ACTIVE_NOT_RECRUITING

Cardiovascular Structure and Function in the Mucopolysaccharidoses

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This study's investigators previously demonstrated the potential utility of non-invasive carotid ultrasonography to calculate carotid intima media thickness (cIMT) and stiffness (as measured by the three parameters, carotid cross-sectional distensibility \[cCSD\], carotid cross-sectional compliance \[cCSC\], and carotid incremental elastic modulus \[cIEM\]) in people with mucopolysaccharidoses (MPS). Investigators also studied arterial gene expression in animal models of MPS, and identified upregulation of a number of markers potentially tied to atherosclerosis and inflammation. These include the atherosclerotic marker known as Clusterin (CLU), Cathepsin S, Elastin, and the inflammatory cytokines interleukin 1-α, interleukin 1-β, interleukin 2, and interleukin 6. Other studies have identified elevation in circulating tumor necrosis factor-α correlating with pain and physical disability in certain mucopolysaccharidoses. Since these studies are cross sectional, and not longitudinal, this study aims to annually measure these previously studied biomarkers (carotid measurements, circulating cytokines, cathepsin S, elastin, and CLU) in a large cohort of MPS patients. This study is a 3-year, prospective, anonymized, longitudinal assessment of cardiovascular structure, function, and circulating biomarkers in patients with mucopolysaccharidoses.

Official Title

Cardiovascular Structure and Function in the Mucopolysaccharidoses

Quick Facts

Study Start:2021-10-13
Study Completion:2026-06-30
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT05063435

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Any patient with a molecularly confirmed diagnosis of mucopolysaccharidosis is eligible to enroll in this study
  2. 2. Parental / patient informed consent
  1. 1. Any reason that the investigators would deem a patient unable to participate in this study
  2. 2. Inability to participate in the assessments required for this study

Contacts and Locations

Principal Investigator

Raymond Wang, M.D.
PRINCIPAL_INVESTIGATOR
CHOC Children's Hospital

Study Locations (Sites)

Children's Hospital of Orange County
Orange, California, 92868
United States

Collaborators and Investigators

Sponsor: Children's Hospital of Orange County

  • Raymond Wang, M.D., PRINCIPAL_INVESTIGATOR, CHOC Children's Hospital

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-10-13
Study Completion Date2026-06-30

Study Record Updates

Study Start Date2021-10-13
Study Completion Date2026-06-30

Terms related to this study

Keywords Provided by Researchers

  • mucopolysaccharidosis
  • carotid
  • cardiac
  • cardiovascular
  • biomarker
  • longitudinal
  • observational

Additional Relevant MeSH Terms

  • Mucopolysaccharidoses
  • Carotid Disease
  • Cardiac Disease
  • Inflammation