A study to assess  an investigational treatment option for people with a rare genetic disorder known as Pompe Disease

The AT2220-ERT Co-Administration Study is evaluating the safety and effectiveness of an investigational, orally-administered pharmacological chaperone for the treatment of Pompe disease.


The AT2220-ERT Co-Administration Study is a Phase 2A clinical trial designed to investigate drug-drug interactions between the investigational pharmacological chaperone AT2220 (duvoglustat HCl) and enzyme replacement therapy (ERT) alglucosidase alfa in subjects with Pompe disease. The key outcome measures of the study will include safety, evaluation of the effects of AT2220 on the plasma pharmacokinetics of alglucosidase alfa (ERT), and measurements of alglucosidase alfa and AT2220 concentrations in skeletal muscle. Study participation will be approximately 3 months for each subject.

The AT2220-ERT Co-Administration Study is open to males and females aged 18-65 with Pompe disease, who are on a stable alglucosidase alfa (ERT) dose and regimen for at least 3 months before screening. (Stable regimen is defined as currently receiving alglucosidase alfa (ERT) every 2 weeks and stable dose defined as not varying by more than 10%).


The AT2220-ERT Co-Administration Study is being conducted at sites in France, United Kingdom, the United States and Canada. Additional locations are continually being added. Check website or contact us for more details.