Bisoprolol for Early Cardiomyopathy in Duchenne Muscular Dystrophy: a Randomized, Controlled Trial
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 42
- 试验地点
- 1
- 主要终点
- Calculate the change of left ventricle global longitudinal strain in cardiac MR
研究概览
简要总结
This study aimed to use cardiac magnetic resonance imaging (CMR) to evaluate the efficacy and safety of bisoprolol therapy for boys with Duchenne muscular dystrophy(DMD) and preserved ejection fraction. On top of angiotensin-converting enzyme inhibitor (ACEI) , half of the participants will receive bisoprolol in combination, while the other half will not receive any beta-blocker.
详细描述
By the age of 20 years, almost all the patients with Duchenne muscular dystrophy(DMD) have experienced dilated cardiomyopathy (DCM), a condition that contributes significantly to their morbidity and mortality. Studies have shown ACEI to be an effective therapy for DMD boys with early cardiomyopathy. Although bisoprolol has been demonstrated as an effective neurohumoral drug for adult patients with DCM, few data exist concerning its safety and efficacy for the patients with DMD. The investigators hypothesize that the treatment with bisoprolol in addition to background ACEI therapy for 12 months in participants with DMD and early myocardial damage evident with late gadolinium enhancement and preserved ejection fraction, would blunt decline in left ventricular systolic performance.
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
- Single (Outcomes Assessor)
盲法说明
The investigator, care providers and participants will know whether the participants use the trial drug（bisoprolol） or not. But the outcome assessor who will analysis the results of the cardiac MR and echocardiography images from the participants will be blinded to the grouping situation.
入排标准
- 年龄范围
- 7 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Older than(including) 7 years old
- •A definite diagnosis of DMD with muscle pathology confirming the expression of dystrophin lower than 5% and/or confirmed mutations in the DMD gene using a clinical accepted technique that completely defines the mutation.
- •Using ACEI or ARB for more than 1 month
- •Confirmed myocardial damage in one or more left ventricular segments evident by late gadolinium enhancement and preserved left ventricular systolic function(>45%) by cine cardiac MR in 45 days
- •Normal renal function
- •Holter and blood pressure shows no contraindication of using bisoprolol
排除标准
- •Having metal implanted in body
- •Having claustrophobia
- •Allergic to gadolinium
- •Complicated with other cardiovascular diseases
- •Medical history or Holter show bradyarrhythmia like II/III degree atrioventricular block, sick sinus syndrome etc.
- •Systolic blood pressure lower than 90mmHg or rest heart rate lower than 75bpm
- •Having COPD or asthma history
- •Having other complications: tumor, endocrine diseases
- •Having beta blockers therapy
- •Planned operation in the future 12 months
- •Allergic to bisoprolol
研究组 & 干预措施
bisoprolol fumarate
In this arm, the participants will receive different dose of bisoprolol fumarate.
干预措施: Bisoprolol Fumarate (Drug)
结局指标
主要结局
Calculate the change of left ventricle global longitudinal strain in cardiac MR
时间窗: baseline and 12 months
Calculate the change of left ventricle global longitudinal strain in cardiac MR from baseline to 12months for each patients
次要结局
- Calculate the change of left ventricular ejection fraction in cardia MR(baseline and 12 months)
- Calculate the change of the level of high-sensitivity cardiac troponin I(baseline and 6months, 12 months)
- Change of the resting heart rate(baseline and 6months, 12 months)
- Calculate the change of the level of NT-proBNP(baseline and 6months, 12 months)
- Calculate the change of E/A ratio assessed by echocardiography(baseline and 12 months)
- Calculate the change of ventricle late gadolinium enhancement area in cardia MR(baseline and 12 months)
研究者
Xiaoxiao Guo
Associated professor
Peking Union Medical College Hospital