Long-Term Open-Label Study of Acoramidis Effects on Heart Function and Amyloid Burden in Adults With Transthyretin Amyloidosis Cardiomyopathy

What this Trial Is About

Researchers are evaluating the long-term effects of acoramidis on heart function and structure in adults aged 18 to 80 with transthyretin amyloidosis cardiomyopathy ATTR-CM. This study focuses on assessing how acoramidis impacts cardiac amyloid burden and improvements using cardiac MRI and echocardiogram imaging techniques over time. All participants will receive acoramidis 712 mg orally twice daily, equivalent to 800 mg of acoramidis hydrochloride for 36 months in an open-label setting. The study includes up to approximately 150 participants and lasts about 38 months per person, including up to 45 days for screening, 36 months of treatment, and a 1-month follow-up after the last dose. Clinic visits occur at Day 1, Day 28, Month 6, and every 6 months up to Month 36, with phone visits at Month 3 and every 6 months until Month 33. Participants will be followed throughout treatment and for 30 days after the last dose. Assessments include cardiac imaging to monitor heart structure and amyloid buildup, safety monitoring, and evaluation of cardiac function improvements by cardiac MRI at Month 36. The research team will also evaluate various cardiac parameters and amyloid burden to understand acoramidis effects over the study period.

Cardiac Magnetic Resonance and Echocardiography Study With Acoramidis