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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
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

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: 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

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 November 20, 2009