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Towards Optimal Treatment of Marfan Syndrome

Towards Optimal Treatment of Marfan Syndrome

Status
Completed
Phases
Unknown
Study type
Observational
Source
ClinicalTrials.gov
Registry ID
NCT05944614
Acronym
TOWER
Enrollment
148
Registered
2023-07-13
Start date
2023-02-23
Completion date
2025-04-16
Last updated
2025-07-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Marfan Syndrome

Keywords

Magnetic Resonance Imaging, Aortic disease

Brief 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.

Interventions

3D CINE and 4 dimensional (4D) flow MRI sequence

Sponsors

Netherlands Organisation for Scientific Research
CollaboratorOTHER_GOV
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Lead SponsorOTHER

Study design

Observational model
COHORT
Time perspective
PROSPECTIVE

Eligibility

Sex/Gender
ALL
Age
18 Years to 50 Years
Healthy volunteers
Yes

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

Design outcomes

Primary

MeasureTime frameDescription
Difference in 3D aortic displacement in millimeters between Marfan patients (MFS) and healthy volunteers.Baselineaortic displacement in the thoracic aorta. normal values unknown, but single paper: Marfan patients range: 1.77 to 10.03 millimeters, volunteers 7.34 ± 1.69 millimeters. lower is worse.
Difference in pulse wave velocity, in meters per second between Marfan patients and healthy volunteers.Baselinepulse wave velocity expressed in m/s in the thoracic aorta, normal values age dependent: but range +/- 4 - 30 m/s, higher is worse
Difference in oscillatory shear index (dimensionless) expressed in m/s between Marfan patients and healthy volunteersBaselineoscillatory shear index (dimensionless) in the thoracic aorta, range between 0 - 0.5 (No normal values defined), higher is worse
Change in aortic displacement expressed in millimeters , between MFS patient aortas prior to compared to after PEARS procedure or conventional aortic surgery.Change from baseline to 6 months after surgeryaortic displacement expressed in millimeters in the thoracic aorta. Pre-post change unknown, however operated 3.60 +/- 1.0 millimeter versus native aorta 7.01 +/- 3.09 millimeters. Lower is worse
Change in aortic pulse wave velocity (meters/second), between MFS patient aortas prior to compared to after PEARS procedure or conventional aortic surgery.Change from baseline to 6 months after surgerypulse wave velocity in the thoracic aorta expressed in meters/second. Higher is worse. normal values for change unknown.
Change in oscillatory shear index (dimensionless) between MFS patient aortas prior to compared to after PEARS procedure or conventional aortic surgery.Change from baseline to 6 months after surgeryoscillatory shear index (dimensionless) in the thoracic aorta. Higher is worse range between 0 -5.

Secondary

MeasureTime frameDescription
Difference in pulse wave velocity, expressed in m/s between Marfan patients with an aortic root diameter >4.5 cm versus <4.5 cmBaselinepulse wave velocity expressed in m/s in the thoracic aorta. Higher is worse. Normal values unknown.
Difference in pulse wave velocity, expressed in m/s between Marfan patients with a haploinsufficient fibrillin-1 mutation versus a dominant negative mutationBaselinepulse wave velocity expressed in m/s in the thoracic aorta. Higher is worse. Normal values unknown.
Difference in pulse wave velocity, expressed in m/s between male and female Marfan patientsBaselinepulse wave velocity expressed in m/s in the thoracic aorta Higher is worse. Normal values unknown.
Difference in pulse wave velocity, expressed in m/s between Marfan patients based on antihypertensive medication usedBaselinepulse wave velocity expressed in m/s in the thoracic aorta
Difference in oscillatory shear index (dimensionless) expressed in m/s between Marfan patients with and without a native aortaBaselineoscillatory shear index (dimensionless) in the thoracic aorta. Higher is worse. Normal values unknown.
Difference in 3D aortic displacement expressed in mm between Marfan patients with and without a native aortaBaselineaortic displacement in the thoracic aorta, lower is worse. operated 3.60 +/- 1.0 millimeter versus native aorta 7.01 +/- 3.09 millimeters
Difference in oscillatory shear index (dimensionless) expressed in m/s between Marfan patients with a haploinsufficient fibrillin 1 mutation versus dominant negative mutationBaselineoscillatory shear index (dimensionless) in the thoracic aorta. Higher is worse. Normal values unknown.
Difference in oscillatory shear index (dimensionless) expressed in m/s between male versus female Marfan patientsBaselineoscillatory shear index (dimensionless) in the thoracic aorta. Higher is worse. Normal values unknown.
Difference in oscillatory shear index (dimensionless) expressed in m/s between Marfan patients based on antihypertensive medication usedBaselineoscillatory shear index (dimensionless) in the thoracic aorta. Higher is worse. Normal values unknown.
The correlation between regional wall shear stress and elastic fibre thickness in resected aorta tissue of MFS patients.Baseline MRI with aortic surgery tissueCorrelation of locally MRI measured wall shear stress and histologic findings in the thoracic aorta. Neither is worse. Normal values dependent on location of measurement, exploratory outcome. .
Difference in oscillatory shear index (dimensionless) expressed in m/s between Marfan patients with aortic root diameter >4.5cm versus <4.5 cmBaselineoscillatory shear index (dimensionless) in the thoracic aorta. Higher is worse. Normal values unknown.
Difference in 3D aortic displacement expressed in mm between Marfan patients with aortic root diameter >4.5cm versus <4.5 cmBaselineaortic displacement in the thoracic aorta, lower is worse. Normal values unknown.
Difference in 3D aortic displacement expressed in mm between Marfan patients with a haploinsufficient fibrillin 1 mutation versus dominant negative mutationBaselineaortic displacement in the thoracic aorta. Lower is worse. Normal values unknown.
Difference in 3D aortic displacement expressed in mm between male versus female Marfan patientsBaselineaortic displacement in the thoracic aorta,Lower is worse. Normal values unknown.
Difference in 3D aortic displacement expressed in mm between Marfan patients based on antihypertensive medication usedBaselineaortic displacement in the thoracic aorta, Lower is worse. Normal values unknown.
Difference in pulse wave velocity, expressed in meters per second between Marfan patients with and without a native aortaBaselinepulse wave velocity expressed in m/s in the thoracic aorta, Lower is worse. Normal values unknown. Single study in general aneurysm population: pre surgery: 18.0 +/-4.0 meters per second post surgery 20.6 +/- 6.0 meters per second. higher is worse

Countries

Netherlands

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026