Testing a new injection to slow liver scarring from a rare protein disorder

This trial is testing fazirsiran, an injectable medication designed to reduce liver scarring in people with alpha-1 antitrypsin deficiency—a rare genetic condition where the body makes an abnormal protein that builds up in the liver and causes damage. Researchers want to see if this medication can slow the progression of liver scarring and prevent further liver injury compared to placebo (a dummy injection with no active ingredient). The study will track changes in liver scarring using imaging, blood tests, and small tissue samples from the liver.

Alpha-1 antitrypsin deficiency is a serious genetic disorder where harmful protein accumulates in the liver, gradually causing scarring and liver failure. There is currently no cure, and existing treatments are limited, so researchers are testing this medication to see if it can slow or stop the damage before it becomes life-threatening.