Clinical Trial: Circulating Transforming Growth Factor Beta (TGF-β) in Individuals With Marfan Syndrome

Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional

Official Title: Circulating Transforming Growth Factor Beta (TGF-β) in Individuals With

Brief Summary: Transforming Growth Factor Beta (TGF-β) is a protein that controls proliferation, cellular differentiation, and other functions in most cells. TGF-β levels play a major role in the pathogenesis of Marfan syndrome, a disease characterized by disproportionate height, long extremities, lens dislocation in the eyes and heart complications such as mitral valve prolapse and aortic enlargement increasing the likelihood of aortic dissection. While the underlying defect in Marfan syndrome is faulty synthesis of the glycoprotein fibrillin I, normally an important component of elastic fibers it has been shown that the Marfan syndrome phenotype can be relieved by addition of a TGF-β antagonist in affected mice.

Detailed Summary: Transforming Growth Factor Beta (TGF-β) is a protein that controls proliferation, cellular differentiation, and other functions in most cells. TGF-β levels play a major role in the pathogenesis of Marfan syndrome, a disease characterized by disproportionate height, long extremities, lens dislocation in the eyes and heart complications such as mitral valve prolapse and aortic enlargement increasing the likelihood of aortic dissection. While the underlying defect in Marfan syndrome is faulty synthesis of the glycoprotein fibrillin I, normally an important component of elastic fibers it has been shown that the Marfan syndrome phenotype can be relieved by addition of a TGF-β antagonist in affected mice. This suggest that while the symptoms of Marfan syndrome may seem consistent with a connective tissue disorder, the mechanism is more likely related to reduced sequestration of TGF-β by fibrillin.
Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago

Current Primary Outcome: To determine if circulating levels of TGF-β correlate with treatment arms: Atenolol vs. Losartan. [ Time Frame: 1 year ]

Original Primary Outcome: Same as current

Current Secondary Outcome: To determine if circulating levels of TGF-β correlate with clinical outcomes within a treatment group or independent treatment groups. [ Time Frame: 1 year ]

These clinical outcomes may be a change in aortic root Z-score, final aortic root dimension, final aortic root Z-score and other clinical outcomes in the main Marfan trial.


Original Secondary Outcome: Same as current

Information By: Ann & Robert H Lurie Children's Hospital of Chicago

Dates:
Date Received: May 25, 2011
Date Started: April 2011
Date Completion:
Last Updated: February 4, 2016
Last Verified: February 2016