Circulating Transforming Growth Factor Beta (TGF-β) in Individuals With Marfan Syndrome
- Conditions
- Marfan Syndrome
- Registration Number
- NCT01361087
- 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 Description
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.
Recruitment & Eligibility
- Status
- WITHDRAWN
- Sex
- All
- Target Recruitment
- Not specified
- Individual with Marfan syndrome consented in to the Main Atenolol Vs. Losartan NIH study.
- Subjects in the main PHN Marfan trial who have not achieved the maintenance drug dosing or who have stopped taking study drug.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Primary Outcome Measures
Name Time Method To determine if circulating levels of TGF-β correlate with treatment arms: Atenolol vs. Losartan. 1 year
- Secondary Outcome Measures
Name Time Method To determine if circulating levels of TGF-β correlate with clinical outcomes within a treatment group or independent treatment groups. 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.
Trial Locations
- Locations (1)
Children's Memorial Hospital Chicago
🇺🇸Chicago, Illinois, United States