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
Start Date
3/6/2023
Completion Date
12/12/2031
Summary
The main aim of this study is to learn if fazirsiran reduces liver scarring (fibrosis) compared to placebo. Other aims are to learn if fazirsiran slows down the disease worsening in the liver, to get information on how fazirsiran affects the body (called pharmacodynamics), to learn if fazirsiran reduces other liver injury (inflammation) and the abnormal Z-AAT protein in the liver, to get information on how the body processes fazirsiran (called pharmacokinetics), to test how well fazirsiran works compared with a placebo in improving measures of liver scarring including imaging and liver biomarkers (substances in the blood that the body normally makes and help show if liver function is improving, staying the same, or getting worse) as well as to check for side effects in participants treated with fazirsiran compared with those who received placebo. 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.
Eligibility Criteria
Age Range: 18 years to 75 years
Interventions
Fazirsiran Injection
Placebo
Conditions
Locations
University of Alabama at Birmingham
Birmingham, Alabama 35233
United States
St. Joseph's Hospital and Medical Center
Phoenix, Arizona 85013
United States
Mayo Clinic
Phoenix, Arizona 85054-4502
United States
University of Arizona Thomas D. Boyer Liver Institute
Tucson, Arizona 85724-5136
United States
Gastroenterology & Liver Institute
Escondido, California 92025
United States
University of California San Diego, Altman Clinical and Translational Institute
La Jolla, California 92093
United States
UCLA Pulmonary and Critical Care
Los Angeles, California 90095-8344
United States
Stanford University
Palo Alto, California 94303
United States
University of California Benioff Children's Hospital
San Francisco, California 94143
United States
Peak Gastroenterology Associates, PC
Colorado Springs, Colorado 80907
United States
University of Florida
Gainesville, Florida 32611
United States
Schiff Center for Liver Diseases/University of Miami
Miami, Florida 33136-1051
United States
Children's Healthcare of Atlanta
Atlanta, Georgia 30329
United States
Indiana University School of Medicine - Indianapolis
Indianapolis, Indiana 46202
United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa 52242
United States
Ochsner Medical Center
New Orleans, Louisiana 70121-2429
United States
University of Maryland Medical Center
Baltimore, Maryland 21201-1504
United States
Brigham and Womens Hospital
Boston, Massachusetts 02115
United States
Boston Medical Center
Boston, Massachusetts 02118-2908
United States
University of Michigan Hospital
Ann Arbor, Michigan 48109
United States
Henry Ford Medical Center - Columbus
Novi, Michigan 48377-3600
United States
Mayo Clinic - PPDS
Rochester, Minnesota 55905-0001
United States
Cardinal Glennon Children's Hospital
St Louis, Missouri 63104-1003
United States
Washington University School of Medicine in St. Louis
St Louis, Missouri 63110
United States
NYU Langone Health
New York, New York 10016
United States
Morgan Stanley Childrens Hospital of New York Presbyterian (CHONY) - PIN
New York, New York 10032-1559
United States
Columbia University Irving Medical Center
New York, New York 10032-3722
United States
University Hospitals Cleveland Medical Center
Cleveland, Ohio 44106-1716
United States
Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania 17033
United States
Temple University Hospital
Philadelphia, Pennsylvania 19140-5103
United States
