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

Primary Purpose

Marfan Syndrome

Status
Withdrawn
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Blood draw
Sponsored by
Ann & Robert H Lurie Children's Hospital of Chicago
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional diagnostic trial for Marfan Syndrome focused on measuring TGF-B blood levels Atenolol vs, Losartan Study, NIH, NIHLB and NMF

Eligibility Criteria

6 Months - 24 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Children's Memorial Hospital Chicago

Outcomes

Primary Outcome Measures

To determine if circulating levels of TGF-β correlate with treatment arms: Atenolol vs. Losartan.

Secondary Outcome Measures

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.

Full Information

First Posted
May 25, 2011
Last Updated
February 4, 2016
Sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Collaborators
Johns Hopkins University
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1. Study Identification

Unique Protocol Identification Number
NCT01361087
Brief Title
Circulating Transforming Growth Factor Beta (TGF-β) in Individuals With Marfan Syndrome
Official Title
Circulating Transforming Growth Factor Beta (TGF-β) in Individuals With
Study Type
Interventional

2. Study Status

Record Verification Date
February 2016
Overall Recruitment Status
Withdrawn
Why Stopped
No outside recruitment of subjects from Main Atenolol VS Losartan NIH study
Study Start Date
April 2011 (undefined)
Primary Completion Date
December 2014 (Actual)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Collaborators
Johns Hopkins University

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Marfan Syndrome
Keywords
TGF-B blood levels Atenolol vs, Losartan Study, NIH, NIHLB and NMF

7. Study Design

Primary Purpose
Diagnostic
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)

8. Arms, Groups, and Interventions

Intervention Type
Other
Intervention Name(s)
Blood draw
Intervention Description
This study includes one blood draw to measure circulating blood levels of TGF-B.
Primary Outcome Measure Information:
Title
To determine if circulating levels of TGF-β correlate with treatment arms: Atenolol vs. Losartan.
Time Frame
1 year
Secondary Outcome Measure Information:
Title
To determine if circulating levels of TGF-β correlate with clinical outcomes within a treatment group or independent treatment groups.
Description
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
24 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Facility Information:
Facility Name
Children's Memorial Hospital Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60614
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
Undecided

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

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