The study aims to evaluate the effect of acoramidis on cardiac functional and structural improvement in participants with ATTR-CM.
Participants aged 18 to 80
Confirmed diagnosis of ATTR-CM requiring targeted therapy
At screening: New York Heart Association (NYHA) Class I/II; estimated glomerular filtration rate (eGFR) ≥40 mL/min/1.73m2; left ventricular ejection fraction (LVEF) >35%; left ventricular (LV) wall thickness ≥12 mm.
Heart failure condition has been clinically stable for at least 6 weeks prior to screening.
Current or planned implantation of a cardiac implantable electronic device (eg, pacemaker, implantable cardiac defibrillator).
Acute myocardial infarction, acute coronary syndrome or coronary revascularization, or experienced stroke or transient ischemic attack within 90 days prior to screening.
Hemodynamic instability.
Likely to undergo heart transplantation within 12 months from screening.
Single group
Acoramidis (AG10) oral tablet
DrugStudy drug is defined as acoramidis 712 mg (equivalent to 800 mg acoramidis hydrochloride) taken orally twice daily for 36 months of open label treatment.
To assess the effect of acoramidis on cardiac function improvement by CMR in participants with ATTR-CM.
Change from baseline in left ventricular ejection fraction (LVEF) or left ventricular stroke volume index (LVSVi) at Month 36.
To assess the effect of acoramidis on cardiac function improvement or stabilization by CMR in participants with ATTR-CM.
Change from baseline in LVEF and LVSVi at Month 36
To assess the effect of acoramidis on cardiac function stabilization by CMR in participants with ATTR-CM.
Change from baseline in LVEF and LVSVi at Month 36
To evaluate the effect of treatment with acoramidis on individual parameters of left ventricular systolic function by CMR in participants with ATTR-CM
Mean change from baseline in LVEF, LVSVi, left ventricular global longitudinal strain (LVGLS) at Month 36
To evaluate the effect of acoramidis on cardiac structure by CMR in participants with ATTR-CM
Mean change from baseline in left ventricular mass index (LVMi) at Month 36
Exploratory Outcome Measure
To evaluate the effect of acoramidis on various biomarkers and cardiac transthoracic echocardiogram
Click on the lead sponsor to view all of their trials.
