Resveratrol as potential aortic growth inhibitor in patients with Marfan Syndrome

Phase 2

Recruiting

Conditions: connective tissue disease
Marfan syndrome
10010394
10007510

Registration Number: NL-OMON49268

Lead Sponsor: Academisch Medisch Centrum

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 100

Inclusion Criteria

- Marfan patients with a known FBN1 mutation
- Between 18-50 years of age
- Native aortic root (n<=50) or aortic root replacement (n<=50)
- At least 2 echocardiography measurements and 1 MRI scan of the aorta prior to
the study
- Sinus rythm

Exclusion Criteria

- More than one vascular prosthesis
- Aortic root diameter > 45 mm
- Aortic surgery likely within 6 months of inclusion
- Aortic surgery in the last 6 months prior to inclusion
- Aortic dissection
- Contraindications for MR imaging
- Mental retardation

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>There are three main study parameters/endpoints:<br /><br>1. A decrease in aortic aneurysm expansion rate in > 90% of the study subjects<br /><br>as measured by echo between baseline (at timepoint of inclusion in the study)<br /><br>and after twelve months of follow-up (after resveratrol treatment).<br /><br>2. Any statistical significant beneficial change in aortic elasticity or aortic<br /><br>wall shear rate as measured by magnetic resonance imaging (MRI) between<br /><br>baseline (at timepoint of inclusion in the study) and after twelve months of<br /><br>follow-up (after resveratrol treatment).<br /><br>3. Any statistical significant beneficial change in endothelial function as<br /><br>measured by Flow Mediated Dilatation (FMD) </p><br>

Secondary Outcome Measures

Name	Time	Method
<p>1. To evaluate functional cardiac parameters by echocardiography in Marfan<br /><br>patients upon treatment with resveratrol.<br /><br>2. To evaluate changes in blood pressure/heart rate in patients with Marfan<br /><br>syndrome treated with resveratrol.<br /><br>3. To evaluate changes in endothelial cell function in patients with Marfan<br /><br>syndrome treated with resveratrol by flow mediated dilation measurement (FMD).<br /><br>4. To evaluate differences between Marfan patients who have had aortic<br /><br>replacement and Marfan patients with a native aorta, before or after<br /><br>resveratrol.<br /><br>5. To evaluate differences between Marfan patients who have a haploinsufficient<br /><br>(HI) FBN1 mutation or a dominant negative (DN) FBN1 mutation. Geneticists<br /><br>determine the HI or DN status, however, skin cultures may provide conclusive<br /><br>results when the mutation effect is unpredictable.</p><br>