Amyloidosis (ATTR) Cardiomyopathy (CM) is a progressive, fatal disease in which amyloid deposits build up slowly, over the course of many years to cause organ damage. This study is evaluating the safety and tolerability of a new drug called AG10 administered to adult patients with symptomatic transthyretin amyloid cardiomyopathy. The participant will be randomized to the study drug or placebo and take the study drug two times per day for 30 months.
The purpose of this research is to test a new experimental drug called "AG10" made by Eidos Therapeutics, to find out if this new drug is safe and if it works to help patients with Amyloidosis Cardiomyopathy. This is a follow up of the AG10-201 study in which participants were randomized to either study drug or placebo. It is only open to participants who completed the AG10-201 study. It will consist of 4 clinic visits and take less than three months for the participants to complete.
The purpose of this research study is to test if ARRY-371797 is safe and effective in treating dilated cardiomyopathy caused by a genetic mutation to the LMNA gene. This will be demonstrated by the functional capacity of patients in their 6-minute walk tests and quality of life questionnaires.
The purpose of this study is to identify gene changes that cause DCM and gene differences that influence the development and severity of DCM. These genetic studies may identify a change in a gene that has already been linked with DCM, a change in a gene not previously associated with DCM, or these studies may identify a gene or genes that affect the development and severity of the DCM. With this knowledge we hope to have a better understanding of how genes and gene changes cause DCM.