A Randomized Double-Blind Placebo-Controlled Phase 3 Study to Evaluate the Efficacy and Safety of Fazirsiranin the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease with METAVIR Stage F2 to F4 Fibrosis

Brief description of study

This is a phase 3, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of fazirsiran (TAK-999, previously called ARO-AAT) in the treatment of subjects aged 12 to 75 years (inclusive) with AATD-LD moderate to advanced fibrosis and compensated cirrhosis. The study will enroll subjects with PiZZ AATD-LD with METAVIR stage F2, F3, or F4 liver fibrosis. Approximately 126 up to 140 subjects are planned to be randomized 1:1 to receive either placebo or fazirsiran administered subcutaneously (SC). Subject randomization will be stratified according to METAVIR stage (F2 or F3 vs F4). The F4cc population will be capped at 25% of the total subjects with the remaining 75% being comprised of F2 and F3 subjects.


Clinical Study Identifier: s22-01352
ClinicalTrials.gov Identifier: NCT05677971
Principal Investigator: Viviana Figueroa Diaz.
Other Investigators: Monique Julia Carasso, Nina M Gardner, Flordelis Cruz Reyes.


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