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Alpha1-Antitrypsin DeficiencyPhase 3NCT06165341

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)

A Phase 3 study testing Fazirsiran Injection in people with Alpha1-Antitrypsin Deficiency, run by Takeda at 41 sites in 13 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

The liver produces a protein called alpha-1 antitrypsin (AAT). AAT is normally released into the bloodstream. In some people, the liver makes an abnormal version of the AAT protein, called Z-AAT. Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Alpha1-Antitrypsin Deficiency
What is being tested?
Fazirsiran Injection
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
50 participants
Where
41 sitesin 13 countries
Study dates
1 Mar 2024to 26 Aug 2028
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • In the opinion of the investigator, the participant is capable of understanding and fully complying with the protocol requirements and adhering to the protocol schedule.
  • The participant is able to read, understand, and complete the study questionnaires electronically per the investigator's judgment.
  • The participant signs and dates a written Informed Consent Form (ICF). Any required privacy authorization should also be signed before the initiation of any study procedures.
  • The participant, of any sex, is aged 18 to 75 years, inclusive.
  • The participant must have a diagnosis of the protease inhibitor Z mutation (PiZZ) genotype AATD. A diagnosis of PiZZ 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's liver biopsy core samples collected as per protocol requirements.
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  • The participant has evidence of METAVIR stage F1 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 from a previous biopsy conducted within 1 year 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 that meets the protocol requirements.
  • It must be confirmed that the participant does not have hepatocellular carcinoma (HCC).
  • Any participant who is taking statins, angiotensin-converting enzyme inhibitors, angiotensin II receptor blockers, or beta-1 selective adrenergic receptor inhibitors must have been receiving a stable dose of these medications for at least 8 weeks before randomization. All attempts are to be made for the participant to continue the same dose of the medication for the duration of study participation.
  • An adult participant must have a body mass index (BMI) between 18 and 39 kilogram per meter square (kg/m^2), inclusive.
  • The participant has a 12-lead electrocardiogram at screening that, in the opinion of the investigator, has no abnormalities that could compromise the participant's safety in this study.
  • The participant is a nonsmoker.
  • If the participant was being treated with any respiratory medications including inhaled bronchodilators, inhaled anticholinergics, inhaled corticosteroids, or low-dose systemic corticosteroids (prednisone less than or equal to [<=10] milligrams per day [mg/d] or its equivalent), the doses of the participant's medications must have remained unchanged for greater than or equal to (>=) 4 weeks before screening.
  • The participant must have suitable venous access for blood sampling.
  • A person of childbearing potential (POCBP) must have a negative serum pregnancy test at screening and a negative urine pregnancy test on Day 1 before dosing.
  • The participant must use appropriate contraception methods (that is, highly effective methods for female and medically appropriate methods for male study participants) for the entire duration of the study and for 6 months after the last dose of study medication. The participant must not donate sperm for at least 6 months after the last dose of study medication.

