Actively Recruiting
A Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Fazirsiran in Adults With Alpha-1 Antitrypsin Deficiency-Associated Liver Disease and Fibrosis
Led by Takeda · Updated on 2026-05-13
160
Participants Needed
89
Research Sites
151 weeks
Total Duration
On this page
Sponsors
T
Takeda
Lead Sponsor
T
Takeda Development Center Americas, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating fazirsiran, an investigational drug, to see if it can reduce liver scarring (fibrosis) and slow disease progression in people with liver disease caused by an abnormal alpha-1 antitrypsin protein. The study compares fazirsiran to a placebo and aims to understand how the drug affects the body, including its impact on liver inflammation and abnormal protein levels. Liver biopsies will be taken twice to assess changes in liver scarring. Participants will receive either fazirsiran or a placebo through subcutaneous injections. The dosing schedule includes an injection on Day 1, at Week 4, and then every 12 weeks up to Week 196. The study is randomized and double-blind, meaning neither participants nor researchers know who receives the drug or placebo during the treatment period. During the study, participants will undergo liver biopsies and various assessments including imaging techniques and blood tests to monitor liver function, protein levels, liver stiffness, and inflammation. Researchers will also track side effects, lung function, vital signs, and laboratory results from the start of treatment through up to Week 230. The main focus is on measuring changes in liver fibrosis after 106 weeks, with ongoing safety and efficacy evaluations throughout the study.
CONDITIONS
Brief Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of Z allele homozygotes (PiZZ) genotype Alpha-1 Antitrypsin Deficiency confirmed by medical records or screening test
- Aged 18 to 75 years, any sex
- Liver biopsy sample meeting protocol requirements
- Evidence of METAVIR stage F2, F3, or F4 liver fibrosis by centrally read biopsy within 6 months
- Pulmonary status meeting protocol requirements
- No evidence of hepatocellular carcinoma (HCC) confirmed by screening tests
- Body mass index (BMI) of 18.0 kg/m2 or higher
- Nonsmoker for at least 6 months before screening
You will not qualify if you...
- History of liver decompensating events such as encephalopathy, ascites, or gastrointestinal bleeding
- History or presence of medium or large varices or varices with red wale signs within 6 months
- Chronic liver disease from other causes like viral hepatitis B or C, autoimmune hepatitis, or alcoholic hepatitis
- Alanine transaminase (ALT) or aspartate transaminase (AST) levels above 250 U/L
- Platelet count below 60,000 per mm3
- Albumin level less than or equal to 2.8 g/dL
- International normalized ratio (INR) of 1.7 or higher
- Expected severe exposure to inhaled pulmonary toxins during the study
- History of drug abuse within 1 year or positive drug screen at screening
- Previous treatment with fazirsiran or RNAi therapies for this condition
- Portal vein thrombosis
- Prior transjugular portosystemic shunt procedure
- History of malignancy within 5 years except certain treated skin or bladder cancers and approved curative cases without metastasis
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 196 weeks
Participants receive subcutaneous injections of either fazirsiran or placebo on Day 1, at Week 4, and then every 12 weeks thereafter up to Week 196.
Multiple visits on Day 1, Week 4, and every 12 weeks up to Week 196
Duration - Up to Week 230
Participants are monitored for safety and long-term effects after the treatment period ends.
