Towards Optimal Treatment of Marfan Syndrome

Marfan Syndrome Clinical Trial

— TOWER  

Official title:

Towards Optimal Treatment of Marfan Syndrome

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05944614
• Other study ID #: NL82669.018.22
• Status: Recruiting
• Start date: February 23, 2023
• Est. completion date: June 2025

Study information

• Verified date: July 2023
• Source: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
• Contact: Daan Bosshardt, MD
• Phone: +31 (0) 20 566 9111
• Email: d.bosshardt[@]amsterdamumc.nl
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: June 2025
• Est. primary completion date: June 2025
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 50 Years

Eligibility

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

Related Conditions & MeSH terms

• Marfan Syndrome
• Syndrome

Intervention

Other:
• Magnetic Resonance imaging examination
3D CINE and 4D flow MRI sequence

Locations

Country	Name	City	State
Netherlands	Amsterdam UMC, location AMC	Amsterdam	Meibergdreef 9
Netherlands	University Medical Center Groningen	Groningen
Netherlands	Leiden University Medical Center	Leiden	Noord-Holland
Netherlands	Radboud University Medical Center	Nijmegen	Gelderland

Sponsors (2)

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

Country where clinical trial is conducted

Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Difference in 3D aortic displacement expressed in mm between Marfan patients and healthy volunteers	aortic displacement in the thoracic aorta	Baseline
Primary	Difference in pulse wave velocity, expressed in m/s between Marfan patients and healthy volunteers	pulse wave velocity expressed in m/s in the thoracic aorta	Baseline
Primary	Difference in oscillatory shear index (dimensionless) expressed in m/s between Marfan patients and healthy volunteers	oscillatory shear index (dimensionless) in the thoracic aorta	Baseline
Primary	Change in aortic displacement expressed in mm, between MFS patient aortas prior to compared to after PEARS procedure or conventional aortic surgery.	aortic displacement expressed in mm in the thoracic aorta	Change from baseline to 6 months after surgery
Primary	Change in aortic pulse wave velocity, between MFS patient aortas prior to compared to after PEARS procedure or conventional aortic surgery.	pulse wave velocity in the thoracic aorta	Change from baseline to 6 months after surgery
Primary	Change in oscillatory shear index (dimensionless) between MFS patient aortas prior to compared to after PEARS procedure or conventional aortic surgery.	oscillatory shear index (dimensionless) in the thoracic aorta	Change from baseline to 6 months after surgery
Secondary	Difference in 3D aortic displacement expressed in mm between Marfan patients with and without a native aorta	aortic displacement in the thoracic aorta	Baseline
Secondary	Difference in 3D aortic displacement expressed in mm between Marfan patients with aortic root diameter >4.5cm versus <4.5 cm	aortic displacement in the thoracic aorta	Baseline
Secondary	Difference in 3D aortic displacement expressed in mm between Marfan patients with a haploinsufficient fibrillin 1 mutation versus dominant negative mutation	aortic displacement in the thoracic aorta	Baseline
Secondary	Difference in 3D aortic displacement expressed in mm between male versus female Marfan patients	aortic displacement in the thoracic aorta	Baseline
Secondary	Difference in 3D aortic displacement expressed in mm between Marfan patients based on anithypertensive medication used	aortic displacement in the thoracic aorta	Baseline
Secondary	Difference in pulse wave velocity, expressed in m/s between Marfan patients with and without a native aorta	pulse wave velocity expressed in m/s in the thoracic aorta	Baseline
Secondary	Difference in pulse wave velocity, expressed in m/s between Marfan patients with an aortic root diameter >4.5 cm versus <4.5 cm	pulse wave velocity expressed in m/s in the thoracic aorta	Baseline
Secondary	Difference in pulse wave velocity, expressed in m/s between Marfan patients with a haploinsufficient fibrillin-1 mutation versus a dominant negative mutation	pulse wave velocity expressed in m/s in the thoracic aorta	Baseline
Secondary	Difference in pulse wave velocity, expressed in m/s between male and female Marfan patients	pulse wave velocity expressed in m/s in the thoracic aorta	Baseline
Secondary	Difference in pulse wave velocity, expressed in m/s between Marfan patients based on anithypertensive medication used	pulse wave velocity expressed in m/s in the thoracic aorta	Baseline
Secondary	Difference in oscillatory shear index (dimensionless) expressed in m/s between Marfan patients with and without a native aorta	oscillatory shear index (dimensionless) in the thoracic aorta	Baseline
Secondary	Difference in oscillatory shear index (dimensionless) expressed in m/s between Marfan patients with aortic root diameter >4.5cm versus <4.5 cm	oscillatory shear index (dimensionless) in the thoracic aorta	Baseline
Secondary	Difference in oscillatory shear index (dimensionless) expressed in m/s between Marfan patients with a haploinsufficient fibrillin 1 mutation versus dominant negative mutation	oscillatory shear index (dimensionless) in the thoracic aorta	Baseline
Secondary	Difference in oscillatory shear index (dimensionless) expressed in m/s between male versus female Marfan patients	oscillatory shear index (dimensionless) in the thoracic aorta	Baseline
Secondary	Difference in oscillatory shear index (dimensionless) expressed in m/s between Marfan patients based on anithypertensive medication used	oscillatory shear index (dimensionless) in the thoracic aorta	Baseline
Secondary	The correlation between regional wall shear stress and elastic fibre thickness in resected aorta tissue of MFS patients.	Correlation of locally MRI measured wall shear stress and histologic findings in the thoracic aorta	Baseline MRI with aortic surgery tissue