| NCT ID | NCT07361536 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Children's Hospital of Orange County |
| Condition | MPS I |
| Study Type | OBSERVATIONAL |
| Enrollment | 240 participants |
| Start Date | 2025-09-01 |
| Primary Completion | 2030-04-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 240 participants in total. It began in 2025-09-01 with a primary completion date of 2030-04-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Inclusion Criteria: 1. Any Participant with a molecularly confirmed diagnosis of mucopolysaccharidosis is eligible to enroll in this study 2. Any healthy participant without a diagnosis of mucopolysaccharidosis whose age and biological sex can be matched with an enrolled mucopolysaccharidosis participant 3. Parental / patient informed consent Exclusion Criteria: 1. Any reason that the investigators would deem a patient not eligible to participate in this study 2. Inability to participate in the assessments required for this study
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07361536 clinical trial?
This trial is open to participants of all sexes, aged 0 Years or older, up to 99 Years, studying MPS I. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT07361536 currently recruiting?
Yes, NCT07361536 is actively recruiting participants. Contact the research team at Metabolic.CRC@choc.org for enrollment information.
Where is the NCT07361536 trial being conducted?
This trial is being conducted at Orange, United States, Minneapolis, United States, St Louis, United States.
Who is sponsoring the NCT07361536 clinical trial?
NCT07361536 is sponsored by Children's Hospital of Orange County. The trial plans to enroll 240 participants.