RECRUITINGINTERVENTIONAL

Marfan Syndrome Moderate Exercise Trial II

Clinical Trial On The Effects Of Moderate Physical Activity On Health And Well-Being In Adolescents And Young Adults With Marfan Syndrome

About This Trial

Marfan syndrome (MFS) is a distinctive connective tissue disorder that 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.

Who May Be Eligible (Plain English)

Who May Qualify: - Male and female patients diagnosed with Marfan syndrome (defined by Ghent criteria and either a pathogenic variant in FBN1 or ectopia lentis) - Age 10-25 years at enrollment Who Should NOT Join This Trial: 1. History of aortic surgery 2. History of spinal surgery with implanted materials that may negatively impact MRI safety or imaging quality. 3. Diagnosis of major congenital heart disease (ASD, VSD, bicuspid aortic valve, and mitral valve prolapse will not qualify as exclusion criteria) 4. Condition limiting the ability to perform moderate exercise. 5. Major concurrent diagnosis that may confound the interpretation of the effect of the proposed intervention on the proposed outcome measures. 6. Aortic dilation meeting threshold for prophylactic aortic surgical intervention (\>/= 4.5 cm in diameter) Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language

Inclusion Criteria: * Male and female patients diagnosed with Marfan syndrome (defined by Ghent criteria and either a pathogenic variant in FBN1 or ectopia lentis) * Age 10-25 years at enrollment Exclusion Criteria: 1. History of aortic surgery 2. History of spinal surgery with implanted materials that may negatively impact MRI safety or imaging quality. 3. Diagnosis of major congenital heart disease (ASD, VSD, bicuspid aortic valve, and mitral valve prolapse will not qualify as exclusion criteria) 4. Condition limiting the ability to perform moderate exercise. 5. Major concurrent diagnosis that may confound the interpretation of the effect of the proposed intervention on the proposed outcome measures. 6. Aortic dilation meeting threshold for prophylactic aortic surgical intervention (\>/= 4.5 cm in diameter)

Treatments Being Tested

OTHER

Exercise Intervention Group

Patients will be educated on methods of self-evaluating exertion and cardiovascular effort by assessing respiratory rate and perceived exertion. Next, patients will be given options for cardiovascular activities and complete at a moderate level of activity for a minimum of 150 minutes per week. Patients will perform a combination of exercises under the supervision of a physical therapist until a mod intensity level is reached and sustained. Patients will be taught to use the activity tracker to record their heart rate, activity, and PES. Patients will have a phone call every week to assess status, answer questions, and provide guidance on progressing intensity or duration of exercise. At the end of 12 weeks all patients will return for re-assessment.

OTHER

Control Group

Patients in the control group will be instructed to continue with baseline physical activities over the duration of the study and be given instructions on the use of the activity tracker. At the end of 12 weeks all patients will return for re-assessment.

Locations (1)

Texas Children's Hospital

Houston, Texas, United States