Efficacy and Safety of Fazirsiran in patients with liver disease
Research type
Research Study
Full title
A Randomized, Double-blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Fazirsiran in the Treatment of Alpha-1 Antitrypsin Deficiency–Associated Liver Disease With METAVIR Stage F2 to F4 Fibrosis
IRAS ID
1006841
Contact name
Nirav Desai
Contact email
Sponsor organisation
Takeda Development Center Americas, Inc.
Eudract number
2021-002702-27
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 study has been designed to show whether fazirsiran reduces liver scarring compared with a placebo (looks like fazisiran but does not have any medicine in it).
Patients with AATD-LD who already have some liver scarring will take part in the study. They will be checked to make sure they meet the rules for taking part in the study which can take up to 10 weeks. Eligible patients will be randomly chosen to be treated with fazirsiran or placebo. They will receive study treatment as an injection under the skin on Day 1, Week 4, then every 12 weeks for 196 weeks (about 4 years). Study visits will take place every 4 weeks for the first year and then every 12 weeks thereafter. Patients will have follow-up visits 6, 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, liver imaging, CT lung densitometry, esophagogastroduodenoscopy and liver biopsy.
This study is sponsored by Takeda Development Center Americas, Inc. Up to 160 patients will participate in this study worldwide with about 10 patients from 5 hospitals in the UK.
REC name
London - Westminster Research Ethics Committee
REC reference
23/LO/0287
Date of REC Opinion
11 Sep 2023
REC opinion
Further Information Favourable Opinion