Comparison of Two Medications Aimed at Slowing Aortic Root Enlargement in Individuals With Marfan Syndrome--Pediatric Heart Network

• Status: Completed
• Trial end date: 2014-02-01
• Target enrollment: 0
• Participant gender: All

Summary

Marfan syndrome is a hereditary connective tissue disorder. Many individuals with this condition die because of the associated heart and blood vessel abnormalities. This study will compare the effectiveness of two medications, losartan and atenolol, at slowing aortic root enlargement in individuals with Marfan syndrome.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: HealthCore-NERI; New England Research Institutes
• Collaborators: FDA Office of Orphan Products Development; National Heart, Lung, and Blood Institute (NHLBI); National Marfan Foundation
• Treatments: Angiotensin Receptor Antagonists; Atenolol; Losartan

Criteria

Inclusion Criteria:

• Diagnosis of Marfan syndrome, according to Ghent criteria (more information can be found in Appendix D of the protocol)

• Aortic root Z-score greater than 3.0

Exclusion Criteria:

• Prior aortic surgery

• Aortic root dimension at the sinuses of Valsalva greater than 5 cm

• Planned aortic surgery within 6 months of study entry

• Aortic dissection

• Shprintzen-Goldberg syndrome

• Loeys-Dietz syndrome

• Therapeutic (i.e., for arrhythmia, ventricular dysfunction, or valve regurgitation) rather than prophylactic use of angiotensin-converting enzyme (ACE) inhibitor, beta-blocker, or calcium channel blocker

• History of angioedema while taking an ACE inhibitor or beta-blocker

• Intolerance to losartan or other angiotensin II receptor blocker (ARB) that resulted in termination of therapy

• Intolerance to atenolol or other beta-blocker that resulted in termination of therapy

• Kidney dysfunction (i.e., creatinine greater than the upper limit of age-related normal values)

• Asthma of sufficient severity to prohibit the use of a beta-blocker

• Chronic use of steroids and/or beta-adrenergic agents with exacerbations of asthma that are frequent (averaging three or more per year) or severe (requiring hospitalization)

• Diabetes mellitus

• Pregnant or planning to become pregnant within 36 months of study entry

• Inability to complete study procedures, including history of poor acoustic windows (i.e., inability to obtain accurate measurement of aortic root)