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An Open-label Expanded Access Trial of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease Who Require Enzyme Replacement Therapy

18 Years
Open (Enrolling)
Gaucher Disease

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Trial Information

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.

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

- 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.

Type of Study:

Expanded Access

Study Design:



United States: Food and Drug Administration

Study ID:




Start Date:

Completion Date:

Related Keywords:

  • Gaucher Disease
  • glucocerebrosidase
  • enzyme replacement therapy
  • Gaucher disease
  • plant cell culture
  • splenomegaly
  • hepatomegaly
  • anemia
  • thrombocytopenia
  • Gaucher Disease



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