A Single-Arm, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of AB-1009 in Adult Participants With Late Onset Pompe Disease (LOPD)
This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB-1009 in adult participants with late-onset Pompe disease (LOPD).
• Participant must be ≥18 years of age at the time of signing the informed consent form.
• Confirmed GAA enzyme deficiency from any tissue source and/or confirmed biallelic GAA gene mutations.
• Undergone enzyme replacement treatment (ERT) (either alglucosidase alfa (Lumizyme®) or avalglucosidase alfa-ngpt (Nexviazyme®)), for at least 6 months (at least 10 infusions) before signing the initial informed consent form. During the screening process, participants need to remain on their current ERT until close to dosing;
• FVC in the upright position ≥30% and ≤80% of predicted;
• Capable of walking at least 100 meters in the 6MWT (use of a cane, quad cane, or standard walker is permitted);
• Contraceptive/barrier use by men and women requirements as per protocol.
• Capable of giving informed consent and able to understand and comply with all study procedures.