New drug shows promise for slowing liver damage in rare genetic disease

NCT ID NCT05899673

First seen Oct 31, 2025 · Last updated May 16, 2026 · Updated 24 times

Summary

This study checks if the drug fazirsiran is safe and works over the long term for people with liver disease caused by Alpha-1 antitrypsin deficiency. About 31 adults who were in earlier studies get fazirsiran every 3 months. Researchers track side effects and check if the drug slows liver scarring.

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Contacts and locations

Locations

  • Addenbrooke's Hospital

    Cambridge, CB2 0QQ, United Kingdom

  • Hospital Nélio Mendonça

    Funchal, 9000-168, Portugal

  • Medical University of South Carolina - Hollings Cancer Center - PPDS

    Charleston, South Carolina, 29425-8900, United States

  • Medizinische Universitat Wien (Medical University of Vienna)

    Vienna, A-1090, Austria

  • Royal Infirmary of Edinburgh - PPDS

    Edinburgh, EH16 4SA, United Kingdom

  • Stanford Medicine Outpatient Center

    Redwood City, California, 94063, United States

  • UCSD Altman Clinical and Translational Research Institute

    La Jolla, California, 92037-1337, United States

  • UF Clinical and Translational Science Institute

    Gainesville, Florida, 32610-3010, United States

  • University Of Iowa Hospitals And Clinics

    Iowa City, Iowa, 52242-1009, United States

  • Universitätsklinikum der RWTH Aachen

    Aachen, North Rhine-Westphalia, 52074, Germany

Conditions

Explore the condition pages connected to this study.