A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101

Exclusion Criteria

• •A clinically significant disease, severe complications from Gaucher disease, or serious intercurrent illness that may preclude participation in the study in the opinion of the Investigator;
• •During the screening/wash out period, any clinically significant findings, based on physical exam, medical history review, lab assessment, vital sign assessment and/or other significant finding which would compromise the safety of the subject, or preclude the subject from completing the study as deemed by the Investigator;
• •Partial or total splenectomy;
• •History of pulmonary hypertension or Gaucher-related lung disease;
• •History of allergy or sensitivity to the study drug or any excipients, including any prior serious allergic reaction to iminosugars;
• •Screening or Day 1 (before dosing) 12-lead ECG demonstration QTc \>450 msec in males or \>470 in females;
• •Pregnant or breast-feeding;
• •Current/recent drug or alcohol abuse within the past 12 months;
• •Treatment with any investigational product, including investigational forms of ERT, in the 90 days before study entry;
• •Treatment in the previous 90 days with any drug known to have a well-defined potential for toxicity to a major organ;
• •Presence or sequelae of gastrointestinal, liver or kidney disease, or other conditions known to interfere with the absorption, distribution, metabolism, or excretion of drugs; or
• •Subject is otherwise unsuitable for the study in the opinion of the Investigator.