Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
- Conditions
- Gaucher Disease
- Registration Number
- NCT00962260
- Lead Sponsor
- Pfizer
- 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.
- 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.
Recruitment & Eligibility
- Status
- NO_LONGER_AVAILABLE
- Sex
- All
- Target Recruitment
- Not specified
- Males and females, 18 years or older
- Diagnosis of Gaucher disease treated historically with imiglucerase
- Able to provide written informed consent
- 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.
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (15)
Sha'are Zedek Medical Center
🇮🇱Jerusalem, Israel
University Research Foundation for Lysosomal Storage Diseases, Inc.
🇺🇸Coral Springs, Florida, United States
University of Colorado Denver
🇺🇸Aurora, Colorado, United States
Department of Human Genetics, Emory University School of Medicine
🇺🇸Decatur, Georgia, United States
Orchard Healthcare Research Inc.
🇺🇸Skokie, Illinois, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Massachusetts General Hospital Cancer Center
🇺🇸Boston, Massachusetts, United States
Neurogenetics, NYU at Rivergate
🇺🇸New York, New York, United States
Department of Medical Genetics, Children's Hospital of Pittsburgh of UPMC
🇺🇸Pittsburgh, Pennsylvania, United States
Division of Medical Genetics, Duke University Medical Center
🇺🇸Durham, North Carolina, United States
Baylor University Medical Center at Dallas, Institute of Metabolic Disease
🇺🇸Dallas, Texas, United States
Center for Clinical Trials
🇺🇸Springfield, Virginia, United States
University of Washington, Department of Pediatrics
🇺🇸Seattle, Washington, United States
La Jolla Village Family Medical Group
🇺🇸La Jolla, California, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States