Phase 1/2Recruiting
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)
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)
- Sponsor
- AskBio Inc (Industry)
- Enrolment
- 12 planned
- Conditions
- Pompe Disease (Late-onset); Pompe Disease Late-Onset; LOPD
- Interventions
- AB-1009 (GAA Gene)
- Ages
- adults, older adults
- Registry
- NCT07282847
Full record on ClinicalTrials.gov
From the registry
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).
Primary outcomes
- Incidence and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) during the primary observation period
History
Study completion expected
Primary completion expected
Last registry update
Started
Registered