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
Condition(s) studied
Investigational drug(s) / intervention(s)
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.
Study 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
Primary outcome measure(s)
- Reduction From Baseline of at Least 1 Stage of Histologic Fibrosis (METAVIR Staging) in the Centrally Read Liver Biopsy at Week 106 in AATD-LD With METAVIR Stage F2 and F3 Fibrosis — Baseline, Week 106
Reduction from baseline of at least 1 stage of histologic fibrosis METAVIR staging in the centrally read liver biopsy at Week 106 in AATD-LD with METAVIR stage F2 and F3 fibrosis will be assessed.
Trial sites (89)
| Facility | City | Region | Status |
|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | Recruiting |
| St. Joseph's Hospital and Medical Center | Phoenix | Arizona | Recruiting |
| Mayo Clinic | Phoenix | Arizona | Recruiting |
| University of Arizona Thomas D. Boyer Liver Institute | Tucson | Arizona | Withdrawn |
| Gastroenterology & Liver Institute | Escondido | California | Withdrawn |
| University of California San Diego, Altman Clinical and Translational Institute | La Jolla | California | Recruiting |
| UCLA Pulmonary and Critical Care | Los Angeles | California | Recruiting |
| Stanford University | Palo Alto | California | Recruiting |
| University of California Benioff Children's Hospital | San Francisco | California | Recruiting |
| Peak Gastroenterology Associates, PC | Colorado Springs | Colorado | Recruiting |
| University of Florida | Gainesville | Florida | Recruiting |
| Schiff Center for Liver Diseases/University of Miami | Miami | Florida | Recruiting |
| Children's Healthcare of Atlanta | Atlanta | Georgia | Withdrawn |
| Indiana University School of Medicine - Indianapolis | Indianapolis | Indiana | Recruiting |
| University of Iowa Hospitals and Clinics | Iowa City | Iowa | Recruiting |
| Ochsner Medical Center | New Orleans | Louisiana | Recruiting |
| University of Maryland Medical Center | Baltimore | Maryland | Recruiting |
| Brigham and Womens Hospital | Boston | Massachusetts | Recruiting |
| Boston Medical Center | Boston | Massachusetts | Recruiting |
| University of Michigan Hospital | Ann Arbor | Michigan | Recruiting |
| Henry Ford Medical Center - Columbus | Novi | Michigan | Recruiting |
| Mayo Clinic - PPDS | Rochester | Minnesota | Recruiting |
| Cardinal Glennon Children's Hospital | St Louis | Missouri | Recruiting |
| Washington University School of Medicine in St. Louis | St Louis | Missouri | Recruiting |
| NYU Langone Health | New York | New York | Recruiting |
| Morgan Stanley Childrens Hospital of New York Presbyterian (CHONY) - PIN | New York | New York | Withdrawn |
| Columbia University Irving Medical Center | New York | New York | Recruiting |
| University Hospitals Cleveland Medical Center | Cleveland | Ohio | Recruiting |
| Penn State Health Milton S. Hershey Medical Center | Hershey | Pennsylvania | Recruiting |
| Temple University Hospital | Philadelphia | Pennsylvania | Recruiting |
| Medical University of South Carolina | Charleston | South Carolina | Recruiting |
| Vanderbilt University Medical Center | Nashville | Tennessee | Recruiting |
| Texoma Liver Center | Denison | Texas | Recruiting |
| Baylor College of Medicine Medical Center | Houston | Texas | Recruiting |
| The Texas Liver Institute | San Antonio | Texas | Recruiting |
| Bon Secours St. Mary's Hospital | Richmond | Virginia | Recruiting |
| VCU Medical Center North Hospital | Richmond | Virginia | Recruiting |
| Royal Adelaide Hospital | Adelaide | South Australia | Withdrawn |
| St Vincents Hospital Melbourne - PPDS | Fitzroy | Victoria | Recruiting |
| LKH-Universitätsklinikum Graz | Graz | Austria | Recruiting |
+ 49 more sites — see the full list on the official registry below.
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Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05677971 on ClinicalTrials.gov ↗ ← All trials in France