🏛️ Verified NIH Record • Updated 2026-06-09
Gene Transfer Study in Patients With Late Onset Pompe Disease (NCT04174105)
A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients With Late Onset Pompe Disease
🔵 Active, Not Recruiting Phase1 / Phase2 📋 Clinical Research Protocol Sponsor: Astellas Gene Therapies
🩺 Plain-English Summary (8th-Grade Level)
This is a phase 1/2 open-label, ascending dose, multicenter clinical study to evaluate the safety and efficacy of AT845 in adult (aged ≥ 18 years) subjects, ambulatory or nonambulatory, with Late Onset Pompe Disease (LOPD).
Who can join: Review the eligibility criteria below.
Location: University of California Irvine, Department of Neurology — Orange, California
Age range: 18 Years to 80 Years
🧪 Interventions in This Study
- zocaglusagene nuzaparvovec (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 (4 Sites)
- University of California Irvine, Department of Neurology — Orange, California
- Stanford University — Palo Alto, California
- University of Utah, Division of Medical Genetics — Salt Lake City, Utah
- Newcastle Upon Tyne Hospitals Foundation Trust Clinical Research Facility — Newcastle upon Tyne,
💰 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
- 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
- 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) — Phoenix, Arizona
Explore by condition: All Pompe Disease (Late-onset) clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.