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New drug could slow liver damage in rare genetic disease

NCT ID NCT05677971

Disease control Sponsor: Takeda Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests an experimental drug called fazirsiran in 160 adults with Alpha-1 antitrypsin deficiency who have moderate to severe liver scarring. The goal is to see if fazirsiran can reduce fibrosis and slow disease progression compared to a placebo. Participants will receive either the drug or a placebo and undergo two liver biopsies over about two years.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 160 people

The number the study aims to enrol. It can still change while the study runs.

Started

Mar 2023

Expected to finish

Dec 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion criteria: * The participant must have a diagnosis of the Z allele homozygotes (PiZZ) genotype AATD. PiZZ diagnosis from source verifiable medical records is permitted. Otherwise, participants must undergo PiZZ confirmatory testing (genotyping for PiS and PiZ alleles) at screening. PiMZ or PiSZ genotypes are not permitted. * The participant, of any sex, is aged 18 to 75 years, inclusive. * The participant's liver biopsy core sample collected should meet the requirements of the protocol. * The participant has evidence of METAVIR stage F2, F3, or F4 liver fibrosis, evaluated by a centrally read baseline liver biopsy during the screening period; or confirmed as meeting all the entry criteria by central reading of a previous biopsy conducted within 6 months before the estimated enrollment date using an adequate liver biopsy and slides as defined in the study laboratory manual. * The participant has a pulmonary status meeting the protocol's requirements. * It must be confirmed that the participant does not have HCC. Participants will be screened for HCC with alpha-fetoprotein (AFP) and abdominal ultrasound. If the participant has any of the following, they will be required to have contrast-enhanced CT or MRI imaging to exclude HCC before randomization. * An adult participant must have a body mass index (BMI) greater than or equal to (\>=) 18.0 kilograms per meter square (kg\^m2). * The participant is a nonsmoker for at least 6 months before screening. Exclusion Criteria * The participant has a history of liver decompensating events (overt hepatic encephalopathy \[West Haven Grade \>=2\] documented by a physician or healthcare professional, clinically significant ascites, spontaneous bacterial peritonitis, GI bleeding from varices, hepatopulmonary syndrome, hepatorenal syndrome, portal pulmonary hypertension, or bleeding portal hypertensive gastropathy). * The participant has a history of the presence of medium or large varices or varices with red wale signs based on a previous esophagogastroduodenoscopy (EGD) within 6 months before the estimated enrollment date. For certain participants, an EGD will be required at screening if there is no EGD available within 6 months before the estimated enrollment date. Presence of small varices with no red wale signs on EGD and no history of bleeding will be acceptable for study eligibility. * The participant has evidence of chronic liver disease attributable to other diseases, including viral hepatitis B or C, primary biliary cholangitis, primary sclerosing cholangitis, Wilson disease, alcoholic hepatitis, hemochromatosis, liver cancer, history of biliary diversion, or autoimmune hepatitis. * The participant has alanine transaminase (ALT) or aspartate transaminase (AST) levels \>250 units per liter (U/L). * The participant has a platelet count \<60,000 per cubic millimeter (mm\^3) (\<60 × 10\^9 per liter \[10\^9/L\]). * The participant has albumin \<=2.8 gram per deciliter (g/dL) (28 grams per liter \[g/L\]). * The participant has international normalized ratio (INR) \>=1.7. * The participant is expected to have severe and unavoidable high-level exposure to inhaled pulmonary toxins during the study such as may occur with occupational exposure to mineral dusts or metals. * The participant has a history of drug abuse (such as cocaine, phencyclidine) within 1 year before the screening visit or has a positive urine drug screen at screening. * The participant has previously been treated with fazirsiran or any other RNAi for AATD-LD. * The participant has portal vein thrombosis. * The participant has a prior transjugular portosystemic shunt procedure. * The participant has a history of malignancy within the last 5 years, except for adequately treated basal cell carcinoma, squamous cell skin cancer, superficial bladder tumors, or in situ cervical cancer. Participants with other curatively treated malignancies who have no evidence of metastatic disease and a greater than 1-year disease-free interval may be entered after approval by the medical monitor.

