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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)
EU CTIS ID: 2023-504198-19-00
What this study is testing
To evaluate the long-term safety and tolerability of fazirsiran compared with placebo, with an emphasis on lung safety, including measures of pulmonary function.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. The participant must have a diagnosis of the 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.
- 2. The participant, of any sex, is aged 18 to 75 years, inclusive.
- 3. The participant has evidence of METAVIR stage 1 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.
- 4. The participant has a pulmonary status meeting the protocol requirements
You likely can't join if
- 1. Evidence of ≥ F2 fibrosis based on liver biopsy during the screening period.
- 2. The participant has a history of liver decompensating events (overt hepatic encephalopathy documented by a physician or trained professional, clinically significant ascites, spontaneous bacterial peritonitis, gastrointestinal (GI) bleeding from varices, hepatopulmonary syndrome, hepatorenal syndrome, portal pulmonary hypertension, or bleeding portal hypertensive gastropathy).
- 3. The participant has evidence of other forms of chronic liver 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. Additional diagnosis will be allowed as per the protocol.
- 4. 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 a greater than 1 year disease-free interval may be enrolled after approval by the medical monitor.
- 5. 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.
- 6. 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.
See the full eligibility criteria
Who can join
- 1. The participant must have a diagnosis of the 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.
- 2. The participant, of any sex, is aged 18 to 75 years, inclusive.
- 3. The participant has evidence of METAVIR stage 1 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.
- 4. The participant has a pulmonary status meeting the protocol requirements
What rules you out
- 1. Evidence of ≥ F2 fibrosis based on liver biopsy during the screening period.
- 2. The participant has a history of liver decompensating events (overt hepatic encephalopathy documented by a physician or trained professional, clinically significant ascites, spontaneous bacterial peritonitis, gastrointestinal (GI) bleeding from varices, hepatopulmonary syndrome, hepatorenal syndrome, portal pulmonary hypertension, or bleeding portal hypertensive gastropathy).
- 3. The participant has evidence of other forms of chronic liver 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. Additional diagnosis will be allowed as per the protocol.
- 4. 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 a greater than 1 year disease-free interval may be enrolled after approval by the medical monitor.
- 5. 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.
- 6. 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.
- 7. The participant has a history of frequent pulmonary exacerbations (≥2 moderate or severe exacerbations within 52 weeks before screening).
- 8. The participant is experiencing a pulmonary exacerbation at the time of screening (participant may be rescreened after the clinical resolution of an exacerbation).
- 9. 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 (eg, for the management of acute chronic obstructive pulmonary disease [COPD] exacerbation) or CPAP for obstructive sleep apnea.
The study team makes the final eligibility decision.
Where it's taking place
- Switzerland
- Canada
- United States
- United Kingdom
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, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Switzerland; Canada; United States; United Kingdom. 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 EU CTIS; the study team decides eligibility.