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

NO_LONGER_AVAILABLE

Conditions studied: Gaucher Disease

In brief

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.

Key facts

Study ID

NCT00962260

Run by

Pfizer

Last updated by the study team

2018-09-07

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Males and females, 18 years or older
• Diagnosis of Gaucher disease treated historically with imiglucerase
• Able to provide written informed consent

You may not qualify if…

• 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.

Where it is running

• 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

Full record on ClinicalTrials.gov

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