Active — Not RecruitingPhase 3

An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease

United States, Austria, Germany31 participantsStarted 2023-08-08

Plain-language summary

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.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Participants must meet all of the following criteria to be eligible for inclusion in the study: * The participant is willing and able to understand and fully comply with study procedures and requirements, in the opinion of the investigator. * The participant is able to read, understand, and complete the study questionnaires electronically per investigator's judgment. * The participant 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. Note: For participants who comprehend, can provide informed consent, and are unable to sign the ICF, an impartial witness may sign the ICF on behalf of the participant after the participant has orally consented to the participant's participation in the study. * The participant enrolling in this open-label extension (OLE) study will have participated in a previously qualifying study, and will be considered for eligibility based on the following study-specific criteria: * AROAAT2001: * Participants with fibrosis may roll over into this OLE study after they reach their next regularly scheduled, Q12W visit. * Participants with fibrosis who have completed the AROAAT2001 study may be enrolled into the OLE study. * AROAAT2002: * Participants in Cohorts 1 and 1b may roll over after completing the 24-week primary study period. * Participants in Cohort 2 may roll over after completing the 48…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Number of Participants With Adverse Events (AEs) and Serious AEs (SAEs)

Timeframe: From start of study drug administration (in current study) up to End of study (EOS) (current study [up to 10 years])

Number of Participants With Clinically Significant Changes From Baseline in Pulmonary Function Parameters

Timeframe: Baseline (current study), up to EOS (current study up to 10 years])

Number of Participants With Clinically Significant Changes in Vital Signs

Timeframe: From start of study drug administration (in current study) up to EOS (current study [up to 10 years])

Number of Participants With Clinically Significant Changes in Laboratory Parameters

Timeframe: From start of study drug administration (in current study) up to EOS (current study [up to 10 years])

Trial details

NCT IDNCT05899673

SponsorTakeda

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2033-05-02

View on ClinicalTrials.gov More Alpha1-Antitrypsin Deficiency trials