Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network
NCT ID: NCT01586871
Last Updated: 2021-02-24
Study Results
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View full resultsBasic Information
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COMPLETED
1147 participants
OBSERVATIONAL
2012-03-31
2017-06-30
Brief Summary
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The research questions are:
1. Is the artery health of MPS I, II and VI patients different than healthy controls?
2. Is the artery health of MPS VI patients different than MPS I and II patients?
It is hypothesized that MPS patients will have poorer outcomes of artery health compared to healthy controls.
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Detailed Description
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Specific Aim #1: Compare carotid artery intima-media thickness and carotid stiffness in individuals with MPS I, II, and VI (treated and non-treated) vs. healthy age-and gender-matched controls. It is hypothesized that MPS patients will have increased carotid artery thickness and reduced carotid compliance and distensibility compared to healthy controls.
Specific Aim #2: Compare carotid artery intima-media thickness and carotid stiffness in individuals with MPS VI vs. I and II and between MPS I patients clinically treated with HSCT vs. ERT. It is hypothesized that MPS VI will have decreased carotid thickness and increased carotid compliance and distensibility compared to MPS I and II and that MPS I patients treated with ERT will have increased carotid thickness and reduced carotid compliance and distensibility compared to MPS I patients treated with HSCT.
Conditions
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Study Design
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CASE_CONTROL
RETROSPECTIVE
Eligibility Criteria
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Inclusion Criteria
* Be diagnosed with MPS I, MPS II or MPS VI
Exclusion Criteria
3 Years
18 Years
ALL
No
Sponsors
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Rare Diseases Clinical Research Network
NETWORK
National Center for Advancing Translational Sciences (NCATS)
NIH
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
University of Minnesota
OTHER
Responsible Party
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Principal Investigators
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Aaron S Kelly, Ph.D.
Role: PRINCIPAL_INVESTIGATOR
University of Minnesota
Locations
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Children's Hospital of Orange County
Orange, California, United States
University of Minnesota
Minneapolis, Minnesota, United States
Countries
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Related Links
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Rare Disease Clinical Research Network
Other Identifiers
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1202M09721
Identifier Type: -
Identifier Source: org_study_id
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