Authorised Therapeutic confirmatory (Phase III) Alpha-1 Antitrypsin Deficiency-Associated Liver Disease

An Open-Label Extension Study of Fazirsiran

EU CTIS ID: 2023-503497-21-00

What this study is testing

The primary objective of the study is to evaluate the safety and tolerability of the long-term treatment of fazirsiran in participants with AATD-LD.

  • 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

  • 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. -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 be enrolled into the OLE study. Note: If the first dose of fazirsiran in the OLE study will be delayed by more than 24 weeks after the last dose in AROAAT2001 or AROAAT2002, the rollover should be discussed with the medical monitor.

You likely can't join if

  • 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 cholangitis, primary sclerosing cholangitis, Wilson disease, alcoholic hepatitis, hemochromatosis, liver cancer, history of biliary diversion, or autoimmune hepatitis. 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 intervention, 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 (that is, highly effective methods for female and medically appropriate methods for male study participants) through the conclusion of study participation.

The study team makes the final eligibility decision.

Where it's taking place

  • 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, 18-64 years, 65+ years. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include 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.