Breakthrough trial aims to reverse liver damage in genetic disorder
NCT ID NCT05677971
Summary
This study is testing whether an investigational drug called fazirsiran can reduce liver scarring (fibrosis) in people with Alpha-1 antitrypsin deficiency, a genetic condition that can damage the liver and lungs. About 160 adults with moderate to advanced liver scarring will receive either fazirsiran or a placebo injection, and researchers will compare liver biopsy results after two years. The main goal is to see if the drug can improve liver health by reducing the buildup of abnormal protein and slowing disease progression.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Addenbrooke's Hospital
RECRUITINGCambridge, CB2 0QQ, United Kingdom
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Amsterdam UMC - VUmc - De Boelelaan
WITHDRAWNAmsterdam, 1081 HV, Netherlands
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Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
RECRUITINGPalermo, 90127, Italy
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Baylor College of Medicine Medical Center
RECRUITINGHouston, Texas, 77030-4202, United States
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Beaumont Hospital
RECRUITINGDublin, D09 YD60, Ireland
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Bon Secours St. Mary's Hospital
RECRUITINGRichmond, Virginia, 23226, United States
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Boston Medical Center
RECRUITINGBoston, Massachusetts, 02118-2908, United States
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Brigham and Womens Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
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CCA Hospital Braga
RECRUITINGBraga, 4710-243, Portugal
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Cardinal Glennon Children's Hospital
RECRUITINGSt Louis, Missouri, 63104-1003, United States
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Centre Francois Magendie
RECRUITINGPessac, 33064, France
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Centro Hospitalar do Porto
RECRUITINGPorto, 4099-003, Portugal
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Charité - Campus Virchow-Klinikum-Ostring 1
RECRUITINGBerlin, 13353, Germany
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Children's Healthcare of Atlanta
WITHDRAWNAtlanta, Georgia, 30329, United States
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Columbia University Irving Medical Center
RECRUITINGNew York, New York, 10032-3722, United States
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Derriford Hospital
RECRUITINGPlymouth, PL6 8DH, United Kingdom
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Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
RECRUITINGMilan, 20122, Italy
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Fondazione IRCCS Policlinico San Matteo di Pavia
RECRUITINGPavia, 27100, Italy
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GI Research Institute
RECRUITINGVancouver, British Columbia, V6Z 2K5, Canada
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Gastroenterology & Liver Institute
WITHDRAWNEscondido, California, 92025, United States
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Hannover Medical School
RECRUITINGHanover, 30625, Germany
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Henry Ford Medical Center - Columbus
RECRUITINGNovi, Michigan, 48377-3600, United States
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Hopital PONTCHAILLOU CHU de Rennes
RECRUITINGRennes, 35000, France
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Hospital Nélio Mendonça
RECRUITINGFunchal, 9000-168, Portugal
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Hospital Purpan
RECRUITINGToulouse, 31000, France
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Hospital Sirio-Libanes
RECRUITINGSão Paulo, 01308-050, Brazil
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Hospital Universitario Marques de Valdecilla
RECRUITINGSantander, 39008, Spain
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Hospital Universitario Vall d'Hebron - PPDS
RECRUITINGBarcelona, 8025, Spain
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Hospital Universitario Virgen de la Victoria
RECRUITINGMálaga, 29071, Spain
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Hospital Universitario Virgen del Rocio - PPDS
RECRUITINGSeville, 41013, Spain
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Hospital de Base do Distrito Federal
RECRUITINGBrasília, 71692-000, Brazil
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Hvidovre Hospital
RECRUITINGHvidovre, 2650, Denmark
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Hôpital Beaujon
RECRUITINGClichy, 92110, France
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Hôpital Paul Brousse
RECRUITINGVal-de-Marne, 94805, France
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Hôpital de La Croix Rousse
RECRUITINGLyon, 69317, France
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ID Clinic Arkadiusz Pisula
WITHDRAWNŚląskie, 41-400, Poland
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IRCCS Istituto Clinico Humanitas
RECRUITINGRozzano, 20089, Italy
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Indiana University School of Medicine - Indianapolis
RECRUITINGIndianapolis, Indiana, 46202, United States
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Inselspital Bern
RECRUITINGBern, 3010, Switzerland
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Inspiration Research Limited
RECRUITINGToronto, Ontario, M5T 3A9, Canada
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Institut klinicke a experimentalni mediciny
RECRUITINGPrague, 140 21, Czechia
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Karolinska Universitetssjukhuset Huddinge
RECRUITINGHuddinge, 14186, Sweden
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King's College Hospital
RECRUITINGLondon, SE5 9RS, United Kingdom
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Klinikum Klagenfurt Am Wörthersee
RECRUITINGKlagenfurt, 9020, Austria
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LKH-Universitätsklinikum Graz
RECRUITINGGraz, 8036, Austria
