Rationale and design of a randomized clinical trial (Marfan Sartan) of angiotensin II receptor blocker therapy versus placebo in individuals with Marfan syndrome

Arch Cardiovasc Dis. 2010 May;103(5):317-25. doi: 10.1016/j.acvd.2010.04.008. Epub 2010 Jul 1.


Background: Recent studies have demonstrated that blockade of the angiotensin II type 1 receptor with losartan decreases aortic damage in an animal model of Marfan syndrome (a KI mouse model with a pathogenic mutation in the gene coding for fibrillin-1).

Aims: To demonstrate a beneficial effect of losartan on aortic dilatation when added to optimal therapy in patients with Marfan syndrome.

Methods: This is a multicentre, randomized, placebo-controlled, double-blind, clinical trial with a 2-year inclusion period and a 3-year follow-up period. Aortic root diameter will be measured using two-dimensional echocardiography. Secondary endpoints will include incidence of aortic dissection, aortic root surgery, death, quality of life, tolerance and compliance with treatments. We aim to enroll a total of 300 patients aged > or =10 years who fulfil the Ghent criteria for Marfan syndrome. Analyses will be based on intention to treat.

Conclusion: The results of this clinical trial could lead to profound modification of the management of aortic risk and complications in patients with Marfan syndrome and possibly in patients with thoracic aortic aneurysms of other aetiologies.

Trial registration: ClinicalTrials.gov NCT00763893.

Publication types

  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Angiotensin II Type 1 Receptor Blockers / therapeutic use*
  • Animals
  • Aortic Aneurysm / diagnostic imaging
  • Aortic Aneurysm / etiology
  • Aortic Aneurysm / prevention & control*
  • Disease Models, Animal
  • Double-Blind Method
  • France
  • Humans
  • Losartan / therapeutic use*
  • Marfan Syndrome / complications
  • Marfan Syndrome / diagnostic imaging
  • Marfan Syndrome / drug therapy*
  • Mice
  • Placebo Effect
  • Research Design*
  • Treatment Outcome
  • Ultrasonography


  • Angiotensin II Type 1 Receptor Blockers
  • Losartan

Associated data

  • ClinicalTrials.gov/NCT00763893