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

This study has been withdrawn prior to enrollment.
(No outside recruitment of subjects from Main Atenolol VS Losartan NIH study)
Johns Hopkins University
Information provided by:
Ann & Robert H Lurie Children's Hospital of Chicago Identifier:
First received: May 25, 2011
Last updated: June 30, 2011
Last verified: May 2011
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

Resource links provided by NLM:

Further study details as provided by Ann & Robert H Lurie Children's Hospital of Chicago:

Primary Outcome Measures:
  • To determine if circulating levels of TGF-β correlate with treatment arms: Atenolol vs. Losartan. [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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)
Intervention Details:
    Other: Blood draw
    This study includes one blood draw to measure circulating blood levels of TGF-B.

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01361087

United States, Illinois
Children's Memorial Hospital Chicago
Chicago, Illinois, United States, 60614
Sponsors and Collaborators
Ann & Robert H Lurie Children's Hospital of Chicago
Johns Hopkins University
  More Information

No publications provided

Responsible Party: Luciana T. Young M.D., Childrens Memorial Hospital 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
Abnormalities, Multiple
Bone Diseases
Bone Diseases, Developmental
Cardiovascular Abnormalities
Cardiovascular Diseases
Congenital Abnormalities
Connective Tissue Diseases
Genetic Diseases, Inborn
Heart Defects, Congenital
Heart Diseases
Limb Deformities, Congenital
Musculoskeletal Abnormalities
Musculoskeletal Diseases processed this record on November 27, 2015