Induced Pluripotent Stem CellsID-STEMNCT05214742
Developing Derived Induced Pluripotent Stem Cells as a Model to Understand Imprinted Disorders
An observational study of Induced Pluripotent Stem Cells, run by Institute of Cardiometabolism and Nutrition, France at 1 site in France.
Trial summary
Fetal and postnatal growth is finely regulated by genetic, epigenetic and environmental mechanisms. Parental imprinting is a regulatory mechanism that allows monoallelic expression of certain genes from a single parental allele through differential DNA methylation. Read more about this study
- Who is it for?
- Ages 3 months and older
- Conditions the study is for
- Induced Pluripotent Stem Cells
- What is being tested?
- Diagnostic Test
- How many people
- 20 participants
- Where
- 1 siteFrance
- Study dates
- 19 Jan 2022to Dec 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 3 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: During their follow-up visit, parents and patients who meet the inclusion criteria will be asked to participate in the study. In the case of minors, their personal adhesion will be sought if their capacity of comprehension allows it after adapted written information.
Who may be able to join
- Minor or young adult patients treated in the department, suffering from rare growth diseases: Silver-Russell syndrome (SRS), Beckwith-Wiedemann syndrome (BWS) and Temple syndrome (TS)
- For minors, the patient's weight must be ≥ 5 kg
Who cannot join
- Patients unable to express their opposition to the use of their personal data.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Developing Derived Induced Pluripotent Stem Cells From Blood as a Model for the Study to Understand Imprinted Disorders
Fetal and postnatal growth is finely regulated by genetic, epigenetic and environmental mechanisms. Parental imprinting is a regulatory mechanism that allows monoallelic expression of certain genes from a single parental allele through differential DNA methylation. Imprinted genes play a very important role in the control of fetal and postnatal growth. The pathophysiological mechanisms of these epimutations are largely unknown.
Studying the consequences of these epimutations on the molecular signature of the imprinted gene network in these patients would provide a better understanding of the epigenetic mechanisms regulating fetal growth. As these genes are weakly expressed in fibroblasts, these studies will be carried out on pluripotent stem cells or IPSCs (Induced Pluripotent Stem Cells).
- Conditions
- Induced Pluripotent Stem Cells
- Being tested
- Diagnostic Test
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Diagnostic TestOther
Molecular diagnosis carried out in the context of care
| Study group | Role | What they receive |
|---|---|---|
| Silver-Russell Syndrome | Not described Receives: Other: Diagnostic Test | |
| Beckwith-Wiedemann Syndrome | Not described Receives: Other: Diagnostic Test | |
| Temple Syndrome | Not described Receives: Other: Diagnostic Test |
What is being measured
The primary outcome is the main question the study is designed to answer.
The objective of the study is to understand the consequences of epimutations found
The objective of the study is to understand the consequences of epimutations found at 11p15 or 14q32 in these three syndromes (SRS, BWS and TS) on the network of genes subject to parental imprinting, in order to progress in the understanding of the mechanisms governing the epigenetic regulation of fetal growth.
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2022
- Study start19 Jan 2022
- Primary completion30 Jan 2022
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Induced Pluripotent Stem CellsStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Institute of Cardiometabolism and Nutrition, France
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Irène NETCHINE, Principal investigator, Institut National de la Santé Et de la Recherche Médicale, France
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05214742
- Sponsor's protocol ID
- 2021-A01597-34
- Acronym
- ID-STEM
- Status last verified
- Jan 2022
- First submitted
- 17 Jan 2022
- Last updated
- 28 Apr 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: IPD will be sahred with partners
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Developing Derived Induced Pluripotent Stem Cells From Blood as a Model for the Study to Understand Imprinted Disorders
- ClinicalTrials.gov ID
- NCT05214742
- Acronym / sponsor ID
- ID-STEM / 2021-A01597-34
- Conditions
- Induced Pluripotent Stem Cells
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Institute of Cardiometabolism and Nutrition, FranceUniversity, hospital or other
- Study start
- 19 Jan 2022 (actual)
- Primary completion
- 30 Jan 2022 (actual)
- Study completion
- Dec 2026 (estimated)
- First posted
- 31 Jan 2022 (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.
Fetal and postnatal growth is finely regulated by genetic, epigenetic and environmental mechanisms. Parental imprinting is a regulatory mechanism that allows monoallelic expression of certain genes from a single parental allele through differential DNA methylation. Imprinted genes play a very important role in the control of fetal and postnatal growth. The pathophysiological mechanisms of these epimutations are largely unknown.
Studying the consequences of these epimutations on the molecular signature of the imprinted gene network in these patients would provide a better understanding of the epigenetic mechanisms regulating fetal growth. As these genes are weakly expressed in fibroblasts, these studies will be carried out on pluripotent stem cells or IPSCs (Induced Pluripotent Stem Cells).
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Silver-Russell Syndrome | – | Other: Diagnostic Test |
| Beckwith-Wiedemann Syndrome | – | Other: Diagnostic Test |
| Temple Syndrome | – | Other: Diagnostic Test |
Who may qualify
- Ages
- 3 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- During their follow-up visit, parents and patients who meet the inclusion criteria will be asked to participate in the study. In the case of minors, their personal adhesion will be sought if their capacity of comprehension allows it after adapted written information.
Key inclusion criteria (2 listed)
- Minor or young adult patients treated in the department, suffering from rare growth diseases: Silver-Russell syndrome (SRS), Beckwith-Wiedemann syndrome (BWS) and Temple syndrome (TS)
- For minors, the patient's weight must be ≥ 5 kg
Key exclusion criteria (1 listed)
- Patients unable to express their opposition to the use of their personal data.
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 |
|---|---|---|
| Diagnostic TestMolecular diagnosis carried out in the context of care | Other | Beckwith-Wiedemann Syndrome, Silver-Russell Syndrome, Temple Syndrome |
Primary outcome
The objective of the study is to understand the consequences of epimutations found
Time frame: 1 day
The objective of the study is to understand the consequences of epimutations found at 11p15 or 14q32 in these three syndromes (SRS, BWS and TS) on the network of genes subject to parental imprinting, in order to progress in the understanding of the mechanisms governing the epigenetic regulation of fetal growth.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2022.
- Individual participant dataSharing planned: Yes. IPD will be sahred with partners
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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.
Irène NETCHINE principal investigator
Institut National de la Santé Et de la Recherche Médicale, France
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Induced Pluripotent Stem Cells, run by Institute of Cardiometabolism and Nutrition, France 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 2 inclusion and 1 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: enrolling by invitation, as of the sponsor's last update on 28 Apr 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?
This study is still under way. Results are not available in the source record yet.
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?
The objective of the study is to understand the consequences of epimutations found (time frame: 1 day). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 28 Apr 2022.