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 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).
• Subject is aged ≥ 18 years.
• Subject has a documented clinical diagnosis of Pompe disease by genetic testing.
• Subject has received enzyme replacement therapy (ERT) with rhGAA for the previous ≥ 2 years.
• Subject has been on a stable standard dose (at least 20 mg/kg every 2 weeks) of ERT with rhGAA for at least the previous 6 months.
• Subject or legally authorized representative(s) (LAR) (if applicable) provides written informed consent.
• Subject and LAR(s) are willing and able to comply with study visits and study procedures.
• Subject must agree to refrain from blood or blood products donation and sperm or egg donation from the time of AT845 administration until the later of 90 days or 3 consecutive negative viral shedding samples
• Subjects enrolled in previous protocol versions 1 through 9: Subject has upright FVC ≥ 30% of predicted normal value. Subjects enrolled in protocol version 10: Subject has upright FVC ≥ 30% and ≤ 85% of predicted normal value.
• Subjects enrolled in protocol version 10: Subject who is able to ambulate ≥ 40 m without stopping and without the use of an assistive device. The use of an assistive device for community ambulation is acceptable. (Subjects enrolled under previous protocol versions 1 through 9 will not be excluded if they do not meet this criterion during re-screening visit).