Active — Not RecruitingPhase 3

An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease

United States31 participantsStarted 2023-08-08

Plain-language summary

The main aim of this study is to learn if fazirsiran is safe during long-term use in people with liver disease caused by the abnormal Z-alpha-1 antitrypsin (Z-AAT) protein. People who have taken part in previous fazirsiran studies (AROAAT2001 \[NCT03945292\] or AROAAT2002 \[NCT03946449\]) can continue to receive fazirsiran every 3 months as long as they participate in this study, the study is ongoing or until health authorities in their country approve fazirsiran to be publicly available. The study may also provide information on whether fazirsiran has a long-term effect in reducing liver fibrosis or slowing down the progression of liver fibrosis in people with liver disease due to the abnormal Z-AAT protein.

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Participants must meet all of the following criteria to be eligible for inclusion in the study: * The participant is willing and able to understand and fully comply with study procedures and requirements, in the opinion of the investigator. * The participant is able to read, understand, and complete the study questionnaires electronically per investigator's judgment. * The participant has provided informed consent (ICF) (that is, in writing, documented via a signed and dated ICF) and any required privacy authorization before the initiation of any study procedures. Note: For participants who comprehend, can provide informed consent, and are unable to sign the ICF, an impartial witness may sign the ICF on behalf of the participant after the participant has orally consented to the participant's participation in the study. * The participant enrolling in this open-label extension (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…

What they're measuring

Number of Participants With Adverse Events (AEs) and Serious AEs (SAEs)

Timeframe: From start of study drug administration (in current study) up to End of study (EOS) (current study [up to 10 years])

Number of Participants With Clinically Significant Changes From Baseline in Pulmonary Function Parameters

Timeframe: Baseline (current study), up to EOS (current study up to 10 years])

Number of Participants With Clinically Significant Changes in Vital Signs

Timeframe: From start of study drug administration (in current study) up to EOS (current study [up to 10 years])

Number of Participants With Clinically Significant Changes in Laboratory Parameters

Timeframe: From start of study drug administration (in current study) up to EOS (current study [up to 10 years])

Trial details

NCT IDNCT05899673

SponsorTakeda

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2033-05-02

View on ClinicalTrials.gov More Alpha1-Antitrypsin Deficiency trials