A Study to Evaluate Acoramidis Treatment Effects on Heart Biomarkers and Symptoms in Adults With Transthyretin-type Cardiac Amyloidosis

What this Trial Is About

Researchers are evaluating the effects of acoramidis in people with transthyretin-type cardiac amyloidosis ATTR-CM, a condition affecting the heart. The study aims to confirm whether acoramidis can prevent the worsening of disease progression markers and assess how these markers reflect disease status. This phase 4 post-marketing study is sponsored by Alexion Pharmaceuticals, Inc. and focuses on real-life treatment outcomes. Participants will take 800 mg of acoramidis twice daily for 18 months. This single-arm, open-label study involves monitoring changes in cardiac biomarkers and clinical features related to ATTR-CM. The study does not include a placebo group, and all participants receive the same treatment throughout the study period. During the study, participants will undergo regular assessments including blood tests for markers like NT-proBNP and high-sensitivity cardiac troponin T, kidney function tests, and questionnaires measuring heart-related quality of life. Researchers will track disease progression over 12 months and follow participants up to 18 months. Safety and treatment effects will be closely monitored throughout this time.

Real-Life Clinical Efficacy of Acoramidis in Participants With ATTR-CM and Association With Cardiac Biomarkers