Cardiac Structure and Function in MPS

Recruiting now

Conditions studied: MPS I, MPS IV A, MPS - Mucopolysaccharidosis, MPS IVA

In brief

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.

Key facts

Study ID

NCT07361536

Run by

Children's Hospital of Orange County

People needed

240

Starts

2025-09-01

Expected to finish

2030-04-30

Last updated by the study team

2026-01-26

Who can join

Age: any, up to 99. Sex: any. Healthy volunteers: accepted.

You may qualify if…

• Any Participant with a molecularly confirmed diagnosis of mucopolysaccharidosis is eligible to enroll in this study
• Any healthy participant without a diagnosis of mucopolysaccharidosis whose age and biological sex can be matched with an enrolled mucopolysaccharidosis participant
• Parental / patient informed consent

You may not qualify if…

• Any reason that the investigators would deem a patient not eligible to participate in this study
• Inability to participate in the assessments required for this study

Where it is running

• Children'S Hospital of Orange County — Orange, California, United States (enrolling)
• University of Minnesota — Minneapolis, Minnesota, United States
• Saint Louis University — St Louis, Missouri, United States

Full record on ClinicalTrials.gov

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