A clinical trial to test a new treatment for Marfan syndrome

A prospective, randomised, placebo-controlled double blind, multi-centre study of the effects of Irbesartan on aortic dilation in Marfan syndrome (AIMS Study)

Marfan syndrome is a life-threatening inherited disorder that causes the major blood vessel from the heart – the aorta – to expand, increasing the risk of dissection (where the aorta tears). Drugs called beta blockers slow the expansion of the aorta. But many people with the condition, such as those who also have asthma, cannot tolerate beta blockers and experience side effects, such as dizziness, nightmares and lethargy. Moreover, the drugs do not combat the underlying process that weakens the aorta wall, and eventually patients need surgery to replace the weakened vessel. Previous studies have suggested that irbesartan, a drug normally used to treat high blood pressure, might also be able to slow the expansion of the aorta. With this grant, researchers at Imperial College, London will conduct a clinical trial to test how effective irbesartan is in patients with Marfan syndrome. They will examine 490 patients aged between 6 and 40, over five and a half years. If irbesartan proves to be effective and tolerable, it could be an important treatment advance for people with Marfan syndrome. The researchers will also analyse the DNA sequence of the gene that causes Marfan syndrome and genes that affect how irbesartan is metabolised to see if some mutations make the person more or less likely to respond to treatment. The outcome could help doctors make the best choices for individual patients in the future.