Using Novel Blood and Imaging Biomarkers to Better Understand the Pathophysiology of Paediatric Dilated Cardiomyopathy
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 试验地点
- 1
- 主要终点
- Fibrosis
研究概览
简要总结
This will be a cross-sectional, observational study.
Null hypothesis:
There is no difference in the amount of extracellular volume (ECV or scarring) in the hearts of patients with heart failure as compared to control subjects.
Heart failure occurs when the heart muscle has become too weak to work properly. It is associated with an increase in the amount of connective tissue (collagen) which replaces dead heart muscle cells (scarring). Currently a biopsy of the muscle is the only way to measure the amount of scarring. This is invasive and rarely done in children. Because of this, it is difficult to measure the amount of scarring in a particular patient or disease process, which is important for improving our understanding and treatment of the disease.
Cardiac magnetic resonance imaging (MRI) is a non-invasive imaging tool which is routinely used to look at areas of local scarring in heart muscle. Because the scarring is so widespread in paediatric patients, we have not been able to use this method previously. Now new imaging techniques allow us to look at widespread scarring but these have not yet been validated in children.
We plan to use late gadolinium enhancement (T1 mapping) to measure the amount of scarring in patients with heart failure (we have evidence that their heart biopsies show increased amounts of scar tissue) and children having MRI scans for other reasons. We will use measures of function including echocardiography and 6 minute walk test to compare to the amount of scarring. This will help us to know whether the amount of scarring will be clinically useful.
We will look at the amount of various proteins in the blood of patients and control subjects which are related to the scarring and cell death processes. We already use blood tests to monitor heart failure and these tests may help us to refine our testing and improve timing of treatment (e.g. transplantation).
This study will help us to design further research in this field.
详细描述
All patients will be under the care of Great Ormond Street Hospital.
Patient Groups:
There will be three main patient groups:
-
Heart Failure Patients (MRI Clinically indicated)
-
Heart Failure Patients (Voluntarily recruited from clinic)
-
8-16 years of age
-
Non-GA only
-
Control subjects (Clinically indicated brain MRI with contrast)
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Cross Sectional
入排标准
- 年龄范围
- — 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Established diagnosis of DCM for 3 months
- •Ability to cooperate with MRI scan without general anaesthesia (volunteers over 8 years), or any age if cardiac MRi clinically indicated
- •Provides written, informed consent
排除标准
- •Patient exclusion criteria:
- •Estimated GFR <30mls/min
- •Contraindication to MRI (see appendix 1)
- •Chronic inflammation/ malignancy/ connective tissue disease
- •Structural congenital heart disease/ previous cardiac surgery
- •Control Group Exclusion Criteria:
- •History of heart failure or congenital heart disease
- •Contraindication to MRI (see below)
- •Chronic inflammation/ malignancy/ connective tissue disease
- •Previous malignancy
- •Exclusion criteria for MRI
- •Central nervous system aneurysm clips
- •Implanted neural stimulator
- •Implanted cardiac pacemaker or defibrillator
- •Cochlear implant
- •Ocular foreign body e.g. metal shavings
- •Other implanted medical devices e.g. drug infusion ports
- •Insulin pump
- •Metal shrapnel or bullet
- •Pregnant women (patients who are uncertain will be required to have a urinary or blood screening test)
结局指标
主要结局
Fibrosis
时间窗: 6 months
Higher fibrosis score (ECV) in heart failure patients in comparison to control subjects
次要结局
- Disease Severity(6 months)
- Biomarkers(6 months)