International Consortium for Multimodality Phenotyping in Adults With Non-compaction
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 600
- 试验地点
- 10
- 主要终点
- Incidence of hard embolic adverse events
研究概览
简要总结
Non-compaction cardiomyopathy (NCCM) is a heterogeneous, poorly understood disorder characterized by a prominent inner layer of loose myocardial tissue, and associated with heart failure, stroke, severe rhythm irregularities and death. For a growing population diagnosed with NCCM there is a need for better risk stratification to appropriately allocate (or safely withhold) these impactful preventive measures. The goal of this international consortium is to improve care of patients with non-compaction cardiomyopathy. We hypothesize that comprehensive analysis of clinical, genetic, structural and functional information will improve risk stratification. In addition, we hypothesize that detailed structural analysis will allow for differentiation of pathological and benign patterns of non-compaction. In a large cohort of adult patients with suspected NCCM we will perform in-depth phenotyping, including clinical information, pedigree data, genetics, echocardiography and MRI, and follow patients for up to 3 years. We will apply machine-learning based analytics to develop predictive models and compare their performance to currently used models and treatment criteria. Secondly, in a subset of patients we will perform high-resolution cardiac CT for detailed structural characterization of the myocardial wall. We will investigate associations between myocardial structure and regional contractile function, as assessed by echo and MRI. The aim of this proposal is to identify a structural signature associated with pathological non-compaction and improve developed risk prediction models. Discovery of pathological structural signatures through innovative imaging techniques, in relation to myocardial contractility, will advance our understanding of NCCM.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •≥18 years old
- •Hypertrabeculation of the left ventricle fulfilling the echo-based Jenni criteria of NCCM
- •Clinical cardiac MRI examination performed or planned
排除标准
- •(general cohort):
- •Complex congenital disease (including transposition great arteries, tetralogy of Fallot, tricuspid atresia, truncus arteriosis, single ventricle, hypoplastic left heart, pulmonary atresia, double-outlet RV), neuromuscular disorders or isolated RV non-compaction
- •Inability to provide informed consent
- •Contra-indications to MRI, which apply if the clinical cardiac MRI has not yet been performed at the time of study enrollment: permanent pacemakers/ICDs, MRI contrast medium allergy, significant arrhythmia with highly irregular RR intervals, severe dyspnea with inability to lay flat/breath hold, inability to communicate with the MRI technician or follow commands for any reason (psychosis, agitation, etc.), other site-specific contra-indications to clinical MRI of the heart.
- •Exclusion Criteria (cardiac CT examination):
- •Age <21 years
- •Decompensated heart failure, or otherwise clinically unstable
- •BMI>40 kg/m2
- •Pregnancy (or cannot be ruled out)
- •Known iodine contrast medium allergy
- •Kidney dysfunction: eGFR<45 ml/min
- •Thyroid disease: toxic multinodular goiter, Graves' disease, Hashimoto's thyroiditis
研究组 & 干预措施
Multimodality imaging
Patients who have undergone echocardiography and cardiac MRI as part of their clinical management A research cardiac CT scan will be performed in eligible participants
干预措施: Echocardiography (Diagnostic Test)
Multimodality imaging
Patients who have undergone echocardiography and cardiac MRI as part of their clinical management A research cardiac CT scan will be performed in eligible participants
干预措施: Magnetic resonance imaging (MRI) (Diagnostic Test)
Multimodality imaging
Patients who have undergone echocardiography and cardiac MRI as part of their clinical management A research cardiac CT scan will be performed in eligible participants
干预措施: Computed tomography (CT) (Diagnostic Test)
结局指标
主要结局
Incidence of hard embolic adverse events
时间窗: Up to 4 years after enrollment
Clinical neuro/systemic embolic event by autopsy, imaging or specialist evaluation
Incidence of hard arrhythmic adverse events
时间窗: Up to 4 years after enrollment
Sudden death (aborted), appropriate ICD discharge or VT/VF on ECG or rhythm/device monitoring
次要结局
- Incidence of composite of adverse events(Up to 4 years after enrollment)
- Incidence of hard heart failure related adverse events(Up to 4 years after enrollment)
- Incidence of composite of hard adverse events(Up to 4 years after enrollment)
- Incidence of all embolic adverse events(Up to 4 years after enrollment)
- Incidence of all arrhythmic adverse events(Up to 4 years after enrollment)
- Incidence of all heart failure related adverse events(Up to 4 years after enrollment)
研究者
Koen Nieman
Associate Professor of Medicine
Stanford University