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RecruitingPhase 3

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

Takeda · with Takeda Development Center Americas, Inc. · NCT05677971

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Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 18–75 · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 3
Target enrollment
160
US sites recruiting
33
Study start
March 6, 2023
Record last updated
May 13, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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.

Being studied: Fazirsiran Injection; Placebo

Conditions: Alpha1-Antitrypsin Deficiency

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full criteria

Inclusion criteria

  • The participant must have a diagnosis of the Z allele homozygotes (PiZZ) genotype AATD. PiZZ diagnosis from source verifiable medical records is permitted. Otherwise, participants must undergo PiZZ confirmatory testing (genotyping for PiS and PiZ alleles) at screening. PiMZ or PiSZ genotypes are not permitted.
  • The participant, of any sex, is aged 18 to 75 years, inclusive.
  • The participant's liver biopsy core sample collected should meet the requirements of the protocol.
  • The participant has evidence of METAVIR stage F2, F3, or F4 liver fibrosis, evaluated by a centrally read baseline liver biopsy during the screening period; or confirmed as meeting all the entry criteria by central reading of a previous biopsy conducted within 6 months before the estimated enrollment date using an adequate liver biopsy and slides as defined in the study laboratory manual.
  • The participant has a pulmonary status meeting the protocol's requirements.
  • It must be confirmed that the participant does not have HCC. Participants will be screened for HCC with alpha-fetoprotein (AFP) and abdominal ultrasound. If the participant has any of the following, they will be required to have contrast-enhanced CT or MRI imaging to exclude HCC before randomization.
  • An adult participant must have a body mass index (BMI) greater than or equal to (>=) 18.0 kilograms per meter square (kg\^m2).
  • The participant is a nonsmoker for at least 6 months before screening.

Exclusion Criteria

  • The participant has a history of liver decompensating events (overt hepatic encephalopathy [West Haven Grade >=2] documented by a physician or healthcare professional, clinically significant ascites, spontaneous bacterial peritonitis, GI bleeding from varices, hepatopulmonary syndrome, hepatorenal syndrome, portal pulmonary hypertension, or bleeding portal hypertensive gastropathy).
  • The participant has a history of the presence of medium or large varices or varices with red wale signs based on a previous esophagogastroduodenoscopy (EGD) within 6 months before the estimated enrollment date. For certain participants, an EGD will be required at screening if there is no EGD available within 6 months before the estimated enrollment date. Presence of small varices with no red wale signs on EGD and no history of bleeding will be acceptable for study eligibility.
  • The participant has evidence of chronic liver disease attributable to other diseases, including viral hepatitis B or C, primary biliary cholangitis, primary sclerosing cholangitis, Wilson disease, alcoholic hepatitis, hemochromatosis, liver cancer, history of biliary diversion, or autoimmune hepatitis.
  • The participant has alanine transaminase (ALT) or aspartate transaminase (AST) levels >250 units per liter (U/L).
  • The participant has a platelet count \<60,000 per cubic millimeter (mm\^3) (\<60 × 10\^9 per liter [10\^9/L]).
  • The participant has albumin \<=2.8 gram per deciliter (g/dL) (28 grams per liter [g/L]).
  • The participant has international normalized ratio (INR) >=1.7.
  • The participant is expected to have severe and unavoidable high-level exposure to inhaled pulmonary toxins during the study such as may occur with occupational exposure to mineral dusts or metals.
  • The participant has a history of drug abuse (such as cocaine, phencyclidine) within 1 year before the screening visit or has a positive urine drug screen at screening.
  • The participant has previously been treated with fazirsiran or any other RNAi for AATD-LD.
  • The participant has portal vein thrombosis.
  • The participant has a prior transjugular portosystemic shunt procedure.
  • The participant has a history of malignancy within the last 5 years, except for adequately treated basal cell carcinoma, squamous cell skin cancer, superficial bladder tumors, or in situ cervical cancer. Participants with other curatively treated malignancies who have no evidence of metastatic disease and a greater than 1-year disease-free interval may be entered after approval by the medical monitor.

Recruiting US locations

  • University of Alabama at Birmingham

    Birmingham, AL 35233

    Site contact: Site Contact · 205-934-9999 · grayme@uab.edu

  • St. Joseph's Hospital and Medical Center

    Phoenix, AZ 85013

    Site contact: Site Contact · 602-406-7791 · nilofar.najafian@commonspirit.org

  • Mayo Clinic

    Phoenix, AZ 85054-4502

    Site contact: Site Contact · 507-284-9523 · vargas.hugo@mayo.edu

  • University of California San Diego, Altman Clinical and Translational Institute

