Phase 3 Study to Evaluate Safety and Effectiveness of Fazirsiran for Liver Disease and Scarring in Alpha-1 Antitrypsin Deficiency Patients Aged 18 to 75

What this Trial Is About

Researchers are evaluating fazirsiran, an investigational drug, to see if it can reduce liver scarring fibrosis and slow disease progression in people with liver disease caused by an abnormal alpha-1 antitrypsin protein. The study compares fazirsiran to a placebo and aims to understand how the drug affects the body, including its impact on liver inflammation and abnormal protein levels. Liver biopsies will be taken twice to assess changes in liver scarring. Participants will receive either fazirsiran or a placebo through subcutaneous injections. The dosing schedule includes an injection on Day 1, at Week 4, and then every 12 weeks up to Week 196. The study is randomized and double-blind, meaning neither participants nor researchers know who receives the drug or placebo during the treatment period. During the study, participants will undergo liver biopsies and various assessments including imaging techniques and blood tests to monitor liver function, protein levels, liver stiffness, and inflammation. Researchers will also track side effects, lung function, vital signs, and laboratory results from the start of treatment through up to Week 230. The main focus is on measuring changes in liver fibrosis after 106 weeks, with ongoing safety and efficacy evaluations throughout the study.

Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein