Research

Beta Blocker Therapy for Marfan Syndrome

Trial of Beta Blocker Therapy vs Angiotensin II Receptor Blocker Therapy in Individuals with Marfan Syndrome

Protocol Description

This multi-center study being conducted through the Pediatric Heart Network investigates whether losartan, an angiotensin II receptor blocker, or atenolol, a beta blocker, is more effective at slowing the rate of aortic root enlargement in Marfan syndrome patients. Marfan syndrome is a connective tissue disorder, which sometimes has as a symptom dilation of the aortic root, the part where the aorta exits the heart. This study will compare effectiveness and side effects of the two drugs, with an aim of determining which is best in treatment of children to potentially improve heart function and reduce risk of life-threatening aortic tears.

Eligibility Criteria

Subject to certain exclusion criteria, males and females up to age 25 who have a diagnosis of Marfan syndrome with an aortic root measurement of greater than 3.0 cm are eligible to participate.
Males: 6 months to 25 years
Females: 6 months to 25 years

Requirements
Following an initial screening examination, participants will be randomly assigned a prescription of either losartan or atenolol, the dosage for which will be adjusted based on the patient’s response and weight changes during the duration of the study. Use of a Holter monitor to provide heart rate information over a 24-hour period will be required after each dosage increase in the dose of the study drug and also at the 6, 12, 24 and 36-month visits. Blood sampling and echocardiograms will also be required at various intervals.
Visits: 5 or more, depending on individual response
Duration: Up to 3 years

Status: Enrolls Only from Current Patient Cases

Sources of Support
National Heart, Lung and Blood Institute
Food and Drug Administration Office of Orphan Products Development
National Marfan Foundation

Additional Resources
Study Description at Pediatric Heart Network
Study Description at National Institutes of Health