    La Jolla, CA 92093

    Site contact: Site Contact · 858-657-7076 · roloomba@health.ucsd.edu

  • UCLA Pulmonary and Critical Care

    Los Angeles, CA 90095-8344

    Site contact: Site Contact · 310-825-6170 · ibarjaktarevic@mednet.ucla.edu

  • Stanford University

    Palo Alto, CA 94303

    Site contact: Site Contact · 650-723-6381 · pkwo@stanford.edu

  • University of California Benioff Children's Hospital

    San Francisco, CA 94143

    Site contact: Site Contact · 415-353-7052 · PROSENTH@ucsf.edu

  • Peak Gastroenterology Associates, PC

    Colorado Springs, CO 80907

    Site contact: Site Contact · 719-310-6719 · bpatel@peakgastro.com

  • University of Florida

    Gainesville, FL 32611

    Site contact: Site Contact · 352-294-5152 · briana.foerman@medicine.ufl.edu

  • Schiff Center for Liver Diseases/University of Miami

    Miami, FL 33136-1051

    Site contact: Site Contact · 305-243-1020 · eschiff@med.miami.edu

  • Indiana University School of Medicine - Indianapolis

    Indianapolis, IN 46202

    Site contact: Site Contact · 317-278-6215 · rvuppala@iu.edu; mandcruz@iu.edu

  • University of Iowa Hospitals and Clinics

    Iowa City, IA 52242

    Site contact: Site Contact · 319-356-2577 · tomohiro-tanaka@uiowa.edu

  • Ochsner Medical Center

    New Orleans, LA 70121-2429

    Site contact: Site Contact · 504-842-3925 · gtherapondos@ochsner.org

  • University of Maryland Medical Center

    Baltimore, MD 21201-1504

    Site contact: Site Contact · 410-328-1358 · kshetty@som.umaryland.edu

  • Brigham and Womens Hospital

    Boston, MA 02115

    Site contact: Site Contact · 617-525-1267 · NHASHEMI@BWH.HARVARD.EDU

  • Boston Medical Center

    Boston, MA 02118-2908

    Site contact: Site Contact · 617-638-8000 · amohanty@bu.edu

  • University of Michigan Hospital

    Ann Arbor, MI 48109

    Site contact: Site Contact · 734-232-3741 · rfontana@med.umich.edu

  • Henry Ford Medical Center - Columbus

    Novi, MI 48377-3600

    Site contact: Site Contact · 248-344-6688 · sgordon3@hfhs.org

  • Mayo Clinic - PPDS

    Rochester, MN 55905-0001

    Site contact: Site Contact · 507-284-0686 · malhi.harmeet@mayo.edu

  • Cardinal Glennon Children's Hospital

    St Louis, MO 63104-1003

    Site contact: Site Contact · 314-577-5633 · jeffrey.teckman@slucare.ssmhealth.com

  • Washington University School of Medicine in St. Louis

    St Louis, MO 63110

    Site contact: Site Contact · 314-454-8025 · smeduchenry@wustl.edu

  • NYU Langone Health

    New York, NY 10016

    Site contact: Site Contact · 212-263-3643 · viviana.figueroadiaz@nyulangone.org

  • Columbia University Irving Medical Center

    New York, NY 10032-3722

    Site contact: Site Contact · 212-305-2862 · mpg2124@cumc.columbia.edu

  • University Hospitals Cleveland Medical Center

    Cleveland, OH 44106-1716

    Site contact: Site Contact · 216-983-0879 · seth.sclair@uhhospitals.org

  • Penn State Health Milton S. Hershey Medical Center

    Hershey, PA 17033

    Site contact: Site Contact · 717-531-6525 · tcraig@pennstatehealth.psu.edu

  • Temple University Hospital

    Philadelphia, PA 19140-5103

    Site contact: Site Contact · 215-707-5067 · yedidya.saiman@tuhs.temple.edu

  • Medical University of South Carolina

    Charleston, SC 29425

    Site contact: Site Contact · 843-792-5300 · strangec@musc.edu

  • Vanderbilt University Medical Center

    Nashville, TN 37232-0028

    Site contact: Site Contact · 615-322-8748 · roman.perri@vumc.org

  • Texoma Liver Center

    Denison, TX 75020

    Site contact: Site Contact · 903-957-0417 · h.elgouhari@somacinicaltrials.com

  • Baylor College of Medicine Medical Center

    Houston, TX 77030-4202

    Site contact: Site Contact · 832-355-1400 · george.cholankeril@bcm.edu

  • The Texas Liver Institute

    San Antonio, TX 78215

    Site contact: Site Contact · 210-253-3426 · lawitz@txliver.com

  • Bon Secours St. Mary's Hospital

    Richmond, VA 23226

    Site contact: Site Contact · 804-977-8920 · mitchell_shiffman@bshsi.org

  • VCU Medical Center North Hospital

    Richmond, VA 23298-5028

    Site contact: Site Contact · 212-824-7587 · amon.asgharpour@vcuhealth.org

Study contacts (from the official record)

  • Takeda Contact · 1-877-825-3327 · medinfoUS@takeda.com
Study to Check the Safety of Fazirsiran and Learn if Fazirsiran… (NCT05677971) | Bored Studies