New drug could slow liver damage in rare genetic disease

NCT ID NCT05677971

Recruiting now Disease control Sponsor: Takeda Source: ClinicalTrials.gov ↗

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.

Get updates

Get notified about this study

Our safety recommendation!

By submitting, you agree to our Terms of use

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

Contacts and locations

Show contact details

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WITHDRAWN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WITHDRAWN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

More trials for these conditions

Other studies related to the condition(s) this trial covers.