Visits continue through end of study up to Week 230
Trial Site Locations
Total: 89 locations
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
St. Joseph's Hospital and Medical Center
Phoenix, Arizona, United States, 85013
Actively Recruiting
3
Mayo Clinic
Phoenix, Arizona, United States, 85054-4502
Actively Recruiting
4
University of Arizona Thomas D. Boyer Liver Institute
Tucson, Arizona, United States, 85724-5136
Withdrawn
5
Gastroenterology & Liver Institute
Escondido, California, United States, 92025
Withdrawn
6
University of California San Diego, Altman Clinical and Translational Institute
La Jolla, California, United States, 92093
Actively Recruiting
7
UCLA Pulmonary and Critical Care
Los Angeles, California, United States, 90095-8344
Actively Recruiting
8
Stanford University
Palo Alto, California, United States, 94303
Actively Recruiting
9
University of California Benioff Children's Hospital
San Francisco, California, United States, 94143
Actively Recruiting
10
Peak Gastroenterology Associates, PC
Colorado Springs, Colorado, United States, 80907
Actively Recruiting
11
University of Florida
Gainesville, Florida, United States, 32611
Actively Recruiting
12
Schiff Center for Liver Diseases/University of Miami
Miami, Florida, United States, 33136-1051
Actively Recruiting
13
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30329
Withdrawn
14
Indiana University School of Medicine - Indianapolis
Indianapolis, Indiana, United States, 46202
Actively Recruiting
15
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States, 52242
Actively Recruiting
16
Ochsner Medical Center
New Orleans, Louisiana, United States, 70121-2429
Actively Recruiting
17
University of Maryland Medical Center
Baltimore, Maryland, United States, 21201-1504
Actively Recruiting
18
Brigham and Womens Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
19
Boston Medical Center
Boston, Massachusetts, United States, 02118-2908
Actively Recruiting
20
University of Michigan Hospital
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
21
Henry Ford Medical Center - Columbus
Novi, Michigan, United States, 48377-3600
Actively Recruiting
22
Mayo Clinic - PPDS
Rochester, Minnesota, United States, 55905-0001
Actively Recruiting
23
Cardinal Glennon Children's Hospital
St Louis, Missouri, United States, 63104-1003
Actively Recruiting
24
Washington University School of Medicine in St. Louis
St Louis, Missouri, United States, 63110
Actively Recruiting
25
NYU Langone Health
New York, New York, United States, 10016
Actively Recruiting
26
Morgan Stanley Childrens Hospital of New York Presbyterian (CHONY) - PIN
New York, New York, United States, 10032-1559
Withdrawn
27
Columbia University Irving Medical Center
New York, New York, United States, 10032-3722
Actively Recruiting
28
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States, 44106-1716
Actively Recruiting
29
Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 17033
Actively Recruiting
30
Temple University Hospital
Philadelphia, Pennsylvania, United States, 19140-5103
Actively Recruiting
31
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Actively Recruiting
32
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232-0028
Actively Recruiting
33
Texoma Liver Center
Denison, Texas, United States, 75020
Actively Recruiting
34
Baylor College of Medicine Medical Center
Houston, Texas, United States, 77030-4202
Actively Recruiting
35
The Texas Liver Institute
San Antonio, Texas, United States, 78215
Actively Recruiting
36
Bon Secours St. Mary's Hospital
Richmond, Virginia, United States, 23226
Actively Recruiting
37
VCU Medical Center North Hospital
Richmond, Virginia, United States, 23298-5028
Actively Recruiting
38
Royal Adelaide Hospital
Adelaide, South Australia, Australia, 5000
Withdrawn
39
St Vincents Hospital Melbourne - PPDS
Fitzroy, Victoria, Australia, 3065
Actively Recruiting
40
LKH-Universitätsklinikum Graz
Graz, Austria, 8036
Actively Recruiting
41
Medizinische Universität Innsbruck
Innsbruck, Austria, 6020
Actively Recruiting
42
