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) (NCT07282847)
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)
🩺 Plain-English Summary (8th-Grade Level)
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).
Who can join: Review the eligibility criteria below.
Location: Barrow Neurological Institute — Phoenix, Arizona
Age range: 18 Years to 65 Years
🧪 Interventions in This Study
- AB-1009 (GAA Gene) (GENETIC)
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (9 Sites)
- Barrow Neurological Institute — Phoenix, Arizona
- University of California, Irvine (UCI) — Irvine, California
- Stanford Neuroscience Health Center — Palo Alto, California
- NYU Langone — New York, New York
- Duke University — Durham, North Carolina
- Hospital of the University of Pennsylvania — Philadelphia, Pennsylvania
- University of Pittsburgh Medical Center (UPMC) — Pittsburgh, Pennsylvania
- University of Texas Southwest Medical Center — Dallas, Texas
- Virginia Commonwealth University (VCU) — Richmond, Virginia
📞 Contact the Study Coordinator
Phone: (919) 561-6210
Email: AskFirst@AskBio.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Pompe Disease (Late-onset)
- A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease — Atlanta, Georgia
- Gene Transfer Study in Patients With Late Onset Pompe Disease — Orange, California
- ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD — Gainesville, Florida
Explore by condition: All Pompe Disease (Late-onset) clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.