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 (NCT05677971)
A Randomized, Double-blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Fazirsiran in the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease With METAVIR Stage F2 to F4 Fibrosis
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: University of Alabama at Birmingham — Birmingham, Alabama
Age range: 18 Years to 75 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (89 Sites)
Showing the first 25 of 89 sites.
- University of Alabama at Birmingham — Birmingham, Alabama
- St. Joseph's Hospital and Medical Center — Phoenix, Arizona
- Mayo Clinic — Phoenix, Arizona
- University of Arizona Thomas D. Boyer Liver Institute — Tucson, Arizona
- Gastroenterology & Liver Institute — Escondido, California
- University of California San Diego, Altman Clinical and Translational Institute — La Jolla, California
- UCLA Pulmonary and Critical Care — Los Angeles, California
- Stanford University — Palo Alto, California
- University of California Benioff Children's Hospital — San Francisco, California
- Peak Gastroenterology Associates, PC — Colorado Springs, Colorado
- University of Florida — Gainesville, Florida
- Schiff Center for Liver Diseases/University of Miami — Miami, Florida
- Children's Healthcare of Atlanta — Atlanta, Georgia
- Indiana University School of Medicine - Indianapolis — Indianapolis, Indiana
- University of Iowa Hospitals and Clinics — Iowa City, Iowa
- Ochsner Medical Center — New Orleans, Louisiana
- University of Maryland Medical Center — Baltimore, Maryland
- Brigham and Womens Hospital — Boston, Massachusetts
- Boston Medical Center — Boston, Massachusetts
- University of Michigan Hospital — Ann Arbor, Michigan
- Henry Ford Medical Center - Columbus — Novi, Michigan
- Mayo Clinic - PPDS — Rochester, Minnesota
- Cardinal Glennon Children's Hospital — St Louis, Missouri
- Washington University School of Medicine in St. Louis — St Louis, Missouri
- NYU Langone Health — New York, New York
📞 Contact the Study Coordinator
Phone: (877) 825-3327
Email: medinfoUS@takeda.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Alpha1-Antitrypsin Deficiency
- An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease — La Jolla, California
- A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems — Gainesville, Florida
- Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis) — Phoenix, Arizona
Explore by condition: All Alpha1-Antitrypsin Deficiency clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.