An Open-Label Extension Study of Fazirsiran
Research type
Research Study
Full title
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
IRAS ID
1007739
Contact name
Nirav Desai
Contact email
Sponsor organisation
Takeda Development Center Americas, Inc.
Eudract number
2023-503497-21
Research 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 AAT, called Z-AAT. Z-AAT builds up in liver cells, and over time leads to different stages of liver disease, such as liver scarring (fibrosis), severe liver scarring (cirrhosis), and eventually end-stage liver disease. There is currently no available cure for AAT deficiency-associated liver disease (AATD-LD).
Fazirsiran is a treatment that has been designed to reduce the making of the abnormal Z-AAT protein and its build-up in the liver.
This open-label extension (OLE) study will allow patients who have taken part in two previous studies with fazirisiran as the study drug to continue to take fazirsiran. Data collected from this OLE study will provide more information on how fazirsiran works and more long-term safety data on fazirsiran.
Patients with AATD-LD who have taken part in one of two studies with fazirsiran (AROAAT2001 or AROAAT2002) will be checked to make sure they continue to meet the criterion for taking part in the OLE study which can take up to 2 weeks. All eligible patients will be treated with fazirsiran. They will receive study treatment as an injection under the skin on Day 1 of the OLE study then every 12 weeks for 96 weeks (about 2 years). Study visits will take place every 12 weeks. Patients will then have follow-up visits 12 and 24 weeks after their last dose of study treatment.
Study procedures include vital signs, physical examination, quality of life questionnaires, blood and urine samples, breathing tests, liver imaging and liver biopsy.
This study is sponsored by Takeda Development Center Americas, Inc. Up to 13 study sites will take part in this OLE study worldwide; the total number of sites and patients will depend on the number of patients that roll over into this study. There will be 2 hospital study sites in the UK taking part.REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
23/EM/0149
Date of REC Opinion
21 Aug 2023
REC opinion
Further Information Favourable Opinion