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

December 13, 2022 updated by: University Hospital, Montpellier

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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

Interventional

Enrollment (Actual)

24

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
    • Occitanie
      • Montpellier, Occitanie, France, 34090
        • CHU Arnaud de Villeneuve

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 15 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Basic Science
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: Healthy children
Blood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography.
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
Other: Cardiomyopathic children
Blood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography.
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
hiPSC-cardiomyocytes culture
Time Frame: Inclusion visit
Blood test with generated hiPSC-cardiomyocytes
Inclusion visit

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University Hospital, Montpellier

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 25, 2017

Primary Completion (Actual)

January 20, 2021

Study Completion (Actual)

January 20, 2021

Study Registration Dates

First Submitted

August 7, 2018

First Submitted That Met QC Criteria

October 3, 2018

First Posted (Actual)

October 4, 2018

Study Record Updates

Last Update Posted (Actual)

December 14, 2022

Last Update Submitted That Met QC Criteria

December 13, 2022

Last Verified

July 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 9806
  • 2017-A01589-44 (Registry Identifier: ID-RCB)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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