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Antisense Oligonucleotide

Fazirsiran for Alpha-1 Antitrypsin Deficiency

Phase 3

Waitlist Available

Research Sponsored by Takeda

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Participants must not have hepatocellular carcinoma (HCC) and must undergo screening for HCC before enrollment

Be older than 18 years old

Must not have

Participants with history of thromboembolic disease or taking chronic anticoagulants

Participants experiencing a pulmonary exacerbation at enrollment

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline (current study), weeks 12, 24, 36, 48, 60, 72, 84, 96, eos (current study [week 120])

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

"This trial aims to determine if fazirsiran is safe for long-term use in individuals with liver disease caused by a specific protein. Participants who were part of previous fazirsiran studies can continue in this

Who is the study for?

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.

What is being tested?

The 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.

What are the potential side effects?

Specific side effects are not listed here, but since this is a safety study, all potential side effects will be closely monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

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I am currently having a lung flare-up.

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My blood pressure is very high and not well controlled.

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I cannot commit to returning for all scheduled study visits.

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I currently have or might have COVID-19.

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My liver functions within a specific range.

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I have a chronic liver disease.

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I stopped a trial medication because it caused side effects.

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My cancer is either newly diagnosed or has come back.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline (current study), weeks 12, 24, 36, 48, 60, 72, 84, 96, eos (current study [week 120])

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline (current study), weeks 12, 24, 36, 48, 60, 72, 84, 96, eos (current study [week 120])

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline (current study), weeks 12, 24, 36, 48, 60, 72, 84, 96, eos (current study [week 120]) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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

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

Number of Participants With Clinically Significant Changes in Laboratory Parameters

+1 more

Secondary study objectives

Change from Baseline in Hepatic Stiffness Assessed by Magnetic Resonance Elastography (MRE) at Weeks 48 and 96

Change from Baseline in Intrahepatic Portal Inflammation Score at Week 102 in Liver Biopsy

Change from Baseline in Intrahepatic Z-AAT Protein Polymer Burden Assessed by Periodic Acid Schiff Plus Diastase (PAS+D) Staining in Liver Biopsy at Week 102

+3 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

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 for up to 96 weeks or until participant withdraws from the study or the sponsor terminates the study.

0 / 10Eligibility criteria met