- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03567460
Children and Adolescents With Marfan Syndrome: 10,000 Healthy Steps and Beyond
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.
Study Overview
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.
Study Type
Enrollment (Actual)
Phase
- Not Applicable
Contacts and Locations
Study Locations
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California
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Palo Alto, California, United States, 94306
- Stanford Children's Health
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 10-19 years of age,
- MFS by revised Ghent criteria,
- Cardiac clearance to exercise by the primary cardiologist.
Exclusion Criteria:
- Ventricular dysfunction,
- Prior history of aortic surgery.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Prevention
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: 10,000 Steps/day for Pediatric Marfan Patients
Participants will be given a Garmin VivoFit and asked to take at least 10,000 steps per day.
A study coordinator will reach out at least once per week to check in on progress made and help make weekly goals.
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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.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
Time Frame: 6 months of intervention
|
The investigators' primary outcome measure is aortic stiffness measured by arterial tonometry (pulse wave velocity).
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6 months of intervention
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
To determine if 6 months of regular physical activity improves aortic stiffness and the biomarker profile in MFS patients.
Time Frame: 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.
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6 months of intervention
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To determine if 6 months of regular physical activity improves aortic stiffness and the biomarker profile in MFS patients.
Time Frame: 6 months of intervention
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Secondary outcomes will be expression levels of TGF-β, Ang-II, MMP-2 & -9, ROS levels.
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6 months of intervention
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To determine if 6 months of regular physical activity decreases aortic stiffness and rate of aortic root dilation in Marfan mice.
Time Frame: 6 months of intervention
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Secondary outcomes will be expression levels of TGF-β, MMP-2, MMP-9, and ROS.
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6 months of intervention
|
To determine if 6 months of regular physical activity improves coping skills in Marfan patients.
Time Frame: 6 months of intervention
|
The outcome measure is the COPE inventory score.
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6 months of intervention
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Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Seda Tierney, MD, Stanford University
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Heart Diseases
- Cardiovascular Diseases
- Disease
- Congenital Abnormalities
- Genetic Diseases, Inborn
- Musculoskeletal Diseases
- Connective Tissue Diseases
- Bone Diseases
- Heart Defects, Congenital
- Cardiovascular Abnormalities
- Musculoskeletal Abnormalities
- Abnormalities, Multiple
- Bone Diseases, Developmental
- Limb Deformities, Congenital
- Syndrome
- Marfan Syndrome
- Arachnodactyly
Other Study ID Numbers
- 37176
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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