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Leiden University Medical Center
RECRUITINGLeiden, 2333 ZA, Netherlands
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Mayo Clinic
RECRUITINGPhoenix, Arizona, 85054-4502, United States
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Mayo Clinic - PPDS
RECRUITINGRochester, Minnesota, 55905-0001, United States
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Medical University of South Carolina
RECRUITINGCharleston, South Carolina, 29425, United States
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Medizinische Universität Innsbruck
RECRUITINGInnsbruck, 6020, Austria
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Medizinische Universität Wien (Medical University of Vienna)
RECRUITINGVienna, 1090, Austria
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Morgan Stanley Childrens Hospital of New York Presbyterian (CHONY) - PIN
WITHDRAWNNew York, New York, 10032-1559, United States
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NYU Langone Health
RECRUITINGNew York, New York, 10016, United States
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Ochsner Medical Center
RECRUITINGNew Orleans, Louisiana, 70121-2429, United States
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Peak Gastroenterology Associates, PC
RECRUITINGColorado Springs, Colorado, 80907, United States
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Penn State Health Milton S. Hershey Medical Center
RECRUITINGHershey, Pennsylvania, 17033, United States
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Queen Elizabeth II Health Sciences Center
RECRUITINGHalifax, Nova Scotia, B3H 2Y9, Canada
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Queen's Medical Centre
WITHDRAWNNottingham, NG7 2UH, United Kingdom
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Royal Adelaide Hospital
WITHDRAWNAdelaide, South Australia, 5000, Australia
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Royal Free Hospital
RECRUITINGLondon, NW3 2Q, United Kingdom
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Royal Infirmary of Edinburgh
RECRUITINGEdinburgh, EH16 4SA, United Kingdom
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Schiff Center for Liver Diseases/University of Miami
RECRUITINGMiami, Florida, 33136-1051, United States
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St Vincents Hospital Melbourne - PPDS
RECRUITINGFitzroy, Victoria, 3065, Australia
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St. Joseph's Hospital and Medical Center
RECRUITINGPhoenix, Arizona, 85013, United States
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Stanford University
RECRUITINGPalo Alto, California, 94303, United States
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Temple University Hospital
RECRUITINGPhiladelphia, Pennsylvania, 19140-5103, United States
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Texoma Liver Center
RECRUITINGDenison, Texas, 75020, United States
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The Texas Liver Institute
RECRUITINGSan Antonio, Texas, 78215, United States
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UCLA Pulmonary and Critical Care
RECRUITINGLos Angeles, California, 90095-8344, United States
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UZ Antwerpen
RECRUITINGAntwerp, 2650, Belgium
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UZ Leuven
RECRUITINGLeuven, 3000, Belgium
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Universidade Estadual Paulista Julio de Mesquita Filho Faculdade de Medicina Campus de Botucatu
RECRUITINGSão Paulo, 18618-687, Brazil
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Universitetssjukhuset i Linköping
RECRUITINGLinköping, 581 85, Sweden
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University Hospitals Cleveland Medical Center
RECRUITINGCleveland, Ohio, 44106-1716, United States
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University of Alabama at Birmingham
RECRUITINGBirmingham, Alabama, 35233, United States
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University of Arizona Thomas D. Boyer Liver Institute
WITHDRAWNTucson, Arizona, 85724-5136, United States
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University of California Benioff Children's Hospital
RECRUITINGSan Francisco, California, 94143, United States
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University of California San Diego, Altman Clinical and Translational Institute
RECRUITINGLa Jolla, California, 92093, United States
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University of Florida
RECRUITINGGainesville, Florida, 32611, United States
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University of Iowa Hospitals and Clinics
RECRUITINGIowa City, Iowa, 52242, United States
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University of Maryland Medical Center
RECRUITINGBaltimore, Maryland, 21201-1504, United States
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University of Michigan Hospital
RECRUITINGAnn Arbor, Michigan, 48109, United States
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Universitätsklinikum Schleswig-Holstein - Campus Kiel
RECRUITINGKiel, 24105, Germany
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Universitätsklinikum Tübingen
RECRUITINGTübingen, 72076, Germany
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Universitätsklinikum der RWTH Aachen
RECRUITINGAachen, 52074, Germany
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VCU Medical Center North Hospital
RECRUITINGRichmond, Virginia, 23298-5028, United States
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Vanderbilt University Medical Center
RECRUITINGNashville, Tennessee, 37232-0028, United States
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WIP Warsaw IBD Point Profesor Kierkus
RECRUITINGWarsaw, 00-728, Poland
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Washington University School of Medicine in St. Louis
RECRUITINGSt Louis, Missouri, 63110, United States
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Conditions
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