PROTALIX : Accés étendu à l'essai de Glucocerebrosidase (prGCD) Recombinant dans la maladie de Gaucher

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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, August 2009

   

    First Received: August 18, 2009            No Changes Posted       

                                                                                     

Sponsored by:	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:

 
 

    Genetics Home Reference related topics:             Chanarin-Dorfman syndrome                 cholesteryl ester storage disease                 Farber lipogranulomatosis                 Gaucher disease                 primary carnitine deficiency                 succinic semialdehyde dehydrogenase deficiency          

       

    MedlinePlus related topics:             Gaucher's Disease          

       

    Drug Information available for:             Alglucerase                 Imiglucerase          

   

    U.S. FDA Resources  

         
 

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:	Bot

           

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

                  Sponsors and Collaborators
Protalix        

  More Information

 
      Publications:  

            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. Epub 2007 May 24.          

       

            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. Epub 2009 Mar 11.          

           
                                                                                                                                                                                            

Responsible Party:	Protalix Ltd ( Einat Brill Almon, PhD )
Study ID Numbers:	PB-06-004
Study First Received:	August 18, 2009
Last Updated:	August 18, 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

         
  Study placed in the following topic categories:                                                   

Lipid Metabolism, Inborn Errors                     Sphingolipidoses                     Metabolic Diseases                     Lysosomal Storage Diseases                     Anemia                     Sphingolipidosis                     Central Nervous System Diseases                     Brain Diseases                     Lymphatic Diseases                     Metabolism, Inborn Errors

Thrombocytopenia                     Genetic Diseases, Inborn                     Splenomegaly                     Lipidoses                     Brain Diseases, Metabolic, Inborn                     Gaucher Disease                     Metabolic Disorder                     Hepatomegaly                     Lipid Metabolism Disorders                     Brain Diseases, Metabolic

     
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 August 21, 2009
   

 

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  http://www.clinicaltrials.gov/ct2/show/NCT00962260?term=prgcd