Clinical trial NCT03567460: Children and Adolescents With Marfan Syndrome: 10,000 Healthy Steps and Beyond
Official title: Children and Adolescents With Marfan Syndrome: 10,000 Healthy Steps and Beyond
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Marfan Syndrome
Lead sponsor: Stanford University
Sponsor class: OTHER
Collaborators: name: Midwestern University
First posted: 2018-06-25
Enrollment: 24
Sex: ALL
Minimum age (years): 10
Maximum age (years): 19
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

Brief summary: Marfan patients are at risk of sudden death due to weakening of the wall of the large blood vessel leading from the heart (aorta). The wall of the aorta weakens and dilates which can rupture, leading to death, and sometimes during intense exercise.

There is some evidence in Marfan patients that a stiffer aorta increases risk for rupture.

For some time, clinical care has focused on what type of exercise these patients should avoid due to risk for aortic dissection. Little clinical emphasis has been placed on encouraging patients to engage in routine and safe exercise such as walking. Informed by this evidence, the investigators propose to collaboratively investigate whether regular exercise improves aortic health in adolescent Marfan patients.

Detailed description: Marfan syndrome is an inherited disorder of the connective tissue, which provides material and support for the skeleton, muscles, and blood vessels. Marfan patients are at risk of sudden death due to weakening of the wall of the large blood vessel leading from the heart (aorta). The wall of the aorta weakens and dilates which can rupture, leading to death, and sometimes during intense exercise.

Typically, the weakening process starts when elastin fibers in the aorta become fragmented. There is some evidence in Marfan patients that a stiffer aorta increases risk for rupture.

For some time, clinical care has focused on what type of exercise these patients should avoid due to risk for aortic dissection. Little clinical emphasis has been placed on encouraging patients to engage in routine and safe exercise such as walking. These young patients also frequently choose sedentary lifestyles, most likely due to limitations imposed by parents as well as adolescents' own perceptions of what is safe for them and their physical capabilities. Another complicating factor is that these patients often experience difficulty coping with their diagnosis. Informed by this evidence, the investigators propose to collaboratively investigate whether regular exercise improves aortic health and coping skills in adolescent Marfan patients.

Eligibility criteria: Inclusion Criteria:

1. 10-19 years of age,
2. MFS by revised Ghent criteria,
3. Cardiac clearance to exercise by the primary cardiologist.

Exclusion Criteria:

1. Ventricular dysfunction,
2. Prior history of aortic surgery.

Primary outcomes:
measure: To determine if 6 months of regular physical activity improves aortic stiffness in MFS patients. exercise, a decrease in expressed biomarkers, and an increase in COPE scores of MFS patients.
timeFrame: 6 months of intervention
Secondary outcomes:
measure: To determine if 6 months of regular physical activity improves aortic stiffness and the biomarker profile in MFS patients.
timeFrame: 6 months of intervention
measure: To determine if 6 months of regular physical activity improves aortic stiffness and the biomarker profile in MFS patients.
timeFrame: 6 months of intervention
measure: To determine if 6 months of regular physical activity decreases aortic stiffness and rate of aortic root dilation in Marfan mice.
timeFrame: 6 months of intervention
measure: To determine if 6 months of regular physical activity improves coping skills in Marfan patients.
timeFrame: 6 months of intervention
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford Children's Health
state: California
Study officials:
affiliation: Stanford University
name: Seda Tierney, MD
role: PRINCIPAL_INVESTIGATOR
References: