Cardiac Structure and Function in MPS
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age0-99
SponsorChildren's Hospital of Orange County
About this trial
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
Qualifiers
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
Disqualifiers
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
Trial design
Treatments tested in this trial
- Not listed
Trial groups
240 Participants
are grouped into 2 trial groupsSponsors and collaborators
Children's Hospital of Orange County
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Collaborator
Source ClinicalTrials.gov