The Ghent Marfan Trial--a randomized, double-blind placebo controlled trial with @entity9886 in Marfan XXXX treated with b-blockers.

@entity1 @entity339 @entity384 @entity1614 @entity2178 @entity9886 @entity397 @entity2533 @entity432 @entity65
BACKGROUND: @entity384 , dissection and @entity397 are major clinical problems in @entity1614 ( @entity1614 ). Although b-blockers remain the standard of preventive treatment, preliminary results from animal studies and a selected group of severely affected @entity1614 @entity1 show significant benefit from treatment with @entity9886 , an @entity2178 receptor blocker with @entity2533 inhibiting potential. Large-scale @entity1 trials are now needed to confirm these results. This trial aims to evaluate the combined effect of both drugs. METHODS: We are conducting a prospective randomized placebo controlled double blind phase III study aiming to include 174 @entity1614 @entity1 (age >=  10 years and z-score >=  2). @entity1 already taking b-blockers are randomized for weight-adjusted treatment with @entity9886 versus placebo. The primary endpoint is decrease in aortic root growth rate. Secondary endpoints are aortic dissection/surgery, progression of aortic/ @entity339 , @entity432 , left ventricular systolic/diastolic function, quality of life and genetic modifiers. Echocardiography, vascular echo-Doppler and quality of life assessment will be performed at baseline and at 6-monthly follow-ups for 3 years. MRI evaluation will be performed at baseline and at the end of the trial. CONCLUSION: This trial will study new therapeutic strategies for the prevention of serious @entity65 in @entity1614 . The uniqueness in our trial is that the additive effect of @entity9886 and b-blocker will be evaluated in a large spectrum of disease severity. A combination of ultrasound and MRI will allow detailed evaluation of anatomic and functional properties of the aorta and left ventricle.
