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 to ≤65 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®), avalglucosidase alfa-ngpt (Nexviazyme®)), or cipaglucosidase alfa (Pombiliti®) 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;
Sequential
AB-1009 (GAA Gene)
GeneticA single intravenous infusion of AB-1009
Incidence and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) during the primary observation period
Area under the curve (AUC) of GAA activity in serum
Viral shedding (whole blood, saliva, urine)
AUC of urinary Glc4
Change from baseline in GAA activity in muscle biopsy tissue at Week 52
Click on the lead sponsor to view all of their trials.
AskBio Inc
Lead sponsor
Bayer
Collaborator
