Experimental drug tested for rare genetic lung disease

NCT ID NCT05643495

Summary

This study tested whether the drug VX-864 could help people with a specific genetic form of Alpha-1 antitrypsin deficiency. Researchers enrolled 14 participants with the PiZZ genotype to receive the drug for 48 weeks. The trial was terminated early, and researchers measured changes in protein levels in the blood and monitored safety.

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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

Conditions

Explore the condition pages connected to this study.