NCT07361289招募中不适用
A Single-Arm Observational Study to Assess the Real-World Effectiveness of Mavacamten in Adult Patients With Obstructive Hypertrophic Cardiomyopathy in China
Bristol-Myers Squibb15 个研究点 分布在 1 个国家目标入组 500 人开始时间: 2026年1月15日最近更新:
适应症
干预措施
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 500
- 试验地点
- 15
- 主要终点
- Change from baseline in Valsalva left ventricular outflow tract (LVOT) gradient
研究概览
简要总结
The purpose of this study is to assess the effectiveness of mavacamten treatment in Chinese adults with symptomatic obstructive hypertrophic cardiomyopathy (HCM) in real-world clinical practice
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
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
- •Participants aged ≥ 18 years (participants enrolled retrospectively: at the time of initial mavacamten prescription), irrespective of gender.
- •Participants who have initiated mavacamten (for whom enrolled retrospectively) or are scheduled to initiate mavacamten (for whom enrolled prospectively) based on clinical therapeutic needs.
- •Diagnosed with obstructive hypertrophic cardiomyopathy (HCM) consistent with current American College of Cardiology Foundation/American Heart Association, European Society of Cardiology, and Chinese guidelines for diagnosis and treatment of patients with hypertrophic cardiomyopathy, i.e., satisfy criteria below:
- •Has a documented diagnosis of HCM prior to enrollment, and
- •Peak left ventricular outflow tract (LVOT) gradient ≥ 30 mmHg at rest or with provocation in the most recent medical record within 3 months prior to enrollment as assessed by echocardiography.
- •Has documented left ventricular ejection fraction (LVEF) ≥ 55%, as measured by resting transthoracic echocardiography (TTE) in the most recent medical record within 3 months prior to enrollment.
- •New York Heart Association (NYHA) class II or III symptoms in the most recent medical record within 3 months prior to enrollment.
- •For participants enrolled retrospectively, essential baseline information* and critical data** must be traceable and available. At least one key follow-up time points*** is required for inclusion.
- •* Essential baseline information, including age, gender, resting or provoked LVOT peak gradient, LVEF, indices of cardiac structure (e.g., maximum LV wall thickness, atrial and ventricular chamber size and volumes), as well as systolic and diastolic function, NYHA functional class.
- •Critical data, including resting or provoked LVOT gradient, LVEF, cardiac structure, systolic and diastolic function, dose of mavacamten.
- •Key follow-up time points: including weeks 4, 8, 12, 24, 36, 48, 72 and
- •Voluntary sign informed consent form. Note: For participants enrolled retrospectively, the most recent medical record within 3 months as mentioned in the above requirements refer to the most recent medical record within 3 months prior to the initial mavacamten prescription.
排除标准
- •Known HCM phenocopy disease (e.g., Fabry disease, amyloidosis).
- •Participants who are expected to undergo major cardiac surgery during the study.
- •Prior treatment of obstructive HCM with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA] or septal radiofrequency ablation) within 6 months prior to enrollment (participants enrolled retrospectively: within 6 months prior to initial mavacamten prescription); participants with an unsuccessful myectomy or percutaneous ASA or septal radiofrequency ablation performed >6 months prior to enrollment (participants enrolled retrospectively: within 6 months prior to initial mavacamten prescription) may be enrolled.
- •Currently treated with disopyramide or ranolazine (within 14 days prior to enrollment [participants enrolled retrospectively: within 14 days prior to initial mavacamten prescription]) or participants who are expected to be taking disopyramide, ranolazine, verapamil in combination with β-receptor blockers, or diltiazem in combination with β-receptor blockers during the study.
- •Presence of other diseases that may affect completion of 96 weeks follow-up as assessed by the investigator.
- •Participants who are using or are expected to be using moderate to strong CYP2C19 inhibitors/inducers, or strong CYP3A4 inhibitors, moderate to strong CYP3A4 inducers during the study.
- •Participants who are participating in other interventional clinical studies.
研究组 & 干预措施
Cohort 1
Participants with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) receiving mavacamten
干预措施: Mavacamten (Drug)
结局指标
主要结局
Change from baseline in Valsalva left ventricular outflow tract (LVOT) gradient
时间窗: Week 48, Week 96
Change from baseline in resting valsalva left ventricular outflow tract (LVOT) gradient
时间窗: Week 48, Week 96
次要结局
- Change from baseline in New York Heart Association (NYHA) class(Week 4, Week 8, Week 12, Week 24, Week 36, Week 48, Week 60, Week 72, Week 84, and Week 96)
- Number and proportion of participants with a resting/provoked left ventricular outflow tract (LVOT) gradient < 30/50 mmHg(Week 12, Week 36, Week 48, Week 72, and Week 96)
- Number and proportion of participants achieving complete response (defined as all left ventricular outflow tract (LVOT) gradients < 30 mmHg and New York Heart Association (NYHA) Class I)(Week 12, Week 36, Week 48, Week 72, and Week 96)
研究者
研究点 (15)
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