Who cannot join

  • The participant has evidence of >= F2 fibrosis based on liver biopsy during the screening period.
  • The participant has a history of liver decompensating events.
  • The participant has a history of varices based on a previous esophagogastroduodenoscopy.
  • The participant has portal vein thrombosis.
  • The participant has undergone a prior trans-jugular portosystemic shunt procedure.
  • The participant has evidence of other forms of chronic liver diseases.
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  • 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 curatively treated malignancies who have no evidence of metastatic disease and disease-free interval greater than (>) 1 year may be enrolled after approval by the medical monitor.
  • The participant has an abnormal finding of clinical relevance at the screening evaluation and before administration of the first dose of study dosing that, in the opinion of the investigator, could adversely impact participant safety during the study or adversely impact study results.
  • The participant has any laboratory abnormalities at screening and before the first dose of the study drug that meet protocol parameters.
  • 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 recent lower respiratory tract infection, such as pneumonia, within the last 6 months before screening. The participant may be screened earlier based on principal investigator (PI) assessment of clinical recovery and return to baseline pulmonary function in discussion with the medical monitor.
  • The participant has a history of frequent pulmonary exacerbations (>=2 moderate or severe exacerbations within 52 weeks before screening).
  • The participant is experiencing a pulmonary exacerbation at the time of screening (participant may be rescreened after the clinical resolution of an exacerbation).
  • The participant is receiving long-term, around-the-clock oxygen supplementation or supplemental oxygen with continuous positive airway pressure (CPAP) or bilevel positive airway pressure for acute respiratory failure. The following conditions are allowable for the participant to enter screening: short-term use of oxygen supplementation (example, for the management of acute chronic obstructive pulmonary disease [COPD] exacerbation) or CPAP for obstructive sleep apnea.
  • The participant has human immunodeficiency virus (HIV) infection as shown by the presence of anti-HIV antibody (seropositive).
  • The participant is seropositive for hepatitis B virus (HBV surface antigen positive and/or HBV core antibody positive without HBV surface antibody at screening) or hepatitis C virus (HCV) (detectable HCV Ribonucleic Acid [RNA] at screening). Cured HCV (positive antibody test without detectable HCV RNA for at least 6 months after treatment) is acceptable.
  • The participant has unstable, poorly controlled, or severe hypertension. Participants may be rescreened once their blood pressure (BP) is successfully controlled.
  • The participant has a history of torsades de pointes, ventricular rhythm disturbances (example, ventricular tachycardia), heart block (excluding first-degree block, being PR interval prolongation only), congenital long QT syndrome or new ST-segment elevation or depression or a new Q wave on ECG. Participants with a history of atrial arrhythmias should be discussed with the medical monitor.
  • The participant has symptomatic heart failure (per New York Heart Association guidelines), unstable angina, myocardial infarction, severe cardiovascular disease (ejection fraction less than [<] 20 percent [%]), transient ischemic attack, or cerebrovascular accident within 6 months before screening.
  • The participant has a history of major surgery within 12 weeks of screening (or longer, at the discretion of the investigator).
  • The participant has a history of more than moderate alcohol consumption within 12 months before the screening visit.
  • 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 RNA interference (RNAi) for alpha-1 antitrypsin deficiency-associated liver disease (AATD-LD).
  • The participant has a history of hypersensitivity or allergies with any associated excipients of fazirsiran.
  • The participant has received an investigational agent or device within 30 days, or 5 half-lives, whichever is longer, before the dosing of study medication or is currently participating in an investigational study involving a therapeutic intervention.
  • The participant has donated >=500 milliliter (mL) of blood within 1 month of the administration of study treatment.
  • The participant has any concomitant medical or psychiatric condition or social situation that would make it difficult to comply with protocol requirements or put the participant at additional safety risk. The participant has a history of clinically significant hematologic, renal, hepatic, pulmonary, neurologic, psychiatric, gastrointestinal (GI), systemic inflammatory, metabolic, or endocrine disorder or any other condition that, in the opinion of the investigator, rendered the participant a poor candidate for inclusion into the study.
  • The participant has a history of thromboembolic disease (including deep vein thrombosis or pulmonary embolism), within 6 months before screening, or is taking chronic anticoagulants.
  • This participant is unable to return for all scheduled study visits.
  • The participant has known or suspected coronavirus disease 2019 (COVID-19) that, in the opinion of the sponsor and investigator, does not resolve during screening. Positive antibody testing for COVID-19 without other evidence of current or recent active infection does not exclude participation. Enrollment of participants who fail inclusion due to COVID-19 infection may be temporarily delayed at the discretion of the sponsor and investigator. If the participant has a positive polymerase chain reaction (PCR) with no other evidence of infection, a retest may be allowed; however, to enroll in the study the participant must have a negative PCR.
  • The participant is a study site employee involved in the conduct of this study, an immediate family member (example, spouse, parent, child, sibling), is in a dependent relationship with study site employee who is involved in the conduct of this study or may consent under duress.
  • The participant takes or is required to take excluded medications.
  • The participant is pregnant or breastfeeding or intending to become pregnant before participating in this study, during the study, or within 6 months after last dose of the study drug; or the participant is intending to donate ova during such time period.

