Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)

Phase 2 Completed 16 enrolled Arrowhead Pharmaceuticals

Non-randomizedParallel-groupOpen-labelTreatment

Summary

The purpose of this study is to evaluate the the safety and efficacy of the investigational product, fazirsiran (TAK-999, ARO-AAT), administered subcutaneously to patients with alpha-1 antitrypsin deficiency associated liver disease (AATD).

Timeline

Start: 2019-10-31
Primary completion: 2022-04-28
Completion: 2023-12-14

Publications

Strnad P, Mandorfer M, Choudhury G, Griffiths W, Trautwein C, Loomba R, Schluep T, Chang T, Yi M, Given BD, Hamilton JC, San Martin J, Teckman JH. Fazirsiran for Liver Disease Associated with Alpha1-Antitrypsin Deficiency. N Engl J Med. 2022 Aug 11;387(6):514-524. doi: 10.1056/NEJMoa2205416. Epub 2022 Jun 25.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Fazirsiran	Unknown	100 mg	Subcutaneous
Subject	Fazirsiran	Unknown	200 mg	Subcutaneous

Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)

Summary

Timeline

Publications

Drugs

Indications