Inflammation Clinical Trial
Official title:
Cardiovascular Structure and Function in the Mucopolysaccharidoses
NCT number | NCT05063435 |
Other study ID # | 200107 |
Secondary ID | |
Status | Recruiting |
Phase | |
First received | |
Last updated | |
Start date | October 13, 2021 |
Est. completion date | March 31, 2025 |
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.
Status | Recruiting |
Enrollment | 30 |
Est. completion date | March 31, 2025 |
Est. primary completion date | October 31, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
United States | Children's Hospital of Orange County | Orange | California |
Lead Sponsor | Collaborator |
---|---|
Children's Hospital of Orange County | Ultragenyx Pharmaceutical Inc |
United States,
Wang RY, Braunlin EA, Rudser KD, Dengel DR, Metzig AM, Covault KK, Polgreen LE, Shapiro E, Steinberger J, Kelly AS. Carotid intima-media thickness is increased in patients with treated mucopolysaccharidosis types I and II, and correlates with arterial stiffness. Mol Genet Metab. 2014 Feb;111(2):128-32. doi: 10.1016/j.ymgme.2013.11.001. Epub 2013 Nov 12. — View Citation
Wang RY, Covault KK, Halcrow EM, Gardner AJ, Cao X, Newcomb RL, Dauben RD, Chang AC. Carotid intima-media thickness is increased in patients with mucopolysaccharidoses. Mol Genet Metab. 2011 Dec;104(4):592-6. doi: 10.1016/j.ymgme.2011.09.004. Epub 2011 Sep 10. — View Citation
Wang RY, Rudser KD, Dengel DR, Braunlin EA, Steinberger J, Jacobs DR, Sinaiko AR, Kelly AS. The Carotid Intima-Media Thickness and Arterial Stiffness of Pediatric Mucopolysaccharidosis Patients Are Increased Compared to Both Pediatric and Adult Controls. Int J Mol Sci. 2017 Mar 15;18(3):637. doi: 10.3390/ijms18030637. — View Citation
Wang RY, Rudser KD, Dengel DR, Evanoff N, Steinberger J, Movsesyan N, Garrett R, Christensen K, Boylan D, Braddock SR, Shinawi M, Gan Q, Montano AM. Abnormally increased carotid intima media-thickness and elasticity in patients with Morquio A disease. Orphanet J Rare Dis. 2020 Mar 17;15(1):73. doi: 10.1186/s13023-020-1331-y. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Cardiovascular Event | A cardiovascular event is defined by new onset of clinically significant aortic or mitral valve disease, aortic root dilatation, cardiomyopathy, reduction in cardiac contractile function, myocardial ischemia, myocardial infarction, or cerebrovascular accident | 3 years | |
Secondary | Age | Change of age of subject (unit: years) | 3 years | |
Secondary | Height | Change of height of subject (unit: meters) | 3 years | |
Secondary | Weight | Change of weight of subject (unit: kilograms) | 3 years | |
Secondary | Blood pressure | Change in systolic and diastolic blood pressure over the duration of the study (unit: millimeters of mercury) | 3 years | |
Secondary | Carotid structure | Change in carotid intima media thickness over the duration of the study (mm). | 3 years | |
Secondary | Carotid stiffness | Change in carotid cross-sectional distensibility over the duration of the study (unit: %). This metric is acquired in the carotid ultrasound, and measures the stiffness of the carotid artery via quantitation of the difference between end-systolic and end-diastolic carotid diameter. | 3 years | |
Secondary | Cardiac structure (left ventricle) | Change in left ventricular mass index over the duration of the study (unit: grams / m2 body surface area). This metric is acquired in the echocardiogram, and quantitates the amount of myocardium in the left ventricle. | 3 years | |
Secondary | Cardiac structure (aortic root diameter) | Change in aortic root measurement over the duration of the study (unit: mm). This metric is acquired in the echocardiogram, and quantitates the width of the aortic root. | 3 years | |
Secondary | Mitral valve function | Assessment of changes in mitral valve function over the duration of the study (unit: 4 point Likert scale from 0 [none] to 4 [severe]). This metric is acquired during the echocardiogram, and assess the degree of mitral valve insufficiency. | 3 years | |
Secondary | Aortic valve function | Assessment of changes in aortic valve function over the duration of the study (unit: 4 point Likert scale from 0 [none] to 4 [severe]). This metric is acquired during the echocardiogram, and assess the degree of aortic valve insufficiency. | 3 years | |
Secondary | Inflammatory biomarkers (Tumor Necrosis Factor - alpha) | Change in plasma TNFa over the duration of the study. | 3 years | |
Secondary | Inflammatory biomarkers (Cathepsin S) | Change in plasma Cathepsin S over the duration of the study. (Unit: mcg/L) | 3 years | |
Secondary | Inflammatory biomarkers (Elastin) | Change in plasma Elastin levels over the duration of the study. (Unit: ng/mL) | 3 years | |
Secondary | Inflammatory biomarkers (Clusterin) | Change in plasma clusterin levels over the duration of the study. (Unit: mcg/mL) | 3 years | |
Secondary | Inflammatory biomarkers (lipidomics) | Change in plasma lipid levels over the duration of the study. (Unit: mcmol/L) | 3 years |
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