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
NCT ID: NCT05677971
Last Updated: 2025-11-25
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE3
160 participants
INTERVENTIONAL
2023-03-06
2030-08-14
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Participants will either receive fazirsiran or placebo. Liver biopsies, a way of collecting a small tissue sample from the liver, will be taken twice during this study.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)
NCT06165341
Phase 1b/2, Open Label, Repeat Dose, Dose Escalation Study of ND-L02-s0201 Injection in Subjects With Moderate to Extensive Fibrosis (METAVIR F3-4)
NCT02227459
A Study About Fazirsiran in People With and Without Liver Problems
NCT05891158
Metabolic Interventions to Resolve Non-alcoholic Steatohepatitis (NASH) With Fibrosis (MIRNA)
NCT04321031
FibroScan ® Study: Assessing Liver Fibrosis in Patients Using Methotrexate for Psoriasis
NCT03820934
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Fazirsiran
Participants will receive fazirsiran 200 milligram per milliliter (mg/ml) subcutaneous (SC) injection on Day 1, at Week 4, and then every 12 weeks (Q12 W) thereafter up to Week 196.
Fazirsiran Injection
Participants will receive fazirsiran 200 mg/ml SC injection on Day 1, at Week 4, and Q12 W thereafter up to Week 196.
Placebo
Participants will receive placebo on Day 1, at Week 4, and Q12 W thereafter up to Week 196.
Placebo
Participants will receive placebo (sterile normal saline \[0.9% NaCl\]) SC injection on Day 1, at Week 4, and Q12 W thereafter up to Week 196.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Fazirsiran Injection
Participants will receive fazirsiran 200 mg/ml SC injection on Day 1, at Week 4, and Q12 W thereafter up to Week 196.
Placebo
Participants will receive placebo (sterile normal saline \[0.9% NaCl\]) SC injection on Day 1, at Week 4, and Q12 W thereafter up to Week 196.
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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) between 18.0 and 39.0 kilograms per meter square (kg\^m2), inclusive.
* The participant is a nonsmoker for at least 6 months before screening.
Exclusion Criteria
* 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 other forms of chronic liver 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 deciliter \[g/L\]).
* 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.
18 Years
75 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Takeda Development Center Americas, Inc.
INDUSTRY
Takeda
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Study Director
Role: STUDY_DIRECTOR
Takeda
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
University of Alabama at Birmingham
Birmingham, Alabama, United States
St. Joseph's Hospital and Medical Center
Phoenix, Arizona, United States
Mayo Clinic
Phoenix, Arizona, United States
University of Arizona Thomas D. Boyer Liver Institute
Tucson, Arizona, United States
Gastroenterology & Liver Institute
Escondido, California, United States
University of California San Diego, Altman Clinical and Translational Institute
La Jolla, California, United States
UCLA Pulmonary and Critical Care
Los Angeles, California, United States
Stanford University
Palo Alto, California, United States
University of California Benioff Children's Hospital
San Francisco, California, United States
University of Florida
Gainesville, Florida, United States
Schiff Center for Liver Diseases/University of Miami
Miami, Florida, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Indiana University School of Medicine - Indianapolis
Indianapolis, Indiana, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States
University of Maryland Medical Center
Baltimore, Maryland, United States
Brigham and Womens Hospital
Boston, Massachusetts, United States
Boston Medical Center
Boston, Massachusetts, United States
University of Michigan Hospital
Ann Arbor, Michigan, United States
