An Open-Label, Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112

Summary

This is an open-label, dose escalation study to evaluate the safety of oral PRX-112 and pharmacokinetics of GCD in subjects with Gaucher disease naive to enzyme replacement therapy. The dose levels of PRX-112 are 50 units, 100 units, 200 units and 400 units GCD. Subjects will receive once daily oral administrations of PRX-112 for 5 consecutive days at each dose level with a 2-day washout period between doses.

Timeline

Start

2014-04

Primary completion

2015-12

Completion

2015-12

Drugs

Indications