A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease

Other illness

• Subject plans to receive gene therapy or participate in another interventional study for Pompe disease
• Subject has a hypersensitivity to any of the excipients in ATB200 or AT2221, or has a medical condition or any other extenuating circumstance that may, in the opinion of the investigator or medical monitor, pose an undue safety risk to the subject or may compromise his/her ability to comply with or adversely impact protocol requirements. This includes clinical depression (as diagnosed by a psychiatrist or other mental health professional) with uncontrolled or poorly controlled symptoms

Other

• Subject, if female, is pregnant or breastfeeding
• Subject, whether male or female, is planning to conceive a child during the study

• Subject must provide signed informed consent prior to any study- related procedures being performed. If the subject is under 20 years of age, the subject must provide written informed consent
• Subject must have completed Study ATB200-03. Note: Subjects who were forced to withdraw from Study ATB200-03 for a logistical reason not related to the efficacy or safety of ATB200/AT2221 (eg, hospitalization for a car accident, COVID-19 pandemic, or emergency surgery) and which resulted in several consecutive missed doses may be eligible to participate in this study upon approval by the Amicus medical monitor
• Female subjects of childbearing potential and male subjects must agree to use medically accepted methods of contraception during the study and for 90 days after the last dose of study drug