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

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ClinicalTrials.gov Identifier: NCT03696628
Recruitment Status : Recruiting
First Posted : October 4, 2018
Last Update Posted : January 24, 2019
Sponsor:
Information provided by (Responsible Party):
University Hospital, Montpellier

Brief 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.

Condition or disease Intervention/treatment Phase
Cardiomyopathy, Familial Other: Electrocardiogram Other: physical examination Other: echocardiography Biological: blood test Not Applicable

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).


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
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: None (Open Label)
Masking Description: Open : no masking us used. All involved know the identity of the intervention assignment.
Primary Purpose: Basic Science
Official Title: Modeling and Pharmacological Targeting of Genetic Cardiomyopathy in Children Via Cardiomyocytes Derived From Induced Pluripotent Stem Cells
Actual Study Start Date : September 25, 2017
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cardiomyopathy

Arm Intervention/treatment
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

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




Primary Outcome Measures :
  1. hiPSC-cardiomyocytes culture [ Time Frame: Inclusion visit ]
    Blood test with generated hiPSC-cardiomyocytes



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   up to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03696628


Contacts
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Contact: Pascal AMEDRO, PhD +33 (0) 4 67 33 66 39 p-amedro@chu-montpellier.fr

Locations
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France
CHU Arnaud de Villeneuve Recruiting
Montpellier, Occitanie, France, 34090
Contact: Pascal AMEDRO, PhD    +33 (0) 4 67 33 66 39    p-amedro@chu-montpellier.fr   
Sponsors and Collaborators
University Hospital, Montpellier

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Responsible Party: University Hospital, Montpellier
ClinicalTrials.gov Identifier: NCT03696628     History of Changes
Other Study ID Numbers: 9806
2017-A01589-44 ( Registry Identifier: ID-RCB )
First Posted: October 4, 2018    Key Record Dates
Last Update Posted: January 24, 2019
Last Verified: January 2019

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

Keywords provided by University Hospital, Montpellier:
Cardiomyocytes
Induced pluripotent stem cells
Genetic cardiomyopathy

Additional relevant MeSH terms:
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Cardiomyopathies
Heart Diseases
Cardiovascular Diseases