Status: Recruiting
A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT 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).
Participants needed: 12
Trial details
Phase: Phase 1, Phase 2Age: 18+Biological sex: AllType: InterventionalSponsor: AskBio IncUpdated: May 28, 2026Locations: 9
Eligibility criteria
Participant must be ≥18 years of age at the time of signing the informed consent... [+6]
Severe cardiomyopathy, defined as left ventricular ejection fraction (LVEF) <40%... [+9]