TAK-999-3001
A Randomized Double-Blind Placebo-Controlled Phase 3 Study to Evaluate the Efficacy and Safety of Fazirsiranin the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease with METAVIR Stage F2 to F4 Fibrosis.
- Sex at Birth: Any
- Age: Adult (18 - 64), Older Adult (65+)
- Accepting Healthy People: No
- Type: Interventional / Therapeutic
- Trial Phase: Phase III
- Conditions Being Studied: Alpha 1 Antitrypsin Deficiency
Study Purpose
The purpose of this study is to: • Test how well fazirsiran works as compared with a placebo in improving your AATD LD. • Check the safety (how many side effects you and other patients have) and tolerability (how tolerable the side effects are) of fazirsiran as compared to a placebo. • Get information on how fazirsiran moves in your body, how long it stays, and how long it takes to eliminate it, this is called pharmacokinetics or PK. • Get information on what fazirsiran does to your body, this is called pharmacodynamics or PD. • Test how well fazirsiran works as compared with a placebo in improving liver biopsy results. • Test how well fazirsiran works as compared with a placebo in improving liver biomarker results. Biomarkers are substances in blood that your body normally makes and will help show if your liver function is improving, staying the same, or getting worse.
Locations
UH Cleveland Medical Center
11100 Euclid Avenue
Cleveland OH, 44106
- UH IRB: STUDY20230061
- StudyID: 2022-0932
- ClinicalTrials.gov: NCT05677971
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