A Phase 3 Study of Acoramidis to Prevent Transthyretin Amyloidosis in Adults Aged 18 to 75 With a Genetic Risk

What this Trial Is About

Transthyretin amyloidosis ATTR is a condition where the transthyretin protein breaks down and forms amyloid plaques that build up in organs, causing damage. There are two types wild-type ATTR, which occurs with aging, and variant ATTR caused by inherited defective TTR genes. This study focuses on adults who have inherited a pathogenic TTR gene variant but do not yet show symptoms, aiming to see if early treatment can prevent or delay disease development. The trial is a Phase 3, randomized, double-blind, placebo-controlled study evaluating prevention of ATTR cardiomyopathy and polyneuropathy. Participants will be randomly assigned to receive either acoramidis, a drug designed to stabilize the TTR protein and prevent amyloid formation, or a matching placebo. The study drug is taken orally twice daily at a dose equivalent to 800 mg acoramidis HCl BID. Participants will be adults aged 18 to 75 years whose age falls within 10 years younger or older than their predicted age of disease onset based on family history or actuarial tables. The trial will last approximately 7 years from randomization or until study end. During the study, participants will be monitored regularly with evaluations to detect the development of ATTR cardiomyopathy or polyneuropathy. Researchers will assess time to disease onset using centrally adjudicated criteria. Safety and adherence will be tracked throughout the trial. This long-term study will help determine if early treatment with acoramidis can delay or prevent symptoms in asymptomatic carriers of pathogenic TTR variants.

Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant