Modeling and Pharmacological Targeting of Genetic Cardiomyopathy in Children Via Cardiomyocytes Derived From Induced Pluripotent Stem Cells (DMDstem)
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University Hospital, Montpellier
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| 追踪信息 | |||
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| 首次提交日期 ICMJE | August 7, 2018 | ||
| 首次发布日期e ICMJE | October 4, 2018 | ||
| 最后更新发布日期 | October 4, 2018 | ||
| 预计研究开始日期 ICMJE | September 25, 2017 | ||
| 预计主要完成日期 | December 2018 (主要结果测量的最终数据收集日期) | ||
| 目前主要观察指标 ICMJE |
hiPSC-cardiomyocytes culture[ Time Frame: Inclusion visit ] Blood test with generated hiPSC-cardiomyocytes |
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| 原始主要观察测量 ICMJE | 与当前相同 | ||
| 目前的二级观察 ICMJE |
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| 描述性信息 | |||
| 简略标题 ICMJE | Modeling and Pharmacological Targeting of Genetic Cardiomyopathy in Children Via Cardiomyocytes Derived From Induced Pluripotent Stem Cells (DMDstem) | ||
| 正式标题 ICMJE | Modeling and Pharmacological Targeting of Genetic Cardiomyopathy in Children Via Cardiomyocytes Derived From Induced Pluripotent Stem Cells | ||
| 简要概况 | 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. |
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| 详细说明 | 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). | ||
| 研究类型 ICMJE | Interventional | ||
| 研究阶段 | N/A | ||
| 研究设计 ICMJE | 分配: Non-Randomized 干预模型: Parallel Assignment 干预模型描述: All participants receive the same intervention throughout the protocol, no matter if they are "healthy children" or "cardiomyopathic children". 盲法: Interventional 盲法描述:Open : no masking us used. All involved know the identity of the intervention assignment. 主要目的: Basic Science |
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| 适用条件 ICMJE | |||
| 干预项目 ICMJE |
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| 研究工具 |
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| 招募信息 | |||
| 招募状态 ICMJE | Recruiting | ||
| 预计入组 ICMJE |
24 | ||
| 原始预计入组 ICMJE | 与当前相同 | ||
| 预计研究完成日期 | December 2020 | ||
| 预计主要完成日期 | December 2018 (主要结果测量的最终数据收集日期) | ||
| 合格标准 ICMJE | Group 1 : Child with genetic cardiomyopathy Inclusion criteria - 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) | ||
| 性别 |
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| 年龄 | 最小年龄：N/A ，最大年龄：17 Years | ||
| 接受健康的志愿者 | 没有 | ||
| 可入组国家 ICMJE | France | ||
| 管理信息 | 数据检测委员会 | No | |
| 研究涉及美国FDA监管的产品 |
研究美国FDA监管的药品: No 研究涉及美国FDA监管的设备产品: No |
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| IPD 共享声明 |
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| 责任方 | ， | ||
| 研究赞助商 ICMJE | University Hospital, Montpellier | ||
| 合作者 ICMJE | |||
| 研究员 ICMJE |
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| PRS 账户 | |||
| 验证日期 | October 2018 | ||
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ICMJE 国际医学期刊编辑委员会和 世界卫生组织 ICTRP 要求的元素 |
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