NCT06165341

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)

A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Safety and Efficacy of Fazirsiran in the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease With METAVIR Stage F1 Fibrosis

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Takeda · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

The liver produces a protein called alpha-1 antitrypsin (AAT). AAT is normally released into the bloodstream. In some people, the liver makes an abnormal version of the AAT protein, called Z-AAT. Making an abnormal version of the AAT protein can result in liver disease as Z-AAT builds up in liver cells, which leads to liver problems such as liver scarring (fibrosis), continuing liver damage (cirrhosis), and eventually end stage liver disease. Fazirsiran is a medicine that reduces the creation of the Z-AAT protein and thus the build-up of this abnormal protein in the liver. People with this type of liver disease who already have mild liver scarring will take part in the study. They will be treated with fazirsiran or a placebo for about 2 years. This study will check the long-term safety of fazirsiran, whether participants tolerate the treatment and if there are any effects on liver scarring. A liver biopsy, a way of collecting a small tissue sample from the liver, will be taken twice during the study.

Conditions

• Alpha1-Antitrypsin Deficiency

Interventions

Type	Name	Description
DRUG	Fazirsiran Injection	Fazirsiran will be injected subcutaneously.
DRUG	Placebo	Fazirsiran matching placebo.

Timeline

• Start date: 2024-03-01
• Primary completion: 2028-08-26
• Completion: 2028-08-26
• First posted: 2023-12-11
• Last updated: 2026-03-05

Locations

41 sites across 13 countries: United States, Austria, Belgium, Canada, France, Germany, Italy, Poland, Portugal, Spain, Sweden, Switzerland, United Kingdom

Regulatory

• FDA-regulated drug study