One-Shot gene therapy trial offers new hope for men with rare genetic disease
NCT ID NCT06270316
Summary
This is an early-stage study testing a single-dose gene therapy called AMT-191 in adult men with classic Fabry disease. The main goals are to find a safe dose and see how the body processes the treatment. The therapy aims to help the liver produce a missing enzyme, potentially offering long-term control of the disease with just one infusion.
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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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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: •••••@•••••
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Conditions
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