Circulating Transforming Growth Factor Beta (TGF-β) in Individuals With Marfan Syndrome
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Purpose
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.
| Condition | Intervention |
|---|---|
|
Marfan Syndrome |
Other: Blood draw |
| Study Type: | Interventional |
- To determine if circulating levels of TGF-β correlate with treatment arms: Atenolol vs. Losartan. [ Time Frame: 1 year ] [ Designated as safety issue: No ]
- To determine if circulating levels of TGF-β correlate with clinical outcomes within a treatment group or independent treatment groups. [ Time Frame: 1 year ] [ Designated as safety issue: No ]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.
| Study Start Date: | April 2011 |
| Estimated Primary Completion Date: | December 2014 (Final data collection date for primary outcome measure) |
-
Other: Blood draw
Eligibility| Ages Eligible for Study: | 6 Months to 24 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Individual with Marfan syndrome consented in to the Main Atenolol Vs. Losartan NIH study.
Exclusion Criteria:
- Subjects in the main PHN Marfan trial who have not achieved the maintenance drug dosing or who have stopped taking study drug.
Contacts and Locations More Information
No publications provided
| Responsible Party: | Luciana T. Young M.D., Childrens Memorial Hospital |
| ClinicalTrials.gov Identifier: | NCT01361087 History of Changes |
| Other Study ID Numbers: | IRB # 2011-14507 |
| Study First Received: | May 25, 2011 |
| Last Updated: | June 30, 2011 |
| Health Authority: | United States: Institutional Review Board |
Keywords provided by Ann & Robert H Lurie Children's Hospital of Chicago:
|
To measure TGF-B blood levels in individuals with who are also participating in the Atenolol vs, Losartan Study through NIH, NIHLB and NMF |
Additional relevant MeSH terms:
|
Marfan Syndrome Arachnodactyly Bone Diseases, Developmental Bone Diseases Musculoskeletal Diseases Heart Defects, Congenital Cardiovascular Abnormalities Cardiovascular Diseases |
Heart Diseases Abnormalities, Multiple Congenital Abnormalities Genetic Diseases, Inborn Connective Tissue Diseases Limb Deformities, Congenital Musculoskeletal Abnormalities |
ClinicalTrials.gov processed this record on May 23, 2013