Study of Acoramidis Treatment in Adults With Transthyretin Amyloid Cardiomyopathy Previously Treated With Tafamidis Phase 4, Single-Group Study Measuring Blood Transthyretin Levels Over 6 Months

What this Trial Is About

Transthyretin amyloid cardiomyopathy ATTR-CM is a condition where a protein called transthyretin TTR breaks down and forms harmful clumps called amyloid, which deposit in the heart wall and impair its ability to pump blood properly. This can lead to heart failure and may be caused by age-related changes or genetic factors. Researchers are studying acoramidis, a drug that attaches to TTR to stabilize it and reduce amyloid formation, in participants who were previously treated with tafamidis, another TTR stabilizer. The goal is to see if switching to acoramidis increases blood TTR levels beyond those achieved with tafamidis. Participants will take acoramidis as two 356 mg tablets twice daily by mouth for up to 6 months. Before starting, all participants will continue their tafamidis treatment during a screening period. The study involves a single treatment group receiving acoramidis, and the main measurement is the change in serum TTR levels from baseline to 6 months or earlier if treatment stops. Secondary measures include heart function markers, kidney and thyroid assessments, and quality of life scores. Participants will be involved in nine check-ins, including two visits at the study site at screening and the end of treatment, six home visits by a study nurse during the first month and at 3 months, and a final phone check-in. Assessments include electrocardiograms, blood pressure, heart rate, and collection of blood and urine samples to monitor various health markers. The total participation time is about 8 months, during which researchers will monitor safety and treatment effects closely.

A Study to Learn More About the Change in the Blood Levels of Transthyretin When Participants With Transthyretin Amyloidosis With Cardiomyopathy Switch From Tafamidis to Acoramidis