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Conditions

The condition(s) this trial relates to.

alpha 1-antitrypsin deficiency

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ALPHA1-antitrypsin deficiency

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    81 sites in 19 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Addenbrooke's Hospital

    RECRUITING

    Cambridge, CB2 0QQ, United Kingdom

  • Amsterdam UMC - VUmc - De Boelelaan

    WITHDRAWN

    Amsterdam, 1081 HV, Netherlands

  • Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone

    RECRUITING

    Palermo, 90127, Italy

  • Baylor College of Medicine Medical Center

    RECRUITING

    Houston, Texas, 77030-4202, United States

  • Beaumont Hospital

    RECRUITING

    Dublin, D09 YD60, Ireland

  • Bon Secours St. Mary's Hospital

    RECRUITING

    Richmond, Virginia, 23226, United States

  • Boston Medical Center

    RECRUITING

    Boston, Massachusetts, 02118-2908, United States

  • Brigham and Womens Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • CCA Hospital Braga

    RECRUITING

    Braga, 4710-243, Portugal

  • Cardinal Glennon Children's Hospital

    RECRUITING

    St Louis, Missouri, 63104-1003, United States

  • Centre Francois Magendie

    RECRUITING

    Pessac, 33064, France

  • Centro Hospitalar do Porto

    RECRUITING

    Porto, 4099-003, Portugal

  • Charité - Campus Virchow-Klinikum-Ostring 1

    RECRUITING

    Berlin, 13353, Germany

  • Children's Healthcare of Atlanta

    WITHDRAWN

    Atlanta, Georgia, 30329, United States

  • Columbia University Irving Medical Center

    RECRUITING

    New York, New York, 10032-3722, United States

  • Derriford Hospital

    RECRUITING

    Plymouth, PL6 8DH, United Kingdom

  • Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

    RECRUITING

    Milan, 20122, Italy

  • Fondazione IRCCS Policlinico San Matteo di Pavia

    RECRUITING

    Pavia, 27100, Italy

  • GI Research Institute

    RECRUITING

    Vancouver, British Columbia, V6Z 2K5, Canada

  • Gastroenterology & Liver Institute

    WITHDRAWN

    Escondido, California, 92025, United States

  • Hannover Medical School

    RECRUITING

    Hanover, 30625, Germany

  • Henry Ford Medical Center - Columbus

    RECRUITING

    Novi, Michigan, 48377-3600, United States

  • Hopital PONTCHAILLOU CHU de Rennes

    RECRUITING

    Rennes, 35000, France

  • Hospital Nélio Mendonça

    RECRUITING

    Funchal, 9000-168, Portugal

  • Hospital Purpan

    RECRUITING

    Toulouse, 31000, France

  • Hospital Sirio-Libanes

    RECRUITING

    São Paulo, 01308-050, Brazil

  • Hospital Universitario Marques de Valdecilla

    RECRUITING

    Santander, 39008, Spain

  • Hospital Universitario Vall d'Hebron - PPDS

    RECRUITING

    Barcelona, 8025, Spain

  • Hospital Universitario Virgen de la Victoria

    RECRUITING

    Málaga, 29071, Spain

  • Hospital Universitario Virgen del Rocio - PPDS

    RECRUITING

    Seville, 41013, Spain

  • Hospital de Base do Distrito Federal

    RECRUITING

    Brasília, 71692-000, Brazil

  • Hvidovre Hospital

    RECRUITING

    Hvidovre, 2650, Denmark

  • Hôpital Beaujon

    RECRUITING

    Clichy, 92110, France

  • Hôpital Paul Brousse

    RECRUITING

    Val-de-Marne, 94805, France

  • Hôpital de La Croix Rousse

    RECRUITING

    Lyon, 69317, France

  • ID Clinic Arkadiusz Pisula

    WITHDRAWN

    Śląskie, 41-400, Poland

  • IRCCS Istituto Clinico Humanitas

    RECRUITING

    Rozzano, 20089, Italy

  • Indiana University School of Medicine - Indianapolis

    RECRUITING

    Indianapolis, Indiana, 46202, United States

  • Inselspital Bern

    RECRUITING

    Bern, 3010, Switzerland

  • Inspiration Research Limited

    RECRUITING

    Toronto, Ontario, M5T 3A9, Canada

  • Institut klinicke a experimentalni mediciny

    RECRUITING

    Prague, 140 21, Czechia

  • Karolinska Universitetssjukhuset Huddinge

    RECRUITING

    Huddinge, 14186, Sweden

  • King's College Hospital

    RECRUITING

    London, SE5 9RS, United Kingdom

  • Klinikum Klagenfurt Am Wörthersee

    RECRUITING

    Klagenfurt, 9020, Austria

  • LKH-Universitätsklinikum Graz

    RECRUITING

    Graz, 8036, Austria

  • Leiden University Medical Center

    RECRUITING

    Leiden, 2333 ZA, Netherlands

  • Mayo Clinic

    RECRUITING

    Phoenix, Arizona, 85054-4502, United States

  • Mayo Clinic - PPDS

    RECRUITING

    Rochester, Minnesota, 55905-0001, United States

  • Medical University of South Carolina

    RECRUITING

    Charleston, South Carolina, 29425, United States

  • Medizinische Universität Innsbruck

    RECRUITING

    Innsbruck, 6020, Austria

  • Medizinische Universität Wien (Medical University of Vienna)

