Assessing cardiovascular health in patients with mucopolysaccharidoses
Cardiovascular Structure and Function in the Mucopolysaccharidoses
This study looks at the heart and blood vessel health of kids with mucopolysaccharidoses to see how their condition affects them over time.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 30 (estimated) |
| Sex | All |
| Sponsor | Children's Hospital of Orange County Academic / other |
| Locations | 1 site (Orange, California) |
| Trial ID | NCT05063435 on ClinicalTrials.gov |
What this trial studies
This observational study aims to evaluate cardiac and carotid structure and function in individuals diagnosed with mucopolysaccharidoses (MPS) using non-invasive carotid ultrasonography. The study will annually measure carotid intima media thickness, stiffness, and various biomarkers associated with atherosclerosis and inflammation. A total of 30 subjects will be enrolled at CHOC Children's Hospital, where eligible patients will be identified from the existing patient population and future referrals. Informed consent will be obtained, and participants will undergo assessments including echocardiography and blood tests.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals with a molecularly confirmed diagnosis of mucopolysaccharidosis.
Not a fit: Patients who are unable to participate in the required assessments or have conditions that preclude their involvement may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could enhance understanding of cardiovascular risks in patients with mucopolysaccharidoses, leading to improved monitoring and management strategies.
How similar studies have performed: Previous studies have shown potential utility in similar approaches, but this specific longitudinal assessment in MPS patients is novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Any patient with a molecularly confirmed diagnosis of mucopolysaccharidosis is eligible to enroll in this study 2. Parental / patient informed consent Exclusion Criteria: 1. Any reason that the investigators would deem a patient unable to participate in this study 2. Inability to participate in the assessments required for this study
Where this trial is running
Orange, California
- Children's Hospital of Orange County — Orange, California, United States (Recruiting)
Study contacts
- Principal investigator: Raymond Wang, M.D. — CHOC Children's Hospital
- Study coordinator: Nina Movsesyan, Ph.D.
- Email: nmovsesyan@choc.org
- Phone: (714) 509-3008
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.