Observational Study of Endothelial Dysfunction in Phenylketonuria
NCT ID: NCT02176603
Last Updated: 2017-02-23
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
51 participants
OBSERVATIONAL
2014-05-31
2017-01-01
Brief Summary
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Detailed Description
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Endothelial dysfunction will be measured by venous occlusion plethysmography on the lower legs in the PKU patients and in the control group. Wave velocity, aortic stiffness, and intima media thickness of the common carotid artery will be measured by vicorder and duplex ultrasound scanner in the PKU patients and in the control group. Analyses of phenylalanine metabolites and renal function will be performed only in the PKU patients, analyses of oxidative stress markers in the PKU patients and in the control group. Dietary protocols of the patients with PKU will be calculated for their daily phenylalanine, total protein, and calorie intake. Results of the study will be kept in the medical records and paper case report forms. Data will be analyzed only in pseudonymized form.
Conditions
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Study Design
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CASE_CONTROL
CROSS_SECTIONAL
Study Groups
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Patients with Phenylketonuria
Patients with Phenylketonuria due to phenylalanine hydroxylase deficiency
No interventions assigned to this group
Control group
Control group of healthy volunteers
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
* age ≥16 years
Exclusion Criteria
* pregnancy or maternity
* late-treated phenylketonuria
* phenylketonuria due a defect in BH4 synthesis or BH4 regeneration
16 Years
ALL
Yes
Sponsors
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Johannes Gutenberg University Mainz
OTHER
Charite University, Berlin, Germany
OTHER
Responsible Party
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Uwe Querfeld
Prof. Dr.
Principal Investigators
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Julia B Hennermann, MD
Role: PRINCIPAL_INVESTIGATOR
Johannes Gutenberg University Mainz, Germany
Uwe Querfeld, MD
Role: PRINCIPAL_INVESTIGATOR
Charite University, Berlin, Germany
References
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Briese S, Claus M, Querfeld U. Arterial stiffness in children after renal transplantation. Pediatr Nephrol. 2008 Dec;23(12):2241-5. doi: 10.1007/s00467-008-0894-y. Epub 2008 Jun 27.
Ribas GS, Sitta A, Wajner M, Vargas CR. Oxidative stress in phenylketonuria: what is the evidence? Cell Mol Neurobiol. 2011 Jul;31(5):653-62. doi: 10.1007/s10571-011-9693-2. Epub 2011 Apr 23.
Kracht D, Shroff R, Baig S, Doyon A, Jacobi C, Zeller R, Querfeld U, Schaefer F, Wuhl E, Schmidt BM, Melk A; 4C Study Consortium. Validating a new oscillometric device for aortic pulse wave velocity measurements in children and adolescents. Am J Hypertens. 2011 Dec;24(12):1294-9. doi: 10.1038/ajh.2011.147. Epub 2011 Aug 25.
Hennermann JB, Roloff S, Gellermann J, Vollmer I, Windt E, Vetter B, Plockinger U, Monch E, Querfeld U. Chronic kidney disease in adolescent and adult patients with phenylketonuria. J Inherit Metab Dis. 2013 Sep;36(5):747-56. doi: 10.1007/s10545-012-9548-0. Epub 2012 Nov 9.
Hennermann JB, Querfeld U. Unknown pathomechanisms of renal impairment in PKU. J Inherit Metab Dis. 2013 Nov;36(6):1087-8. doi: 10.1007/s10545-013-9652-9. Epub 2013 Sep 17. No abstract available.
Other Identifiers
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PKU-EnDys
Identifier Type: -
Identifier Source: org_study_id
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