A Phase 3 interventional study of Blood draw in Marfan Syndrome, sponsored by Ann & Robert H Lurie Children's Hospital of Chicago. Withdrawn at 1 site in United States. Open to participants aged 6 Months to 24 Years. Per ClinicalTrials.gov, last updated 2016-02-05.
Sponsored by Ann & Robert H Lurie Children's Hospital of Chicago · Phase 3, Interventional, and Diagnostic
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.
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.
73 studies on the registry are indexed under Marfan Syndrome; 16 are open to participants now.
Browse Marfan Syndrome studies →Ann & Robert H Lurie Children's Hospital of Chicago is the lead sponsor of 180 studies on the registry; 30 are open to participants now.
Of its 10 completed or terminated interventional studies of FDA-regulated products, 5 (50%) have results posted.
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This study includes one blood draw to measure circulating blood levels of TGF-B.
To determine if circulating levels of TGF-β correlate with treatment arms: Atenolol vs. Losartan.
Time frame: 1 year
To determine if circulating levels of TGF-β correlate with clinical outcomes within a treatment group or independent treatment groups.
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.
Time frame: 1 year
Plan to share: Undecided
No publications or documents are linked to this record.
The record gives a start of Apr 2011 and a primary completion of Dec 2014. It does not say whether these dates are actual or estimated.
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This study is withdrawn, as verified in Feb 2016. You cannot join it, but the record below documents what was studied.
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Ann & Robert H Lurie Children's Hospital of Chicago →