A new phase 3, open-label extension study is set to evaluate the long-term safety and efficacy of fazirsiran in patients with alpha-1 antitrypsin deficiency (AATD) who have previously participated in one of the qualifying studies.

The study participants will be administered 200 mg subcutaneous injections of fazirsiran once every 12 weeks for up to 96 weeks and will be followed for an additional 6 months.

The study aims to enroll approximately 37 participants between the ages of 18 and 75 years. Patients with fibrosis who have taken part in the AROAAT2001 study may enroll after their upcoming once-every-12-week visit or within 12 to 24 weeks of the final dose.

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As for the AROAAT2002 study, participants belonging to cohorts 1 and 1b may enter the open-label extension after completing the 24-week primary study period, whereas those from cohort 2 need to first complete the 48-week primary study period. Those who have received the final dose may roll over within 12 to 24 weeks of the final dose.

Read more about AATD experimental therapies

The exclusion criteria cite any other forms of chronic liver disease, newly diagnosed or recurrent malignancy, pulmonary exacerbation, upcoming major surgeries, as well as unstable, poorly controlled, or severe hypertension.  

Participants must not smoke or consume alcohol for the study duration. Using highly effective contraception during the study and 24 weeks after the last dose is obligatory.

The researchers will monitor the number of participants with adverse events and serious adverse events, as well as clinically significant changes from baseline in pulmonary function parameters, vital signs, and laboratory parameters as primary outcome measures.

The study, sponsored by Takeda, is set to start on July 14, 2023, and the estimated primary and study completion date is set for May 29, 2026.


An extension study to learn about the long-term safety of fazirsiran and if fazirsiran can help people with alpha-1 antitrypsin liver disease. ClinicalTrials.gov. Last updated June 18, 2023. Accessed July 12, 2023.