New drug shows promise for rare liver condition

NCT ID NCT03946449

First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 28 times

Summary

This study tested a new drug called fazirsiran in 16 people with alpha-1 antitrypsin deficiency, a genetic condition that can cause liver damage. The goal was to see if the drug is safe and can reduce harmful protein buildup in the liver. Participants received multiple doses of the drug by injection, and researchers measured changes in liver protein levels and other health markers over 24 to 48 weeks.

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Contacts and locations

Locations

  • Research Center 1

    Vienna, 1090, Austria

  • Research Center 1

    Aachen, 52074, Germany

  • Research Center 2

    Edinburgh, EH19 3BJ, United Kingdom

  • Research Center 3

    Cambridge, CB2 0QQ, United Kingdom

Conditions

Explore the condition pages connected to this study.