Marfan SyndromeMYLIFENCT04731493
Effect on the Quality of Life of a Therapeutic Education Program in Patients With Marfan Syndrome
An observational study of Marfan Syndrome, run by University Hospital, Toulouse at 1 site in France.
Trial summary
This is a before-after, observational, prospective, multicenter cohort study. The study will consist of 2 phases: an initial observational phase of a minimum of 3 months before the Therapeutic Education Program (TEP) intervention, then a phase of evolution analysis of at least 3 months. Read more about this study
- Who is it for?
- Ages 7 to 25 years
- Conditions the study is for
- Marfan Syndrome
- What is being tested?
- Therapeutic Education Program
- How many people
- 60 participants
- Where
- 1 siteFrance
- Study dates
- 2 Dec 2020to 31 Jan 2024
- Phase
- Observational
- 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 7 to 25 years
- All sexes
- Child, Adult
Study population: Patient with Marphan Syndrom
Who may be able to join
- Patient aged between 7 and 25 years old with Marfan Syndrome or related according to Ghent criteria
- For minors, no oral opposition from the holders of parental authority and consent of the minor.
- For adults, no oral opposition collected
- Patient affiliated to a social security scheme or equivalent
Who cannot join
- Inability of the patient to understand the content of the TEP sessions or the questionnaire about quality of life (non-French speaking, severe intellectual disability)
- Patient who has already participated in a TEP session for his pathology.
- Protected adult: patient under legal guardianship or curator protection
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Effect on the Quality of Life of a Therapeutic Education Program in Patients With Marfan Syndrome: an Observational, Prospective and Multicenter Study
This is a before-after, observational, prospective, multicenter cohort study. The study will consist of 2 phases: an initial observational phase of a minimum of 3 months before the Therapeutic Education Program (TEP) intervention, then a phase of evolution analysis of at least 3 months.
Read the detailed description
During the first observational phase, the quality of life assessment will be carried out 3 months apart without intervention. The data obtained during this first phase will make it possible to characterize the functional impact in patients, to verify the reproducibility of our evaluations, and to evaluate the spontaneous evolution of the parameters over a period of 3 months.
During the second phase of evolution analysis, the quality of life will be assessed at 3 month intervals (M9, M12) after the therapeutic Education Program (M6). The comparison between the evolution during the two phases, before and after the TEP will allow us to assess the benefits of the TEP.
- Conditions
- Marfan Syndrome
- Being tested
- Therapeutic Education Program
- Medical subject headings
- Marfan Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Therapeutic Education ProgramOther
- needs assessment
- educational group workshop
- specialized workshops by age group: child / adolescent / transition / adult / entourage / separated parents and in parallel
- socio-administrative workshop
- individual summary (accompanied or not by his entourage / family)
- satisfaction questionnaire
| Study group | Role | What they receive |
|---|---|---|
| therapeutic Education Program | individual and group sessions will be set up with educational workshops specialized by age group (child / adolescent / transition / adult / entourage / parents), socio-administrative workshops, etc.
The objectives will be adapted to each age group and to each patient individually. Receives: Other: Therapeutic Education Program |
What is being measured
The primary outcome is the main question the study is designed to answer.
quality of life of children and young adults with Marfan
Answer to the question of PedsQL
Measured: Base line
quality of life of children and young adults with Marfan syndrome after therapeutic education program (ETP),
Answer to the question of PedsQL
Measured: 3 months
quality of life of children and young adults with Marfan syndrome after therapeutic education program (ETP),
Answer to the question of PedsQL
Measured: 9 months
quality of life of children and young adults with Marfan syndrome after therapeutic education program (ETP),
Answer to the question of PedsQL
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Feb 2021
- Study start2 Dec 2020
- Primary completion31 Jan 2024 (estimated)
- Study completion31 Jan 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Marfan SyndromeStudies of the same condition, open and under way.
- More trials run by University Hospital, ToulouseThe 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Toulouse
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Yves DULAC, MD, Principal investigator, University Hospital, Toulouse
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04731493
- Sponsor's protocol ID
- RC31/19/0505
- Acronym
- MYLIFE
- Status last verified
- Jan 2021
- First submitted
- 3 Dec 2020
- Last updated
- 1 Feb 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect on the Quality of Life of a Therapeutic Education Program in Patients With Marfan Syndrome: an Observational, Prospective and Multicenter Study
- ClinicalTrials.gov ID
- NCT04731493
- Acronym / sponsor ID
- MYLIFE / RC31/19/0505
- Conditions
- Marfan Syndrome
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- University Hospital, ToulouseUniversity, hospital or other
- Study start
- 2 Dec 2020 (actual)
- Primary completion
- 31 Jan 2024 (estimated)
- Study completion
- 31 Jan 2024 (estimated)
- First posted
- 1 Feb 2021 (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.
