Gene therapy trial hopes to tame rare enzyme disorder
NCT ID NCT06270316
First seen Oct 31, 2025 · Last updated Apr 29, 2026 · Updated 24 times
Summary
This study tests a single-dose gene therapy called AMT-191 in 12 adult men with classic Fabry disease, a rare genetic condition. The main goals are to check if the treatment is safe and to see how it behaves in the body. Participants receive the therapy through an IV, and researchers monitor side effects and how long the genetic material stays in body fluids.
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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
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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
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Emory University School of Medicine
RECRUITINGAtlanta, Georgia, 30322, United States
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Lysosomal & Rare Disorders Research and Treatment Center, Inc
RECRUITINGFairfax, Virginia, 22030, United States
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MHealth Fairview University of Minnesota Medical Center East Bank
RECRUITINGMinneapolis, Minnesota, 55455, United States
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NYC Health + Hospitals/Metropolitan
RECRUITINGNew York, New York, 10029, United States
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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
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University of Utah, Clinical and Translational Sciences Institute
RECRUITINGSalt Lake City, Utah, 84108, United States
Contact
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Contact Email: •••••@•••••
Conditions
Explore the condition pages connected to this study.