Gene therapy shot could free pompe patients from weekly infusions
NCT ID NCT07282847
First seen Jan 04, 2026 · Last updated May 22, 2026 · Updated 24 times
Summary
This early-stage trial is testing a single intravenous dose of a gene therapy called AB-1009 in 12 adults with late-onset Pompe disease. Participants must have been on standard enzyme replacement therapy for at least 6 months. The main goal is to check safety and see if the treatment can reduce or replace the need for ongoing 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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Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Barrow Neurological Institute
NOT_YET_RECRUITINGPhoenix, Arizona, 85013, United States
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Duke University
NOT_YET_RECRUITINGDurham, North Carolina, 27705, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hospital of the University of Pennsylvania
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
Contact Email: •••••@•••••
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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
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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
Explore the condition pages connected to this study.