Cardiac Structure and Function in MPS
Part of paid clinical trials in Orange, California.
- Sponsor
- Children's Hospital of Orange County
- Study ID
- NCT07361536
- Status
- Recruiting
Conditions
- MPS - Mucopolysaccharidosis
- MPS I
- MPS IV A
- MPS IVA
Eligibility Criteria
- Sex
- ALL
- Age
- 0 Years - 99 Years
- Healthy Volunteers
- Accepted
Study Details
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 Dates
- Start date
- Sep 1, 2025
- Status verified
- Jan 2026
- Primary completion
- Apr 30, 2030
- Completion
- Apr 30, 2030
Study Design
- Enrollment
- 240 participants (estimated)
Arms
- Arm: MPSParticipants diagnosed with MPS I or MPS IVA
- Arm: ControlParticipants who do not have an MPS diagnosis
Primary Outcome Measure
Cardiac and Carotid Structure and Function [ Time Frame: Year 1, Year 2, Year 3, and Year 4 ]
Central Contacts
- Nina Movsesyan714-509-3008
Locations (3)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| Children'S Hospital of Orange County | Orange | California | 92868 | |
| University of Minnesota | Minneapolis | Minnesota | 55455 | |
| Saint Louis University | St Louis | Missouri | 63104 |
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