RecruitingPHASE3INTERVENTIONAL

Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein

This study is looking into a new treatment called fazirsiran for people who have Alpha-1 Antitrypsin Deficiency, a condition that can cause liver scarring. Researchers want to find out if fazirsiran can reduce this scarring compared to a dummy drug (placebo). They will also check if it can slow down the disease getting worse in the liver and reduce other liver problems like inflammation. The study will also help understand how fazirsiran acts in the body and if it reduces the abnormal protein in the liver. Safety is a key focus, so they will be carefully looking for any side effects. Participants will either receive fazirsiran or the placebo, and small liver tissue samples will be taken twice during the study.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Takeda

Enrolment target

160

Start

06 Mar 2023

Estimated completion

12 Dec 2031

Alpha1-Antitrypsin Deficiency

What is this study about?

This important study is for people with a specific type of liver disease caused by Alpha-1 Antitrypsin Deficiency. In this condition, an abnormal protein can build up in the liver, leading to scarring, also known as fibrosis. Over time, this scarring can become a serious health issue.

The main goal of this study is to see if a new drug, fazirsiran, can actually reduce this liver scarring. Researchers are also keen to learn if fazirsiran can stop the liver disease from getting worse. They will also look at whether it can lessen inflammation in the liver and reduce the amount of the harmful abnormal protein. Understanding how the drug works in the body and how the body handles it is also a key part of the research.

To do this, some participants will receive the active drug, fazirsiran, while others will be given a 'placebo', which looks like the drug but contains no active medicine. This allows researchers to compare the two groups and clearly see if fazirsiran is making a difference. Throughout the study, doctors will closely monitor participants for any changes in their liver health using various tests, and they will also pay close attention to any possible side effects.

Key takeaways

This study tests a new drug, fazirsiran, for liver scarring in Alpha-1 Antitrypsin Deficiency.
It aims to see if the drug reduces scarring and slows liver disease progression.
Some participants will get the actual drug, others a dummy drug (placebo).
Liver biopsies will be done twice to check for changes in scarring.
The study will also monitor for side effects and how the drug works in the body.

Who may be eligible?

To join this study, you must be between 18 and 75 years old and have a specific diagnosis of Alpha-1 Antitrypsin Deficiency (called PiZZ genotype). Your liver must show signs of scarring, which will be confirmed by looking at a small tissue sample (biopsy).

There are also some other health checks. For example, your lungs need to be healthy enough for the study, and you must not have certain serious liver complications, like a history of internal bleeding from swollen veins in the gut. Your doctors will also check to make sure you don't have liver cancer (HCC).

You won't be able to join if you have experienced certain severe liver problems in the past, or if you have specific types of enlarged veins in your food pipe (varices). You also need to have a Body Mass Index (BMI) of 18 or higher, and you must have stopped smoking at least six months before the screening tests.

Quick self-check

Are you between 18 and 75 years old?
Have you been diagnosed with PiZZ Alpha-1 Antitrypsin Deficiency?
Has your doctor told you that you have liver scarring (fibrosis)?
Have you avoided smoking for at least the past six months?
Do you not have a history of severe liver complications or liver cancer?

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

What does participation involve?

If you decide to take part, you will either receive Fazirsiran or a placebo (a dummy drug). Both will be given as injections. Over the course of the study, you will have regular visits to the clinic for various checks. These checks might include blood tests, imaging scans of your liver, and other assessments to see how your liver is responding to the treatment.

A key part of the study involves having a liver biopsy twice. This is a procedure where a small tissue sample is taken from your liver to be examined under a microscope. This helps doctors see any changes in your liver scarring. The study will also track how the drug affects your body and look for any side effects. Your participation will last for a certain period, which the study team will explain to you in detail.

Potential risks and benefits

Taking part in a clinical trial offers the potential benefit of accessing a new treatment before it's widely available. While fazirsiran might help reduce liver scarring and improve your liver health, it's also possible it won't work for you, or that you might receive the placebo. All medications carry potential risks, and fazirsiran could have side effects, which the study team will monitor closely. You will be fully informed about all known risks before you decide to join, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (89)

University of Alabama at Birmingham
Birmingham, United States· Recruiting
St. Joseph's Hospital and Medical Center
Phoenix, United States· Recruiting
Mayo Clinic
Phoenix, United States· Recruiting
University of Arizona Thomas D. Boyer Liver Institute
Tucson, United States· Withdrawn
Gastroenterology & Liver Institute
Escondido, United States· Withdrawn
University of California San Diego, Altman Clinical and Translational Institute
La Jolla, United States· Recruiting
UCLA Pulmonary and Critical Care
Los Angeles, United States· Recruiting
Stanford University
Palo Alto, United States· Recruiting
University of California Benioff Children's Hospital
San Francisco, United States· Recruiting
Peak Gastroenterology Associates, PC
Colorado Springs, United States· Recruiting
University of Florida
Gainesville, United States· Recruiting
Schiff Center for Liver Diseases/University of Miami
Miami, United States· Recruiting

+77 more sites — see the official record for the full list.

Common questions

What is Alpha-1 Antitrypsin Deficiency?

It's a genetic condition where your body doesn't produce enough of a protective protein. This can lead to lung and liver damage, including scarring in the liver.

What is a placebo?

A placebo is a substance that looks exactly like the real drug but doesn't contain any active medicine. It helps researchers compare the effects of the active drug.

What is a liver biopsy?

A liver biopsy is a procedure where a very small piece of your liver tissue is taken, usually with a needle, to be examined under a microscope. It helps doctors see how healthy your liver is.

Will I know if I'm getting the drug or the placebo?

No, this is usually a 'blinded' study, meaning neither you nor your doctors will know whether you are receiving fazirsiran or the placebo until after the study is complete. This ensures fair results.

What are 'liver biomarkers'?

These are substances in your blood that your body normally produces. Doctors measure them to get clues about how well your liver is working and if its condition is changing.

How to find out more

Takeda Contact

medinfoUS@takeda.com 1-877-825-3327 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.