An Open-label Expanded Access Trial of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease Who Require Enzyme Replacement Therapy
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.
Expanded Access
N/A
United States: Food and Drug Administration
PB-06-004
NCT00962260
Name | Location |
---|---|
Massachusetts General Hospital Cancer Center | Boston, Massachusetts 02114 |
University of Kansas Medical Center | Kansas City, Kansas 66160-7353 |
University of Minnesota | Minneapolis, Minnesota 55455 |
University of Colorado Denver | Denver, Colorado 80262 |
Orchard Healthcare Research Inc. | Skokie, Illinois 60076 |
La Jolla Village Family Medical Group | La Jolla, California 92037 |
University Research Foundation for Lysosomal Storage Diseases, Inc. | Coral Springs, Florida 33065 |
Department of Human Genetics, Emory University School of Medicine | Decatur, Georgia 30033 |
Neurogenetics, NYU at Rivergate | New York, New York 10016 |
Division of Medical Genetics, Duke University Medical Center | Durham, North Carolina 27710 |
Department of Medical Genetics, Children's Hospital of Pittsburgh of UPMC | Pittsburgh, Pennsylvania 15213 |
Baylor University Medical Center at Dallas, Institute of Metabolic Disease | Dallas, Texas 75246 |
Center for Clinical Trials | Springfield, Virginia 22152 |
University of Washington, Department of Pediatrics | Seattle, Washington 98195 |