Trial locations and contacts

41 sites in 13 countries. Contact the study team or a site near you to ask about taking part.

Takeda Contact contact

+1-877-825-3327

medinfoUS@takeda.com

41 sites in 13 countries

United States20 sites, 20 recruiting

  • University of Michigan Hospital - 1500 E Medical Center Dr

    Ann Arbor, Michigan, 48109

    Site Contact, 734-232-3741

    Robert Fontana

    Recruiting

  • Boston Medical Center

    Boston, Massachusetts, 02118-2335

    Site Contact, 617-638-6525, arpan.mohanty@bmc.org

    Arpan Mohanty

    Recruiting

  • University Hospitals Cleveland Medical Center

    Cleveland, Ohio, 44106-1716

    Site Contact, 216-983-0879

    Seth Sclair

    Recruiting

  • Peak Gastroenterology Associates

    Colorado Springs, Colorado, 80907

    Site Contact, 719-636-1201

    Bhaktasharan Patel

    Recruiting

  • Penn State Health Milton S. Hershey Medical Center

    Hershey, Pennsylvania, 17033-2360

    Site Contact, 717-531-7532, tcraig@pennstatehealth.psu.edu

    Timothy Craig

    Recruiting

  • Indiana University School of Medicine-Indianapolis

    Indianapolis, Indiana, 46202-2266

    Site Contact, 317-278-4607

    Raj Vuppalanchi

    Recruiting

  • University Of Iowa Hospitals And Clinics

    Iowa City, Iowa, 52242-1009

    Site Contact, 319-356-2577, Tomohiro-tanaka@uiowa.edu

    Tomohiro Tanaka

    Recruiting

  • University of California San Diego

    La Jolla, California, 92037-1337

    Site Contact, 858-534-2555, roloomba@health.ucsd.edu

    Rohit Loomba

    Recruiting

  • UCLA Pulmonary and Critical Care

    Los Angeles, California, 90095-3075

    Site Contact, 310-825-8061

    Igor Barjaktarevic

    Recruiting

  • Schiff Center for Liver Diseases/University of Miami

    Miami, Florida, 33136

    Site Contact, 305-243-5185, idsresearch@med.miami.edu

    Eugene Schiff

    Recruiting

  • NYU Langone Medical Center

    New York, New York, 10016-6402

    Site Contact, 212-263-3643, viviana.figueroadiaz@nyumc.org

    Viviana Figueroa Diaz

    Recruiting

  • Columbia University Irving Medical Center

    New York, New York, 10032-3722

    Monica Goldklang

    Recruiting

  • Bon Secours St. Mary's Hospital

    Newport, Virginia, 23602-4414

    Site Contact, 804-977-8920, mitchell_shiffman@bshsi.org

    Mitchell Schiffman

    Recruiting

  • Henry Ford Health System

    Novi, Michigan, 48377-3600

    Site Contact, 248-344-6688, sgordon3@hfhs.org

    Stuart Gordon

    Recruiting

  • St Joseph's Hospital and Medical Center

    Phoenix, Arizona, 85013-4224

    Site Contact, 602-274-7195

    Justin Reynolds

    Recruiting

  • Mayo Clinic - PPDS

    Phoenix, Arizona, 85054-4502

    Site Contact, 480-301-6990, vargas.hugo@mayo.edu

    Hugo Vargas

    Recruiting

  • Mayo Clinic PPDS

    Rochester, Minnesota, 55905

    Site Contact, 507-284-0141

    Harmeet Malhi

    Recruiting

  • Texas Liver Institute American Research Corporation

    San Antonio, Texas, 78215

    Site Contact, 210-253-3426, lawitz@txliver.com

    Eric Lawitz

    Recruiting

  • University of California Benioff Children's Hospital

    San Francisco, California, 94143-2203

    Site Contact, 415-476-5892, PROSENTH@ucsf.edu

    Philip Rosenthal

    Recruiting

  • University of Arizona Thomas D. Boyer Liver Institute

    Tucson, Arizona, 85724-0001

    Site Contact, 520-626-3005, gdblock@deptofmed.arizona.edu

    Geoffrey Block

    Recruiting

Other trials in United States

Germany4 sites, 4 recruiting