This is a before-after, observational, prospective, multicenter cohort study. The study will consist of 2 phases: an initial observational phase of a minimum of 3 months before the Therapeutic Education Program (TEP) intervention, then a phase of evolution analysis of at least 3 months.
Detailed description
During the first observational phase, the quality of life assessment will be carried out 3 months apart without intervention. The data obtained during this first phase will make it possible to characterize the functional impact in patients, to verify the reproducibility of our evaluations, and to evaluate the spontaneous evolution of the parameters over a period of 3 months.
During the second phase of evolution analysis, the quality of life will be assessed at 3 month intervals (M9, M12) after the therapeutic Education Program (M6). The comparison between the evolution during the two phases, before and after the TEP will allow us to assess the benefits of the TEP.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| therapeutic Education Program | – | Other: Therapeutic Education Programindividual and group sessions will be set up with educational workshops specialized by age group (child / adolescent / transition / adult / entourage / parents), socio-administrative workshops, etc. The objectives will be adapted to each age group and to each patient individually. |
Who may qualify
- Ages
- 7 to 25 years
- Sex
- All
- Age groups
- Child, Adult
- Study population
- Patient with Marphan Syndrom
Key inclusion criteria (4 listed)
- Patient aged between 7 and 25 years old with Marfan Syndrome or related according to Ghent criteria
- For minors, no oral opposition from the holders of parental authority and consent of the minor.
- For adults, no oral opposition collected
- Patient affiliated to a social security scheme or equivalent
Key exclusion criteria (3 listed)
- Inability of the patient to understand the content of the TEP sessions or the questionnaire about quality of life (non-French speaking, severe intellectual disability)
- Patient who has already participated in a TEP session for his pathology.
- Protected adult: patient under legal guardianship or curator protection
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 |
|---|---|---|
| Therapeutic Education Program* needs assessment * educational group workshop * specialized workshops by age group: child / adolescent / transition / adult / entourage / separated parents and in parallel * socio-administrative workshop * individual summary (accompanied or not by his entourage / family) * satisfaction questionnaire | Other | therapeutic Education Program |
Primary outcome
quality of life of children and young adults with Marfan
Time frame: Base line
Answer to the question of PedsQL
quality of life of children and young adults with Marfan syndrome after therapeutic education program (ETP),
Time frame: 3 months
Answer to the question of PedsQL
quality of life of children and young adults with Marfan syndrome after therapeutic education program (ETP),
Time frame: 9 months
Answer to the question of PedsQL
quality of life of children and young adults with Marfan syndrome after therapeutic education program (ETP),
Time frame: 12 months
Answer to the question of PedsQL
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Feb 2021.
- Individual participant dataSharing planned: No
Related trials for Marfan Syndrome
RecruitingObservational study
Asprosin and miRNA Expression in Young Women With Metabolic Syndrome
Metabolic Syndrome, Insulin Resistance
Enrolling by invitationObservational study
Advanced Ultrasound Signal Processing of Suspicious Breast Images
Breast Cancer, Breast Cyst
RecruitingObservational study
Pregnancy in Women With Rare Multisystemic Vascular Diseases: COGRare5 Study
Vascular Anomaly, Osler Rendu Disease
Not yet recruitingObservational study
Asprosin in Behect Patients
Behcet Disease
Not yet recruitingObservational study
Serum Asprosin, Metabolic Syndrome, and Cardiometabolic Risk in Rheumatoid Arthritis
Rheumatoid Arthritis, Metabolic Syndrome
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.
Yves DULAC, MD principal investigator
University Hospital, Toulouse
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 Marfan Syndrome, run by University Hospital, Toulouse 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 4 inclusion and 3 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: status not confirmed, as of the sponsor's last update on 1 Feb 2021. 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?
Results are not available in the source record.
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?
quality of life of children and young adults with Marfan (time frame: Base line). The study lists 4 primary outcomes in all. 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 1 Feb 2021.