Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy

Summary

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Description

Transthyretin amyloid cardiomyopathy (ATTR-CM) is an underdiagnosed condition believed to affect more than 400,000 people worldwide. In ATTR-CM, the accumulation of transthyretin (TTR) amyloid results in thickening and stiffening of the heart, which often leads to heart failure or even death. There ...

Transthyretin amyloid cardiomyopathy (ATTR-CM) is an underdiagnosed condition believed to affect more than 400,000 people worldwide. In ATTR-CM, the accumulation of transthyretin (TTR) amyloid results in thickening and stiffening of the heart, which often leads to heart failure or even death. There are two forms of ATTR-CM: Wild Type* This form of the condition primarily develops in older individuals who do not carry gene mutations Hereditary* This form of the condition comes from gene mutations passed down in families In this study we are researching the investigational drug AG10 800mg administered orally twice a day. Through the study, we want to evaluate the efficacy and safety of AG10 in patients with ATTR-CM versus placebo This is a 30 month, placebo-controlled study. This means that, during the 30 month study, investigators conducting the research and study participants will not know whether the study participant is receiving AG10 or placebo The primary outcomes of the study are: The impact of AG10 versus placebo on the 6 minute walk test after 12 months of treatment The impact of AG10 versus placebo on the frequencies of deaths and cardiovascular-related hospitalizations after 30 months of treatment At the end of 30 months, participants may be eligible to receive investigational AG10, and there is no placebo. This is called an "open label extension." This part of the study may help us better understand the safety related to taking AG10 over a longer period of time

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