Danzhou

Researchers are evaluating the effects of nucresiran compared to a placebo in people with transthyretin amyloidosis with cardiomyopathy, a condition affecting the heart. This Phase 3 study aims to see if nucresiran can reduce death from any cause and cardiovascular events such as hospitalizations or urgent visits for heart failure. The study also looks at how the treatment impacts patients health status and quality of life. Participants are randomly assigned to receive either nucresiran 300 mg or a placebo, both administered by subcutaneous injection once every six months during the double-blind period. After this period, all participants receive nucresiran 300 mg every six months during an open-label extension. The double-blind period is expected to last about 32 months, with a maximum duration of up to 5 years. During the study, participants will have regular assessments to monitor outcomes like mortality, cardiovascular events, and patient-reported health measures using the Kansas City Cardiomyopathy Questionnaire. The primary outcome is a combined measure of all-cause mortality and recurrent cardiovascular events from the start of the study through the double-blind period. Safety and health status will be closely followed throughout the trial, which may continue until 2032.