Children and Adolescents With Marfan Syndrome: 10,000 Healthy Steps and Beyond

Status

Completed

Phases

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03567460

Enrollment

Registered

2018-06-25

Start date

2017-01-01

Completion date

2019-12-31

Last updated

2020-07-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Marfan Syndrome

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.

Interventions

BEHAVIORALExercise

Across studies in adolescents in general population, 60 minutes of recommended daily exercise level is achieved, on average, within a total volume of 10,000-11,700 steps. The investigators will assess baseline physical activity quantitatively by using an accelerometer worn on the wrist during waking hours over a 7-day period. Each participant will be given a Garmin device to place on their wrist that will track their steps. Then patients will be asked to complete 10,000 steps daily for 6 months (acclimation over a week), which will be encouraged by daily text messages or e-mail, and weekly phone calls by the intervention team in addition to a Garmin and Facebook peer group.

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

PREVENTION

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

10 Years to 19 Years

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
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.	6 months of intervention	The investigators' primary outcome measure is aortic stiffness measured by arterial tonometry (pulse wave velocity).

Secondary

Measure	Time frame	Description
To determine if 6 months of regular physical activity improves coping skills in Marfan patients.	6 months of intervention	The outcome measure is the COPE inventory score.
To determine if 6 months of regular physical activity improves aortic stiffness and the biomarker profile in MFS patients.	6 months of intervention	The secondary outcomes include aortic stiffness at the aortic root at the level of the sinuses of Valsalva and ascending aorta.
To determine if 6 months of regular physical activity decreases aortic stiffness and rate of aortic root dilation in Marfan mice.	6 months of intervention	Secondary outcomes will be expression levels of TGF-β, MMP-2, MMP-9, and ROS.

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 10, 2026