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)

Phase 1

Recruiting

• Conditions: Alpha-1 Antitrypsin Deficiency-Associated Liver Disease; MedDRA version: 23.1Level: LLTClassification code: 10001806Term: Alpha-1 anti-trypsin deficiency Class: 10010331; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: CTIS2023-504198-19-00
• Lead Sponsor: Takeda Development Center Americas Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-11-27
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

1. The participant must have a diagnosis of the PiZZ genotype AATD., 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 screening period using an adequate liver biopsy and slides as defined in the study laboratory manual., 4. The participant has a pulmonary status meeting the protocol's requirements.

Exclusion Criteria

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, clinically significant ascites, spontaneous bacterial peritonitis, gastrointestinal (GI) bleeding from varices, hepatopulmonary syndrome, hepatorenal syndrome, portal pulmonary hypertension, or portal 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., 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 24 weeks before screening., 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.

Study & Design

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