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 Protei

Phase 1

Recruiting

• Conditions: Alpha-1 Antitrypsin Deficiency-Associated Liver Disease; MedDRA version: 23.0Level: PTClassification code: 10001806Term: Alpha-1 anti-trypsin deficiency Class: 10010331; Therapeutic area: Not possible to specify; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: CTIS2022-501943-34-00
• Lead Sponsor: Takeda Development Center Americas Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-01-17
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 191

Inclusion Criteria

1. The participant must have a diagnosis of the PiZZ genotype AATD. PiZZ diagnosis from source verifiable medical records is permitted. Otherwise, participants must undergo PiZZ confirmatory testing (genotyping for PiS and protease inhibitor Z 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 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. 4. The participant has a pulmonary status meeting the criteria listed on the Protocol

Exclusion Criteria

1. The participant has a history of liver decompensating events (overt hepatic encephalopathy [West Haven Grade =2] documented by a physician, clinically significant ascites, spontaneous bacterial peritonitis, GI bleeding from varices, hepatopulmonary syndrome, hepatorenal syndrome, portal pulmonary hypertension, or portal gastropathy). 2.The participant has a history of the presence of medium or large varices or varices with no red wale signs based on a previous EGD within 6 months before the estimated enrollment date. 3. The participant has portal vein thrombosis. 4.The participant has a prior transjugular portosystemic shunt procedure. 5. The participant has evidence of other forms of chronic liver diseases, including viral hepatitis B or C, primary biliary cirrhosis, primary sclerosing cholangitis, Wilson disease, alcoholic hepatitis, hemochromatosis, liver cancer, history of biliary diversion, or autoimmune hepatitis. 6. 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. For detailed exclusion criteria, refer to the protocol.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified