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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

Tracking Information
First Submitted Date  ICMJE August 7, 2018
First Posted Date  ICMJE October 4, 2018
Last Update Posted Date January 24, 2019
Actual Study Start Date  ICMJE September 25, 2017
Estimated Primary Completion Date December 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 3, 2018)
hiPSC-cardiomyocytes culture [ Time Frame: Inclusion visit ]
Blood test with generated hiPSC-cardiomyocytes
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03696628 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
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 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).

Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
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: None (Open Label)
Masking Description:
Open : no masking us used. All involved know the identity of the intervention assignment.
Primary Purpose: Basic Science
Condition  ICMJE Cardiomyopathy, Familial
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  ICMJE
  • Healthy children
    Blood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography.
    Interventions:
    • Other: Electrocardiogram
    • Other: physical examination
    • Other: echocardiography
    • Biological: blood test
  • Cardiomyopathic children
    Blood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography.
    Interventions:
    • Other: Electrocardiogram
    • Other: physical examination
    • Other: echocardiography
    • Biological: blood test
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 3, 2018)
24
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2020
Estimated Primary Completion Date December 2019   (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  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE
Contact: Pascal AMEDRO, PhD +33 (0) 4 67 33 66 39 p-amedro@chu-montpellier.fr
Listed Location Countries  ICMJE France
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03696628
Other Study ID Numbers  ICMJE 9806
2017-A01589-44 ( Registry Identifier: ID-RCB )
Has Data Monitoring Committee No
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  ICMJE Not Provided
Responsible Party University Hospital, Montpellier
Study Sponsor  ICMJE University Hospital, Montpellier
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account University Hospital, Montpellier
Verification Date January 2019

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