The Therapeutic Value of Mavacamten in Hypertrophic Cardiomyopathy With Mid-to-Apical Left Ventricular Obstruction: A Prospective, Interventional, Real-World Clinical Study.
试验速览
- 阶段
- 4 期
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 132
- 试验地点
- 2
- 主要终点
- Percentage change in pressure gradient during the Valsalva maneuver
研究概览
简要总结
This study is a prospective interventional cohort study aimed at evaluating the therapeutic efficacy and clinical utility of Mavacamten-a targeted myosin inhibitor specifically developed for obstructive hypertrophic cardiomyopathy (HCM)-in patients with HCM characterized by mid-to-apical left ventricular obstruction.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients diagnosed with HCM according to the 2023 Chinese Guidelines for the Diagnosis and Treatment of Adult Hypertrophic Cardiomyopathy, meeting one of the following:
- •Left ventricular wall thickness ≥15 mm at end-diastole in any segment as assessed by echocardiography or cardiac magnetic resonance imaging (CMR);
- •Left ventricular wall thickness ≥13 mm in individuals with a confirmed pathogenic gene mutation or in genetically affected family members;
- •Exclusion of other cardiovascular, systemic, or metabolic disorders that may cause ventricular hypertrophy.
- •Symptomatic non-outflow tract obstructive HCM patients (meeting criterion a and at least one of b or c):
- •Presence of clinical symptoms such as dyspnea, chest pain, dizziness, palpitations, or syncope, with New York Heart Association (NYHA) functional class II-III;
- •Maximal pressure gradient (PGmax) >30 mmHg in the mid-ventricle under resting or Valsalva maneuver as assessed by echocardiography;
- •PGmax >30 mmHg in the apical region under resting or Valsalva maneuver on echocardiography.
- •③Ability to provide written informed consent (ICF) and any required privacy authorization prior to study enrollment.
排除标准
- •Obstructive hypertrophic cardiomyopathy (HCM), defined as a maximal left ventricular outflow tract pressure gradient (LVOT-PGmax) ≥30 mmHg at rest and during the Valsalva maneuver on echocardiography;
- •Maximal right ventricular outflow tract pressure gradient (RVOT-PGmax) ≥16 mmHg at rest; ③ Left ventricular ejection fraction (LVEF) <50% on echocardiography;
- •Uncontrolled primary hypertension;
- •Moderate or severe aortic valve stenosis and/or primary mitral valve disease with severe mitral regurgitation; ⑥ Known infiltrative or storage disorders mimicking the HCM phenotype (e.g., Fabry disease, cardiac amyloidosis); ⑦ Presence of severe infections, hepatic dysfunction, renal impairment, or other serious conditions significantly affecting life expectancy.
研究组 & 干预措施
No mavacamten
Guideline-directed standard medical therapy group
干预措施: Beta Blocker (BB) - metoprolol, bisoprolol, carvedilol (Drug)
Mavacamten
Add-on use of mavacamten on top of guideline-directed standard medical therapy
干预措施: diltiazem (Drug)
Mavacamten
Add-on use of mavacamten on top of guideline-directed standard medical therapy
干预措施: mavacamten (Drug)
Mavacamten
Add-on use of mavacamten on top of guideline-directed standard medical therapy
干预措施: Beta Blocker (BB) - metoprolol, bisoprolol, carvedilol (Drug)
No mavacamten
Guideline-directed standard medical therapy group
干预措施: diltiazem (Drug)
结局指标
主要结局
Percentage change in pressure gradient during the Valsalva maneuver
时间窗: Week 36
The percentage reduction in the pressure gradient at the site of left ventricular obstruction during the Valsalva maneuver at week 36, compared to baseline, as measured by echocardiography.
次要结局
- New York Heart Association (NYHA) functional classification(Week 36)
- Percentage change in resting pressure gradient(Week 36)
- Absolute change in pressure gradient during the Valsalva maneuver(Week 36)
- KCCQ-CSS(Week 36)
- The proportion of patients with a pressure gradient <30 mmHg during the Valsalva maneuver(Week 36)
- BNP(Week 36)
- Troponin T(Week 36)
- New-onset atrial fibrillation(From baseline to week 36)
- LVEF<50%(From baseline to week 36)
- LVEF<40%(From baseline to week 36)
- Anterior papillary muscle to the interventricular septum distance(Week 36)
- Left atrial global longitudinal strain(Week 36)
- Left ventricular global longitudinal strain(Week 36)
研究者
xie xiaojie
MD, PhD
Second Affiliated Hospital, Zhejiang University, School of Medicine