NCT05174416进行中（未招募）3 期
A Phase III, Randomized, Double-blinded, Placebo-controlled Clinical Study With A Long-term Extension to Evaluate the Efficacy and Safety of Mavacamten in Chinese 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
- 进行中（未招募）
- 发起方
- LianBio LLC
- 入组人数
- 81
- 试验地点
- 1
- 主要终点
- Change From Baseline to Week 30 in Valsalva Left Ventricular Outflow Tract (LVOT) Peak Gradient
研究概览
简要总结
Mavacamtenis a novel, small molecule, selective allosteric inhibitor of cardiac-specific myosin, for the treatment of patients with symptomatic oHCM. This study will assess the efficacy and safety of mavacamten in Chinese adults with symptomatic oHCM.
详细描述
This is a randomized, double-blinded, placebo-controlled clinical study witha long-term extension to evaluate the efficacy and safety of mavacamten in Chinese adults with symptomatic oHCM. Approximately 81eligible participants will be enrolled and randomized in a 2:1 ratio (mavacamten:placebo). Participants will receive mavacamten or matching placebofor 30 weeks indouble-blinded manner. After 30-week double-blinded placebo-controlled treatment, eligible participants will receive mavacamten for additional 48 weeks (placebogroup: switch from placebo to mavacamten, mavacamten group: maintain on 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 99 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Is at least 18 years old at screening.
- •Body weight is greater than 45 kg at screening.
- •Has adequate acoustic windows to enable accurate TTEs
- •Diagnosed with oHCM
- •Has documented LVEF ≥ 55% at rest.
- •Has a valid measurement of Valsalva LVOT peak gradient at screening
- •Has NYHA Class II or III symptoms at screening
- •Female participants must not be pregnant or lactating
- •Able to understand and comply with the study procedures, understand the risks involved in the study, and provide written informed consent according to national, local, and institutional guidelines before the first study specific procedure.
排除标准
- •Participated in a clinical trial in which the participant received any investigational drug (or is currently using an investigational device) within 30 days prior to screening, or at least 5 times the respective elimination half-life (if known), whichever is longer.
- •Causing cardiac hypertrophy in other reasons
- •Previously participated in a clinical study with mavacamten.
- •Hypersensitivity to any of the components of the mavacamten formulation.
- •Current treatment (within 14 days prior to screening) or planned treatment during the double-blinded treatment with a combination of beta-blockers and verapamil or a combination of beta-blockers and diltiazem.
- •Has been successfully treated with invasive septal reduction
- •Has documented obstructive coronary artery disease
- •Has known moderate or severe (as per investigator's judgment) aortic valve stenosis, constrictive pericarditis, or clinically significant congenital heart disease at screening.
- •Has any acute or serious comorbid condition that, in the judgment of the investigator, could lead to premature termination of study participation or interfere with the measurement or interpretation of the efficacy and safety assessments in the study.
- •History of malignant disease within 10 years of screening
- •Has safety laboratory parameters outside normal limits at screening as assessed by the local laboratory
- •Has a positive serologic test at screening for infection with human immunodeficiency virus, hepatitis C virus, or hepatitis B virus surface antigen.
- •Known uncured COVID-19 (coronavirus disease 2019) infection or with severe complication before screening.
- •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 cardio toxic agents.
- •Unable to comply with the study requirements, including the number of required visits to the clinical site.
- •Is a first degree relative of personnel directly affiliated with the study at the clinical study site, any study vendor, or the study sponsor.
- •Identified as alcohol addicts.
研究组 & 干预措施
Mavacamten
Experimental
Mavacamten Capsules
干预措施: Mavacamten (Drug)
placebo
Placebo Comparator
Matching Placebo Capsules
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline to Week 30 in Valsalva Left Ventricular Outflow Tract (LVOT) Peak Gradient
时间窗: 30 weeks
To compare the effect of a 30-week course of mavacamten with placebo on Valsalva LVOT peak gradient as determined by Doppler echocardiography
次要结局
- Change From Baseline to Week 30 in Resting LVOT Peak Gradient(30 weeks)
- Proportion of Participants Achieving a Valsalva LVOT Peak Gradient < 30 mmHg at Week 30(30 weeks)
- Proportion of Participants Achieving a Valsalva LVOT Peak Gradient < 50 mmHg at Week 30.(30 weeks)
- Proportion of Participants With at Least 1 Class Improvement in New York Heart Association (NYHA) Functional Classification From Baseline to Week 30(30 weeks)
- Change From Baseline to Week 30 in Kansas City Cardiomyopathy Questionnaire (KCCQ) Clinical Summary Score (CSS)(30 weeks)
- Change From Baseline to Week 30 in N-terminal Pro-B-type Natriuretic Peptide (NT-proBNP)(30 weeks)
- Change From Baseline to Week 30 in Cardiac Troponin(30 weeks)
- Change From Baseline to Week 30 in Left Ventricular (LV) Mass Index(30 weeks)
研究者
研究点 (1)
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