Transthyretine amyloidose cardiomyopathie (ATTR-CM) blocker
Sponsor: eidos therapeutics
Transthyretin amyloidosis characterised by cardiomyopathy (ATTR-CM) occurs because the protein transthyretin breaks down into individual pieces (monomers) that stick together and form amyloid, the deposits in the heart leading to heart failure. AG10 stabilises the protein transthyretin and can thus inhibit the formation of amyloid. This study will investigate whether further deterioration of cardiac function in cardiac ATTR-CM can be inhibited with AG10 in a double-blind, placebo-controlled, multicentre phase 3 study, followed by an open-label extension.
Team:
SI: drs. A.Tubben