A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101
Status:
Terminated
Trial end date:
2009-07-01
Target enrollment:
Participant gender:
Summary
This is an open-label study designed to assess if AT2101 is safe in patients with Gaucher
disease and how AT2101 gets through the body after it is taken by mouth. The study is being
offered to adult patients with type 1 Gaucher disease who are currently receiving a stable
dose of enzyme replacement therapy (ERT) with imiglucerase. During the study, subjects will
not be receiving ERT (up to 35 days).
The study consists of a screening period (~14 days), a treatment period (12 days) and a
follow-up period (7 days after last dose). At two points in the study, subjects will be
housed in an in-patient treatment facility for 3 days/2 nights to accommodate all necessary
blood draws.