An Open-Label Study to Investigate the Long-Term Effects of Acoramidis on Cardiac Function, Structure and Cardiac Amyloid Burden in Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 发起方
- 入组人数
- 150
- 试验地点
- 1
- 主要终点
- To assess the effect of acoramidis on cardiac function improvement by CMR in participants with ATTR-CM.
研究概览
简要总结
The study aims to evaluate the effect of acoramidis on cardiac functional and structural improvement in participants with ATTR-CM.
详细描述
Study AG10-404 is an open-label study to investigate the long-term effects of acoramidis on cardiac functional and structural improvement, and the effects on cardiac amyloid burden in participants with ATTR-CM using two types of imaging, cardiac MRI and echocardiograms to track changes in heart structure and amyloid buildup over time.
Up to approximately 150 participants (ages 18-80) will be enrolled. Participants will receive study drug (712 mg equivalent to 800 mg of acoramidis hydrochloride) orally twice daily from Day 1 through Month 36. The study duration for an individual participant is approximately 38 months, including up to 45 days for screening, 36 months for open-label treatment and 1 month follow-up after the last dose of study drug. In-clinic visits will occur at Day 1, Day 28, Month 6, and every 6 months up to Month 36. Phone visits will occur at Month 3 and every 6 months thereafter up to Month 33. Participants will be followed throughout their participation in this study and for 30 days (+7 days) after the last dose of study drug.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
- •Confirmed diagnosis of AL amyloidosis at screening.
- •At screening: NYHA Class III/IV; National Amyloidosis Center (NAC) Stage
- •Prior or current treatment with acoramidis.
研究组 & 干预措施
Acoramidis (AG10)
Open-label study where all participants will receive acoramidis (AG10) during the study
干预措施: Acoramidis (AG10) oral tablet (Drug)
结局指标
主要结局
To assess the effect of acoramidis on cardiac function improvement by CMR in participants with ATTR-CM.
时间窗: Month 36
Change from baseline in left ventricular ejection fraction (LVEF) or left ventricular stroke volume index (LVSVi) at Month 36.
次要结局
- To assess the effect of acoramidis on cardiac function improvement or stabilization by CMR in participants with ATTR-CM.(Month 36)
- To assess the effect of acoramidis on cardiac function stabilization by CMR in participants with ATTR-CM.(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(Month 36)
- To evaluate the effect of acoramidis on cardiac structure by CMR in participants with ATTR-CM(Month 36)
- To evaluate the effect of treatment with acoramidis on cardiac amyloid burden by CMR in participants with ATTR-CM(Month 36)