Evaluation of Arrhythmic Risk in Myotonic Dystrophy Type I (DM 1)
试验速览
- 阶段
- 3 期
- 入组人数
- 537
- 试验地点
- 2
- 主要终点
- Evaluate incidence of: major cardiac events (sudden death
研究概览
简要总结
This is a prospective multicentric Italian study to evaluate the arrhythmic risk in myotonic dystrophy type 1.
详细描述
Myotonic dystrophy type 1 (DM1, Steinert disease) is a multisystem disorder that affects, beside muscle, several other organs, including the heart.
Cardiac involvement represents a major problem in the clinical management of patients, so that cardiac complications represent one of the primary causes of premature death in DM1. In particular there is a high incidence of sudden death, ranging from 2 to 30% of cases, so far principally related to the development of conduction blocks. However, literature reports of sudden death in patients implanted with pacemakers, as well as of spontaneous ventricular tachycardia would suggest a potential etiologic role also for ventricular arrhythmias. The lack of clinical research studies conducted on a large number of patients does not make available definite data regarding the etiology and the epidemiology of arrhythmic events in DM1. For the same reasons, other considerable topics, such as prognostic stratification of the arrhythmic risk and clinical management of life-threatening arrhythmias in DM1 patients, are still undefined.
To clarify these issues, the investigators propose a clinical research study performed on a large cohort of DM1 patients enrolled through a multicenter collaboration that also involves 5 cardiological-neurological Italian centres.
Aims of this study are:
- To estimate the incidence of arrhythmias and to characterize the brady-tachyarrhythmic mechanisms underlying the occurrence of cardiac sudden death in DM1;
- To verify by statistical analysis the reliability of data obtained from both non invasive and invasive diagnostic procedures as indexes useful for estimating the arrhythmic risk in DM1;
- To identify more adequate therapeutic guidelines in order to prevent the occurrence of life-threatening arrhythmias.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 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 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient affected by myotonic dystrophy type I (MD1).
- •Patient willing to provide a signed informed consent.
排除标准
- •Age < 18 years old or >70 years old.
- •Ischemic cardiomyopathy
- •Cardiomyopathy due to chronic excess of alcohol consumption (>100 g\day)
- •Congenital heart disease
- •Acquired valvular heart disease
- •Metabolic cardiomyopathy: thyrotoxicosis, hypothyroidism, adrenal cortical insufficiency, pheochromocytoma, acromegaly
- •Familiar storage and infiltrative diseases (hemochromatosis, glycogen storage, Hurler's syndrome, Niemann-Pick disease; primary, secondary, familial and hereditary cardiac amyloidoses)
- •Systemic diseases (connective tissue disorder; sarcoidosis)
- •Peripartum cardiomyopathy
结局指标
主要结局
Evaluate incidence of: major cardiac events (sudden death
resuscitated cardiac arrest
ventricular fibrillation
sustained ventricular tachycardia
sinoatrial and atrioventricular [AV] blocks)
次要结局
- signal-averaged ECG
- Evaluate with diagnostic non-invasive (standard electrocardiogram [ECG]
- 24-hour monitoring ECG
- echocardiography) and invasive procedures (electrophysiology study [EPS] and implantable loop recorders) the risk to develop cardiac arrhythmias in DM patients