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.
This exploratory, open-label safety and pharmacokinetic (PK) study is designed to assess the delivery of prGCD after oral administration of PRX-112 in Gaucher subjects. Subjects will receive an oral dose of PRX-112 in a single administration and followed by 3 consecutive daily administrations at the same dose. prGCD levels in plasma will be determined at selected time points. Safety parameters will also be assessed at selected time points. Enrollment will proceed into the next dosage cohort after the pharmacokinetic and safety data of the previous cohort have been reviewed. A different dosage may be selected based on the pharmacokinetic results of the first cohort.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
16
Single dose level, four doses per cohort
Rambam Medical Center
Haifa, Israel
Shaare Zedek Medical Center
Jerusalem, Israel
Adverse Events
Spontaneous reports of adverse events, or events identified during physical examination or clinical laboratory testing
Time frame: 3 days after the last dose
Area Under the Curve (AUC)
Area under the GCD level curve 0-30 hours (AUC0-30h)
Time frame: From start of infusion to 30 hours after infusion
Maximum Concentration (Cmax)
Time frame: From start of infusion to 30 hours after infusion
Time of maximum prGCD concentration (Tmax)
Time frame: From start of infusion to 30 hours after infusion
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