Experimental drug VX-864 aims to fix rare lung and liver disease
NCT ID NCT05643495
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 26 times
Summary
This study tested an experimental drug called VX-864 in 14 adults with Alpha-1 antitrypsin deficiency who have the PiZZ genetic type. The goal was to see if the drug could raise levels of a protective protein in the blood and reduce harmful protein clumps in the liver. The study was stopped early, so results are limited.
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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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Contacts and locations
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Locations
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Central Florida Pulmonary Group, P.A.
Orlando, Florida, 32803, United States
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Columbia University Irving Medical Center
New York, New York, 10032, United States
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Hannibal Regional Healthcare System
Hannibal, Missouri, 63401, United States
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Inova Fairfax Medical Campus
Falls Church, Virginia, 22042, United States
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King's College Hospital
London, United Kingdom
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Marsico Clinical Research Center at UNC Pulmonary Clinic
Chapel Hill, North Carolina, 27517, United States
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Renovatio Clinical
Houston, Texas, 77380, United States
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Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital
Beaumont, Ireland
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The University of Iowa Hospitals and Clinics: Adult Pulmonary Clinic
Iowa City, Iowa, 52242, United States
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University Hospital RWTH Aachen
Aachen, Germany
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University of Florida
Gainesville, Florida, 32610, United States
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University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
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University of Utah Health
Salt Lake City, Utah, 84108, United States
Conditions
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