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: MPS
    Participants diagnosed with MPS I or MPS IVA
  • Arm: Control
    Participants 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

Locations (3)

FacilityCityStateZIPSite coordinators
Children'S Hospital of Orange CountyOrangeCalifornia92868
Nina Movsesyan
714-509-3008
University of MinnesotaMinneapolisMinnesota55455
Donald Dengel, PhD
612-626-9701
Saint Louis UniversitySt LouisMissouri63104
Adriana M Montano, PhD
314-977-9104

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