2025-521831-35-00招募中4 期
(23026) A prospective, single-arm, Phase 4 study to evaluate the course of serum transthyretin (TTR) level with acoramidis in adult patients with variant or wild-type transthyretin amyloidosis with cardiomyopathy (ATTR-CM) previously treated with tafamidis.
适应症
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 32
- 试验地点
- 6
- 主要终点
- Change in serum TTR level from baseline to month 6 or premature discontinuation of treatment
研究概览
简要总结
To evaluate the change of serum TTR level associated with acoramidis treatment in patients with ATTR-CM switching from tafamidis.
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Participant must be 18 to 90 years of age inclusive, at the time of signing the informed consent.
- •Diagnosis of ATTR-CM; disease defining examination, i.e., SPECT or SPECT/CT or biopsy, within 24 months prior to V1
- •Participants must currently be treated with tafamidis and have used tafamidis for at least the previous 3 months prior to V1 and have adhered to tafamidis therapy
- •NYHA class ≤ II at V1
- •eGFR ≥ 30 mL/min/1.73m2 at V1
- •NT-proBNP > 300 and ≤ 7000 pg/mL at V1
排除标准
- •Prior liver or heart transplantation or planned within the next 12 months
- •Initiation of treatment with a diuretic or increase in diuretic dose within 3 months before V1
- •Treatment with calcium channel blockers (e.g., verapamil, diltiazem) or digitalis required
- •Recent CV hospitalization within 3 months before V1
- •Known hypersensitivity to acoramidis or to any of the excipients
- •A condition that, as judged by the investigator, would preclude compliance with the study protocol, such as a history of substance abuse, alcoholism, or a psychiatric condition
- •Known or suspected liver disorder and bile secretion/flow (cholestasis, also history of it)
- •Abnormal liver function tests at V1, defined as ALT (GPT) or AST (GOT) ≥ 3 x ULN or total bilirubin ≥ 3 x ULN at V1
- •Current or planned use of ventricular assist device
- •Active cancer or other disease that decreases the life expectancy to less than one year
- •Heart failure due to ischemic heart disease
- •Myocardial infarction, CV surgery, or unstable angina within the last 90 days prior to V1
- •Confirmed diagnosis of light-chain amyloidosis
- •Dialysis or severe renal impairment as reflected by UACR > 300 mg/g at V1
- •Major surgery 90 days prior to V1
- •Recent initiation of SGLT2i within 3 months before V1
结局指标
主要结局
Change in serum TTR level from baseline to month 6 or premature discontinuation of treatment
Change in serum TTR level from baseline to month 6 or premature discontinuation of treatment
次要结局
- Serum TTR level at baseline, week 1, 2, 3, 4 and at month 3.
- Occurrence of TEAEs and type of TEAEs and serious TEAEs.
- Change from baseline to month 6 of NT-proBNP, hs-TnT, hs-CRP, RBP4.
- Signs of remodeling from baseline to month 6 in ECHO.
- Change from baseline to month 6 in 6MWD
- Change from baseline to month 6 in kidney and thyroid biomarkers: eGFR, Creatinine, Cystatin C, UACR, and TSH.
- Change from baseline to month 6 in KCCQ-OS score and in EQ-5D-5L index score.
研究者
Chief Medical officer
Scientific
Bayer Consumer Care AG
研究点 (6)
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