Cardiovascular Assessment for Mucopolysaccharidosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to better understand heart and blood vessel health in individuals with mucopolysaccharidoses (MPS), a group of rare genetic disorders affecting various body parts. Researchers will regularly check specific blood markers and use non-invasive scans to measure the flexibility and thickness of the carotid artery walls, a major neck blood vessel. These checks, part of the Cardiovascular Structure and Function Assessment, will occur annually for three years to observe any changes over time. Individuals with a confirmed MPS diagnosis who can participate in the study assessments may be eligible to join. As an unphased trial, this study offers a unique opportunity to enhance the understanding of cardiovascular health in MPS patients.
Do I need to stop my current medications to join the trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Why are researchers excited about this trial?
Researchers are excited about the trial focusing on cardiovascular structure and function in people with mucopolysaccharidoses (MPS) because it aims to understand how these conditions impact the heart and blood vessels in ways not yet fully explored. Unlike existing treatments that primarily address the enzyme deficiencies in MPS, this assessment focuses on the cardiovascular system, potentially uncovering new insights into how these disorders affect heart health. This could pave the way for more targeted therapies that not only manage symptoms but also improve overall cardiovascular outcomes for patients with MPS.
Who Is on the Research Team?
Raymond Wang, M.D.
Principal Investigator
CHOC Children's Hospital
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Initial Entry
Collection of demographic data, medical history, and initial measurements including carotid ultrasonography and echocardiography
Annual Assessment
Annual assessment of cardiac and carotid structure and function, and measurement of circulating biomarkers
Follow-up
Participants are monitored for safety and effectiveness after the main assessments
What Are the Treatments Tested in This Trial?
Interventions
- Cardiovascular Structure and Function Assessment
Find a Clinic Near You
Who Is Running the Clinical Trial?
Children's Hospital of Orange County
Lead Sponsor
Ultragenyx Pharmaceutical Inc
Industry Sponsor
Dr. Emil D. Kakkis
Ultragenyx Pharmaceutical Inc
Chief Executive Officer since 2010
MD/PhD in Biological Chemistry from UCLA
Dr. Eric Crombez
Ultragenyx Pharmaceutical Inc
Chief Medical Officer since 2023
MD from Wayne State University School of Medicine
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