TerminatedPhase 2

A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype

Stopped: Study drug dosing was permanently discontinued at the Sponsor's discretion based on frequency and nature of skin and subcutaneous tissue disorders.

United States, Germany, Ireland14 participantsStarted 2023-02-23

Plain-language summary

The purpose of this study evaluates the efficacy and safety of VX-864 in participants with the PiZZ genotype over 48 weeks.

Who can participate

Age range

18 Years – 80 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Change in Blood Functional Alpha-1 Antitrypsin (AAT) Levels

Timeframe: From Baseline at Week 48

Trial details

NCT IDNCT05643495

SponsorVertex Pharmaceuticals Incorporated

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-08-19

Results submitted2025-08-19

View on ClinicalTrials.gov More Alpha-1 Antitrypsin Deficiency trials

Who can participate

Age range

18 Years – 80 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.