2023-505797-15-00招募中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
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 175
- 试验地点
- 53
- 主要终点
- Dual primary endpoints of: • Change in KCCQ-CSS from baseline to Week 36 • Change in pVO2 from baseline to Week 36
研究概览
简要总结
To evaluate the effect of aficamten compared with placebo on participant health status and maximal exercise capacity
研究设计
- 分配方式
- Randomized
- 主要目的
- Treatment Period 2
- 盲法
- Double (Investigator, Subject)
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Between 18–85 years of age at screening
- •Body mass index < 40 kg/m2
- •Diagnosed with nHCM and has a screening echocardiogram with the following: • End-diastolic 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
- •New York Heart Association (NYHA) class II or III
- •Respiratory exchange ratio of ≥ 1.00 at screening by CPET and predicted peak oxygen uptake (pVO2) of ≤ 90% for age and sex
- •KCCQ-CSS score ≤ 85
- •N-terminal prohormone brain natriuretic peptide (NT-proBNP) of: • ≥ 300 pg/mL or ≥ 900 pg/mL if in atrial fibrillation or atrial flutter OR • For Black participants, ≥ 225 pg/mL or ≥ 675 pg/mL if in atrial fibrillation or atrial flutter
- •Hemoglobin ≥ 10 g/dL
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
- •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
- •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 randomization • rate control and anticoagulation have not been achieved for at least 3 months prior to screening
- •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)
- •Known or suspected infiltrative, genetic or storage disorder causing cardiac hypertrophy that mimics nHCM (e.g., Noonan syndrome, Fabry disease, amyloidosis)
- •Known current unrevascularized coronary artery stenosis of ≥ 70% or documented history of Type 1 myocardial infarction.
- •History of LV systolic dysfunction (LVEF < 45%) or stress cardiomyopathy
- •Inability to exercise on a treadmill or bicycle (e.g., 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 the following events with exercise within 3 months prior to screening • syncope, • symptomatic ventricular arrhythmia, or • sustained ventricular tachyarrhythmia
结局指标
主要结局
Dual primary endpoints of: • Change in KCCQ-CSS from baseline to Week 36 • Change in pVO2 from baseline to Week 36
Dual primary endpoints of: • Change in KCCQ-CSS from baseline to Week 36 • Change in pVO2 from baseline to Week 36
次要结局
- Proportion of participants with ≥ 1 class improvement in NYHA Functional Class from baseline to Week 36
- Change in the composite of two Z-scores of CPET parameters from baseline to Week 36: – pVO2 (maximal exercise capacity) – VE/VCO2 slope (sub-maximal exercise capacity)
- Change in NT-proBNP from baseline to Week 36
- Change in LAVI from baseline to Week 36
- Time to first event of cardiovascular death, heart transplantation or left ventricular assist device, aborted sudden cardiac death, non-fatal stroke, heart failure hospitalization, or cardiac arrhythmia (atrial fibrillation or ventricular tachyarrhythmia) requiring treatment or hospitalization
研究者
Cytokinetics Inc. Medical Affairs
Scientific
Cytokinetics Inc.
研究点 (53)
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