durham, North Carolina, United States, 27710
Contact: Priya Kishnani, MD     919-681-1945        
Principal Investigator: Priya Kishnani, MD            
United States, Pennsylvania
Department of Medical Genetics, Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15213
Contact: David Finegold, MD     412-692-5070     david.finegold@chp.edu    
Principal Investigator: David Finegold, MD            
United States, Washington
University of Washington, Department of Pediatrics
Seattle, Washington, United States, 98195
Contact: C. Ronald Scott, MD     206-543-3370     crscott@u.washington.edu    
Principal Investigator: C. Ronald Scott, MD            
Israel
Sha'are Zedek Medical Center
Jerusalem, Israel, 91031
Contact: Ari Zimran, MD     +972-2-655-5673     zimran@md.huji.ac.il    
Principal Investigator: Ari Zimran, MD            
Sponsors and Collaborators
Protalix
  More Information

Publications:
Responsible Party: Protalix Ltd ( Einat Brill Almon, PhD )
Study ID Numbers: PB-06-004
Study First Received: August 18, 2009
Last Updated: November 24, 2009
ClinicalTrials.gov Identifier: NCT00962260     History of Changes
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:
Lipid Metabolism, Inborn Errors
Sphingolipidoses
Metabolic Diseases
Reticuloendotheliosis
Lysosomal Storage Diseases, Nervous System
Lysosomal Storage Diseases
Nervous System Diseases
Central Nervous System Diseases
Brain Diseases
Lymphatic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Brain Diseases, Metabolic, Inborn
Lipidoses
Gaucher Disease
Lipid Metabolism Disorders
Brain Diseases, Metabolic

ClinicalTrials.gov processed this record on February 08, 2010