Cardiac Magnetic Resonance and Echocardiography Study With Acoramidis (ASCEND-ATTR)
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)
研究概览
地位
详细说明
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.
研究类型
注册 (估计的)
阶段
- 第四阶段
联系人和位置
学习联系方式
- 姓名:Medical Information
- 电话号码:1-844-550-2246
- 邮箱:medinfo@bridgebio.com
学习地点
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Missouri
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St Louis、Missouri、美国、63110
- 招聘中
- Washington University in St. Louis
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-
参与标准
资格标准
适合学习的年龄
- 成人
- 年长者
接受健康志愿者
描述
Key Inclusion Criteria:
- 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.
Key Exclusion Criteria:
- 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 3.
- Prior or current treatment with acoramidis.
学习计划
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:不适用
- 介入模型:单组作业
- 屏蔽:无(打开标签)
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
实验性的:Acoramidis (AG10)
Open-label study where all participants will receive acoramidis (AG10) during the study
|
Study drug is defined as acoramidis 712 mg (equivalent to 800 mg acoramidis hydrochloride) taken orally twice daily for 36 months of open label treatment.
|
研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
To assess the effect of acoramidis on cardiac function improvement by CMR in participants with ATTR-CM.
大体时间:Month 36
|
Change from baseline in left ventricular ejection fraction (LVEF) or left ventricular stroke volume index (LVSVi) at Month 36.
|
Month 36
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
To assess the effect of acoramidis on cardiac function improvement or stabilization by CMR in participants with ATTR-CM.
大体时间:Month 36
|
Change from baseline in LVEF and LVSVi at Month 36
|
Month 36
|
|
To assess the effect of acoramidis on cardiac function stabilization by CMR in participants with ATTR-CM.
大体时间:Month 36
|
Change from baseline in LVEF and LVSVi at Month 36
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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
大体时间:Month 36
|
Mean change from baseline in LVEF, LVSVi, left ventricular global longitudinal strain (LVGLS) at Month 36
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Month 36
|
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To evaluate the effect of acoramidis on cardiac structure by CMR in participants with ATTR-CM
大体时间:Month 36
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Mean change from baseline in left ventricular mass index (LVMi) at Month 36
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Month 36
|
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To evaluate the effect of treatment with acoramidis on cardiac amyloid burden by CMR in participants with ATTR-CM
大体时间:Month 36
|
Mean change from baseline in extracellular volume (ECV) at Month 36
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Month 36
|
其他结果措施
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Exploratory Outcome Measure
大体时间:At Month 12, 24 and 36
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To evaluate the effect of acoramidis on various biomarkers and cardiac transthoracic echocardiogram
|
At Month 12, 24 and 36
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合作者和调查者
研究记录日期
研究主要日期
学习开始 (估计的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
与本研究相关的术语
其他相关的 MeSH 术语
其他研究编号
- AG10-404
- 2026-526068-19-00 (克蒂斯)
- U1111-1339-5700 (其他标识符:WHO UTN)
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)?
药物和器械信息、研究文件
研究美国 FDA 监管的药品
研究美国 FDA 监管的设备产品
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