Active not recruitingPHASE3INTERVENTIONAL

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

This research study is looking at a medicine called fazirsiran. Its main purpose is to understand if fazirsiran is safe to use for a long time in people who have liver disease because of a genetic condition called Alpha-1 Antitrypsin Deficiency. This condition causes a problem with a protein called Z-alpha-1 antitrypsin, which can damage the liver. People who have already taken part in similar fazirsiran studies can continue to receive the medicine every three months if they join this new study. The study also wants to see if fazirsiran can help reduce scarring in the liver (called fibrosis) or slow down its progression over time. This could potentially help improve liver health for those affected by this condition.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Takeda

Enrolment target

Start

08 Aug 2023

Estimated completion

02 May 2033

Alpha1-Antitrypsin Deficiency

What is this study about?

This study is focused on a genetic condition called Alpha-1 Antitrypsin Deficiency, specifically when it causes liver problems. Our bodies make a protein called alpha-1 antitrypsin, which helps protect our lungs and liver. However, in some people, a faulty version of this protein, called Z-alpha-1 antitrypsin, doesn't work properly and can build up in the liver, leading to damage and disease.

The medicine being studied is called fazirsiran. This is an 'extension study,' which means it's a continuation for people who have already been taking part in earlier research studies on fazirsiran. The main goal is to find out if this medicine is safe when used over a longer period of time. Medical experts want to monitor participants to ensure there are no unexpected side effects or long-term issues.

Beyond just safety, the study also hopes to gather information on whether fazirsiran can help the liver itself. They are particularly interested in seeing if it can reduce or slow down the development of liver scarring, known as fibrosis. Liver fibrosis can lead to serious liver problems, so finding a way to manage it would be a big step forward for people with this condition.

Key takeaways

This study is for long-term safety and effects of fazirsiran.
It's for people with liver disease from Alpha-1 Antitrypsin Deficiency.
Only previous fazirsiran study participants can join.
Researchers want to see if it helps with liver scarring over time.
Participants will receive injections every three months.
You can leave the study at any time.

Who may be eligible?

To join this study, you need to have already taken part in one of the previous fazirsiran studies (called AROAAT2001 or AROAAT2002). This study is for adults aged 18 and over. You must be able to understand what the study involves and be willing to follow the study's instructions and attend appointments.

It's important that you are able to fill out questionnaires, which might be done on a computer or tablet. You'll also need to provide written consent, showing that you understand and agree to be part of the study. If you have trouble signing but can understand and agree, a special witness can sign for you.

Finally, for your own health, you must not be a daily cigarette smoker and should not have smoked daily for at least 24 weeks before joining. We will check this with a urine test. E-cigarettes are also not allowed. If you use nicotine replacement products like patches or gum, that's generally okay.

Quick self-check

Have I participated in a previous fazirsiran study?
Am I 18 years old or older?
Am I able to read and understand study materials?
Am I willing to follow study instructions and attend appointments?
Do I currently not smoke cigarettes daily?
Am I willing to provide written consent?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, you will continue to receive the fazirsiran injection regularly, usually every three months. An injection is when the medicine is given with a needle. You will have regular appointments where study staff will check how you are doing, monitor your health, and ask you questions about any changes you experience. These appointments are important for tracking the safety and potential effects of the medicine over time. The study will continue as long as it is ongoing, or until fazirsiran becomes officially available to the public in your country, allowing people to access it outside of a study. The total duration of your participation will depend on these factors.

Potential risks and benefits

Participating in this study might offer a potential benefit: continuing to receive fazirsiran, which may help with your liver condition by potentially reducing or slowing down liver scarring. However, as with any medication, there are always potential risks or side effects that are not yet fully known, especially with long-term use. The study aims to gather more information on these. You will be closely monitored for any unwanted effects. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (10)

UCSD Altman Clinical and Translational Research Institute
La Jolla, United States
Stanford Medicine Outpatient Center
Redwood City, United States
UF Clinical and Translational Science Institute
Gainesville, United States
University Of Iowa Hospitals And Clinics
Iowa City, United States
Medical University of South Carolina - Hollings Cancer Center - PPDS
Charleston, United States
Medizinische Universitat Wien (Medical University of Vienna)
Vienna, Austria
Universitätsklinikum der RWTH Aachen
Aachen, Germany
Hospital Nélio Mendonça
Funchal, Portugal
Addenbrooke's Hospital
Cambridge, United Kingdom
Royal Infirmary of Edinburgh - PPDS
Edinburgh, United Kingdom

Common questions

What is Alpha-1 Antitrypsin Deficiency?

It's a genetic condition where your body doesn't make enough of a special protein that protects your lungs and liver. A faulty version of this protein can build up and damage the liver.

What is fazirsiran?

Fazirsiran is a medicine being tested in studies for people with Alpha-1 Antitrypsin Deficiency. It's usually given as an injection.

Why is this called an 'extension study'?

This study is a continuation for people who have already been taking part in earlier fazirsiran research. It allows them to keep receiving the medicine and helps us learn about its long-term effects.

How often will I receive the medicine?

If you join, you will typically receive the fazirsiran injection every three months.

Can I stop being in the study if I change my mind?

Yes, you can choose to leave the study at any time, and it won't affect your regular medical care.