A Randomized, Double-blind, Placebo-controlled, Concentration-guided, Exploratory Study of Mavacameten in Patients With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM) and Preserved Left Ventricular Ejection Fraction
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 59
- 试验地点
- 32
- 主要终点
- Percentage of Participants Who Experienced at Least One Treatment Emergent Adverse Event (TEAE)
研究概览
简要总结
This is a multicenter, exploratory, randomized, double-blind study of the administration of mavacamten in 60 participants with symptomatic nHCM randomized to receive a 16-week course of mavacamten doses titrated to achieve 1 of 2 target drug concentrations.
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
- •Diagnosed with nHCM (hypertrophied and non-dilated left ventricle in absence of systemic or other known cause), with LV wall thickness ≥ 15mm at Screening or ≥ 13mm with a positive family history of HCM.
- •Age 18 and greater, Body weight > 45kg
- •Documented LVEF ≥ 55% at the Screening as determined by echo central lab
- •LVOT gradient < 30 mmHg at rest AND during Valsalva AND post-exercise
- •NYHA functional class II or III
- •Elevated NT-proBNP at rest
排除标准
- •History of syncope, sustained ventricular tachyarrhythmia with exercise, obstructive coronary artery disease or myocardial infarction within the past 6 months
- •History of resuscitated sudden cardiac arrest at any time or known appropriate implantable cardioverter defibrillator (ICD) discharge within 6 months prior to Screening
- •Current treatment with disopyramide or ranolazine (within 14 days prior to Screening)
- •Current or planned treatment during the study with a combination of beta-blockers and calcium channel blockers
- •Has been treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA]) within 6 months prior to Screening
- •History of resting or post-exercise LVOT >30 mmHg unless subsequently treated by septal reduction
- •Has QTc Fridericia (QTcF) >480 ms or any other ECG abnormality considered by the investigator to pose a risk to participant safety (eg, second-degree atrioventricular block type II)
- •Has persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or not adequately rate-controlled within 1 year of Screening
- •History of clinically significant malignant disease within 10 years such as non-metastatic cutaneous squamous cell or basal cell carcinoma
- •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.
研究组 & 干预措施
Group 1
Active Treatment for participants with base target trough concentration
干预措施: mavacamten (Drug)
Group 2
Active Treatment for participants with higher target trough concentration
干预措施: mavacamten (Drug)
Placebo
Placebo Group
干预措施: Placebo (Drug)
结局指标
主要结局
Percentage of Participants Who Experienced at Least One Treatment Emergent Adverse Event (TEAE)
时间窗: From first dose to 8 weeks following last dose (Up to 24 weeks)
This is the percentage of participants who experienced at least one treatment emergent adverse event (TEAE)
Percentage of Participants Who Experienced at Least One Serious Treatment-emergent Adverse Event (STEAE)
时间窗: From first dose to 8 weeks following last dose (Up to 24 weeks)
This is the percentage of participants who experienced at least one serious treatment-emergent adverse event (STEAE)
次要结局
未报告次要终点