Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network

Completed

Conditions studied: Mucopolysaccharidoses

In brief

Mucopolysaccharidosis (MPS) syndromes are disorders characterized by enzyme deficiencies, and they have been linked to heart health complications. However, there are currently no proven markers of heart and artery health for this population. The main purpose of this observational study is to evaluate the ease and convenience of a non-invasive measurement of artery function in MPS I, MPS II and MPS VI patients compared to healthy control subjects. An observational study is a research design meaning that there is no treatment in this study. 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.

Key facts

Study ID
NCT01586871
Run by
University of Minnesota
People needed
1147
Starts
2012-03-01
Expected to finish
2017-06-01
Last updated by the study team
2021-02-24

Who can join

Age: 3 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

You may not qualify if…

Where it is running

Full record on ClinicalTrials.gov

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