Development of a Blood Test for Marfan Syndrome
1 other identifier
observational
336
1 country
5
Brief Summary
The objective of this study is to determine whether a simple blood test can be a useful clinical tool for monitoring aortic disease in Marfan syndrome and Marfan-related disorders.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started May 2013
Longer than P75 for all trials
5 active sites
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
May 1, 2013
CompletedFirst Submitted
Initial submission to the registry
May 23, 2014
CompletedFirst Posted
Study publicly available on registry
May 28, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2017
CompletedJanuary 5, 2017
January 1, 2017
4.6 years
May 23, 2014
January 4, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
microfibril fragments
Plasma will be tested using sandwich ELISAs and standard curves to quantitate fragments of fibrillin-1, fibrillin-2, and fibulin-4.
measured yearly over 5 years
Secondary Outcomes (1)
Aortic root growth
yearly for five years
Other Outcomes (1)
medical and lifestyle issues
yearly for five years
Study Arms (3)
Marfan
Diagnosis of Marfan syndrome, according to Ghent criteria.
Marfan Related Disorders
Diagnosis of Loeys-Dietz syndrome, vascular Ehlers-Danlos syndrome, or Familial Thoracic Aortic Aneurysm and Dissection.
Control Subjects
Unaffected by Marfan or Marfan related disorders.
Eligibility Criteria
The study population includes 3 groups: (1) individuals with Marfan syndrome; (2) individuals with Marfan-related disorders; (3) unaffected controls. Both genders, ages 4 to 26, will be included. Geographic locations will include the Pacific Northwest, Texas, and the Salt Lake City/Denver areas.
You may qualify if:
- Age 4-26 years of age for all participants
- Able and willing to sign informed consent/assent
- Signed HIPPA compliant research authorization
You may not qualify if:
- Other known cardiovascular disorder for unaffected controls
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Shriners Hospitals for Childrenlead
- Oregon Health and Science Universitycollaborator
- University of Utahcollaborator
- The University of Texas Health Science Center, Houstoncollaborator
Study Sites (5)
Lurie Childrens Hospital
Chicago, Illinois, 60611-2605, United States
University of Nebraska
Omaha, Nebraska, 68114, United States
Oregon Health & Science University
Portland, Oregon, 97239, United States
Shriners Hospital for Children
Portland, Oregon, 97239, United States
University of Texas at Houston
Houston, Texas, 77030, United States
Biospecimen
Plasma Frozen Cells
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Lynn Y. Sakai, Ph.D.
Shriners Hospitals for Children
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- CASE CONTROL
- Time Perspective
- PROSPECTIVE
- Target Duration
- 5 Years
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Sr. Investigator
Study Record Dates
First Submitted
May 23, 2014
First Posted
May 28, 2014
Study Start
May 1, 2013
Primary Completion
December 1, 2017
Study Completion
December 1, 2017
Last Updated
January 5, 2017
Record last verified: 2017-01
Data Sharing
- IPD Sharing
- Will not share