Modeling and Pharmacological Targeting of Genetic Cardiomyopathy in Children Via Cardiomyocytes Derived From Induced Pluripotent Stem Cells
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 24
- 试验地点
- 1
- 主要终点
- hiPSC-cardiomyocytes culture
研究概览
简要总结
Interventional, cross-sectional biomedical study of children with genetic cardiomyopathy and healthy children. The aim is to generate, via induced human pluripotent stem cells (hiPSC), "patient-specific" cardiomyocytes (CMs) (hiPSC-CMs) to study the molecular mechanisms of cardiomyopathies of genetic origin.
详细描述
Interventional, cross-sectional biomedical study of children with genetic cardiomyopathy and healthy children. The aim is to generate, via induced human pluripotent stem cells (hiPSC), "patient-specific" cardiomyocytes (CMs) (hiPSC-CMs) to study the molecular mechanisms of cardiomyopathies of genetic origin.
The study will be proposed to the parents or legal guardians of the children from 0 to 17 included sent in pediatric cardiology consultation to the University Hospital of Montpellier as part of their usual follow-up or a health check (control) .
The only direct intervention performed on the patient is a venous blood sample. The volume of blood collected will be lower than the thresholds defined in the Decree of December 2nd, 2016 on minimal risks in biomedical research (3 ml).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Basic Science
- 盲法
- None
盲法说明
Open : no masking us used. All involved know the identity of the intervention assignment.
入排标准
- 年龄范围
- 0 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Child from 0 to 17 years old included
- •Bearer or at risk of cardiomyopathy of genetic origin. Written and informed consent of parents or guardians of legal guardians
- •Affiliation or beneficiary of a social security scheme
- •Criterion of non-inclusion
- •. Cardiomyopathy of non-genetic origin (metabolic, toxic, malformative, etc.)
- •Group 2 : Healthy child
- •Inclusion criteria
- •Children aged 0 to 17 years old
- •Normal assessment: clinical examination, ECG, echocardiography
- •Written and informed consent
- •Affiliation or beneficiary of a social security scheme
- •Criterion of non-inclusion
- •Heart, muscle or respiratory disease
- •Treatment with cardiac resonance
- •Other chronic diseases (diabetes, neuropathy, kidney failure, tumor)
排除标准
- 未提供
研究组 & 干预措施
Healthy children
Blood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography.
干预措施: physical examination (Other)
Healthy children
Blood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography.
干预措施: echocardiography (Other)
Cardiomyopathic children
Blood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography.
干预措施: Electrocardiogram (Other)
Healthy children
Blood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography.
干预措施: blood test (Biological)
Healthy children
Blood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography.
干预措施: Electrocardiogram (Other)
Cardiomyopathic children
Blood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography.
干预措施: physical examination (Other)
Cardiomyopathic children
Blood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography.
干预措施: echocardiography (Other)
Cardiomyopathic children
Blood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography.
干预措施: blood test (Biological)
结局指标
主要结局
hiPSC-cardiomyocytes culture
时间窗: Inclusion visit
Blood test with generated hiPSC-cardiomyocytes
次要结局
未报告次要终点