The ATB200-02 Study will assess a new investigational treatment option for adults with a rare genetic disorder known as Pompe Disease.
This is an open-label, fixed-sequence, single- and multiple-ascending dose, first-in-human (FIH) study to evaluate the safety, tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD), and efficacy of intravenous ATB200 alone and when co-administered with oral AT2221. The study will be conducted in 3 stages and will have 3 different cohorts.
To learn more about this clinical trial please visit: https://clinicaltrials.gov/ct2/show/NCT02675465.
This study is an international, multi-center, study of patients with Pompe disease and includes those currently receiving enzyme-replacement therapy (ERT), as well as naïve patients. The purpose of this study is to find out if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.