Safety and Pharmacokinetics of Oral PRX-112 in Gaucher Disease Patients
Status:
Completed
Trial end date:
2014-03-01
Target enrollment:
Participant gender:
Summary
Absorption of therapeutic proteins taken orally has remained the major hurdle for treatment
in humans. The proteins are generally degraded by enzymes in the stomach and intestine and
the intestine lining that prevents absorption into the circulation. Administration of
PRX-112, a plant recombinant human glucocerebrosidase (prGCD) using plant cells as carrier
vehicle, may help overcome many of these hurdles. The plant cell wall protects the protein
from degradation in its transport through the upper GI and allows release in the lower
intestine. Studies in animals have shown that prGCD delivered in this way can be found in the
blood stream in an active form.