An Open-Label Extension Study of Fazirsira

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: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: CTIS2023-503497-21-00
• Lead Sponsor: Takeda Development Center Americas Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-03-24
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

The participant enrolling in this OLE study will have participated in a previously qualifying study, and will be considered for eligibility based on the following study-specific criteria: -AROAAT2001: – Participants with fibrosis may roll over into this OLE study after they reach their next regularly scheduled, Q12W visit. – Participants with fibrosis who have completed the AROAAT2001 study may be enrolled into the OLE study if within 12 to 24 weeks of the final dose. -AROAAT2002: – Participants in Cohorts 1 and 1b may roll over after completing the 24 week primary study period. –Participants in Cohort 2 may roll over after completing the 48 week primary study period. – Participants who have completed the study may roll over within 12 to 24 weeks of the final dose.

Exclusion Criteria

1. The participant is likely to require major surgery. Major surgery typically requires at least 1 night in the hospital. Examples include laparoscopic surgery (except cholecystectomy and tubal ligation); GI tract surgery including 1 or more segments of the colon or terminal ileum; open resection of organs; large joint replacements; mastectomy with reconstruction; and spine, thoracic, vascular, or intracranial surgery. Note: participants who are awaiting a liver transplant may roll over into this study and receive treatment until the last regularly scheduled study visit, or 4 weeks before the procedure, whichever occurs later. In an urgent situation, a transplant should not be withheld due to timing of last dose. 2. 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. Participants with nonalcoholic steatohepatitis (NASH) will be allowed to enroll in the study. 3. The participant has abnormal finding(s) of clinical relevance during the evaluation before the first study dosing that, in the opinion of the investigator, could adversely impact participant safety during the study or adversely impact study results. 4. The participant had major protocol deviation(s) (as determined by the sponsor) in AROAAT2001 or AROAAT2002 that would affect the conduct of this study. 5. The participant permanently discontinued investigational product because of an AE, adjudicated as related to the study drug, in AROAAT2001 or AROAAT2002. 6. Female participants who became pregnant during Study AROAAT2001 or AROAAT2002, female participants who are lactating or planning to become pregnant during the study period; or males or female participants of childbearing potential not agreeing to continue using appropriate contraception methods (ie, highly effective methods for female and medically appropriate methods for male study participants) through the conclusion of study participation.

Study & Design

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