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Marfan SyndromeMARF'HOMENCT04553094

Effects of Personalized Training at Home Combining Endurance and Resistance in Patients Suffering From Marfan Syndrome

A study testing Endurance training and Muscle building training in people with Marfan Syndrome, run by French Cardiology Society at 1 site in France.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Marfan syndrome (MS) is an autosomal dominant genetic disorder caused by a mutation in the fibrillin-1 gene (FBN1) encoding the protein fibrillin-1. Fibrillin is the main component of microfibrils, elements found in all of the body's tissues, and this pathology is characterized by the multitude of its clinical… Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Marfan Syndrome
What is being tested?
Endurance training and Muscle building training
Could I receive a placebo?
No, everyone receives an active treatment
How many people
72 participants
Where
1 siteFrance
Study dates
4 Jan 2021to 30 Sep 2022
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 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • patient with Marfan syndrome and followed up at Bichat Claude Bernard hospital, Paris. Marfan syndrome should be diagnosed according to the Ghent nosology revised in 2010
  • Patient taking protective treatment for the aorta (beta blocker, calcium channel blocker, ACE inhibitor, angiotensin II receptor antagonist).
  • Valid patient, able to return to consultation and carry out functional assessments and personalized physical training at home.
  • Patients operated prophylactically on the ascending aorta may be included at a distance > 6 months from their surgery.

Who cannot join

  • Myocardial pathology other than Marfan syndrome.
  • Thoracic aorta dissection.
  • Aortic diameter > 45 millimeters.
  • Uncontrolled high blood pressure at rest (systolic blood pressure > 140 Millimeter of mercury and diastolic blood pressure > 90 Millimeter of mercury)
  • Increase in systolic blood pressure > 160 Millimeter of mercury during exercise.
  • Pregnancy at the time of the study

Trial locations and contacts

1 site in 1 country.

France1 site

  • Groupe Hospitalier Bichat - Claude Bernard

    Paris, 75018

Other trials in France

About this study

Official title: Evaluation of the Effects of Personalized Training at Home Combining Endurance and Resistance in Patients Suffering From Marfan Syndrome

Marfan syndrome (MS) is an autosomal dominant genetic disorder caused by a mutation in the fibrillin-1 gene (FBN1) encoding the protein fibrillin-1. Fibrillin is the main component of microfibrils, elements found in all of the body's tissues, and this pathology is characterized by the multitude of its clinical manifestations. These patients may develop aneurysms in the aortic root and one of the main factors of morbidity in patients with MS is aortic dissection. Prevention mainly involves preventive aortic surgery. However, the repercussions are global and can affect the functioning of other tissues such as skeletal muscle tissue, bone tissue, lung tissue and the eyes. The association of skeletal (scoliosis, hyperlaxity), muscular and ocular disorders is clearly associated with an impairment in the quality of life. These disorders are associated with pain and disability which affect professional activity, leisure and family life.

Physical activity could represent a relevant alternative for these patients. A recent animal study suggests that moderate training is beneficial.

Read the detailed description

The main objective is to show that the quality of life of patients with Marfan syndrome can be improved by personalized training at home. The goal is to be able to offer new non-drug management based on physical activity to these patients.

Improved quality of life; Improvement of muscle strength by a muscle strengthening protocol; Improvement of cardiovascular function parameters. Regular coaching throughout the study.

  1. / Assessment of muscular capacities
  2. / Assessment of cardiovascular and respiratory capacities
  3. / Response to psychometric questionnaires
  4. / Participation in a training circuit (training protocol)
Conditions
Marfan Syndrome
Being tested
Endurance training, Muscle building training
Keywords
Physical training
Medical subject headings
Marfan Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Endurance trainingOther

    Aerobic circuit training

  • Muscle building trainingOther

    Muscle strengthening circuit training

Study groups in NCT04553094
Study groupRoleWhat they receive
Group 1No interventionNo physical training
Group 2ExperimentalEndurance training
Receives: Other: Endurance training
Group 3ExperimentalMuscle building training
Receives: Other: Muscle building training
Group 4ExperimentalTraining combining endurance + muscle building
Receives: Other: Endurance training; Other: Muscle building training

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Quality of life assessment: questionnaire

    Assessed with Medical Outcome Study Short Form 36 questionnaire, a scale scored on a 0 to 100 range so that the lowest is the worth condition and highest is the best condition.

    Measured: 3 months

Secondary outcomes (9)

  1. Pain assessment: Fibromyalgia Rapid Screening Tool

    Assessed with Fibromyalgia Rapid Screening Tool

    Measured: 3 months

  2. Self-perception assessment

    Assessed with Physical Self Inventory questionnaire, a scale scoring 25 items rated on a 6-point scale ranging from 1 (Not at all) to 6 (Entirely) and assessing 6 dimensions (global self-worth (10-50), physical self-worth (10-50), physical condition (10-50), sport competence (10-40), physical attractiveness (10-30), and physical strength (10-30)) of the physical self-conception.

    Measured: 3 months

  3. Aortic diameter

    Evolution of the aortic diameter (in millimeter) in echocardiography, safety cardiovascular parameters

    Measured: 3 months

  4. Blood pressure

    Evolution of the systolic and diastolic blood pressures (in Millimeter of mercury) during exercise, monitoring of physiological parameters

    Measured: 3 months

  5. Heart rate

    Evolution of heart rate (Beats per minute) during exercise, monitoring of physiological parameters

    Measured: 3 months

  6. oxygen consumption

    Evolution of oxygen consumption (in liter) during exercise, monitoring of physiological parameters

    Measured: 3 months

  7. Maximal voluntary contraction

    Evolution of maximal voluntary contraction (in Newtons) during exercise, monitoring of physiological parameters

    Measured: 3 months

  8. Left Ventricular Ejection Fraction (LVEF)

    Evolution of Left Ventricular Ejection Fraction (in percentage) at rest, monitoring of cardiovascular parameters.

    Measured: 3 months

  9. Global longitudinal strain (GLS)

    Evolution of GLS (in percentage) at rest, monitoring of cardiovascular parameters

    Measured: 3 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Sep 2020
  2. Study start4 Jan 2021
  3. Primary completion30 Sep 2022
  4. Study completion30 Sep 2022

Resources

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
Supportive care
Allocation
Randomised
Intervention model
Parallel groups
Model description
3 groups of physical training intervention compared with control group
Masking
None (open label)
Enrolment
72 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04553094
Sponsor's protocol ID
2020-01
Acronym
MARF'HOME
Status last verified
Sep 2022
First submitted
11 Sep 2020
Last updated
3 Oct 2022
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Publications

  • Jouini S, Milleron O, Eliahou L, Jondeau G, Vitiello D. Online Personal Training in Patients With Marfan Syndrome: A Randomized Controlled Study of Its Impact on Quality of Life and Physical Capacity. J Am Heart Assoc. 2024 Oct;13(19):e033024. doi: 10.1161/JAHA.123.033024. Epub 2024 Sep 18.

    PubMed 39291498

Questions about this trial

What is this clinical trial studying?

A study testing Endurance training and Muscle building training in people with Marfan Syndrome, run by French Cardiology Society 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 6 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 3 Oct 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?

Quality of life assessment: questionnaire (time frame: 3 months). 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 NCT04553094, 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 3 Oct 2022.

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