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

NCT ID: NCT00962260

Last Updated: 2018-09-07

Basic Information

• Recruitment Status: NO_LONGER_AVAILABLE
• Study Classification: EXPANDED_ACCESS

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.

Conditions

Gaucher Disease

Interventions

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

Intervention Type: DRUG

Other Intervention Names

taliglucerase alfa

Eligibility 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
• 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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Pfizer

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

La Jolla Village Family Medical Group

La Jolla, California, United States

University of Colorado Denver

Aurora, Colorado, United States

University Research Foundation for Lysosomal Storage Diseases, Inc.

Coral Springs, Florida, 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 Kansas Medical Center

Kansas City, Kansas, United States

Massachusetts General Hospital Cancer Center

Boston, Massachusetts, United States

University of Minnesota

Minneapolis, Minnesota, United States

Neurogenetics, NYU at Rivergate

New York, New York, United States

Division of Medical Genetics, Duke University Medical Center

Durham, North Carolina, United States

Department of Medical Genetics, Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 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

Sha'are Zedek Medical Center

Jerusalem, , Israel

Countries

United States; Israel

References

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. doi: 10.1111/j.1467-7652.2007.00263.x. Epub 2007 May 24.

Reference Type: BACKGROUND

PMID: 17524049 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 19277123 (View on PubMed)

Other Identifiers

PB-06-004

Identifier Type: -

Identifier Source: org_study_id