Efficacy of Mavacamten in Patients With Symptomatic Latent Obstructive Hypertrophic Cardiomyopathy: A Randomized Controlled Trial
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 78
- 试验地点
- 1
- 主要终点
- The change in peak left ventricular outflow tract (LVOT) gradient
研究概览
简要总结
This study aimed to evaluate the efficacy and safety of Mavacamten compared to no treatment in patients with symptomatic latent obstructive hypertrophic cardiomyopathy. The trial was randomized into two groups: Mavacamten group and Non-Mavacamten group. Over the 30-week treatment period, patients underwent a series of assessments at predefined time points, including transthoracic echocardiography, electrocardiogram (ECG), Holter monitoring, NYHA functional classification, Kansas City Cardiomyopathy Questionnaire (KCCQ), and cardiac biomarkers.
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 (Investigator, Outcomes Assessor)
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
- •Age ≥18 years.
- •Weight greater than 45 kg.
- •Adequate acoustic windows to allow for accurate transthoracic echocardiograms (TTEs).
- •Diagnosis of latent obstructive hypertrophic cardiomyopathy, in accordance with the current guidelines of the American College of Cardiology Foundation/American Heart Association, European Society of Cardiology, and Chinese Society of Cardiology.
- •Left ventricular ejection fraction (LVEF) ≥55% at rest, confirmed by the echocardiography core laboratory.
- •New York Heart Association (NYHA) Class II or III symptoms at the time of screening.
- •Resting oxygen saturation ≥90% at the time of screening.
排除标准
- •Any acute or severe comorbidities (e.g., severe infections or hematological, renal, metabolic, gastrointestinal, or endocrine dysfunction).
- •Currently using or having used prohibited medications within 14 days prior to screening, such as cytochrome CYP2C19 inhibitors (e.g., omeprazole or esomeprazole) or strong CYP3A4 inhibitors.
- •Life expectancy of less than 1 year.
- •Pregnant or breastfeeding women.
- •History of syncope or sustained ventricular tachyarrhythmia during exercise within the past 6 months.
- •Atrial fibrillation (AF).
- •Patients currently receiving or planning to receive treatment with disopyramide, cibenzoline, ranolazine, or a combination of beta-blockers with verapamil or diltiazem.
研究组 & 干预措施
Mavacamten Group
Patients in this group were treated with Mavacamten, starting at an initial dose of 2.5 mg, administered orally once daily. Subsequent doses were adjusted based on changes in pressure gradients and cardiac function observed during follow-up.
干预措施: mavacamten (Drug)
结局指标
主要结局
The change in peak left ventricular outflow tract (LVOT) gradient
时间窗: From baseline to week 30.
The change in peak left ventricular outflow tract (LVOT) gradient, determined by Doppler echocardiography, during exercise or pharmacologic provocation from baseline to week 30.
次要结局
- The proportion of patients with at least a one-class improvement in NYHA functional classification.(At week 30.)
- The change in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS).(From baseline to week 30.)
- The change in N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels.(From baseline to week 30.)
- The change in high-sensitivity cardiac troponin I (hs-cTnI) levels.(From baseline to week 30.)
- Key safety endpoints(From baseline to week 30.)
- The proportion of patients with a peak left ventricular outflow tract (LVOT) gradient of less than 50 mmHg.(At week 30.)
- The proportion of patients with a peak left ventricular outflow tract (LVOT) gradient of less than 30 mmHg.(At week 30.)
研究者
Xu Liu
Professor
Shanghai Chest Hospital