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

Cardiomyopathy, Familial Clinical Trial

— DMDstem  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03696628
• Other study ID #: 9806
• Secondary ID: 2017-A01589-44
• Status: Completed
• Phase: N/A
• Start date: September 25, 2017
• Est. completion date: January 20, 2021

Study information

• Verified date: July 2022
• Source: University Hospital, Montpellier
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 24
• Est. completion date: January 20, 2021
• Est. primary completion date: January 20, 2021
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 0 Years to 17 Years

Eligibility

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)

Related Conditions & MeSH terms

• Cardiomyopathies
• Cardiomyopathy, Familial

Intervention

Other:
• Electrocardiogram
heart testing
• physical examination
done by the investigator
• 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

Locations

Country	Name	City	State
France	CHU Arnaud de Villeneuve	Montpellier	Occitanie

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Montpellier

Country where clinical trial is conducted

France, 

References & Publications (1)

Souidi M, Amedro P, Meyer P, Desprat R, Lemaitre JM, Rivier F, Lacampagne A, Meli AC. Generation of three Duchenne Muscular Dystrophy patient-specific induced pluripotent stem cell lines DMD_YoTaz_PhyMedEXp, DMD_RaPer_PhyMedEXp, DMD_OuMen_PhyMedEXp (INSRM — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	hiPSC-cardiomyocytes culture	Blood test with generated hiPSC-cardiomyocytes	Inclusion visit