Gene therapy trial aims to fix enzyme defect in fabry disease
NCT ID NCT06270316
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial is testing a single-dose gene therapy called AMT-191 in 12 adult men with classic Fabry disease. The therapy uses a harmless virus to deliver a working copy of the GLA gene to the liver, so the body can produce the missing enzyme. The main goals are to check safety and see how the drug behaves in the body.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
AMT-191 (a gene therapy using a harmless virus to deliver a working copy of the GLA gene)
What this could lead to
If successful, this could lead to a one-time gene therapy that helps the body produce the missing enzyme, potentially reducing the need for regular enzyme infusions.
What could go wrong
This is an early-phase trial with only 12 participants, so results may not apply to everyone. Gene therapies can have side effects like immune reactions, and long-term effects are unknown.
Disclaimer
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the original study
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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: •••••@•••••
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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: •••••@•••••
Contact
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Lysosomal & Rare Disorders Research and Treatment Center, Inc
RECRUITINGFairfax, Virginia, 22030, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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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: •••••@•••••