Fazirsiran for Alpha-1 Antitrypsin Deficiency

Recruiting in Palo Alto (17 mi)

+11 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Takeda

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

The main aim of this study is to learn if fazirsiran is safe during long-term use in people with liver disease caused by the abnormal Z-alpha-1 antitrypsin (Z-AAT) protein. People who have taken part in previous fazirsiran studies (AROAAT2001 \[NCT03945292\] or AROAAT2002 \[NCT03946449\]) can continue to receive fazirsiran every 3 months as long as they participate in this study, the study is ongoing or until health authorities in their country approve fazirsiran to be publicly available. The study may also provide information on whether fazirsiran has a long-term effect in reducing liver fibrosis or slowing down the progression of liver fibrosis in people with liver disease due to the abnormal Z-AAT protein.

Research Team

Study Director

Principal Investigator

Takeda

Eligibility Criteria

This trial is for people with Alpha-1 Antitrypsin Deficiency causing liver disease, who have already been in previous fazirsiran studies. They must be able to follow the study rules and fill out questionnaires. Smokers or those using e-cigarettes can't join.

Inclusion Criteria

I do not have liver cancer and will be screened for it before joining.

The participant/participant's legally acceptable representative has provided informed consent (ICF) (that is, in writing, documented via a signed and dated ICF) and any required privacy authorization before the initiation of any study procedures.

I am willing and able to follow the study's procedures and requirements.

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Exclusion Criteria

Participants with major protocol deviations in previous studies

I have a history of blood clots or I am on long-term blood thinners.

Participants not meeting specific ALT, AST, and Creatinine criteria

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Treatment Details

Interventions

Fazirsiran (Antisense Oligonucleotide)

Trial OverviewThe trial tests the long-term safety of Fazirsiran injections given every three months over two years, with an additional six-month follow-up. It aims to see if it reduces or slows down liver fibrosis caused by Z-AAT protein.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: Fazirsiran 200 mgExperimental Treatment1 Intervention

Participants who are currently taking part in or who have completed their treatment in parent studies AROAAT2001 (NCT03945292) and AROAAT2002 (NCT03946449) may rollover in this study to receive fazirsiran, 200 milligrams (mg), injection, subcutaneously on Day 1 and once every 12 weeks (Q12W) thereafter until fazirsiran receives approval and is commercially available in the participant's country, until a participant withdraws from the study or the sponsor decides to terminate the study.