AB-1009 Gene Therapy for Late-Onset Pompe Disease
This study is testing a new gene therapy called AB-1009 for adults with late-onset Pompe disease (LOPD). AB-1009 is given as a single infusion into a vein. The main goal is to see how safe AB-1009 is and if it causes any side effects. Researchers will also look at how well it works. You might be able to join if you are between 18 and 65 years old, have a confirmed diagnosis of LOPD, and have already received enzyme replacement therapy. The study plans to enroll up to 12 participants.
- Study design
- This is an open-label study, meaning both you and the study team will know which treatment you are receiving. It will include up to 12 participants and test two different doses of AB-1009.
- What's involved
- You would have a screening period of up to 75 days, followed by a primary observation period of 52 weeks after receiving the infusion.
- Compensation
- Not stated in the trial record.
- Follow-up
- After the primary observation period, participants will be followed for an additional 4 years to monitor their health.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
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)
At a glance
Conditions
NCT07282847
Where you'd take part
This study runs at 9 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Duke University
Durham, North Carolinastudy coordinator listed
Recruiting
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvaniastudy coordinator listed
Recruiting
University of California, Irvine (UCI)
Irvine, Californiastudy coordinator listed
Recruiting
University of Texas Southwest Medical Center
Dallas, Texasstudy coordinator listed
Recruiting
Barrow Neurological Institute
Phoenix, Arizonano site contact published
Not yet recruiting
NYU Langone
New York, New Yorkno site contact published
Not yet recruiting
Stanford Neuroscience Health Center
Palo Alto, Californiano site contact published
Not yet recruiting
University of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvaniano site contact published
Not yet recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
What this trial measures
- Incidence and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) during the primary observation periodDay 1 (Dosing) through Week 52 (the end of the primary observation period)