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Clinical Trial Details — Status: No longer available

Administrative data

NCT number NCT00962260
Other study ID # PB-06-004
Secondary ID
Status No longer available
Phase
First received
Last updated

Study information

Verified date September 2018
Source Pfizer
Contact n/a
Is FDA regulated No
Health authority
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
Est. completion date
Est. primary completion date
Accepts healthy volunteers
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.

Study Design


Related Conditions & MeSH terms


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

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