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Antisense Oligonucleotide

Fazirsiran 200 mg 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

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

Study 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.Check my eligibility

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.See study design

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

Select...

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

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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 outcome measures

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

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 / 1Eligibility criteria met

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Who is running the clinical trial?

TakedaLead Sponsor

1,205 Previous Clinical Trials

4,187,932 Total Patients Enrolled

Study DirectorStudy DirectorTakeda

1,214 Previous Clinical Trials

499,477 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the main goals that this investigation aims to achieve?

"The primary aim of this investigation, conducted over a period spanning the commencement of administering the study drug until the end-of-study evaluation at Week 120, is to determine the count of participants manifesting clinically significant alterations in laboratory metrics. Secondary endpoints consist of assessing individuals for any lack of progression compared to baseline levels by at least one stage on histologic fibrosis from liver biopsy findings at Week 102 according to Meta-Analysis of Histological Data in Viral Hepatitis (METAVIR) staging criteria; evaluating changes from baseline values in intrahepatic portal inflammation score as observed during Week 102 through liver biopsy assessments scored between "

Answered by AI

Which individuals meet the criteria for participation in this research study?

"Candidates seeking enrollment in this research study must exhibit alpha-1 antitrypsin deficiency and fall within the age bracket of 18 to 75. The trial aims to recruit a total of 37 participants."

Answered by AI

Are there any available slots for patients to participate in this research study?

"As per clinicaltrials.gov, the ongoing medical investigation is not presently in search of participants. The trial was initially made public on August 8th, 2023 and last modified on February 7th, 2024. Despite the current non-recruitment status of this particular study, there exist a total of 273 other trials welcoming new enrollees at present."

Answered by AI

Has Fazirsiran 200 mg received the official seal of approval from the FDA?

"The safety of Fazirsiran 200 mg is rated as a 3 on a scale from 1 to 3, based on the fact that this trial is in Phase 3. This indicates that there is existing evidence supporting its efficacy and multiple rounds of data demonstrating its safety."

Answered by AI

In how many medical facilities is this examination being conducted?

"In addition to UCSD Altman Clinical and Translational Research Institute in La Jolla, UF Clinical and Translational Science Institute in Gainesville, and University Of Iowa Hospitals And Clinics in Iowa City; there are 5 other sites participating in this clinical trial."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2023. "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. https://clinicaltrials.gov/ct2/show/NCT05899673.

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