Experimental pill for rare lung and liver disease hits snag: trial stopped early
NCT ID NCT05643495
First seen Jun 26, 2026 · Last updated Jun 26, 2026 · Updated 1 time
Summary
This study tested an experimental drug called VX-864 in 14 people with a severe form of alpha-1 antitrypsin deficiency (PiZZ genotype). The goal was to see if the pill could raise protective protein levels in the blood and reduce harmful buildup in the liver. The trial was terminated early, so we have limited data, but the results may still help researchers understand how to treat this condition.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
VX-864 (oral tablets)
What this could lead to
If successful, VX-864 could increase functional alpha-1 antitrypsin levels, potentially slowing lung and liver damage in people with the PiZZ form of the disease.
What could go wrong
This early-stage trial was terminated, so results are limited. The drug may not prove effective or safe in larger studies, and it is not a cure.
Disclaimer
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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.
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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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