Cardiomyopathy, FamilialDMDstemNCT03696628
Modeling and Pharmacological Targeting of Genetic Cardiomyopathy in Children Via Cardiomyocytes Derived From Induced Pluripotent Stem Cells (DMDstem)
A study testing Electrocardiogram, physical examination and echocardiography in people with Cardiomyopathy, Familial, run by University Hospital, Montpellier at 1 site in France.
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. Read more about this study
- Who is it for?
- Ages 0 to 17 yearsHealthy volunteers can take part
- Conditions the study is for
- Cardiomyopathy, Familial
- What is being tested?
- Electrocardiogram, physical examination and echocardiography
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteFrance
- Study dates
- 25 Sep 2017to 20 Jan 2021
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 17 years
- All sexes
- Healthy volunteers accepted
- Child
Who may be able to join
- 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
Show all 14Show fewer
- 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)
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Modeling and Pharmacological Targeting of Genetic Cardiomyopathy in Children Via Cardiomyocytes Derived From Induced Pluripotent Stem Cells
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.
Read the 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
- Being tested
- Electrocardiogram, physical examination, echocardiography, blood test
- Keywords
- Cardiomyocytes, Induced pluripotent stem cells, Genetic cardiomyopathy
- Medical subject headings
- Cardiomyopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ElectrocardiogramOther
heart testing
physical examinationOther
done by the investigator
echocardiographyOther
heart testing
blood testBiological
A sample of blood will be taken to each patient or healthy children in order to generate hiPSC-cardiomyocytes
| Study group | Role | What they receive |
|---|---|---|
| Healthy children | Other | Blood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography. Receives: Other: Electrocardiogram; Other: physical examination; Other: echocardiography; Biological: blood test |
| Cardiomyopathic children | Other | Blood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography. Receives: Other: Electrocardiogram; Other: physical examination; Other: echocardiography; Biological: blood test |
What is being measured
The primary outcome is the main question the study is designed to answer.
hiPSC-cardiomyocytes culture
Blood test with generated hiPSC-cardiomyocytes
Measured: Inclusion visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Oct 2018
- Study start25 Sep 2017
- Primary completion20 Jan 2021
- Study completion20 Jan 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cardiomyopathy, FamilialStudies of the same condition, open and under way.
- More trials run by University Hospital, MontpellierThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- 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)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Montpellier
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03696628
- Sponsor's protocol ID
- 9806
- Other IDs
- 2017-A01589-44
- Acronym
- DMDstem
- Status last verified
- Jul 2022
- First submitted
- 7 Aug 2018
- Last updated
- 14 Dec 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
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.
PubMed 33246213
Study at a glance
From the registry record, last updated by the sponsor on 14 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Modeling and Pharmacological Targeting of Genetic Cardiomyopathy in Children Via Cardiomyocytes Derived From Induced Pluripotent Stem Cells
- ClinicalTrials.gov ID
- NCT03696628
- Acronym / sponsor ID
- DMDstem / 9806
- Conditions
- Cardiomyopathy, Familial
- Keywords
- Cardiomyocytes, Induced pluripotent stem cells, Genetic cardiomyopathy
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- University Hospital, MontpellierUniversity, hospital or other
- Study start
- 25 Sep 2017 (actual)
- Primary completion
- 20 Jan 2021 (actual)
- Study completion
- 20 Jan 2021 (actual)
- First posted
- 4 Oct 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- All participants receive the same intervention throughout the protocol, no matter if they are "healthy children" or "cardiomyopathic children".
| Arm | Type | Receives |
|---|---|---|
| Healthy children | Other | Other: Electrocardiogram, Other: physical examination, Other: echocardiography, Biological: blood testBlood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography. |
| Cardiomyopathic children | Other | Other: Electrocardiogram, Other: physical examination, Other: echocardiography, Biological: blood testBlood test with generated hiPSC-cardiomyocytes Physical Examination. Electrocardiogram. Echocardiography. |
Who may qualify
- Ages
- 0 to 17 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
Key inclusion criteria (14 listed)
- 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.)
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Electrocardiogramheart testing | Other | Cardiomyopathic children, Healthy children |
| physical examinationdone by the investigator | Other | Cardiomyopathic children, Healthy children |
| echocardiographyheart testing | Other | Cardiomyopathic children, Healthy children |
| blood testA sample of blood will be taken to each patient or healthy children in order to generate hiPSC-cardiomyocytes | Biological | Cardiomyopathic children, Healthy children |
Primary outcome
hiPSC-cardiomyocytes culture
Time frame: Inclusion visit
Blood test with generated hiPSC-cardiomyocytes
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Dec 2022.
- Results publication: PubMed 33246213Souidi 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Electrocardiogram, physical examination and echocardiography in people with Cardiomyopathy, Familial, run by University Hospital, Montpellier at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
hiPSC-cardiomyocytes culture (time frame: Inclusion visit). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03696628, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Dec 2022.