NCT02842242已完成2 期
A Phase 2 Open-label Pilot Study to Evaluate Efficacy, Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of MYK-461 in Subjects With Symptomatic Hypertrophic Cardiomyopathy and Left Ventricular Outflow Tract Obstruction
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 21
- 试验地点
- 7
- 主要终点
- Change in Post-exercise Peak LVOT Gradient From Baseline to Week 12
研究概览
简要总结
The purpose of this phase 2 open-label pilot study is to evaluate the efficacy, pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of MYK-461 in subjects with symptomatic HCM and LVOT obstruction aged 18-70 years.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with HCM (hypertrophied and non-dilated left ventricle in absence of systemic or other known cause), with LV wall thickness ≥ 15 mm at time of initial diagnosis or ≥ 13 mm with a positive family history of HCM.
- •Age 18-70
- •BMI 18-37kg/m2
- •Documented LVEF ≥ 55% at the Screening visit as determined by the investigator and the investigational site's echocardiography laboratory.
- •Resting LVOT gradient ≥ 30 mg Hg and post-exercise peak LVOT gradient ≥ 50 mm Hg
- •NYHA functional class II or higher
排除标准
- •History of sustained ventricular tachyarrhythmia.
- •History of syncope with exercise within past 6 months.
- •Active infection.
- •Persistent atrial fibrillation or atrial fibrillation at Screening or history of paroxysmal atrial fibrillation with resting rate document > 100bpm within 1 year of screening.
- •Has QTc Fridericia (QTcF) > 500 ms, or any other ECG abnormality considered by the investigator to pose a risk to subject safety (e.g. second degree atrioventricular block type II).
- •Aortic stenosis or fixed subaortic obstruction.
- •History of LV systolic dysfunction (LVEF < 45%) at any time during their clinical course.
- •History of obstructive coronary artery disease.
- •History or evidence of any other clinically significant disorder, condition, or disease that, in the opinion of the investigator or MyoKardia physician, would pose a risk to subject safety or interfere with the study evaluation, procedures, or completion.
- •Part A: Ongoing therapy with beta blockers, calcium channel blockers, or disopyramide. Subjects on any of these medications who, in the opinion of the investigator, can safely be withdrawn are eligible as long as medication is discontinued at least 14 days prior to the Screening visit.
- •Part B: Ongoing therapy with calcium channel blockers or disopyramide. Subjects on any of these medications who, in the opinion of the investigator, can safely be withdrawn are eligible as long as medication is discontinued at least 14 days prior to the Screening visit.
- •Prior treatment with cardiotoxic agents such as doxorubicin or similar, or current treatment with antiarrhythmic drugs that have negative inotropic activity, e.g. flecainide or propafenone.
研究组 & 干预措施
Open Label
Experimental
MYK-461
干预措施: MYK-461 (Drug)
结局指标
主要结局
Change in Post-exercise Peak LVOT Gradient From Baseline to Week 12
时间窗: Baseline and Week 12
Post-exercise peak LVOT gradients are assessed after a treadmill stress test by echocardiography.
次要结局
- Number of Participants Achieving a Post-exercise Peak LVOT Gradient Response of < 30 mmHg. Gradient < 30 mmHg(Baseline and Week 12)
- Change in Peak VO2 From Baseline to Week 12(Baseline and Week 12)
- Change in Post-exercise Peak LVOT Gradient From Week 12 to Week 16(Weeks 12 and 16)
- Change in VE/VCO2 From Baseline to Week 12(Baseline and Week 12)
- Change in LV Fractional Shortening (LVFS) From Baseline to Week 12(Baseline and Week 12)
- Change in Global Longitudinal Strain (GLS) From Baseline to Week 12(Baseline and Week 12)
- Change in Resting LVEF From Baseline to Week 12(Baseline and Week 12)
- Change in Dyspnea Symptom Score From Baseline to Week 12(Baseline and Week 12)
研究者
研究点 (7)
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