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

Sponsor:

Collaborators:

Information provided by (Responsible Party):

，

Tracking Information

First Submitted Date ^ICMJE

August 7, 2018

First Posted Date ^ICMJE

October 4, 2018

Last Update Posted Date

October 4, 2018

Actual Study Start Date ^ICMJE

September 25, 2017

Estimated Primary Completion Date

December 2018 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

hiPSC-cardiomyocytes culture[ Time Frame: Inclusion visit ]

Blood test with generated hiPSC-cardiomyocytes

Original Primary Outcome Measures ^ICMJE

Same as current

Current Secondary Outcome Measures ^ICMJE

[ Time Frame: ]

Descriptive Information

Brief Title ^ICMJE

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

Official Title ^ICMJE

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

Brief Summary

Detailed Description

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 ^ICMJE

Interventional

Study Phase

N/A

Study Design ^ICMJE

Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: All participants receive the same intervention throughout the protocol, no matter if they are "healthy children" or "cardiomyopathic children".
Masking: Interventional
Masking Description:Open : no masking us used. All involved know the identity of the intervention assignment.
Primary Purpose: Basic Science

Condition ^ICMJE

Intervention ^ICMJE

Other: Electrocardiogram
heart testing
Other: physical examination
done by the investigator
Other: echocardiography
heart testing
Biological: blood test
A sample of blood will be taken to each patient or healthy children in order to generate hiPSC-cardiomyocytes

Study Arms

Other: Healthy children
Blood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography.
Other: Cardiomyopathic children
Blood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

Original Estimated Enrollment ^ICMJE

Same as current

Estimated Study Completion Date

December 2020

Estimated Primary Completion Date

December 2018 (Final data collection date for primary outcome measure)

Eligibility Criteria ^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)

Sex/Gender

Sexes Eligible for Study:

All

Ages

N/A and older (Adult, Older Adult)

Accepts Healthy Volunteers

Listed Location Countries ^ICMJE

France

Removed Location Countries

Administrative Information

Has Data Monitoring Committee

U.S. FDA-regulated Product

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

Plan to Share IPD:

Responsible Party

，

Study Sponsor ^ICMJE

University Hospital, Montpellier

Collaborators ^ICMJE

Investigators ^ICMJE

PRS Account

Verification Date

October 2018

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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