Study on Fazirsiran for Patients with Alpha-1 Antitrypsin Deficiency-Related Liver Disease and Fibrosis

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What is this study about?

This clinical trial is focused on studying a condition known as Alpha-1 Antitrypsin Deficiency-Associated Liver Disease. This disease is linked to an abnormal version of a protein called alpha-1 antitrypsin, which can lead to liver damage and scarring, also known as fibrosis. The study is testing a treatment called Fazirsiran, which is a solution for injection. Fazirsiran is also known by its code name TAK-999 and is designed to target specific liver cells to help reduce the effects of this disease.

The purpose of the study is to evaluate how effective Fazirsiran is compared to a placebo in improving liver health in people with this condition. Participants in the study will receive either Fazirsiran or a placebo through subcutaneous injections, which means the injection is given under the skin. The study will take place over a period of time, with regular check-ups to monitor the health of the liver and the overall safety of the treatment.

Throughout the study, participants will undergo various assessments to track changes in their liver condition. These assessments will help determine if Fazirsiran can reduce liver scarring and improve liver function. The study aims to provide valuable information on whether this treatment can be a beneficial option for people with Alpha-1 Antitrypsin Deficiency-Associated Liver Disease.

1 initial visit

Upon joining the study, an initial visit is scheduled. During this visit, a detailed explanation of the study procedures, potential risks, and benefits is provided. Consent is obtained to participate in the trial.

A comprehensive medical history is taken, and a physical examination is conducted to ensure eligibility based on the study criteria.

2 screening tests

Screening tests are performed to confirm the diagnosis of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease. This includes a liver biopsy to assess the stage of liver fibrosis.

Additional tests may include blood tests and imaging studies to evaluate liver function and structure.

3 randomization

Participants are randomly assigned to receive either the study medication Fazirsiran or a placebo, which is a substance with no active medication, administered as a subcutaneous injection.

4 treatment phase

The treatment phase involves receiving the assigned injection every four weeks. The dosage and frequency are determined by the study protocol.

Regular follow-up visits are scheduled to monitor health status, manage any side effects, and assess the effectiveness of the treatment.

5 mid-study assessment

At approximately week 106, a liver biopsy is conducted to evaluate changes in liver fibrosis. This helps determine the effectiveness of the treatment.

Additional assessments may include blood tests and imaging to monitor liver health.

6 continuation of treatment

Participants continue to receive the assigned treatment and attend regular follow-up visits. Health status and treatment effects are continuously monitored.

7 final assessment

At the end of the study, around week 202, a final liver biopsy is performed to assess the long-term effects of the treatment on liver fibrosis.

Final evaluations include blood tests and imaging studies to provide a comprehensive overview of liver health.

8 study completion

Upon completion of the study, participants receive a summary of their health status and any relevant findings from the trial.

Participants may be offered the opportunity to continue receiving the study medication if it is found to be beneficial and safe.

Who Can Join the Study?

The participant must have a diagnosis of the PiZZ genotype AATD. This means they have a specific genetic type related to Alpha-1 Antitrypsin Deficiency. If this diagnosis is not already in their medical records, they will need to undergo testing to confirm it.
The participant can be of any sex and must be aged between 18 and 75 years old.
The participant must have evidence of METAVIR stage F2, F3, or F4 liver fibrosis. This is a way to measure liver scarring. It will be checked by a liver biopsy, which is a small sample of liver tissue taken for testing. This can be done during the screening period or confirmed by a previous biopsy done within 6 months before joining the study.
The participant must have a lung condition that meets specific criteria listed in the study protocol. This means their lung health will be evaluated to ensure it fits the study requirements.

Who Cannot Join the Study?

Patients with any other serious liver disease cannot participate.
Patients who have had a liver transplant are not eligible.
Patients with a history of alcohol or drug abuse in the past year cannot join the study.
Patients who are pregnant or breastfeeding are not allowed to participate.
Patients with any other medical condition that might interfere with the study cannot take part.
Patients who are currently participating in another clinical trial are not eligible.
Patients who have had a significant allergic reaction to the study medication or similar medications cannot participate.
Patients with uncontrolled high blood pressure are not eligible.
Patients with active infections that require treatment cannot join the study.
Patients with a history of cancer within the last five years, except for certain skin cancers, are not eligible.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Medical University Of Graz	Graz	Austria
Medizinische Universitaet Innsbruck	Innsbruck	Austria
Medizinische Hochschule Hannover	Hanover	Germany
IRCCS Humanitas Research Hospital	Rozzano	Italy
Medical University Of Vienna	Vienna	Austria
Centre Hospitalier Universitaire De Bordeaux	Bordeaux	France
Katholieke Universiteit te Leuven	Leuven	Belgium

