One-Time gene therapy aims to tame rare enzyme disorder
NCT ID NCT06270316
First seen Oct 31, 2025 · Last updated May 08, 2026 · Updated 26 times
Summary
This early-stage study tests a single dose of a gene therapy called AMT-191 in 12 adult men with classic Fabry disease. The treatment uses a harmless virus to deliver a working copy of the GLA gene, which is faulty in Fabry disease. The main goals are to check safety and how the drug behaves in the body.
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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
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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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