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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03567460
Recruitment Status : Recruiting
First Posted : June 25, 2018
Last Update Posted : September 19, 2019
Sponsor:
Collaborator:
Midwestern University
Information provided by (Responsible Party):
Seda Tierney, Stanford University

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.


Condition or disease Intervention/treatment Phase
Marfan Syndrome Behavioral: Exercise Not Applicable

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 25 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: Children and Adolescents With Marfan Syndrome: 10,000 Healthy Steps and Beyond
Actual Study Start Date : January 1, 2017
Estimated Primary Completion Date : December 31, 2019
Estimated Study Completion Date : December 31, 2019


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.
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.




Primary Outcome Measures :
  1. 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).


Secondary Outcome Measures :
  1. 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.

  2. 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 ]
    Secondary outcomes will be expression levels of TGF-β, Ang-II, MMP-2 & -9, ROS levels.

  3. 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 ]
    Secondary outcomes will be expression levels of TGF-β, MMP-2, MMP-9, and ROS.

  4. 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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Ages Eligible for Study:   10 Years to 19 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03567460


Contacts
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Contact: Seda Tierney, MD (650) 725-8246 tierneys@stanford.edu
Contact: Angela Chen, BS 650-307-3595 achen11@stanford.edu

Locations
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United States, California
Stanford Children's Health Recruiting
Palo Alto, California, United States, 94306
Contact: Elif Seda M Selamet Tierney    650-736-8716    tierneys@stanford.edu   
Sponsors and Collaborators
Stanford University
Midwestern University
Investigators
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Principal Investigator: Seda Tierney, MD Stanford University

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Responsible Party: Seda Tierney, Assistant Professor in Pediatrics, Stanford University
ClinicalTrials.gov Identifier: NCT03567460    
Other Study ID Numbers: 37176
First Posted: June 25, 2018    Key Record Dates
Last Update Posted: September 19, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Marfan Syndrome
Arachnodactyly
Syndrome
Disease
Pathologic Processes
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn
Connective Tissue Diseases
Limb Deformities, Congenital
Musculoskeletal Abnormalities