Other Sites

Site Name	City	Country
Region Oestergoetland	Linkoping	Sweden
Universitaetsklinikum Schleswig-Holstein AöR	Kiel	Germany
Hopital Beaujon	Clichy	France
Hospital Universitario Puerta De Hierro De Majadahonda	Majadahonda	Spain
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone	Palermo	Italy
Landeskrankenanstalten-Betriebsgesellschaft Kabeg	Klagenfurt am Wörthersee	Austria
ID Clinic	Myslowice	Poland
Virgen del Rocío University Hospital	Sevilla	Spain
Universitaetsklinikum Tuebingen AöR	Tuebingen	Germany
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Fondazione IRCCS Policlinico San Matteo	Pavia	Italy
Centre Hospitalier Universitaire De Toulouse	Toulouse	France
Karolinska University Hospital	Solna	Sweden
Universitaetsklinikum Aachen AöR	Aachen	Germany
Deutsches Herzzentrum Berlin	Berlin	Germany
CCAB Centro Clinico Academico Braga Associacao	Braga	Portugal
Servico de Saude da Regiao Autonoma Da Madeira EPERAM	Funchal	Portugal
Hospital Paul Brousse	Villejuif	France
Beaumont Hospital	Dublin	Ireland
Hôpital Pontchaillou-CHU Rennes	Rennes	France
Hospital Universitario Virgen De La Victoria	Malaga	Spain
Iziujzpwx Fjw Ctffkofj Avc Ewnnfnfwcdpn Mckocakh	Prague	Czechia
Lnqvl Ukrlsclvkisl Moapgdp Cvtohvf (wwqvk	Leiden	The Netherlands
Hilrftog Udmuemujlhkwn Mptoiap Du Vxmgwsxqva	Santander	Spain
Hkyzoz Heyfpbjh	Herlev	Denmark
Avzjhfmbv Uys	Amsterdam	The Netherlands
Ciqvcc Hjesxvvrvj Uxsfqhstscvlz Do Pylaz Ecvouz	Porto	Portugal
Uitvdtawzb Ot Aathnlg	Edegem	Belgium
Cnd Crskq Rlthylzmzpw	Lyon	France
Hqmqsihi Vops deyitnox	Barcelona	Spain
Wzu Wcrdqz Ikx Pleia Pxuracox Kykfldb	Warsaw	Poland

Trial status

Country	Status	Recruitment Start
Austria	Recruiting	01.05.2023
Belgium	Recruiting	01.05.2023
Czechia	Recruiting	01.05.2023
Denmark	Recruiting	01.05.2023
France	Recruiting	01.05.2023
Germany	Recruiting	01.05.2023
Ireland	Recruiting	01.05.2023
Italy	Recruiting	01.05.2023
Poland	Recruiting	01.05.2023
Portugal	Recruiting	01.05.2023
Spain	Recruiting	01.05.2023
Sweden	Recruiting	01.05.2023
The Netherlands	Recruiting	01.05.2023

Trial locations

Investigated drugs:

Fazirsiran is a medication being studied for its potential to help people with liver disease caused by a condition called Alpha-1 Antitrypsin Deficiency (AATD). This condition can lead to liver damage over time. Fazirsiran is designed to target and reduce the harmful effects of the abnormal protein that builds up in the liver due to AATD. By doing this, it aims to improve the health of the liver and reduce the amount of scarring, or fibrosis, in the liver tissue. The study is looking at how well Fazirsiran works in improving liver health and whether it is safe for patients to use.

Alpha-1 Antitrypsin Deficiency-Associated Liver Disease – This disease is caused by a genetic deficiency of the protein alpha-1 antitrypsin, which is produced in the liver. The deficiency leads to the accumulation of abnormal protein in the liver cells, causing liver damage. Over time, this can result in liver fibrosis, where scar tissue forms in the liver. As the disease progresses, fibrosis can advance to cirrhosis, a condition where the liver is severely scarred and its function is impaired. The progression of liver damage can vary, with some individuals experiencing more rapid advancement than others. The disease is often monitored through liver biopsies and imaging to assess the extent of fibrosis and liver stiffness.

Trial ID:

2022-501943-34-00

Protocol code:

TAK-999-3001

NCT ID:

NCT05677971

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Fazirsiran for Patients with Alpha-1 Antitrypsin Deficiency-Related Liver Disease and Fibrosis

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?