One-Time gene therapy could change pompe disease treatment
NCT ID NCT07282847
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a single intravenous dose of a gene therapy called AB-1009 in 12 adults with late-onset Pompe disease. Participants must have been on enzyme replacement therapy for at least 6 months. The main goal is to check safety and side effects, while also seeing if the treatment helps control the disease.
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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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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
Contact
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Stanford Neuroscience Health Center
NOT_YET_RECRUITINGPalo Alto, California, 94304, United States
Contact
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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
Contact
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University of Texas Southwest Medical Center
RECRUITINGDallas, Texas, 75235, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Virginia Commonwealth University (VCU)
NOT_YET_RECRUITINGRichmond, Virginia, 23298, United States
Contact