Clinical trial NCT05838235: Adapted Physical Activity Program (APA) for Effort Rehabilitation of Children and Teenagers With Marfan Syndrome
Official title: Marfan&Moves (M&M's) : an Adapted Physical Activity Program (APA) for Effort Rehabilitation of Children and Teenagers With Marfan Syndrome: an Interventional, Prospective, Monocentric Study.
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Marfan Syndrome
Lead sponsor: University Hospital, Toulouse
Sponsor class: OTHER
First posted: 2023-05-01
Enrollment: 30
Sex: ALL
Minimum age (years): 7
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

Brief summary: This research study aims to evaluate the effect of a 6-month adapted physical activity program (APA) on the endurance capacities (evaluated as the maximum oxygen consumption \[VO2 peak\] on a cardiopulmonary exercise test) of children and adolescents with Marfan syndrome or related.

Detailed description: Marfan syndrome (MFS) is characterized by the association of multisystem damage related to connective tissue fragility. The risk of aortic dissection has diverted attention from significant musculoskeletal damage despite commonly reported extreme fatigue and pain with impact on the patient's quality of life. In a recent study (Marfanpower, NCT03236571), investigators have observed reduced endurance capacities in children and teenagers with MFS (average VO2 peak at 60% of expected), which were significantly increased by an intensive rehabilitation program. Based on this experience, investigators aim to evaluate the effect of a physical activity program adapted to the daily life of the young patients on their endurance capacities and their quality of life. This program will be set up by an adapted physical activity (APA) coach after a complete assessment, including a cardiopulmonary exercise test with VO2 peak measurement, which will be carried out in the Reference Centre for Marfan syndrome at the Toulouse University Hospital. The VO2 peak evolution will be evaluated after 6 months of program. This is a recognized parameter for the evaluation of endurance and has been validated in children. Investigators hypothesize that the implementation of a physical activity program adapted to the daily life and interests of the child with MFS will efficiently improve endurance, prevent deconditioning and promote long term benefits.

Eligibility criteria: Inclusion Criteria:

* Genetically confirmed Marfan syndrome or related syndrome
* Girl or boy aged 7 to 17
* Informed and written consent signed by at least one of the two holders of parental authority
* Patient affiliated to a social security scheme or equivalent

Exclusion Criteria:

* Cardiac contraindications to the APA program:

  * Severe aortic dilation (aortic diameter \> 45 mm)
  * and/or left ventricular failure (left ventricular ejection fraction \<45%)
  * and/or severe mitral leakage ≥ grade 3
* Pregnancy

Primary outcomes:
measure: Measurement of maximum endurance
timeFrame: At baseline
measure: Measurement of maximum endurance
timeFrame: Up to 6 month
Secondary outcomes:
Other outcomes:
Locations:
city: Toulouse
country: France
facility: CHU Toulouse
Study officials:
affiliation: CHU Toulouse
name: Thomas EDOUARD, MD
role: PRINCIPAL_INVESTIGATOR
References: