Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

Completed · Phase 3

Conditions studied: Gaucher Disease

In brief

Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) 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 is an extension trial to Study NCT00376168 and NCT00712348.

Key facts

Study ID

NCT00705939

Run by

Pfizer

People needed

45

Starts

2008-06-01

Expected to finish

2013-08-01

Last updated by the study team

2018-10-04

Who can join

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

You may qualify if…

• Successful completion of Protocol PB-06-001
• The patient signs informed consent

You may not qualify if…

• Currently taking another experimental drug for any condition
• Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease
• Pregnant or nursing
• 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

• Department of Human Genetics, Emory University School of Medicine — Decatur, Georgia, United States
• Neurogenetics, NYU at Rivergate — New York, New York, United States
• Bone Marrow Transplant Service, The Royal Melbourne Hospital — Parkville, Victoria, Australia
• Mount Sinai Hospital — Toronto, Ontario, Canada
• Pontificia Universidad Catolica de Chile — Santiago, Chile
• Rambam Medical Center — Haifa, Israel
• Shaare Zedek Medical Center — Jerusalem, Israel
• Morningside Medi-Clinic — Morningside, South Africa
• Hospital Universitario Miguel Servet — Zaragoza, Spain
• Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust — Cambridge, United Kingdom
• Royal Free Hospital — London, United Kingdom

Full record on ClinicalTrials.gov

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