NCT07282847

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)

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: AskBio Inc · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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).

Detailed description

This is an open-label study, up to 12 participants will receive a single IV infusion of AB-1009. Participants will be assigned to either cohort 1 (1.0E13 vg/kg) or Cohort 2 (1.5E13 vg/kg) based on enrollment in the study. Study duration will include a screening period of up to 75 days, primary observation of 52 weeks, and a long-term follow-up period of 4 years.

Conditions

• Pompe Disease (Late-onset)
• Pompe Disease Late-Onset
• LOPD

Interventions

Type	Name	Description
GENETIC	AB-1009 (GAA Gene)	A single intravenous infusion of AB-1009

Timeline

• Start date: 2026-02-10
• Primary completion: 2028-09-01
• Completion: 2032-09-01
• First posted: 2025-12-15
• Last updated: 2026-04-09

Locations

10 sites across 1 country: United States

Regulatory

• FDA-regulated drug study