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

Marfan Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03567460
• Other study ID #: 37176
• Status: Completed
• Phase: N/A
• Start date: January 1, 2017
• Est. completion date: December 31, 2019

Study information

• Verified date: July 2020
• Source: Stanford University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 24
• Est. completion date: December 31, 2019
• Est. primary completion date: December 31, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 10 Years to 19 Years

Eligibility

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.

Related Conditions & MeSH terms

• Marfan Syndrome
• Syndrome

Intervention

Behavioral:
• Exercise
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.

Locations

Country	Name	City	State
United States	Stanford Children's Health	Palo Alto	California

Sponsors (2)

Lead Sponsor	Collaborator
Stanford University	Midwestern University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	The investigators' primary outcome measure is aortic stiffness measured by arterial tonometry (pulse wave velocity).	6 months of intervention
Secondary	To determine if 6 months of regular physical activity improves aortic stiffness and the biomarker profile in MFS patients.	The secondary outcomes include aortic stiffness at the aortic root at the level of the sinuses of Valsalva and ascending aorta.	6 months of intervention
Secondary	To determine if 6 months of regular physical activity improves aortic stiffness and the biomarker profile in MFS patients.	Secondary outcomes will be expression levels of TGF-ß, Ang-II, MMP-2 & -9, ROS levels.	6 months of intervention
Secondary	To determine if 6 months of regular physical activity decreases aortic stiffness and rate of aortic root dilation in Marfan mice.	Secondary outcomes will be expression levels of TGF-ß, MMP-2, MMP-9, and ROS.	6 months of intervention
Secondary	To determine if 6 months of regular physical activity improves coping skills in Marfan patients.	The outcome measure is the COPE inventory score.	6 months of intervention