Actively Recruiting
Cardiovascular Structure and Function in Mucopolysaccharidosis Types I and IVA
Led by Children's Hospital of Orange County · Updated on 2026-01-26
240
Participants Needed
3
Research Sites
N/A
Total Duration
On this page
Sponsors
C
Children's Hospital of Orange County
Lead Sponsor
N
National Heart, Lung, and Blood Institute (NHLBI)
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying how heart and blood vessel problems develop in people with Mucopolysaccharidosis (MPS), a rare condition affecting the body. They want to understand if people with MPS experience faster changes in their blood vessels and if certain proteins in their blood are linked to these changes. The study also aims to find reliable blood and urine markers to track heart health and guide future treatments. The study includes two groups: people diagnosed with MPS types I or IVA and healthy participants without MPS who have similar age and biological sex. Participants will undergo yearly tests for four years, including carotid ultrasound to image neck blood vessels, echocardiogram to image the heart, blood draws, and urine collection. These tests help track changes in heart and blood vessel structure and function over time. Participants will visit once a year for four years to complete the tests. Researchers will measure heart and carotid artery structure and function, as well as biomarkers in blood and urine at each visit. The study helps monitor heart health changes and may inform future treatment strategies. Participation is open from birth up to 99 years old, and healthy volunteers are included for comparison.
CONDITIONS
Brief Title
Cardiac Structure and Function in MPS
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants with a confirmed molecular diagnosis of mucopolysaccharidosis
- Healthy participants without MPS matched by age and biological sex to enrolled MPS participants
- Parental or patient informed consent provided
You will not qualify if you...
- Any reason investigators deem the participant ineligible
- Inability to participate in required assessments for the study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - 4 years
Participants undergo assessments to evaluate cardiac and carotid structure and function.
Annual visits for up to 4 years
Trial Site Locations
Total: 3 locations
1
Children'S Hospital of Orange County
Orange, California, United States, 92868
Actively Recruiting
2
University of Minnesota
Minneapolis, Minnesota, United States, 55455
Not Yet Recruiting
3
Saint Louis University
St Louis, Missouri, United States, 63104
Not Yet Recruiting
Research Team
N
Nina Movsesyan
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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