NCT01747980

Safety and Pharmacokinetics of Oral PRX-112 in Gaucher Disease Patients

An Exploratory, Open-label Study to Evaluate the Safety of PRX-112 and Pharmacokinetics of Oral prGCD (Plant Recombinant Human Glucocerebrosidase) in Gaucher Patients

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: Protalix · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

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.

Conditions

• Gaucher Disease

Interventions

Type	Name	Description
DRUG	PRX-112	Single dose level, four doses per cohort

Timeline

• Start date: 2013-03-01
• Primary completion: 2014-01-01
• Completion: 2014-03-01
• First posted: 2012-12-12
• Last updated: 2014-04-07

Locations

2 sites across 1 country: Israel