CompletedNot Applicable

Towards Optimal Treatment of Marfan Syndrome

Netherlands148 participantsStarted 2023-02-23

Plain-language summary

The purpose of this study is to determine whether new magnetic resonance imaging techniques can be used as a biomarker of aortic disease severity in patients with Marfan syndrome.

Who can participate

Age range

18 Years – 50 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * MFS patients with a known FBN1 mutation * Between 18-50 years of age Exclusion Criteria: * Contraindication for MR imaging * Mental retardation * Pregnancy, or planned pregnancy during study period Additional exclusion criteria healthy controls: \- History of aorta disease

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Difference in 3D aortic displacement in millimeters between Marfan patients (MFS) and healthy volunteers.

Timeframe: Baseline

Difference in pulse wave velocity, in meters per second between Marfan patients and healthy volunteers.

Timeframe: Baseline

Difference in oscillatory shear index (dimensionless) expressed in m/s between Marfan patients and healthy volunteers

Timeframe: Baseline

Change in aortic displacement expressed in millimeters , between MFS patient aortas prior to compared to after PEARS procedure or conventional aortic surgery.

Timeframe: Change from baseline to 6 months after surgery

Change in aortic pulse wave velocity (meters/second), between MFS patient aortas prior to compared to after PEARS procedure or conventional aortic surgery.

Timeframe: Change from baseline to 6 months after surgery

Change in oscillatory shear index (dimensionless) between MFS patient aortas prior to compared to after PEARS procedure or conventional aortic surgery.

Timeframe: Change from baseline to 6 months after surgery

Trial details

NCT IDNCT05944614

SponsorAcademisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2025-04-16

View on ClinicalTrials.gov More Marfan Syndrome trials

Who can participate

Age range

18 Years – 50 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Difference in 3D aortic displacement in millimeters between Marfan patients (MFS) and healthy volunteers.

Timeframe: Baseline

Difference in pulse wave velocity, in meters per second between Marfan patients and healthy volunteers.

Timeframe: Baseline

Difference in oscillatory shear index (dimensionless) expressed in m/s between Marfan patients and healthy volunteers

Timeframe: Baseline

Change in aortic displacement expressed in millimeters , between MFS patient aortas prior to compared to after PEARS procedure or conventional aortic surgery.

Timeframe: Change from baseline to 6 months after surgery

Change in aortic pulse wave velocity (meters/second), between MFS patient aortas prior to compared to after PEARS procedure or conventional aortic surgery.

Timeframe: Change from baseline to 6 months after surgery

Change in oscillatory shear index (dimensionless) between MFS patient aortas prior to compared to after PEARS procedure or conventional aortic surgery.

Timeframe: Change from baseline to 6 months after surgery