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

Stopped early · Phase 2

Conditions studied: Alpha-1 Antitrypsin Deficiency

In brief

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

Key facts

Study ID

NCT05643495

Run by

Vertex Pharmaceuticals Incorporated

People needed

14

Starts

2023-02-23

Expected to finish

2024-08-19

Last updated by the study team

2025-09-08

Who can join

Age: 18 and older, up to 80. Sex: any. Healthy volunteers: not accepted.

Where it is running

• University of Florida — Gainesville, Florida, United States
• Central Florida Pulmonary Group, P.A. — Orlando, Florida, United States
• The University of Iowa Hospitals and Clinics: Adult Pulmonary Clinic — Iowa City, Iowa, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• Hannibal Regional Healthcare System — Hannibal, Missouri, United States
• Columbia University Irving Medical Center — New York, New York, United States
• Marsico Clinical Research Center at UNC Pulmonary Clinic — Chapel Hill, North Carolina, United States
• Renovatio Clinical — Houston, Texas, United States
• University of Utah Health — Salt Lake City, Utah, United States
• Inova Fairfax Medical Campus — Falls Church, Virginia, United States
• University Hospital RWTH Aachen — Aachen, Germany
• Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital — Beaumont, Ireland
• King's College Hospital — London, United Kingdom

Full record on ClinicalTrials.gov

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