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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Shaare Zedek Medical Center
Jerusalem, Israel
Area under the curve
Blood samples for GCD level every 2 hours for 24 hours
Time frame: 24 hours
Adverse Events
Reporting of adverse events collected daily
Time frame: 7 Days
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