Klinikum Klagenfurt Am Wörthersee
Klagenfurt, Austria, 9020
Actively Recruiting
43
Medizinische Universität Wien (Medical University of Vienna)
Vienna, Austria, 1090
Actively Recruiting
44
UZ Antwerpen
Antwerp, Belgium, 2650
Actively Recruiting
45
UZ Leuven
Leuven, Belgium, 3000
Actively Recruiting
46
Hospital de Base do Distrito Federal
Brasília, Brazil, 71692-000
Actively Recruiting
47
Hospital Sirio-Libanes
São Paulo, Brazil, 01308-050
Actively Recruiting
48
Universidade Estadual Paulista Julio de Mesquita Filho Faculdade de Medicina Campus de Botucatu
São Paulo, Brazil, 18618-687
Actively Recruiting
49
GI Research Institute
Vancouver, British Columbia, Canada, V6Z 2K5
Actively Recruiting
50
Queen Elizabeth II Health Sciences Center
Halifax, Nova Scotia, Canada, B3H 2Y9
Actively Recruiting
51
Inspiration Research Limited
Toronto, Ontario, Canada, M5T 3A9
Actively Recruiting
52
Institut klinicke a experimentalni mediciny
Prague, Czechia, 140 21
Actively Recruiting
53
Hvidovre Hospital
Hvidovre, Denmark, 2650
Actively Recruiting
54
Hôpital Beaujon
Clichy, France, 92110
Actively Recruiting
55
Hôpital de La Croix Rousse
Lyon, France, 69317
Actively Recruiting
56
Centre Francois Magendie
Pessac, France, 33064
Actively Recruiting
57
Hopital PONTCHAILLOU CHU de Rennes
Rennes, France, 35000
Actively Recruiting
58
Hospital Purpan
Toulouse, France, 31000
Actively Recruiting
59
Hôpital Paul Brousse
Val-de-Marne, France, 94805
Actively Recruiting
60
Universitätsklinikum der RWTH Aachen
Aachen, Germany, 52074
Actively Recruiting
61
Charité - Campus Virchow-Klinikum-Ostring 1
Berlin, Germany, 13353
Actively Recruiting
62
Hannover Medical School
Hanover, Germany, 30625
Actively Recruiting
63
Universitätsklinikum Schleswig-Holstein - Campus Kiel
Kiel, Germany, 24105
Actively Recruiting
64
Universitätsklinikum Tübingen
Tübingen, Germany, 72076
Actively Recruiting
65
Beaumont Hospital
Dublin, Ireland, D09 YD60
Actively Recruiting
66
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
Milan, Italy, 20122
Actively Recruiting
67
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
Palermo, Italy, 90127
Actively Recruiting
68
Fondazione IRCCS Policlinico San Matteo di Pavia
Pavia, Italy, 27100
Actively Recruiting
69
IRCCS Istituto Clinico Humanitas
Rozzano, Italy, 20089
Actively Recruiting
70
Amsterdam UMC - VUmc - De Boelelaan
Amsterdam, Netherlands, 1081 HV
Withdrawn
71
Leiden University Medical Center
Leiden, Netherlands, 2333 ZA
Actively Recruiting
72
ID Clinic Arkadiusz Pisula
Śląskie, Poland, 41-400
Withdrawn
73
WIP Warsaw IBD Point Profesor Kierkus
Warsaw, Poland, 00-728
Actively Recruiting
74
CCA Hospital Braga
Braga, Portugal, 4710-243
Actively Recruiting
75
Hospital Nélio Mendonça
Funchal, Portugal, 9000-168
Actively Recruiting
76
Centro Hospitalar do Porto
Porto, Portugal, 4099-003
Actively Recruiting
77
Hospital Universitario Vall d'Hebron - PPDS
Barcelona, Spain, 8025
Actively Recruiting
78
Hospital Universitario Virgen de la Victoria
Málaga, Spain, 29071
Actively Recruiting
79
Hospital Universitario Marques de Valdecilla
Santander, Spain, 39008
Actively Recruiting
80
Hospital Universitario Virgen del Rocio - PPDS
Seville, Spain, 41013
Actively Recruiting
81
Karolinska Universitetssjukhuset Huddinge
Huddinge, Sweden, 14186
Actively Recruiting
82
Universitetssjukhuset i Linköping
Linköping, Sweden, 581 85
Actively Recruiting
83
Inselspital Bern
Bern, Switzerland, 3010
Actively Recruiting
84
Addenbrooke's Hospital
Cambridge, United Kingdom, CB2 0QQ
Actively Recruiting
85
Royal Infirmary of Edinburgh
Edinburgh, United Kingdom, EH16 4SA
Actively Recruiting
86
Royal Free Hospital
London, United Kingdom, NW3 2Q
Actively Recruiting
87
King's College Hospital
London, United Kingdom, SE5 9RS
Actively Recruiting
88
Queen's Medical Centre
Nottingham, United Kingdom, NG7 2UH
Withdrawn
89
Derriford Hospital
Plymouth, United Kingdom, PL6 8DH
Actively Recruiting
Research Team
T
Takeda Contact
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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