One-Time gene therapy aims to tame rare enzyme disorder
NCT ID NCT06270316
First seen Oct 31, 2025 · Last updated Jun 09, 2026 · Updated 33 times
Summary
This early-stage study tests a single-dose gene therapy called AMT-191 in 12 adult men with classic Fabry disease. The goal is to see if the treatment is safe and how it works in the body. Fabry disease is a rare genetic disorder that can cause pain, organ damage, and shorten life. This approach tries to fix the root cause by delivering a working gene.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
Contact
Contact Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Emory University School of Medicine
RECRUITINGAtlanta, Georgia, 30322, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Lysosomal & Rare Disorders Research and Treatment Center, Inc
RECRUITINGFairfax, Virginia, 22030, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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MHealth Fairview University of Minnesota Medical Center East Bank
RECRUITINGMinneapolis, Minnesota, 55455, United States
Contact Email: •••••@•••••
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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NYC Health + Hospitals/Metropolitan
RECRUITINGNew York, New York, 10029, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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The Kirklin Clinic Of university of Alabama Birmingham Hospital
RECRUITINGBirmingham, Alabama, 35233, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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UPMC Children's Hospital of Pittsburgh
RECRUITINGPittsburgh, Pennsylvania, 15224, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Utah, Clinical and Translational Sciences Institute
RECRUITINGSalt Lake City, Utah, 84108, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Email: •••••@•••••
Conditions
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