Modeling and Pharmacological Targeting of Genetic Cardiomyopathy in Children Via Cardiomyocytes Derived From Induced Pluripotent Stem Cells (DMDstem)

University Hospital, Montpellier24 enrolled

Overview

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).

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

BASIC_SCIENCE

Masking

NONE

Enrollment

Conditions

Cardiomyopathy, Familial

Interventions

ElectrocardiogramOTHER

heart testing

physical examinationOTHER

done by the investigator

echocardiographyOTHER

heart testing

blood test

Eligibility

Sex: ALLMin age: 0 YearsMax age: 17 YearsHealthy volunteers:

Medical Language ↔ Plain English

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)

Locations (1)

CHU Arnaud de Villeneuve

Montpellier, Occitanie, France

Outcomes

Primary Outcomes

hiPSC-cardiomyocytes culture

Blood test with generated hiPSC-cardiomyocytes

Time frame: Inclusion visit

Data from ClinicalTrials.gov

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