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;
Severe cardiomyopathy, defined as left ventricular ejection fraction (LVEF) <40% or New York Heart Association (NYHA) functional class 3 or above;
Require invasive mechanical ventilation, or rely on noninvasive ventilation during the day;
Intolerance to ERT or investigator-assessed intolerance to ERT, prior experience of serious ERT-related infusion-associated reactions (IARs);
Have known intrinsic liver diseases, including hepatitis, HIV-related liver disease, prior diagnosis of portal hypertension, splenomegaly, hepatic encephalopathy, severe fatty liver, cirrhosis or liver fibrosis ≥stage 2, ultrasound-identified liver neoplasms, or laboratory tests suggesting elevated alpha-fetoprotein. Patients with liver function tests including ALT or AST >3× upper limit of normal (ULN) or any total bilirubin above ULN during screening will also be excluded;
AskBio Inc
Lead sponsor
Bayer
Collaborator