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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Could a simple shot replace IV drips for Alpha-1 patients?
- Hidden liver harm: study tracks silent damage in genetic disorder
- Promising liver drug trial halted early: what it means for patients
- Gene-Editing shot aims to fix lung and liver damage in rare disease
- New drug AIR-001 enters first human tests for rare lung condition