N/A Completed N=1,147

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

Mucopolysaccharidoses

Source: ClinicalTrials.gov NCT01586871 ↗

Enrolled (actual)

1,147

Serious AEs

0.0%

Results posted

Feb 2021

Primary outcomePrimary: Carotid Intima-media Thickness, Measured in Millimeters — 0.44; 0.52; 0.56 mm

Summary

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.

Outcome Measures

Outcome	Result	p-value
PRIMARY Carotid Intima-media Thickness, Measured in Millimeters	0.44; 0.52; 0.56	—
SECONDARY Carotid Cross-sectional Distensibility	32.03; 16.33; 28.36	—
SECONDARY Carotid Cross-sectional Compliance	0.16; 0.11; 0.14	—
SECONDARY Carotid Incremental Elastic Modulus	951; 1859; 1341	—

Eligibility Criteria

Inclusion Criteria

Be between the ages of 3 and 18 years old
Be diagnosed with MPS I, MPS II or MPS VI

Exclusion Criteria

None

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01586871). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.