Modeling and Pharmacological Targeting of Genetic Cardiomyopathy in Children Via Cardiomyocytes Derived From Induced Pluripotent Stem Cells (DMDstem)
NCT ID: NCT03696628
Last Updated: 2022-12-14
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
NA
24 participants
INTERVENTIONAL
2017-09-25
2021-01-20
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
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
See the medical conditions and disease areas that this research is targeting or investigating.
Keywords
Explore important study keywords that can help with search, categorization, and topic discovery.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NON_RANDOMIZED
PARALLEL
BASIC_SCIENCE
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Healthy children
Blood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography.
Electrocardiogram
heart testing
physical examination
done by the investigator
echocardiography
heart testing
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.
Electrocardiogram
heart testing
physical examination
done by the investigator
echocardiography
heart testing
blood test
A sample of blood will be taken to each patient or healthy children in order to generate hiPSC-cardiomyocytes
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Electrocardiogram
heart testing
physical examination
done by the investigator
echocardiography
heart testing
blood test
A sample of blood will be taken to each patient or healthy children in order to generate hiPSC-cardiomyocytes
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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
* 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)
0 Years
17 Years
ALL
Yes
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
University Hospital, Montpellier
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
CHU Arnaud de Villeneuve
Montpellier, Occitanie, France
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
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 (INSRMi008-A, INSRMi009-A and INSRMi010-A). Stem Cell Res. 2020 Dec;49:102094. doi: 10.1016/j.scr.2020.102094. Epub 2020 Nov 19.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2017-A01589-44
Identifier Type: REGISTRY
Identifier Source: secondary_id
9806
Identifier Type: -
Identifier Source: org_study_id