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
This trial accepts healthy volunteersQualifiers
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 population
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.
Trial design
Design model
Case-control
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
240 Participants
are grouped into 2 trial groupsGroup A: MPS
Group B: Control
Trial outcomes
Primary outcomes
1
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 4Secondary outcomes
1
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 4Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Children's Hospital of Orange County
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Collaborator
Source ClinicalTrials.gov