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Cardiac Structure and Function in MPS

Children's Hospital of Orange County · with National Heart, Lung, and Blood Institute (NHLBI) · NCT07361536

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 0 mo–99 · All sexes
Healthy volunteers
Accepted
Study type
Observational
Target enrollment
240
US sites recruiting
2
Study start
September 1, 2025
Record last updated
October 21, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study 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:

  • 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?
  • 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: MPS I; MPS IV A; MPS - Mucopolysaccharidosis; MPS IVA

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full criteria

Inclusion Criteria

  • 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

Exclusion Criteria

  • 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

Recruiting US locations

  • Children'S Hospital of Orange County

    Orange, CA 92868

    Site contact: Nina Movsesyan · 714-509-3008 · Metabolic.CRC@choc.org

  • Saint Louis University

    St Louis, MO 63104

    Site contact: Adriana M Montano, PhD · 314-977-9104 · adriana.montano@health.slu.edu

Study contacts (from the official record)

  • Nina Movsesyan · 714-509-3008 · Metabolic.CRC@choc.org
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Cardiac Structure and Function in MPS (NCT07361536) | Bored Studies