A Phase 3, Open-Label, Multicenter, Extension Study of Acoramidis in Patients With Newly Diagnosed Variant Transthyretin Amyloid Cardiomyopathy (ACT-EARLY OLE)
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 250
- 主要终点
- To evaluate the long-term safety and tolerability of acoramidis in participants with newly diagnosed ATTR-CM
研究概览
简要总结
The AG10-504 study is an open-label extension study of acoramidis in participants with newly diagnosed transthyretin amyloid cardiomyopathy (ATTR-CM) or both ATTR-CM and transthyretin amyloid polyneuropathy (ATTR-PN).
详细描述
The AG10-504 study is the extension of the Phase 3 AG10-501 study (ACT-EARLY) which was designed to investigate if the use of acoramidis as a preventative intervention in individuals who are carriers of a known pathogenic transthyretin (TTR) variant but with no clinical evidence of ATTR could prevent or delay the onset of clinically detectable ATTR and the considerable morbidity and mortality that result from this devastating, progressive, and ultimately fatal disease. Only participants who have completed the AG10-501 study (ACT-EARLY) with a diagnosis of ATTR-CM may enroll in this AG10-504 Open Label Extension (OLE) study.
The primary objective of the AG10-504 study is to evaluate the long-term safety and tolerability of acoramidis in participants with newly diagnosed ATTR-CM. The duration of this study will be up to 5 years.
Currently, acoramidis is approved for the treatment of ATTR-CM in some regions including the United States, all member states of the European Union (EU), Albania, Bosnia Herzegovina, Brazil, Iceland, Israel, Japan, Liechtenstein, Montenegro, North Macedonia, Norway, Switzerland, and the United Kingdom (UK).
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must have completed the AG10-501 study (ACT-EARLY study) within the past 60 calendar days with a diagnosis of ATTR-CM (based on the AG10-501 protocol definition of ATTR-CM).
排除标准
- •Participants who completed the AG10-501 study with a diagnosis of ATTR-PN only or who permanently discontinued study drug prior to diagnosis of ATTR-CM in AG10-
- •History of light-chain amyloidosis (AL) or another non-TTR amyloid subtype (eg, ApoA-1, gelsolin).
- •History of a monoclonal paraprotein or abnormal light chains in serum or urine (ie, monoclonal gammopathy of undetermined significance, MGUS) in which AL has not been ruled out.
- •Stage IV or V chronic kidney disease.
- •Active malignancy, except for basal or squamous cell carcinoma of the skin, carcinoma in situ of the cervix that has been successfully treated.
- •History of any organ transplant (with the exception of corneal transplant).
- •Known hypersensitivity to acoramidis or any of its excipients.
- •Major surgery planned during the next 6 months (some exceptions).
- •Treatment of ATTR-CM with any ATTR-oriented on- or off-label or OTC product.
研究组 & 干预措施
Acoramidis
Participants will receive acoramidis 712 mg orally BID (which is equivalent to 800 mg acoramidis HCl BID)
干预措施: Acoramidis (AG10) (Drug)
结局指标
主要结局
To evaluate the long-term safety and tolerability of acoramidis in participants with newly diagnosed ATTR-CM
时间窗: The duration of study participation for the participants will be up to 5 years.
Proportion of participants with: treatment-emergent AEs and SAEs, AEs leading to treatment discontinuation, abnormal physical examination findings of clinical relevance, abnormal vital signs of clinical relevance, abnormal ECG parameters of clinical relevance, changes in clinical safety laboratory parameters of potential concern
次要结局
未报告次要终点