    RECRUITING

    Vienna, 1090, Austria

  • Morgan Stanley Childrens Hospital of New York Presbyterian (CHONY) - PIN

    WITHDRAWN

    New York, New York, 10032-1559, United States

  • NYU Langone Health

    RECRUITING

    New York, New York, 10016, United States

  • Ochsner Medical Center

    RECRUITING

    New Orleans, Louisiana, 70121-2429, United States

  • Peak Gastroenterology Associates, PC

    RECRUITING

    Colorado Springs, Colorado, 80907, United States

  • Penn State Health Milton S. Hershey Medical Center

    RECRUITING

    Hershey, Pennsylvania, 17033, United States

  • Queen Elizabeth II Health Sciences Center

    RECRUITING

    Halifax, Nova Scotia, B3H 2Y9, Canada

  • Queen's Medical Centre

    WITHDRAWN

    Nottingham, NG7 2UH, United Kingdom

  • Royal Adelaide Hospital

    WITHDRAWN

    Adelaide, South Australia, 5000, Australia

  • Royal Free Hospital

    RECRUITING

    London, NW3 2Q, United Kingdom

  • Royal Infirmary of Edinburgh

    RECRUITING

    Edinburgh, EH16 4SA, United Kingdom

  • Schiff Center for Liver Diseases/University of Miami

    RECRUITING

    Miami, Florida, 33136-1051, United States

  • St Vincents Hospital Melbourne - PPDS

    RECRUITING

    Fitzroy, Victoria, 3065, Australia

  • St. Joseph's Hospital and Medical Center

    RECRUITING

    Phoenix, Arizona, 85013, United States

  • Stanford University

    RECRUITING

    Palo Alto, California, 94303, United States

  • Temple University Hospital

    RECRUITING

    Philadelphia, Pennsylvania, 19140-5103, United States

  • Texoma Liver Center

    RECRUITING

    Denison, Texas, 75020, United States

  • The Texas Liver Institute

    RECRUITING

    San Antonio, Texas, 78215, United States

  • UCLA Pulmonary and Critical Care

    RECRUITING

    Los Angeles, California, 90095-8344, United States

  • UZ Antwerpen

    RECRUITING

    Antwerp, 2650, Belgium

  • UZ Leuven

    RECRUITING

    Leuven, 3000, Belgium

  • Universidade Estadual Paulista Julio de Mesquita Filho Faculdade de Medicina Campus de Botucatu

    RECRUITING

    São Paulo, 18618-687, Brazil

  • Universitetssjukhuset i Linköping

    RECRUITING

    Linköping, 581 85, Sweden

  • University Hospitals Cleveland Medical Center

    RECRUITING

    Cleveland, Ohio, 44106-1716, United States

  • University of Alabama at Birmingham

    RECRUITING

    Birmingham, Alabama, 35233, United States

  • University of Arizona Thomas D. Boyer Liver Institute

    WITHDRAWN

    Tucson, Arizona, 85724-5136, United States

  • University of California Benioff Children's Hospital

    RECRUITING

    San Francisco, California, 94143, United States

  • University of California San Diego, Altman Clinical and Translational Institute

    RECRUITING

    La Jolla, California, 92093, United States

  • University of Florida

    RECRUITING

    Gainesville, Florida, 32611, United States

  • University of Iowa Hospitals and Clinics

    RECRUITING

    Iowa City, Iowa, 52242, United States

  • University of Maryland Medical Center

    RECRUITING

    Baltimore, Maryland, 21201-1504, United States

  • University of Michigan Hospital

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • Universitätsklinikum Schleswig-Holstein - Campus Kiel

    RECRUITING

    Kiel, 24105, Germany

  • Universitätsklinikum Tübingen

    RECRUITING

    Tübingen, 72076, Germany

  • Universitätsklinikum der RWTH Aachen

    RECRUITING

    Aachen, 52074, Germany

  • VCU Medical Center North Hospital

    RECRUITING

    Richmond, Virginia, 23298-5028, United States

  • Vanderbilt University Medical Center

    RECRUITING

    Nashville, Tennessee, 37232-0028, United States

  • WIP Warsaw IBD Point Profesor Kierkus

    RECRUITING

    Warsaw, 00-728, Poland

  • Washington University School of Medicine in St. Louis

    RECRUITING

    St Louis, Missouri, 63110, United States

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