Henry Ford Medical Center - Columbus
Novi, Michigan, United States
Mayo Clinic - PPDS
Rochester, Minnesota, United States
Cardinal Glennon Children's Hospital
St Louis, Missouri, United States
Washington University School of Medicine in St. Louis
St Louis, Missouri, United States
NYU Langone Health
New York, New York, United States
Morgan Stanley Childrens Hospital of New York Presbyterian (CHONY) - PIN
New York, New York, United States
Columbia University Irving Medical Center
New York, New York, United States
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States
Temple University Hospital
Philadelphia, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Texoma Liver Center
Denison, Texas, United States
Baylor College of Medicine Medical Center
Houston, Texas, United States
The Texas Liver Institute
San Antonio, Texas, United States
Bon Secours St. Mary's Hospital
Richmond, Virginia, United States
VCU Medical Center North Hospital
Richmond, Virginia, United States
Royal Adelaide Hospital
Adelaide, South Australia, Australia
St Vincents Hospital Melbourne - PPDS
Fitzroy, Victoria, Australia
LKH-Universitätsklinikum Graz
Graz, , Austria
Medizinische Universität Innsbruck
Innsbruck, , Austria
Klinikum Klagenfurt Am Wörthersee
Klagenfurt, , Austria
Medizinische Universität Wien (Medical University of Vienna)
Vienna, , Austria
UZ Antwerpen
Antwerp, , Belgium
UZ Leuven
Leuven, , Belgium
Hospital Sirio-Libanes
São Paulo, , Brazil
Universidade Estadual Paulista Julio de Mesquita Filho Faculdade de Medicina Campus de Botucatu
São Paulo, , Brazil
GI Research Institute
Vancouver, British Columbia, Canada
Queen Elizabeth II Health Sciences Center
Halifax, Nova Scotia, Canada
Inspiration Research Limited
Toronto, Ontario, Canada
Institut klinicke a experimentalni mediciny
Prague, , Czechia
Hvidovre Hospital
Hvidovre, , Denmark
Hôpital Beaujon
Clichy, , France
Hôpital de La Croix Rousse
Lyon, , France
Centre Francois Magendie
Pessac, , France
Hopital PONTCHAILLOU CHU de Rennes
Rennes, , France
Hospital Purpan
Toulouse, , France
Hôpital Paul Brousse
Val-de-Marne, , France
Universitätsklinikum der RWTH Aachen
Aachen, , Germany
Charité - Campus Virchow-Klinikum-Ostring 1
Berlin, , Germany
Hannover Medical School
Hanover, , Germany
Universitätsklinikum Schleswig-Holstein - Campus Kiel
Kiel, , Germany
Universitätsklinikum Tübingen
Tübingen, , Germany
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
Milan, , Italy
Fondazione IRCCS Policlinico San Matteo di Pavia
Pavia, , Italy
IRCCS Istituto Clinico Humanitas
Rozzano, , Italy
Amsterdam UMC - VUmc - De Boelelaan
Amsterdam, , Netherlands
Leiden University Medical Center
Leiden, , Netherlands
ID Clinic Arkadiusz Pisula
Śląskie, , Poland
WIP Warsaw IBD Point Profesor Kierkus
Warsaw, , Poland
CCA Hospital Braga
Braga, , Portugal
Hospital Nélio Mendonça
Funchal, , Portugal
Centro Hospitalar do Porto
Porto, , Portugal
Hospital Universitario Vall d'Hebron - PPDS
Barcelona, , Spain
Hospital Universitario Virgen de la Victoria
Málaga, , Spain
Hospital Universitario Marques de Valdecilla
Santander, , Spain
Hospital Universitario Virgen del Rocio - PPDS
Seville, , Spain
Karolinska Universitetssjukhuset Huddinge
Huddinge, , Sweden
Universitetssjukhuset i Linköping
Linköping, , Sweden
Inselspital Bern
Bern, , Switzerland
Royal Infirmary of Edinburgh
Edinburgh, , United Kingdom
Royal Free Hospital
London, , United Kingdom
King's College Hospital
London, , United Kingdom
Queen's Medical Centre
Nottingham, , United Kingdom
Derriford Hospital
Plymouth, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Related Links
Access external resources that provide additional context or updates about the study.
To obtain more information on the study, click here/on this link
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2022-501943-34-00
Identifier Type: CTIS
Identifier Source: secondary_id
TAK-999-3001
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.