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Tests treatment safety and results for Alpha1-Antitrypsin Deficiency
Official title 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
ClinicalTrials.gov ID: NCT05677971
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, ADS-001.
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 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.
- Phase 3: a large, late-stage study
- 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
- The participant must have a diagnosis of the Z allele homozygotes (PiZZ) genotype AATD. PiZZ diagnosis from source verifiable medical records is...
- 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...
- The participant has a pulmonary status meeting the protocol's requirements.
You likely can't join if
- The participant has a history of liver decompensating events (overt hepatic encephalopathy [West Haven Grade \>=2] documented by a physician or...
- The participant has a history of the presence of medium or large varices or varices with red wale signs based on a previous...
- The participant has evidence of chronic liver disease attributable to other diseases, including viral hepatitis B or C, primary biliary cholangitis...
- 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]).
See the full eligibility 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...
- 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...
- 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...
- 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.
- 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...
- 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...
- 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...
- 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...
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- Phoenix, Arizona, United States
- Tucson, Arizona, United States
- Escondido, California, United States
- La Jolla, California, United States
- Los Angeles, California, United States
- Palo Alto, California, United States
- San Francisco, California, United States
- Colorado Springs, Colorado, United States
- Gainesville, Florida, United States
- Miami, Florida, United States
- Atlanta, Georgia, United States
- Indianapolis, Indiana, United States
- Iowa City, Iowa, United States
- New Orleans, Louisiana, United States
- Baltimore, Maryland, United States
- Boston, Massachusetts, United States
- Ann Arbor, Michigan, United States
- Novi, Michigan, United States
- Rochester, Minnesota, United States
+ 61 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 listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to 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 Birmingham, Alabama, United States; Phoenix, Arizona, United States; Tucson, Arizona, United States; Escondido, California, United States; La Jolla, California, United States; Los Angeles, California, United States and 75 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.