NCT06081894进行中（未招募）3 期
A Phase 3, Multi-Center, Randomized, Double-Blind Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy
Cytokinetics181 个研究点 分布在 1 个国家目标入组 500 人开始时间: 2023年8月31日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 500
- 试验地点
- 181
- 主要终点
- Change in Kansas City Cardiomyopathy Questionnaire - Clinical Summary Score (KCCQ-CSS)
研究概览
简要总结
This clinical trial will study the effects of aficamten (versus placebo) on the quality of life, exercise capacity, and clinical outcomes of patients with non-obstructive hypertrophic cardiomyopathy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Between 18-85 years of age
- •Body mass index < 40 kg/m2
- •Diagnosed with nHCM and has a screening echocardiogram with the following:
- •End-diastolic left ventricular (LV) wall thickness:
- •≥ 15 mm in one or more myocardial segments OR
- •≥ 13 mm in one or more wall segments and a known disease-causing gene mutation or positive family history of HCM AND
- •Resting LVOT-G < 30 mmHg AND Valsalva LVOT-G < 50 mmHg AND
- •LVEF ≥ 60%
- •Participants with a history of intracavitary obstruction are eligible.
- •NYHA class II or III
- •Respiratory exchange ratio of ≥ 1.00 at screening by cardiopulmonary exercise testing (CPET) and predicted peak oxygen uptake (pVO2) ≤ 90% for age and sex
- •KCCQ-CSS score of ≤ 85
- •NT-proBNP of:
- •NT-pro BNP ≥ 300 pg/mL or NT-proBNP ≥ 900 pg/mL if in atrial fibrillation or atrial flutter OR
- •For Black participants, an NT-pro BNP ≥ 225 pg/mL or NT-proBNP ≥ 675 pg/mL if in atrial fibrillation or atrial flutter
排除标准
- •Significant valvular heart disease (per Investigator judgment)
- •Moderate or severe valvular aortic stenosis or fixed subaortic obstruction
- •Moderate or severe mitral regurgitation
- •Known or suspected infiltrative, genetic or storage disorder causing cardiac hypertrophy that mimics nHCM (eg, Noonan syndrome, Fabry disease, amyloidosis)
- •Known current unrevascularized coronary artery stenosis of ≥ 70% or documented history of myocardial infarction.
- •History of LV systolic dysfunction (LVEF < 45%) or stress cardiomyopathy
- •Inability to exercise on a treadmill or bicycle (eg, orthopedic limitations)
- •Documented room air oxygen saturation reading < 90% at screening or history of significant chronic obstructive pulmonary disease or severe/significant pulmonary hypertension
- •History of syncope, symptomatic ventricular arrhythmia, or sustained ventricular tachyarrhythmia with exercise within 3 months prior to screening
- •History of resistant hypertension (persistently elevated blood pressure despite maximal doses of 3 or more classes of medications for hypertension control)
- •Screening diastolic blood pressure ≥ 100 mmHg
- •Received prior treatment with aficamten
- •Received treatment with mavacamten within 3 months prior to screening (must be discussed with the medical monitor prior to screening)
- •Undergone septal reduction therapy < 6 months prior to screening
- •Is being considered for or is likely to be considered for heart transplant listing or left ventricular assist device placement during the study period
- •Paroxysmal or permanent atrial fibrillation is excluded only if:
- •rhythm restoring treatment (e.g., direct-current cardioversion, atrial fibrillation ablation procedure, or antiarrhythmic therapy) has been required ≤ 3 months prior to screening
- •rate control and anticoagulation have not been achieved for at least 3 months prior to screening.
研究组 & 干预措施
Aficamten
Experimental
Participants in this arm will receive a single daily oral dose of 5 mg, 10 mg, 15 mg, or 20 mg of aficamten with dose levels guided by echocardiography assessments, for up to 72 weeks.
干预措施: Aficamten (Drug)
Placebo
Placebo Comparator
Participants in this arm will receive placebo, for up to 72 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Change in Kansas City Cardiomyopathy Questionnaire - Clinical Summary Score (KCCQ-CSS)
时间窗: Baseline to Week 36
Effect of aficamten compared with placebo on participant health status
Change in pVO2
时间窗: Baseline to Week 36
Effect of aficamten compared with placebo on maximal exercise capacity
次要结局
- Change in composite of two Z-scores of CPET parameters (pVO2 and VE/VCO2 slope)(Baseline to Week 36)
- Proportion of participants with ≥ 1 class improvement in NYHA Functional Class(Baseline to Week 36)
- Change in NT-proBNP(Baseline to Week 36)
- Time to first CV event(Baseline to End of Study, Week 72)
- Proportion of participants with ≥ 1 class improvement in New York Heart Association (NYHA) Functional Class(Baseline to Week 36)
- Change in N-terminal prohormone brain natriuretic peptide (NT-proBNP)(Baseline to Week 36)
- Change in Left Atrial Volume Index (LAVI) in participants without atrial fibrillation or flutter at baseline on ECG(Baseline to Week 36)
- Time to first cardiovascular (CV) event(Baseline to End of Study, Week 72)
- Change in LAVI in participants without atrial fibrillation or flutter at baseline on ECG(Baseline to Week 36)
研究者
研究点 (181)
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