An Open Label, Single-armed, Exploratory Study of Simvastatin Therapy on the Cardiac Function in Patients With Dilated Cardiomyopathy.
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound.
研究概览
简要总结
Dilated cardiomyopathy (DCM) is the most common childhood cardiomyopathy and is associated with significant early morbidity and mortality. About half of patients die or require heart transplantation within 5 years of diagnosis. The medical therapy for DCM with heart failure includes anti-congestive medications and antiplatelet therapy. Those who fail to improve within the first year of diagnosis usually deteriorated even upon aggressive anti-congestive medications. The investigators had conducted precision-medicine-based approach to provide strategic approach as drug repurposing to identify new treatments. The investigators have identified the beneficial effects from a statin, simvastatin, to restore the cardiac contractility. The investigators would further assess the efficacy of simvastatin to improve the cardiac function in patients with DCM.
详细描述
Dilated cardiomyopathy (DCM) is the most common childhood cardiomyopathy and is associated with significant early morbidity and mortality. About half of patients die or require heart transplantation within 5 years of diagnosis. The survival advantage from transplantation is limited, particularly in DCM infants.
The medical therapy for DCM with heart failure includes anti-congestive medications and antiplatelet therapy. Those who fail to improve within the first year of diagnosis usually deteriorated even upon aggressive anti-congestive medications. The investigators had conducted precision-medicine-based approach to provide strategic approach as drug repurposing to identify new treatments. The investigators have identified the beneficial effects from a statin, simvastatin, to restore the cardiac contractility in a DCM proband.The initial experience in the proband is promising.
Simvastatin is effective in lowing LDL and cholesterol, thereby to improve the outcome of patients with coronary arterial disease, familiar hypercholesterolemia, etc. For children, though the dosage range and the indication remain unclear, it had been used in children with various diseases. Simvastatin had been given in a small cohort of adult DCM. Patients treated with simvastatin had a lower New York Heart Association functional class compared with those receiving placebo. The LVEF also improved in the simvastatin group. The investigators would further assess the efficacy of simvastatin to improve the cardiac function in patients with DCM.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who have already received anti-congestive medications for at least three months and still have compromised LV function (LVEF < 45% and the Z score of the LV end-diastolic diameter > 2.0).
- •Patients who have persistent or even worsening heart failure after one month of anti-congestive medications.
- •Patients who have positive family history of dilated cardiomyopathy and have received anti-congestive medications for one week.
排除标准
- •Patients who underwent prior cardiac surgery. Those who received DCM related cardiac surgery, such as mitral valve plasty, for longer than a year are not subject to this restriction.
- •Patients who had liver / renal dysfunction.
- •Patients who are pregnant or plan to pregnancy in the period of study.
- •Patients who are intolerance to simvastatin therapy.
研究组 & 干预措施
Simvastatin
Simvastatin, 0.5mg/kg/d(maximum 20mg), once daily
干预措施: Simvastatin (Drug)
结局指标
主要结局
Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound.
时间窗: baseline, 24th month(The 24th month follow-up time tolerates a 1-month window )
Change from baseline in N-terminal pro-brain natriuretic peptide level.natriuretic peptide level.
时间窗: baseline, 24th month(The 24th month follow-up time tolerates a 1-month window )
次要结局
- Number of participants with treatment-related adverse events as assessed by CTCAE v3.0(Up to 24 months)