Actively Recruiting
Cardiac Structure and Function in MPS
Led by Children's Hospital of Orange County · Updated on 2026-01-26
240
Participants Needed
3
Research Sites
243 weeks
Total Duration
On this page
Sponsors
C
Children's Hospital of Orange County
Lead Sponsor
N
National Heart, Lung, and Blood Institute (NHLBI)
Collaborating Sponsor
AI-Summary
What this Trial Is About
The purpose of this study is to better understand how heart and blood vessel problems develop in people with Mucopolysaccharidosis (MPS). The investigators are looking at certain substances in the body called GAGs and proteoglycans to see how they affect the heart. The investigators also want to find reliable blood and urine markers that can help us track heart health and guide future treatments. This study aims to answer two main questions: 1. Do people with MPS show faster changes in their blood vessels over time (such as thickening or stiffening of the carotid artery) compared to people without MPS? 2. Do people with MPS have higher levels of certain proteins in their blood (such as clusterin and inflammatory markers) that are linked to blood vessel changes? What participants will do? Participants will complete the following tests once a year for 4 years: * Carotid ultrasound: an imaging test that looks at the blood vessels in the neck. * Echocardiogram: an ultrasound of the heart. * Blood draw * Urine collection These tests help the investigators track changes in heart and blood vessel health over time.
CONDITIONS
Official Title
Cardiac Structure and Function in MPS
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants with a confirmed molecular diagnosis of mucopolysaccharidosis
- Healthy participants without mucopolysaccharidosis whose age and biological sex match an enrolled MPS participant
- Parental or patient informed consent provided
You will not qualify if you...
- Any condition or reason deemed by investigators that makes a patient ineligible
- Inability to participate in required study assessments
AI-Screening
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Trial Site Locations
Total: 3 locations
1
Children'S Hospital of Orange County
Orange, California, United States, 92868
Actively Recruiting
2
University of Minnesota
Minneapolis, Minnesota, United States, 55455
Not Yet Recruiting
3
Saint Louis University
St Louis, Missouri, United States, 63104
Not Yet Recruiting
Research Team
N
Nina Movsesyan
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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