Marfan Syndrome Clinical Trial
Official title:
Evaluating the Effects of Moderate Physical Activity on Health and Well-being in Adolescents and Young Adults With Marfan Syndrome
Verified date | August 2023 |
Source | Baylor College of Medicine |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
Marfan syndrome (MFS) affects multiple organ systems including the heart, bones, ligaments, and eyes, and is associated with significant risk of aortic dissection. Given limited evidence from in-vitro studies, and theoretical concerns, the majority of patients with MFS are restricted from certain physical activities. The lack of exercise and deconditioning have detrimental effects including increasing weakness, joint pain, decreased endurance, and depressive symptoms. Given the significant paucity of data currently existing on the effects of exercise in humans with MFS, and the recent, optimistic findings in rodent models, this pilot trial was established to assess the effects of moderated dynamic exercise in adolescents and young adults with MFS.
Status | Completed |
Enrollment | 23 |
Est. completion date | December 20, 2022 |
Est. primary completion date | October 30, 2022 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 12 Years to 21 Years |
Eligibility | Inclusion Criteria: - Must be between the ages of 12-21 - Diagnosis of Marfan Syndrome - Must not have other conditions that limit the patients ability to perform exercise Exclusion Criteria: - Patients who have undergone aortic surgery - Patients with major congenital heart disease |
Country | Name | City | State |
---|---|---|---|
United States | Texas Children's Hospital | Houston | Texas |
Lead Sponsor | Collaborator |
---|---|
Baylor College of Medicine | The Marfan Foundation |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Maximum VO2 | Maximum VO2 in ml/kg/minute will be collected via Exercise Stress Test. Range 30-85, higher is better. | 4 months | |
Secondary | Mean systolic blood pressure | mmHg, range 70-200, both low and high are abnormal, goal is age, sex and height-based, goal 10-50 percentile | 4 months | |
Secondary | Mean Diastolic blood pressure | mmHg, range 20-150, both low and high are abnormal, goal is age, sex and height-based, goal 10-50 percentile | 4 months | |
Secondary | Mean pulse pressure | mmHg, systolic blood pressure minus diastolic blood pressure, range 30-70 mmHg, goal is normal range for age and sex | 4 months | |
Secondary | Weight | kg, range 50-300kg, lower is better, excluding underweight patients | 4 months | |
Secondary | BMI | kg/m2, lower is better generally, excluding pts with BMI <5% for age | 4 months | |
Secondary | Left ventricular strain by cardiac MRI | Continuous measure derived from post-processing MRI | 4 months | |
Secondary | Right ventricular strain by cardiac MRI | Continuous measure derived from post-processing MRI | 4 months | |
Secondary | Aortic root strain | Continuous measure %, higher is less stiff, Range 0-40 | 4 months | |
Secondary | Aortic Root Distensibility | ×10-3 mm Hg-1, Continuous measure, range 0.1-10 | 4 months | |
Secondary | Aortic Root ß-Stiffness index | No units, Range 0.1-90 | 4 months | |
Secondary | Maximum aortic root dimension | Measured in cm, range 1-8cm | 4 months | |
Secondary | Aortic root z-score | No units, based on body surface area published references, range -3 to 25 | 4 months | |
Secondary | Aortic pulse wave velocity from MRI | meters/second, range 0-30 | 4 months | |
Secondary | Pulse wave velocity derived from applanation tonometry | meters/second, range 0-30 | 4 months | |
Secondary | Augmentation index | %, range 1-90 | 4 months | |
Secondary | Manual muscle testing score | Grade 0-5, higher is better | 4 months | |
Secondary | Reactive hyperemia index | no units, 0-4 range, higher is worse | 4 months | |
Secondary | Visual analog assessment of pain | no units, scale from 0-6, 6 is worse | 4 months | |
Secondary | Single Leg Stance Test | seconds, higher is better | 4 months | |
Secondary | Single limb squat test score | seconds, higher is better | 4 months | |
Secondary | Star Excursion Balance Test | %, range 0-100, higher is better | 4 months | |
Secondary | 6M Timed Hop Test | milliseconds, range 1 to infinity, lower is better | 4 months | |
Secondary | Scale for Child Anxiety Related Emotional Disorders (SCARED) (ages 12-18 y) | 41 item scale, each with 3 point Likert scale, scale is summed, range 0-123, higher is worse | 4 months | |
Secondary | Quality of Life Scale (QOLS) (ages 19-21 y) | 16 items, each with 7 point Likert, higher is worse | 4 months | |
Secondary | Pediatric Quality of Life Scale (PedsQL) scale scores | reported in 3 domains, each reported on Likert scale, scaled to 0-100 scale, lower is worse | 4 months | |
Secondary | Children's Depression Inventory (CDI) 2 (ages 12-18 y) | 28-item assessment that yields a Total Score, 2 scale scores, and 4 subscale scores. For each item, respondent is presented with 3 choices that correspond to 3 levels of symptomatology: 0 (absence of symptoms), 1 (mild or probable symptom), or 2 (definite symptom). Lower is better | 4 months | |
Secondary | Depression, Anxiety, & Stress Scale (DASS) (ages 19-21 y) | set of 3 self-report scales. Each scale contains 14 items, each with a 4-point severity/frequency scale, higher is worse | 4 months |
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