Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease

Gaucher Disease Clinical Trial

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

Clinical Trial Details — Status: No longer available

Administrative data

• NCT number: NCT00962260
• Other study ID #: PB-06-004
• Status: No longer available

Study information

• Verified date: September 2018
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial 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.

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 information / eligibility

• Status: No longer available
• Enrollment: 0
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Gaucher Disease

Intervention

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

Locations

Country	Name	City	State
Israel	Sha'are Zedek Medical Center	Jerusalem
United States	University of Colorado Denver	Aurora	Colorado
United States	Massachusetts General Hospital Cancer Center	Boston	Massachusetts
United States	University Research Foundation for Lysosomal Storage Diseases, Inc.	Coral Springs	Florida
United States	Baylor University Medical Center at Dallas, Institute of Metabolic Disease	Dallas	Texas
United States	Department of Human Genetics, Emory University School of Medicine	Decatur	Georgia
United States	Division of Medical Genetics, Duke University Medical Center	Durham	North Carolina
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	La Jolla Village Family Medical Group	La Jolla	California
United States	University of Minnesota	Minneapolis	Minnesota
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	University of Washington, Department of Pediatrics	Seattle	Washington
United States	Orchard Healthcare Research Inc.	Skokie	Illinois
United States	Center for Clinical Trials	Springfield	Virginia

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

United States,  Israel, 

References & Publications (2)

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. doi: 10.1371/journal.pone.0004792. Epub 2009 Mar 11. — View Citation

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. — View Citation