ClinicalTrials.gov record NCT07361536

Cardiac Structure and Function in MPS

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

Cardiovascular Structure and Function in Mucopolysaccharidosis Types I and IVA

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
Healthy volunteersAcceptedPeople who do not have the condition being studied who are allowed to take part. Some studies include both people with the condition and healthy volunteers; others may enrol only healthy volunteers.
AgeUnder 1 month old to 99 years old
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This observational study follows people with mucopolysaccharidosis (MPS) — including types I and IVA — and matched healthy control participants. Participants have yearly carotid ultrasound, echocardiogram, blood draws, and urine collection for four years. The study tracks heart and blood vessel structure/function and measures blood and urine proteins over time.

What it is studying. Whether people with MPS show faster changes in carotid artery or heart structure/function over time than matched controls, whether they have higher levels of certain circulating proteins (for example clusterin and inflammatory markers), and which blood or urine markers could track cardiovascular changes.

What the researchers hope to learn. To understand how cardiac and blood vessel problems develop in MPS, to study how GAGs and proteoglycans relate to cardiovascular changes, and to identify reliable blood or urine biomarkers (including clusterin, elastin, cathepsin S, and cytokines) to track heart and vessel health.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants will come for one study visit each year for four years. At each visit they will have a carotid ultrasound (neck blood-vessel imaging), an echocardiogram (heart ultrasound), a blood draw, and a urine collection. These tests and samples are used to track heart and blood vessel structure and to measure blood and urine biomarkers. People who cannot complete these assessments would not be eligible to enroll.

How the study is structured. A prospective, observational case-control study. People with MPS I or MPS IVA and matched healthy participants are followed yearly for four years with carotid ultrasound, echocardiogram, blood draws and urine collection. No treatments are given as part of the study.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Hurler syndrome

Clinical trials10 trials listed for Hurler syndromeEvery trial the registry lists for Hurler syndrome, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Hurler syndrome researchLatest monthly update: September 2026. Includes: Blood smear clue. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Sep 1, 2025
Primary completion
Apr 30, 2030 (estimated)
Study completion
Apr 30, 2030 (estimated)
Target enrollment
240 participants (estimated)
Last updated on registry
Jan 26, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Children's Hospital of Orange County
Collaborators
  • National Heart, Lung, and Blood Institute (NHLBI)

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (3)

Terms used on this page · explanations by MedicalUpdates.ai

Echocardiogram
An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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