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

NCT ID NCT05899673

First seen Oct 31, 2025 · Last updated May 21, 2026 · Updated 26 times

Summary

This study looks at the long-term safety of a drug called fazirsiran for people with liver disease caused by alpha-1 antitrypsin deficiency. About 31 participants who were in earlier studies will receive fazirsiran every 3 months. Researchers will monitor side effects and check if the drug can slow liver scarring.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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.