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

Marfan Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT01361087
• Other study ID #: IRB # 2011-14507
• Status: Withdrawn
• Phase: Phase 3
• First received: May 25, 2011
• Last updated: February 4, 2016
• Start date: April 2011

Study information

• Verified date: February 2016
• Source: Ann & Robert H Lurie Children's Hospital of Chicago
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. primary completion date: December 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Months to 24 Years

Eligibility

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.

Study Design

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Diagnostic

Related Conditions & MeSH terms

• Marfan Syndrome

Intervention

Other:
• Blood draw
This study includes one blood draw to measure circulating blood levels of TGF-B.

Locations

Country	Name	City	State
United States	Children's Memorial Hospital Chicago	Chicago	Illinois

Sponsors (2)

Lead Sponsor	Collaborator
Ann & Robert H Lurie Children's Hospital of Chicago	Johns Hopkins University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To determine if circulating levels of TGF-ß correlate with treatment arms: Atenolol vs. Losartan.		1 year	No
Secondary	To determine if circulating levels of TGF-ß 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.	1 year	No