NCT03696628

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

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

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: University Hospital, Montpellier · Academic / Other
• Sex: All
• Age: 0 Years – 17 Years
• Healthy volunteers: Accepted

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.

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).

Conditions

• Cardiomyopathy, Familial

Interventions

Type	Name	Description
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

Timeline

• Start date: 2017-09-25
• Primary completion: 2021-01-20
• Completion: 2021-01-20
• First posted: 2018-10-04
• Last updated: 2022-12-14

Locations

1 site across 1 country: France