  • Universitätsklinikum der RWTH Aachen

    Aachen, 52074

    +492418035324, pstrnad@ukaachen.de

    Pavel Strnad

    Recruiting

  • Charité - Campus Virchow-Klinikum

    Berlin, 13353

    Site Contact, +4939450553022, frank.tacke@charite.de

    Frank Tacke

    Recruiting

  • Hannover Medical School

    Hanover, 30625

    Site Contact, +495115322730

    Heiner Wedemeyer

    Recruiting

  • Universitätsklinikum Tübingen

    Tübingen, 72076

    Site Contact, +4970712977661

    Christoph Berg

    Recruiting

Other trials in Germany

France3 sites, 3 recruiting

  • Hôpital de La Croix Rousse

    Lyon, 69317

    Site contact, +33145596790, teresa.antonini@aphp.fr

    Teresa Antonini

    Recruiting

  • Hopital PONTCHAILLOU CHU de Rennes

    Rennes, 35000

    Site Contact, +33299284297, edouard.bardou-jacquet@chu-rennes.fr

    Edouard Bardou-Jacquet

    Recruiting

  • Hôpital Paul Brousse

    Val-de-Marne, 94800

    Site Contact, +33145593336, audrey.coilly@aphp.fr

    Audrey Coilly

    Recruiting

Other trials in France

Portugal3 sites, 3 recruiting

  • CCA Hospital Braga

    Braga, 4710-243

    Carla Rolanda

    Recruiting

  • Hospital Dr. Nélio Mendonça

    Funchal, 9000-168

    Site Contact, (351) 291-7056 x00

    Vitor Pereira

    Recruiting

  • Centro Hospitalar de Universitário de Santo António E.P.E

    Porto, 4099-001

    Site Contact, +351918000000

    Luís Maia

    Recruiting

Other trials in Portugal

Austria2 sites, 2 recruiting

  • LKH-Universitätsklinikum Graz

    Graz, 8036

    Site Contact, +4331638580287, martin.wagner@medunigraz.at

    Martin Wagner

    Recruiting

  • KABEG - Klinikum Klagenfurt Am Wörthersee

    Klagenfurt, 9020

    Markus Peck-Radosavljevic

    Recruiting

Other trials in Austria

Belgium2 sites, 2 recruiting

  • UZ Antwerpen

    Antwerp, 2650

    Site Contact, +3238213323, sven.francque@uza.be

    Sven Francque

    Recruiting

  • UZ Leuven

    Leuven, 3000

    Site Contact, +328888888888, jef.verbeek@uzleuven.be

    Jef Verbeek

    Recruiting

Other trials in Belgium

Canada1 site, 1 recruiting

  • Inspiration Research Limited

    Toronto, Ontario, M5T 3A9

    Site Contact, (416) 944-9602

    Kenneth Chapman

    Recruiting

Other trials in Canada

Italy1 site, 1 recruiting

  • Fondazione IRCCS Policlinico San Matteo

    Pavia, 27100

    Site Contact, +390382503777

    Angelo Corsico

    Recruiting

Other trials in Italy

Poland1 site

  • ID Clinic Arkadiusz Pisula

    Mysłowice, 41-400

    Withdrawn

Other trials in Poland

Spain1 site, 1 recruiting

  • Hospital Universitario Virgen del Rocio - PPDS

    Seville, 41018

    Site Contact, +34955012065, mromerogomez@us.es

    Manuel Romero Gómez

    Recruiting

Other trials in Spain

Sweden1 site, 1 recruiting

  • Karolinska Universitetssjukhuset Huddinge

    Huddinge, 14186

    Site Contact, +46858580000

    Staffan Wahlin

    Recruiting

Other trials in Sweden

Switzerland1 site, 1 recruiting

  • Universitätsspital Bern

    Bern, 3010

    Site Contact, +41442663568

    Guido Stirnimann

    Recruiting

Other trials in Switzerland

United Kingdom1 site, 1 recruiting

  • King's College Hospital

    London, SE5 9RS

    Site Contact, +442032993506

    Aftab Ala

    Recruiting

Other trials in United Kingdom

About this study

Official title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Safety and Efficacy of Fazirsiran in the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease With METAVIR Stage F1 Fibrosis

