Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

• Status: Completed
• Trial end date: 2013-08-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Pfizer; Protalix

Criteria

Inclusion Criteria:

• Successful completion of Protocol PB-06-001

• The patient signs informed consent

Exclusion Criteria:

• 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