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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This study has been withdrawn prior to enrollment.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)
Sponsor:
ClinicalTrials.gov Identifier:
NCT01361087
First Posted: May 26, 2011
Last Update Posted: February 5, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Johns Hopkins University
Information provided by (Responsible Party):
Ann & Robert H Lurie Children's Hospital of Chicago
  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.

Condition Intervention Phase
Marfan Syndrome Other: Blood draw Phase 3

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Diagnostic
Official Title: Circulating Transforming Growth Factor Beta (TGF-β) in Individuals With

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 ]

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 ]
    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.


Enrollment: 0
Study Start Date: April 2011
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.
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.
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   6 Months to 24 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01361087


Locations
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

Responsible Party: Ann & Robert H Lurie Children's Hospital of Chicago
ClinicalTrials.gov Identifier: NCT01361087     History of Changes
Other Study ID Numbers: IRB # 2011-14507
First Submitted: May 25, 2011
First Posted: May 26, 2011
Last Update Posted: February 5, 2016
Last Verified: February 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Keywords provided by Ann & Robert H Lurie Children's Hospital of Chicago:
TGF-B blood levels Atenolol vs, Losartan Study, NIH, NIHLB and NMF

Additional relevant MeSH terms:
Marfan Syndrome
Arachnodactyly
Syndrome
Disease
Pathologic Processes
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
Mitogens
Mitosis Modulators
Molecular Mechanisms of Pharmacological Action