The liver produces a protein called alpha-1 antitrypsin (AAT). AAT is normally released into the bloodstream. In some people, the liver makes an abnormal version of the AAT protein, called Z-AAT. Making an abnormal version of the AAT protein can result in liver disease as Z-AAT builds up in liver cells, which leads to liver problems such as liver scarring (fibrosis), continuing liver damage (cirrhosis), and eventually end stage liver disease. Fazirsiran is a medicine that reduces the creation of the Z-AAT protein and thus the build-up of this abnormal protein in the liver. People with this type of liver disease who already have mild liver scarring will take part in the study. They will be treated with fazirsiran or a placebo for about 2 years. This study will check the long-term safety of fazirsiran, whether participants tolerate the treatment and if there are any effects on liver scarring. A liver biopsy, a way of collecting a small tissue sample from the liver, will be taken twice during the study.

Being tested
Fazirsiran Injection, Placebo
Medical subject headings
alpha 1-Antitrypsin Deficiency

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Fazirsiran InjectionDrug

    Also called TAK-999, ARO-AAT, ADS-001

    Fazirsiran will be injected subcutaneously.

  • PlaceboDrug

    Fazirsiran matching placebo.

Study groups in NCT06165341
Study groupRoleWhat they receive
Fazirsiran 200 mgExperimentalParticipants will receive fazirsiran 200 milligrams (mg), injection, subcutaneously on Day 1, at Week 4 and then every 12 weeks (Q12W) for up to Week 100.
Receives: Drug: Fazirsiran Injection
PlaceboPlacebo comparatorParticipants will receive fazirsiran matching placebo injection, subcutaneously on Day 1, at Week 4 and Q12W for up to Week 100.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants With Adverse Events (AEs) and Serious AEs (SAEs)

    An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of the study intervention, whether or not it is considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of the study intervention. An SAE is defined as any untoward medical occurrence that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, congenital anomaly/birth defect, suspected transmission of any infectious agent, an important medical event. AEs and SAEs including any pulmonary AEs or SAEs indicative of worsening pulmonary condition (example, pulmonary exacerbation, respiratory infection, significant pulmonary function test decline) will be reported.

    Measured: From start of study drug administration up to End of study (EOS) (Week 124)

  2. Number of Participants With Clinically Significant Change From Baseline in Pulmonary Function Parameters

    Standard pulmonary function parameters will be used to study lung function. Clinical significance of pulmonary function parameters will be determined at the investigator's discretion.

    Measured: From start of study drug administration up to EOS (Week 124)

  3. Change From Baseline in Whole Lung 15th Percentile Density as Measured by Computed Tomography (CT) Lung Densitometry

    Change from baseline in whole lung 15th percentile density as measured by CT lung densitometry will be assessed.

    Measured: Baseline up to Week 100

  4. Number of Participants With Clinically Significant Changes in Vital Signs

    Vital signs include body temperature, respiratory rate, blood pressure (systolic and diastolic), pulse (beats per minute) and pulse oximetry. Clinical significance of vital signs will be determined at the investigator's discretion.

    Measured: From start of study drug administration up to EOS (Week 124)

  5. Number of Participants with Clinically Significant Changes in Electrocardiogram (ECG) Parameters

    12-lead ECG will be evaluated. Any clinically significant change in ECG assessments will be determined at the investigator's discretion.

