Study on Fazirsiran for Patients with Alpha-1 Antitrypsin Deficiency Liver Disease and Mild 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, which is a genetic disorder that can lead to liver damage. The study is investigating a treatment called Fazirsiran, also known by its code name TAK-999. Fazirsiran is a type of medication designed to target specific genes in the liver to help reduce the damage caused by this disease. The trial will compare the effects of Fazirsiran with a placebo to understand its safety and effectiveness, particularly in people with mild liver scarring, also known as fibrosis.

The main goal of the study is to evaluate the long-term safety of Fazirsiran, with a special focus on lung health, as the condition can also affect the lungs. Participants in the study will receive either Fazirsiran or a placebo through injections. The study will monitor various health indicators, including lung function and liver health, over a period of time to see how the treatment affects these areas. Participants will undergo regular check-ups and tests to track any changes in their condition.

This study is important for understanding how Fazirsiran can help people with Alpha-1 Antitrypsin Deficiency-Associated Liver Disease and to ensure that it is safe for long-term use. By participating in this trial, researchers hope to gather valuable information that could lead to better treatment options for those affected by this genetic disorder.

1 joining the study

Upon joining the study, the participant is confirmed to have a diagnosis of the PiZZ genotype associated with alpha-1 antitrypsin deficiency. The participant is between 18 and 75 years old and has evidence of mild liver scarring, known as METAVIR stage 1 fibrosis, confirmed by a liver biopsy.

2 initial assessment

An initial assessment is conducted to evaluate the participant’s lung function and overall health status. This includes checking vital signs, performing an electrocardiogram (ECG), and conducting laboratory tests such as blood and urine analysis.

3 treatment phase

The participant receives either the study medication, fazirsiran, or a placebo. The medication is administered as a subcutaneous injection, which means it is injected under the skin. The frequency and dosage are determined by the study protocol.

4 ongoing monitoring

Throughout the study, the participant’s health is monitored regularly. This includes checking for any side effects, changes in lung function, and liver health. Pulmonary function tests and computed tomography (CT) scans are performed to assess lung density and function over time.

5 final assessment

At the end of the study, a final assessment is conducted. This includes a comprehensive evaluation of the participant’s lung and liver health, as well as any changes in the levels of specific proteins associated with the condition.

Who Can Join the Study?

  • The participant must have a diagnosis of the PiZZ genotype AATD. This means they have a specific genetic form of Alpha-1 Antitrypsin Deficiency.
  • The participant, of any sex, is aged 18 to 75 years, inclusive.
  • The participant has evidence of METAVIR stage 1 liver fibrosis. This is a mild form of liver scarring, which is checked by a liver biopsy, a procedure where a small piece of the liver is taken to look for disease.
  • The participant has a pulmonary status meeting the protocol’s requirements. This means their lung health must meet certain standards set by the study.

Who Cannot Join the Study?

  • Patients with other serious liver diseases that are not related to Alpha-1 Antitrypsin Deficiency.
  • Patients who have had a liver transplant.
  • Patients with severe lung diseases that are not related to Alpha-1 Antitrypsin Deficiency.
  • Patients who are currently participating in another clinical trial.
  • Patients who have a history of drug or alcohol abuse.
  • Patients who are pregnant or breastfeeding.
  • Patients who have any condition that the study doctors believe would make it unsafe for them to participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Medizinische Hochschule Hannover Hanover Germany
Centre Hospitalier Universitaire De Lille Lille France
Medical University Of Graz Graz Austria
Katholieke Universiteit te Leuven Leuven Belgium

Other Sites

Site Name City Country Status
Universitaetsklinikum Aachen AöR Aachen Germany
CCAB Centro Clinico Academico Braga Associacao Braga Portugal
Servico de Saude da Regiao Autonoma Da Madeira EPERAM Funchal Portugal
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 Policlinico San Matteo Pavia Italy
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Karolinska University Hospital Solna Sweden
Centre Hospitalier Universitaire De Rennes Rennes France
Centro Hospitalar Universitario De Santo Antonio E.P.E. Porto Portugal
Hospital Paul Brousse Villejuif France
Beaumont Hospital Dublin Ireland
Uqjjdvgixm On Aivpjdd Edegem Belgium
Cmz Ckbot Rbvrfiyqlqh Lyon France

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Recruiting
01.05.2024
Belgium Belgium
Recruiting
01.05.2024
France France
Recruiting
01.05.2024
Germany Germany
Recruiting
01.05.2024
Ireland Ireland
Recruiting
01.05.2024
Italy Italy
Recruiting
01.05.2024
Poland Poland
Not recruiting
01.05.2024
Portugal Portugal
Recruiting
01.05.2024
Spain Spain
Recruiting
01.05.2024
Sweden Sweden
Recruiting
01.05.2024

Trial locations

Investigated drugs:

Fazirsiran is a medication being studied for its potential to treat liver disease associated with Alpha-1 Antitrypsin Deficiency. This condition can lead to liver damage due to the accumulation of abnormal proteins. Fazirsiran aims to reduce these harmful proteins in the liver, potentially improving liver function and slowing disease progression. The trial is also focused on assessing the long-term safety of Fazirsiran, particularly its effects on lung health and pulmonary function.

Alpha-1 Antitrypsin Deficiency-Associated Liver Disease – This is a genetic condition where the liver produces an abnormal form of a protein called alpha-1 antitrypsin. This abnormal protein can accumulate in the liver, leading to liver damage over time. The disease may start with mild liver dysfunction and can progress to more significant liver issues. Symptoms can include jaundice, fatigue, and abdominal swelling. The progression of the disease varies among individuals, with some experiencing more severe liver problems than others. It is considered a rare disease, affecting a small percentage of the population.

Trial ID:
2023-504198-19-00
Protocol code:
TAK-999-3002
Trial Phase:
Therapeutic confirmatory (Phase III)

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