Safety and Efficacy Study of Fazirsiran in Adults With Alpha-1 Antitrypsin Deficiency Liver Disease and Mild Fibrosis

What this Trial Is About

This research aims to evaluate the safety and effects of fazirsiran in people with alpha-1 antitrypsin deficiency-associated liver disease who have mild liver scarring (fibrosis). This condition involves the liver producing an abnormal protein called Z-AAT that can build up and damage liver cells, potentially leading to serious liver problems. The study focuses on adults aged 18 to 75 who already have mild liver fibrosis and seeks to understand how well participants tolerate the treatment and its long-term safety. Participants will be randomly assigned to receive either fazirsiran or a placebo. Fazirsiran is given as a subcutaneous injection of 200 mg on Day 1, Week 4, and then every 12 weeks up to Week 100. The placebo group receives matching injections on the same schedule. The study is double-blind, meaning neither participants nor researchers know who receives the active drug or placebo during the study period. During the study, participants will have liver biopsies twice to collect tissue samples and undergo various tests including lung function checks, vital sign monitoring, ECGs, and blood tests to track changes in liver scarring and protein levels. Researchers will closely observe any adverse events and changes in clinical laboratory results from the start of treatment through Week 124. The study will also assess changes in liver inflammation, fibrosis scores, and markers of liver injury to understand the treatment's impact over about two years of participation.

Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)