Marfan Syndrome Moderate Exercise Pilot

NCT04641325 | Completed

• 1 other identifier: H-45475
• Study Type: interventional
• Target: 23
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Marfan Syndrome

Keywords

Cardiovascular; Moderate Exercise; Physical Therapy

Outcome Measures

Primary Outcomes (1)

• Maximum VO2

  Maximum VO2 in ml/kg/minute will be collected via Exercise Stress Test. Range 30-85, higher is better.

  4 months

Secondary Outcomes (27)

• Mean systolic blood pressure

  4 months

• Mean Diastolic blood pressure

  4 months

• Mean pulse pressure

  4 months

• Weight

  4 months

• BMI

  4 months

Study Arms (1)

Exercise Intervention Group

EXPERIMENTAL

Group of up to 20 patients will receive all of the preliminary outcome measure testing (cardiovascular, musculoskeletal, and psychological screening) in addition to exercise intervention education, demonstration, and follow up to ensure compliance and safety.

Other: Exercise Intervention Group

Interventions

Exercise Intervention Group OTHER

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 8 weeks all patients will return for re-assessment.

Exercise Intervention Group

Eligibility Criteria

• Age: 12 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• Patients who have undergone aortic surgery
• Patients with major congenital heart disease

Sponsors & Collaborators

• Baylor College of Medicine: lead
• The Marfan Foundation: collaborator

Study Sites (1)

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Marfan Syndrome

Condition Hierarchy (Ancestors)

Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Connective Tissue Diseases; Skin and Connective Tissue Diseases

Study Officials

• Shaine A Morris, MD MPH

  Baylor College of Medicine

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Masking Details: The primary investigator and the co-PI (physical therapist) will know whether testing is at baseline or follow-up, but will not know the cardiovascular outcome measures. The cardiovascular outcomes assessor will be fully blinded.
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor, Pediatrics-Cardiology

Model Details: The first part of trial was a Randomized Controlled Step-Wedge Pilot Trial as below 2 groups (Exercise Intervention Group and Current Care Group) The first phase of the study will maintain the Current Care Group as the control. At the end of the first phase, the Current Care Group will crossover and receive the Exercise Intervention as the second phase of the trial. With the COVID-19 pandemic causing delays in the study, and decreased enrollment and decreased access to MRI, the study was altered to a single group. comparing the same outcomes at baseline and after the intervention. This eliminates the blinding of the PI, but those interpreting the tests are still blinded.

Study Record Dates

• First Submitted: April 23, 2020
• First Posted: November 23, 2020
• Study Start: November 9, 2020
• Primary Completion: October 30, 2022
• Study Completion: December 20, 2022
• Last Updated: August 14, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)