Cardiac Structure and Function in MPS
Recruiting
Observational Study
MPS I
MPS IV A
MPS - Mucopolysaccharidosis
MPS IVA
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Healthy Volunteers Welcome
You do not need to have the condition being studied to take part.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 0 – 99
- Sex
- Any
- Study type
- Observational
- Participants needed
- 240 (estimated)
- Sponsor
- Children's Hospital of Orange County · Other
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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.
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Trial Locations
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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 parti…
Contacts