One-shot gene therapy aims to free pompe patients from lifelong infusions
NCT ID NCT07282847
First seen Jan 04, 2026 · Last updated May 09, 2026 · Updated 23 times
Summary
This study tests a single intravenous dose of AB-1009 gene therapy in 12 adults with late-onset Pompe disease. Participants must have been on enzyme replacement therapy for at least 6 months. The goal is to see if the treatment is safe and can reduce or replace the need for regular enzyme infusions.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Barrow Neurological Institute
NOT_YET_RECRUITINGPhoenix, Arizona, 85013, United States
Contact
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Duke University
NOT_YET_RECRUITINGDurham, North Carolina, 27705, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Hospital of the University of Pennsylvania
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
Contact Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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NYU Langone
NOT_YET_RECRUITINGNew York, New York, 10016, United States
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Oregon Health and Science University (OHSU)
NOT_YET_RECRUITINGPortland, Oregon, 97239, United States
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Stanford Neuroscience Health Center
NOT_YET_RECRUITINGPalo Alto, California, 94304, United States
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University of California, Irvine (UCI)
RECRUITINGIrvine, California, 92697, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Pittsburgh Medical Center (UPMC)
NOT_YET_RECRUITINGPittsburgh, Pennsylvania, 15213, United States
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University of Texas Southwest Medical Center
NOT_YET_RECRUITINGDallas, Texas, 75235, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Virginia Commonwealth University (VCU)
NOT_YET_RECRUITINGRichmond, Virginia, 23298, United States
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Conditions
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