Within-Patient Comparison of Serum Transthyretin Response to Tafamidis Versus Acoramidis: A Pilot Crossover Study in Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- The primary endpoint of this study is the absolute change in serum TTR concentration (mg/dL) from baseline (pre-dose, day 0) to week 4 (day 28 ±5) after initiation of treatment with tafamidis and acoramidis. The pre-specified primary endpoints are the absolute change, with relative (%) change reported as supportive.
研究概览
简要总结
The primary objective is to evaluate the within-patient difference in serum TTR levels between tafamidis and acoramidis treatment periods, operationalized as the difference between the mean serum TTR concentration during Days 0–28 of each period.
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
- 否
入选标准
- •Age 18-99 years. Confirmed diagnosis of ATTR-CM based on. eGFR ≥30 mL/min/1.73m² (CKD-EPI) at screening. NT-proBNP >300 pg/mL and ≤7000 pg/mL at screening. Clinically stable status at enrolment (no recent acute decompensation or intercurrent infection). Treatment-naive with respect to all TTR-targeted therapies (no prior tafamidis, acoramidis, diflunisal, doxycycline+TUDCA, patisiran, vutrisiran, inotersen, eplontersen, or other TTR-directed agents). Females of childbearing potential: negative pregnancy test and highly effective contraception. Ability and willingness to comply with visits, repeated blood draws, and temporary treatment interruption during washout. Written informed consent.
排除标准
- •Hospitalization for HF or ATTR-CM within 3 months prior to screening. Clinical instability that would make a temporary interruption of ATTR-specific therapy unsafe in the opinion of the investigator. Intercurrent infection or inflammatory flare at baseline. Previous heart or liver transplantation, or planned transplantation within 12 months. Current/planned VAD. Current/planned pregnancy. Life expectancy <1 year. HF primarily due to ischemic heart disease. Confirmed AL amyloidosis. Dialysis or UACR >300 mg/g at screening. Major surgery within 90 days. Significant liver disease; ALT or AST ≥3×ULN or bilirubin ≥3×ULN. Prohibited concomitant ATTR therapies. Known hypersensitivity to either IMP or excipients.
研究组 & 干预措施
Vyndaqel 61 mg soft capsules
干预措施: Vyndaqel 61 mg soft capsules (Drug)
BEYONTTRA 356 mg film-coated tablets
干预措施: BEYONTTRA 356 mg film-coated tablets (Drug)
结局指标
主要结局
The primary endpoint of this study is the absolute change in serum TTR concentration (mg/dL) from baseline (pre-dose, day 0) to week 4 (day 28 ±5) after initiation of treatment with tafamidis and acoramidis. The pre-specified primary endpoints are the absolute change, with relative (%) change reported as supportive.
The primary endpoint of this study is the absolute change in serum TTR concentration (mg/dL) from baseline (pre-dose, day 0) to week 4 (day 28 ±5) after initiation of treatment with tafamidis and acoramidis. The pre-specified primary endpoints are the absolute change, with relative (%) change reported as supportive.
次要结局
未报告次要终点
研究者
Department of Medicine II, Division of Cardiology
Scientific
Medical University Of Vienna