NCT05414175已完成3 期
A Phase 3, Open-label, Single Arm, Clinical Study to Evaluate Efficacy, Safety and Tolerability of Mavacamten in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy
Bristol-Myers Squibb20 个研究点 分布在 1 个国家目标入组 38 人开始时间: 2022年8月19日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 38
- 试验地点
- 20
- 主要终点
- Change From Baseline in Post-exercise Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30
研究概览
简要总结
The purpose of this study is to evaluate the effectiveness, safety, and tolerability of a 30-week course of mavacamten and the long-term effects of mavacamten in Japanese participants with symptomatic obstructive hypertrophic cardiomyopathy (HCM).
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Age 18 and greater, body weight ≥ 35kg
- •Has adequate acoustic windows to enable accurate transthoracic echocardiograms (TTEs)
- •Diagnosed with obstructive hypertrophic cardiomyopathy consistent with current American College of Cardiology Foundation/American Heart Association, European Society of Cardiology, and Japanese Circulation Society guidelines
- •Has documented left ventricular ejection fraction (LVEF) ≥60% NYHA Class II or III
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 atrial fibrillation with atrial fibrillation present at the time of 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 cibenzoline, disopyramide or ranolazine
- •Treatment (within 14 days prior to Screening) or planned treatment during the study with a combination of beta blockers and verapamil or a combination of beta blockers and diltiazem
- •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
- •Other protocol-defined inclusion/exclusion criteria apply
研究组 & 干预措施
Mavacamten
Experimental
干预措施: Mavacamten (Drug)
结局指标
主要结局
Change From Baseline in Post-exercise Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30
时间窗: At Baseline and Week 30
The post-exercise LVOT gradient was measured from echocardiograms obtained at baseline and week 30 following a study-specified exercise protocol and read by the doppler echocardiography. Baseline is defined as the last non-missing assessment prior to the first dose of the study treatment if both the time of the measurement and the time of first dose are available otherwise it is the last non-missing assessment on or prior to the first dose of the study treatment.
次要结局
- Change From Baseline in Kansas City Cardiomyopathy Questionnaire 23-item Version (KCCQ-23) Clinical Summary Score (CSS) at Week 30(At Baseline and Week 30)
- Percentage of Participants With at Least 1 Class Improvement in New York Heart Association (NYHA) Functional Class From Baseline to Week 30(Baseline and at Week 30)
- Change From Baseline in N-terminal Pro B-type Natriuretic Peptide (NT-proBNP) at Week 30(At baseline and week 30)
- Change From Baseline in Cardiac Troponin I at Week 30(At baseline and week 30)
- Change From Baseline in Cardiac Troponin T at Week 30(At baseline and week 30)
研究者
研究点 (20)
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