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Experimental pill for rare lung and liver disease hits snag: trial stopped early

NCT ID NCT05643495

Disease control Sponsor: Vertex Pharmaceuticals Incorporated Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

14 people

The number who actually took part.

Started

Feb 2023

Finished

Aug 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 80 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Participants must have a PiZZ genotype confirmed at screening * Plasma AAT levels indicating severe deficiency at screening Key Exclusion Criteria: * History of a medical condition that could negatively impact the ability to complete the study * Solid organ, or hematological transplantation or is currently on a transplant list * History of use of gene therapy or Ribonucleic acid interference (RNAi) therapy at any time previously * Participants for whom discontinuation of augmentation therapy is not considered to be in their best interest, based on the clinical judgement of the treating physician Other protocol defined Inclusion/Exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

alpha 1-antitrypsin deficiency

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Alpha 1 antitrypsin deficiency

Contacts and locations

Locations

  • Central Florida Pulmonary Group, P.A.

    Orlando, Florida, 32803, United States

  • Columbia University Irving Medical Center

    New York, New York, 10032, United States

  • Hannibal Regional Healthcare System

    Hannibal, Missouri, 63401, United States

  • Inova Fairfax Medical Campus

    Falls Church, Virginia, 22042, United States

  • King's College Hospital

    London, United Kingdom

  • Marsico Clinical Research Center at UNC Pulmonary Clinic

    Chapel Hill, North Carolina, 27517, United States

  • Renovatio Clinical

    Houston, Texas, 77380, United States

  • Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital

    Beaumont, Ireland

  • The University of Iowa Hospitals and Clinics: Adult Pulmonary Clinic

    Iowa City, Iowa, 52242, United States

  • University Hospital RWTH Aachen

    Aachen, Germany

  • University of Florida

    Gainesville, Florida, 32610, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Utah Health

    Salt Lake City, Utah, 84108, United States

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