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

In this study, fazirsiran is being compared with a placebo for patients with alpha-1 antitrypsin deficiency-associated liver disease (AATD LD). A placebo is a liquid similar to fazirsiran, but does not contain any active ingredients.

The purpose of this study is to:

  • Test how well fazirsiran works as compared with a placebo in improving the symptoms of AATD LD.
  • Check the safety (the side effects) and tolerability (how tolerable the side effects are) of fazirsiran as compared to a placebo.
  • Get information on how fazirsiran moves in the body, how long it stays, and how long it takes to eliminate it, this is called pharmacokinetics or PK.
  • Get information on what fazirsiran does to the body, this is called pharmacodynamics or PD.
  • Test how well fazirsiran works as compared with a placebo in improving liver biopsy results.
  • Test how well fazirsiran works as compared with a placebo in improving liver biomarker results. Biomarkers are substances in blood that your body normally makes and will help show if your liver function is improving, staying the same, or getting worse.

Fazirsiran is an investigational drug, which means it has not been approved by any regulatory authority such as the United States (US) Food and Drug Administration (FDA), European Medicines Agency (EMA), Australian Therapeutic Goods Administration (TGA) or Brazil National Health Surveillance Agency (ANVISA). Fazirsiran can only be used in a study like this.


Clinical Study Identifier: s22-01352
Principal Investigator: Viviana Figueroa Diaz.


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