Plicera™ (AT2101) Clinical Trials

Amicus is currently developing AT2101, an experimental, oral medication for the treatment of Gaucher disease.  AT2101 acts as a pharmacological chaperone and may help to selectively stabilize and restore the three-dimensional shape, trafficking, and biological activity of glucocerebrosidase, the enzyme that is defective in Gaucher disease.

Amicus is conducting two Phase 2 clinical trials of AT2101 for Gaucher disease.  One is a 4-week study designed to evaluate the safety and pharmacodynamic effects of AT2101 in Type I Gaucher patients who will discontinue enzyme replacement therapy for the duration of the study.  The second is a 6-month study designed to evaluate the safety of AT2101 and its effect on parameters that are commonly abnormal in Gaucher disease.  This study will be conducted in Type I Gaucher patients who have never received enzyme replacement therapy.  More information regarding these studies can be found at www.clinicaltrials.gov or via the links below:

 

If you are a health care provider interested in referring a patient or learning more about these clinical trials, please send an email correspondence to Amicus at clinicaltrials@amicustherapeutics.com.

If you are a patient interested in participating in one of these trials, please have your personal physician send an email correspondence to Amicus at clinicaltrials@amicustherapeutics.com.