An observational study in MPS I, MPS IV A and MPS - Mucopolysaccharidosis, sponsored by Children's Hospital of Orange County. Recruiting at 3 sites in United States. Open to participants aged 0 Years to 99 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2026-10-21.
Sponsored by Children's Hospital of Orange County · Observational
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:
What participants will do?
Participants will complete the following tests once a year for 4 years:
These tests help the investigators track changes in heart and blood vessel health over time.
101 studies on the registry are indexed under Mucopolysaccharidosis IV; 27 are open to participants now.
This study's planned enrollment of 240 is above the median of 100 across 41 observational studies indexed under Mucopolysaccharidosis IV.
Browse Mucopolysaccharidosis IV studies →Children's Hospital of Orange County is the lead sponsor of 18 studies on the registry; 10 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Participants will be drawn from each participating site's existing patient population. Eligible individuals who meet protocol-defined criteria and are willing to take part in annual study visits will be invited to enroll. The study population will therefore consist of patients receiving care at the participating institutions who consent to ongoing observational follow-up.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants diagnosed with MPS I or MPS IVA
Participants who do not have an MPS diagnosis
Cardiac and Carotid Structure and Function
1\) We aim to annually assess cardiac and carotid structure and function utilizing ultrasonography, in participants with mucopolysaccharidoses and matched control participants
Time frame: Year 1, Year 2, Year 3, and Year 4
Measure biomarkers
2\) We aim to annually measure circulating levels of clusterin, elastin, cathepsin S, and cytokines in participants with mucopolysaccharidoses and matched control participants
Time frame: Year 1, Year 2, Year 3, and Year 4
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Children's Hospital of Orange County