Marfan Syndrome Clinical Trial
— MarfanpowerOfficial title:
Cardiorespiratory and Muscular Rehabilitation of Children and Young Adults With Marfan Syndrome: an Interventional, Prospective, Monocentric Study.
Verified date | July 2022 |
Source | University Hospital, Toulouse |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
Marfan syndrome (MFS) is a rare genetic disease (1/5000) characterized by the association of ocular impairment, cardiovascular disease and musculoskeletal disease. In some chronic conditions, physical activity and training have been shown to be effective in improving muscle strength and functional abilities but also fatigue and quality of life. We hypothesize that the implementation of a personalized exercise rehabilitation program (Personalized Training Program) in children and young adults with MFS, by improving muscle mass, physical endurance, muscle strength, bone mass and quality of life of these patients. In order to test this hypothesis, investigators wish to carry out an interventional, prospective, monocentric study for the first time in children and young adults (<25 years old) presenting an MFS.
Status | Completed |
Enrollment | 28 |
Est. completion date | June 21, 2022 |
Est. primary completion date | June 21, 2022 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 7 Years to 25 Years |
Eligibility | Inclusion Criteria: - Marfan syndrome according to Ghent criteria. - For minors, signed informed consent of at least one of the holders of the parental authority. For majors, signed informed consent. - Patient affiliated to a social security scheme or equivalent. Exclusion Criteria: - Cardiac contraindications to Personal Training Program: O Severe aorta dilatation (aortic diameter> 45 mm) O and / or left ventricular failure (left ventricular ejection fraction <45%) O and / or severe mitral leakage = grade 3 - Pregnancy |
Country | Name | City | State |
---|---|---|---|
France | CHU de Toulouse | Toulouse | Midi-Pyrénées |
Lead Sponsor | Collaborator |
---|---|
University Hospital, Toulouse |
France,
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Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Measurement of the maximum endurance capacities. | Reflected by the maximal oxygen consumption (VO2 peak) during an exercise test. The values of VO2 peak will be compared between the beginning and the end of the rehabilitation. | Month 9 | |
Secondary | Fatigability with effort and quality of life. | Questionnaires and self-assessment test. It will be compared between the beginning and the end of the rehabilitation. | Month 9 | |
Secondary | Muscular force. | Static evaluation (handgrip) and dynamic evaluation by mechanography. It will be compared between the beginning and the end of the rehabilitation. | Month 9 | |
Secondary | Body composition (muscle mass and bone mass). | Bone mineral content, bone mineral density of the entire body and lumbar spine (L2-L4), and muscle mass evaluated by dual-energy xray absorptiometry. It will be compared between the beginning and the end of the rehabilitation. | Month 9 | |
Secondary | Aortic dilation and myocardial function. | Assessed by a cardiac ultrasound 2D and 2D strain. It will be compared between the beginning and the end of the rehabilitation. | Month 9 | |
Secondary | Endothelial function. | Assessed by a high resolution vascular ultrasound. It will be compared between the beginning and the end of the rehabilitation. | Month 9 | |
Secondary | Compliance. | Assessed by questionnaires. It will be compared between the beginning and the end of the rehabilitation. | Month 9 | |
Secondary | Cardiac adverse events. | Evolution of the aorta dimensions, evaluated by ultrasound. It will be compared between the beginning and the end of the rehabilitation. | Month 9 |
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