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Tests treatment safety and results for Alpha1-Antitrypsin Deficiency
Official title 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)
ClinicalTrials.gov ID: NCT06165341
What this study is testing
What is Fazirsiran Injection?
Fazirsiran Injection is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for alpha1-antitrypsin deficiency.
Also referred to as TAK-999, ARO-AAT.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The liver produces a protein called alpha-1 antitrypsin (AAT). AAT is normally released into the bloodstream.
- Phase 3: a large, late-stage study
- Time commitment: about 2 years
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 75
You may be able to join if
- In the opinion of the investigator, the participant is capable of understanding and fully complying with the protocol requirements and adhering to...
- 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...
- 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...
You likely can't join if
- 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.
See the full eligibility criteria
Who can 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...
- The participant's liver biopsy core samples collected as per protocol requirements.
- 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...
- 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...
- 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...
- 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...
What rules you out
- 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.
- 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...
- 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...
- 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...
- 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 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...
- 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...
- 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 [%])...
- 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...
- 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...
- 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...
- 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...
- 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...
The study team makes the final eligibility decision.
Where it's taking place
- Phoenix, Arizona, United States
- Tucson, Arizona, United States
- La Jolla, California, United States
- Los Angeles, California, United States
- San Francisco, California, United States
- Colorado Springs, Colorado, United States
- Miami, Florida, United States
- Indianapolis, Indiana, United States
- Iowa City, Iowa, United States
- Boston, Massachusetts, United States
- Ann Arbor, Michigan, United States
- Novi, Michigan, United States
- Rochester, Minnesota, United States
- New York, New York, United States
- Cleveland, Ohio, United States
- Hershey, Pennsylvania, United States
- San Antonio, Texas, United States
- Newport, Virginia, United States
- Graz, Austria
- Klagenfurt, Austria
+ 19 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 2 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Phoenix, Arizona, United States; Tucson, Arizona, United States; La Jolla, California, United States; Los Angeles, California, United States; San Francisco, California, United States; Colorado Springs, Colorado, United States and 33 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.