This is a phase 1/2 open-label, ascending dose, multicenter clinical study to evaluate the safety and efficacy of AT845 in adult (aged ≥ 18 years) subjects, ambulatory or nonambulatory, with Late Onset Pompe Disease (LOPD).
A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients With Late Onset Pompe Disease
This study (FORTIS) will evaluate the safety and efficacy of an investigational gene replacement therapy, AT845, in adult subjects with LOPD. Subjects will receive a single dose of AT845 delivered via intravenous (IV) infusion. Up to 2 nominal dose levels of AT845 are planned to be evaluated in FORTIS. A single AT845 administration via IV infusion is planned for each subject. The initial dosing cohort will receive a single dose at 3x1013 vg/kg of AT845. The second dose cohort will receive a single dose at 6x1013 vg/kg of AT845. The core observation period will be completed by Week 48 for each subject. Subjects will be followed for a total of 5 years after administration of AT845.