NCT05899673

An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease

A Phase 3, Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Fazirsiran in Participants With Alpha-1 Antitrypsin Deficiency-Associated Liver Disease

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 31 (actual)
• Sponsor: Takeda · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The main aim of this study is to learn if fazirsiran is safe during long-term use in people with liver disease caused by the abnormal Z-alpha-1 antitrypsin (Z-AAT) protein. People who have taken part in previous fazirsiran studies (AROAAT2001 \[NCT03945292\] or AROAAT2002 \[NCT03946449\]) can continue to receive fazirsiran every 3 months as long as they participate in this study, the study is ongoing or until health authorities in their country approve fazirsiran to be publicly available. The study may also provide information on whether fazirsiran has a long-term effect in reducing liver fibrosis or slowing down the progression of liver fibrosis in people with liver disease due to the abnormal Z-AAT protein.

Conditions

• Alpha1-Antitrypsin Deficiency

Interventions

Type	Name	Description
DRUG	Fazirsiran Injection	Fazirsiran will be injected subcutaneously.

Timeline

• Start date: 2023-08-08
• Primary completion: 2033-05-02
• Completion: 2033-05-02
• First posted: 2023-06-12
• Last updated: 2025-10-14

Locations

10 sites across 5 countries: United States, Austria, Germany, Portugal, United Kingdom

Regulatory

• FDA-regulated drug study