Amicus is initiating the multi-national, open-label Phase II clinical trial designed to enroll 18 adult patients diagnosed with Pompe disease. The primary objective of the study is to evaluate the safety and tolerability of different dosing regimens of AT2220 over a 12-week period.
The study will also explore certain pharmacodynamic and pharmacokinetic measures including the effect of treatment with AT2220 on acid alpha-glucosidase activity and on glycogen levels in various cells and tissues.
Additional objectives include preliminary assessments of pulmonary and skeletal muscle function. Participants who complete the study may be eligible to participate in a voluntary extension study that will further evaluate the effect of AT2220 on these functional parameters.
Amicus will conduct the study in adult Pompe patients in clinical centers throughout North America and Europe. AT2220 is the third compound based on Amicus’s pharmacological chaperone technology platform to enter Phase II clinical development.
Barry Byrne, professor of pediatrics, molecular genetics and microbiology at the University of Florida in Gainesville, said: “We look forward to evaluating AT2220 as a potential new oral therapeutic option for patients living with Pompe disease.”