NCT01361087

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.

Trial Health

35
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Geographic Reach
1 country

1 active site

Status
withdrawn

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 1, 2011

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

May 25, 2011

Completed
1 day until next milestone

First Posted

Study publicly available on registry

May 26, 2011

Completed
3.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2014

Completed
Last Updated

February 5, 2016

Status Verified

February 1, 2016

Enrollment Period

3.7 years

First QC Date

May 25, 2011

Last Update Submit

February 4, 2016

Conditions

Marfan Syndrome

Keywords

TGF-B blood levels Atenolol vs, Losartan Study, NIH, NIHLB and NMF

Outcome Measures

Primary Outcomes (1)

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

    1 year

Secondary Outcomes (1)

  • To determine if circulating levels of TGF-β correlate with clinical outcomes within a treatment group or independent treatment groups.

    1 year

Interventions

Blood drawOTHER

This study includes one blood draw to measure circulating blood levels of TGF-B.

Eligibility Criteria

Age6 Months - 24 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Individual with Marfan syndrome consented in to the Main Atenolol Vs. Losartan NIH study.

You may not qualify if:

  • Subjects in the main PHN Marfan trial who have not achieved the maintenance drug dosing or who have stopped taking study drug.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Children's Memorial Hospital Chicago

Chicago, Illinois, 60614, United States

Location

MeSH Terms

Conditions

Marfan Syndrome

Interventions

Blood Specimen Collection

Condition Hierarchy (Ancestors)

Bone Diseases, DevelopmentalBone DiseasesMusculoskeletal DiseasesHeart Defects, CongenitalCardiovascular AbnormalitiesCardiovascular DiseasesHeart DiseasesAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, InbornConnective Tissue DiseasesSkin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

Specimen HandlingClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisPuncturesSurgical Procedures, OperativeInvestigative Techniques
0

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
DIAGNOSTIC
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 25, 2011

First Posted

May 26, 2011

Study Start

April 1, 2011

Primary Completion

December 1, 2014

Last Updated

February 5, 2016

Record last verified: 2016-02

Locations

US(1)