NCT03470545已完成3 期
A Randomized, Double Blind, Placebo Controlled Clinical Study to Evaluate Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 251
- 试验地点
- 71
- 主要终点
- Percentage of Participants Achieving A Clinical Response
研究概览
简要总结
This is a multicenter, international, double-blind study of the administration of mavacamten in participants with symptomatic obstructive HCM (oHCM). Approximately 220 participants will be randomized to receive placebo or mavacamten.
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
- Quadruple (Participant, Care Provider, 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 and greater, body weight ≥ 45kg
- •Has adequate acoustic windows to enable accurate transthoracic echocardiograms (TTEs)
- •Diagnosed with oHCM consistent with current American College of Cardiology Foundation/American Heart Association and European Society of Cardiology guidelines and satisfy both criteria:
- •Has documented left ventricular ejection fraction (LVEF) ≥55%
- •NYHA Class II or III
- •Has documented oxygen saturation at rest ≥90% at Screening
- •Is able to perform an upright CPET and has a respiratory exchange ratio (RER) ≥1.0 at Screening per central reading
排除标准
- •Known infiltrative or storage disorder causing cardiac hypertrophy that mimics oHCM, such as Fabry disease, amyloidosis, or Noonan syndrome with LV hypertrophy
- •History of syncope or sustained ventricular tachyarrhythmia with exercise within 6 months prior to Screening
- •History of resuscitated sudden cardiac arrest (at any time) or known history of appropriate implantable cardioverter defibrillator (ICD) discharge for life-threatening ventricular arrhythmia within 6 months prior to Screening
- •Paroxysmal, intermittent atrial fibrillation with atrial fibrillation present at Screening
- •Persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or not adequately rate controlled within 6 months prior to Screening
- •Treatment (within 14 days prior to Screening) or planned treatment during the study with disopyramide or ranolazine
- •Treatment (within 14 days prior to Screening) or planned treatment during the study with a combination of β-blockers and calcium channel blockers
- •LVOT gradient with Valsalva maneuver <30 mmHg at Screening
- •Has been successfully treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA]) within 6 months prior to Screening or plans to have either of these treatments during the study
- •ICD placement within 2 months prior to Screening or planned ICD placement during the study
- •Has a history or evidence of any other clinically significant disorder, condition, or disease that, in the opinion of the investigator, would pose a risk to participant safety or interfere with the study evaluation, procedures, or completion
- •Prior treatment with cardiotoxic agents such as doxorubicin or similar
研究组 & 干预措施
mavacamten (MYK-461)
Experimental
干预措施: mavacamten (Drug)
Placebo
Placebo Comparator
干预措施: Placebo (Drug)
结局指标
主要结局
Percentage of Participants Achieving A Clinical Response
时间窗: 30 weeks
A positive clinical response (value="YES") is defined as having achieved either an improvement of at least 1.5 mL/kg/min in peak oxygen consumption (pVO2) as determined by cardiopulmonary exercise testing (CPET) and a reduction of one or more class in New York Heart Association (NYHA) functional classification (e.g.I, II, III, or IV) -OR- an improvement of 3.0 mL/kg/min or more in pVO2 with no worsening in NYHA Functional Class.
次要结局
- Change From Baseline to Week 30 in pVO2 as Assessed by CPET(30 weeks)
- Changes From Baseline to Week 30 in Post Exercise in LVOT Peak Gradient.(30 weeks)
- Proportion of Participants With at Least 1 Class Improvement in NYHA Functional Class From Baseline to Week 30(30 weeks)
- Change From Baseline to Week 30 in Participant-reported Health-related Quality of Life as Assessed by the KCCQ Score(30 weeks)
- Change From Baseline to Week 30 in Participant-reported Severity of HCM Symptoms as Assessed by the HCMSQ Score(30 weeks)
研究者
研究点 (71)
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