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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.
Full description
While on the study, subjects will be required to refrain from consuming the following foods and beverages:
Subjects will also be restricted to taking medications or herbal products during the study except if the Principal Investigator permits.
Subjects will not engage in strenuous activity at any time during the in-patient periods.
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1 participants in 1 patient group
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Data sourced from clinicaltrials.gov
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