Medical University of South Carolina
Charleston, South Carolina 29425
United States
Vanderbilt University Medical Center
Nashville, Tennessee 37232-0028
United States
Texoma Liver Center
Denison, Texas 75020
United States
Baylor College of Medicine Medical Center
Houston, Texas 77030-4202
United States
The Texas Liver Institute
San Antonio, Texas 78215
United States
Bon Secours St. Mary's Hospital
Richmond, Virginia 23226
United States
VCU Medical Center North Hospital
Richmond, Virginia 23298-5028
United States
Royal Adelaide Hospital
Adelaide, South Australia 5000
Australia
St Vincents Hospital Melbourne - PPDS
Fitzroy, Victoria 3065
Australia
LKH-Universitätsklinikum Graz
Graz, 8036
Austria
Medizinische Universität Innsbruck
Innsbruck, 6020
Austria
Klinikum Klagenfurt Am Wörthersee
Klagenfurt, 9020
Austria
Medizinische Universität Wien (Medical University of Vienna)
Vienna, 1090
Austria
UZ Antwerpen
Antwerp, 2650
Belgium
UZ Leuven
Leuven, 3000
Belgium
Hospital de Base do Distrito Federal
Brasília, 71692-000
Brazil
Hospital Sirio-Libanes
São Paulo, 01308-050
Brazil
Universidade Estadual Paulista Julio de Mesquita Filho Faculdade de Medicina Campus de Botucatu
São Paulo, 18618-687
Brazil
GI Research Institute
Vancouver, British Columbia V6Z 2K5
Canada
Queen Elizabeth II Health Sciences Center
Halifax, Nova Scotia B3H 2Y9
Canada
Inspiration Research Limited
Toronto, Ontario M5T 3A9
Canada
Institut klinicke a experimentalni mediciny
Prague, 140 21
Czechia
Hvidovre Hospital
Hvidovre, 2650
Denmark
Hôpital Beaujon
Clichy, 92110
France
Hôpital de La Croix Rousse
Lyon, 69317
France
Centre Francois Magendie
Pessac, 33064
France
Hopital PONTCHAILLOU CHU de Rennes
Rennes, 35000
France
Hospital Purpan
Toulouse, 31000
France
Hôpital Paul Brousse
Val-de-Marne, 94805
France
Universitätsklinikum der RWTH Aachen
Aachen, 52074
Germany
Charité - Campus Virchow-Klinikum-Ostring 1
Berlin, 13353
Germany
Hannover Medical School
Hanover, 30625
Germany
Universitätsklinikum Schleswig-Holstein - Campus Kiel
Kiel, 24105
Germany
Universitätsklinikum Tübingen
Tübingen, 72076
Germany
Beaumont Hospital
Dublin, D09 YD60
Ireland
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
Milan, 20122
Italy
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
Palermo, 90127
Italy
Fondazione IRCCS Policlinico San Matteo di Pavia
Pavia, 27100
Italy
IRCCS Istituto Clinico Humanitas
Rozzano, 20089
Italy
Amsterdam UMC - VUmc - De Boelelaan
Amsterdam, 1081 HV
Netherlands
Leiden University Medical Center
Leiden, 2333 ZA
Netherlands
ID Clinic Arkadiusz Pisula
Śląskie, 41-400
Poland
WIP Warsaw IBD Point Profesor Kierkus
Warsaw, 00-728
Poland
CCA Hospital Braga
Braga, 4710-243
Portugal
Hospital Nélio Mendonça
Funchal, 9000-168
Portugal
Centro Hospitalar do Porto
Porto, 4099-003
Portugal
Hospital Universitario Vall d'Hebron - PPDS
Barcelona, 8025
Spain
Hospital Universitario Virgen de la Victoria
Málaga, 29071
Spain
Hospital Universitario Marques de Valdecilla
Santander, 39008
Spain
Hospital Universitario Virgen del Rocio - PPDS
Seville, 41013
Spain
Karolinska Universitetssjukhuset Huddinge
Huddinge, 14186
Sweden
Universitetssjukhuset i Linköping
Linköping, 581 85
Sweden
Inselspital Bern
Bern, 3010
Switzerland
Addenbrooke's Hospital
Cambridge, CB2 0QQ
United Kingdom
Royal Infirmary of Edinburgh
Edinburgh, EH16 4SA
United Kingdom
Royal Free Hospital
London, NW3 2Q
United Kingdom
King's College Hospital
London, SE5 9RS
United Kingdom
Queen's Medical Centre
Nottingham, NG7 2UH
United Kingdom
Derriford Hospital
Plymouth, PL6 8DH
United Kingdom