    Measured: From start of study drug administration up to EOS (Week 124)

  6. Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters

    Laboratory parameters assessments include hematology, biochemistry including liver tests, coagulation, and urinalysis. Clinical significance of laboratory parameters will be determined at the investigator's discretion.

    Measured: From start of study drug administration up to EOS (Week 124)

Secondary outcomes (9)

  1. Change From Baseline in Serum Z-AAT Protein Over Time to Week 106

    Change From Baseline in Serum Z-AAT Protein will be assessed.

    Measured: Baseline up to Week 106

  2. Percent Change From Baseline in Intrahepatic Liver Z-AAT Protein at Week 106

    Percent change from baseline in intrahepatic liver Z-AAT protein will be assessed.

    Measured: Baseline up to Week 106

  3. Change From Baseline in Intrahepatic Z-AAT Protein Polymer Burden Assessed by Periodic Acid Schiff Plus Diastase (PAS+D) Staining in Liver Biopsy at Week 106

    Change from baseline in intrahepatic Z-AAT protein polymer burden assessed by PAS+D staining in liver biopsy will be assessed.

    Measured: Baseline up to Week 106

  4. Number of Participants with No Change or Decrease From Baseline in Intrahepatic Z-AAT Protein Polymer Burden Assessed by PAS+D staining at Week 106

    Number of participants with no change or decrease from baseline in intrahepatic Z-AAT protein polymer burden assessed by PAS+D staining will be reported.

    Measured: Baseline up to Week 106

  5. Change From Baseline in Intrahepatic Portal Inflammation Score in Liver Biopsy at Week 106

    Change in portal inflammation score in liver biopsy is based on pathology slide reads. Inflammation will be assessed on a scale of 0-3, with higher scores showing more severe inflammation.

    Measured: Baseline up to Week 106

  6. Number of Participants with No Change or Decrease From Baseline in Intrahepatic Portal Inflammation Score at Week 106

    No Change or decrease in portal inflammation score is based on pathology slide reads. Inflammation will be assessed on a scale of 0-3, with higher scores showing more severe inflammation. Number of Participants with no change or decrease from baseline in intrahepatic portal inflammation score will be reported.

    Measured: Baseline up to Week 106

  7. Number of Participants With No Change or Decrease From Baseline in Histologic Fibrosis Score (Meta-analysis of Histological Data in Viral Hepatitis [METAVIR] Staging ) at Week 106

    No change or decrease in histologic fibrosis (METAVIR staging) will be assessed using a fibrosis score of F0-F4, where higher scores mean a worse result. Number of participants with no change or decrease from baseline in histologic fibrosis score (by [METAVIR] staging) at Week 106 will be reported.

    Measured: Baseline up to Week 106

  8. Change From Baseline in Vibration-Controlled Transient Elastography (VCTE)-Derived Liver Stiffness

    Change from baseline in VCTE-derived liver stiffness will be assessed.

    Measured: Baseline up to Week 106

  9. Change From Baseline in Markers of Liver Injury

    Change from baseline in marker (alanine aminotransferase [ALT], aspartate aminotransferase [AST], glutamyl transferase [GGT]) of liver injury will be assessed.

    Measured: Baseline up to Week 106

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Dec 2023
  2. Study start1 Mar 2024
  3. Primary completion26 Aug 2028 (estimated)
  4. Study completion26 Aug 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
50 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06165341
Sponsor's protocol ID
TAK-999-3002
Other IDs
2023-504198-19-00, 2023
Status last verified
Mar 2026
First submitted
1 Dec 2023
Last updated
5 Mar 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Fazirsiran Injection in people with Alpha1-Antitrypsin Deficiency, run by Takeda at 41 sites in 13 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 17 inclusion and 33 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 5 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 41 sites in United States, Germany, France, Portugal, Austria and 8 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Takeda Contact (+1-877-825-3327, medinfoUS@takeda.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Adverse Events (AEs) and Serious AEs (SAEs) (time frame: From start of study drug administration up to End of study (EOS) (Week 124)). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06165341.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Mar 2026.

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