Marfan Syndrome Moderate Exercise Trial II

NCT05809323 | Recruiting

• 1 other identifier: H-53417
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

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.

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 (2)

Exercise Intervention Group

EXPERIMENTAL

Group of up to 50 patients will randomly allocated into an experimental group at a 4:1 ratio intervention to controls. The experimental group 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

Control Group

OTHER

Control group will be randomly allocated at a 4:1 ratio, intervention to controls.. The control group will receive all of the preliminary outcome measure testing (cardiovascular, musculoskeletal, and psychological screening) and will be instructed to continue with baseline physical activities. They will be asked to return for a reassessment of all baseline procedures (cardiovascular, musculoskeletal, and psychological screening).

Other: Control 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 12 weeks all patients will return for re-assessment.

Exercise Intervention Group

Control Group OTHER

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.

Also known as: No Intervention

Control Group

Eligibility Criteria

• Age: 10 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• History of aortic surgery
• History of spinal surgery with implanted materials that may negatively impact MRI safety or imaging quality.
• Condition limiting the ability to perform moderate exercise.
• Major concurrent diagnosis that may confound the interpretation of the effect of the proposed intervention on the proposed outcome measures.
• Aortic dilation meeting threshold for prophylactic aortic surgical intervention (\>/= 4.5 cm in diameter)

Sponsors & Collaborators

• Baylor College of Medicine: lead
• Southern Star Research: collaborator

Study Sites (1)

Texas Children's Hospital

Houston, Texas, 77030, United States

RECRUITING

MeSH Terms

Conditions

Marfan Syndrome

Interventions

Control Groups

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

Intervention Hierarchy (Ancestors)

Epidemiologic Research Design; Epidemiologic Methods; Investigative Techniques; Research Design; Methods

Study Officials

• Shaine A Morris, MD, MPH

  Baylor College of Medicine

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Shaine A Morris, MD, MPH

CONTACT

832-826-5692; shainem@bcm.edu

Jennifer Bogardus, PT, MPT, PhD

CONTACT

713-794-2070; jennifer.bogardus@utah.edu

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor, Pediatrics-Cardiology

Model Details: The interventional study will be conducted as a parallel study model, with 2 groups in total: Exercise Intervention Group (interventional group) and Current Care Group (control group). Baseline and follow up testing will occur in both groups at identical time intervals.

Study Record Dates

• First Submitted: March 30, 2023
• First Posted: April 12, 2023
• Study Start: September 1, 2023
• Primary Completion: July 1, 2025
• Study Completion (Estimated): July 1, 2027
• Last Updated: January 24, 2024

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)