Towards Optimal Treatment of Marfan Syndrome
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 148
- 试验地点
- 4
- 主要终点
- Difference in pulse wave velocity, in meters per second between Marfan patients and healthy volunteers.
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 50 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •MFS patients with a known FBN1 mutation
- •Between 18-50 years of age
排除标准
- •Contraindication for MR imaging
- •Mental retardation
- •Pregnancy, or planned pregnancy during study period
- •Additional exclusion criteria healthy controls:
- •History of aorta disease
结局指标
主要结局
Difference in pulse wave velocity, in meters per second between Marfan patients and healthy volunteers.
时间窗: Baseline
pulse 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 volunteers
时间窗: Baseline
oscillatory shear index (dimensionless) in the thoracic aorta, range between 0 - 0.5 (No normal values defined), higher 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.
时间窗: Change from baseline to 6 months after surgery
pulse 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.
时间窗: Change from baseline to 6 months after surgery
oscillatory shear index (dimensionless) in the thoracic aorta. Higher is worse range between 0 -5.
Difference in 3D aortic displacement in millimeters between Marfan patients (MFS) and healthy volunteers.
时间窗: Baseline
aortic 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.
Change in aortic displacement expressed in millimeters , between MFS patient aortas prior to compared to after PEARS procedure or conventional aortic surgery.
时间窗: Change from baseline to 6 months after surgery
aortic 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
次要结局
- Difference in pulse wave velocity, expressed in m/s between Marfan patients with an aortic root diameter >4.5 cm versus <4.5 cm(Baseline)
- Difference in 3D aortic displacement expressed in mm between Marfan patients based on antihypertensive medication used(Baseline)
- Difference in pulse wave velocity, expressed in meters per second between Marfan patients with and without a native aorta(Baseline)
- Difference in pulse wave velocity, expressed in m/s between Marfan patients based on antihypertensive medication used(Baseline)
- Difference in oscillatory shear index (dimensionless) expressed in m/s between Marfan patients with and without a native aorta(Baseline)
- Difference in 3D aortic displacement expressed in mm between Marfan patients with and without a native aorta(Baseline)
- Difference in 3D aortic displacement expressed in mm between male versus female Marfan patients(Baseline)
- Difference in 3D aortic displacement expressed in mm between Marfan patients with aortic root diameter >4.5cm versus <4.5 cm(Baseline)
- Difference in 3D aortic displacement expressed in mm between Marfan patients with a haploinsufficient fibrillin 1 mutation versus dominant negative mutation(Baseline)
- Difference in pulse wave velocity, expressed in m/s between Marfan patients with a haploinsufficient fibrillin-1 mutation versus a dominant negative mutation(Baseline)
- Difference in oscillatory shear index (dimensionless) expressed in m/s between male versus female Marfan patients(Baseline)
- Difference in oscillatory shear index (dimensionless) expressed in m/s between Marfan patients based on antihypertensive medication used(Baseline)
- The correlation between regional wall shear stress and elastic fibre thickness in resected aorta tissue of MFS patients.(Baseline MRI with aortic surgery tissue)
- Difference in pulse wave velocity, expressed in m/s between male and female Marfan patients(Baseline)
- Difference in oscillatory shear index (dimensionless) expressed in m/s between Marfan patients with aortic root diameter >4.5cm versus <4.5 cm(Baseline)
- Difference in oscillatory shear index (dimensionless) expressed in m/s between Marfan patients with a haploinsufficient fibrillin 1 mutation versus dominant negative mutation(Baseline)
研究者
Dr. Ir. Pim van Ooij
Principal Investigator
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)