NCT02994303|UnknownDMC
Podocyturia - Predictor of Renal Dysfunction in Fabry Nephropathy
Podocyturia, a Non-Invasive Predictor of Renal Dysfunction in Fabry Nephropathy
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Podocyturia, a Non-Invasive Predictor of Renal Dysfunction in Fabry Nephropathy
Sponsor study identifier
Secondary identifiers
NIH
observational
58
1 country
3
Study Start
September 2014
Date type not reportedThis date has not been verified as actual or estimated.
Primary Completion
August 2019
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Study Completion
August 2019
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
In patients with Fabry disease, this research study explores the presence of podocytes in their urine as a potential non-invasive biomarker for baseline kidney disease; and explores changes in the quantity of podocytes in their urine over time as a predictor for kidney disease progression. To accomplish this, the investigators will evaluate the quantification of podocytes in the urine of Fabry disease patients at baseline and longitudinally over time. This study requires a single patient visit, during which the patient provides a urine specimen. The research team will then collect the patient's kidney function data proximate to the time of urine collection, and follow the patient's kidney function data longitudinally over the five years of this study by reviewing their medical charts. The study offers no interventions.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
August 2019
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
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Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
September 2014
Date type not reportedThis date has not been verified as actual or estimated.
December 5, 2016
Actual (reported)December 15, 2016
Actual (reported)August 2019
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
August 2019
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Detection of Podocyturia in Subjects Diagnosed with Fabry Disease
In subjects who have been diagnosed with Fabry disease, their urine sample will be examined for the presence of podocyturia at time of enrollment. If podocyturia is present, it will be quantified and recorded.
At time of enrollment
Change from Baseline in Number of Urine Podocytes at Year 1, 2, 3, 4 and 5
Annually at Year 1, Year 2, Year 3, Year 4 and Year 5
Change from Baseline in urine protein/creatinine ratio (PCR) at Year 1, 2, 3, 4 and 5
At time of enrollment, then annually at Year 1, Year 2, Year 3, Year 4 and Year 5
Change from Baseline in urine albumin/creatinine ratio (ACR) at Year 1, 2, 3, 4 and 5
At time of enrollment, then annually at Year 1, Year 2, Year 3, Year 4 and Year 5
Change from Baseline in estimated glomerular filtration rate (eGFR) at Year 1, 2, 3, 4 and 5
At time of enrollment, then annually at Year 1, Year 2, Year 3, Year 4 and Year 5
Change from Baseline in Number of Urine Podocytes at Year 1, 2, 3, 4 or 5, Based On Subject's New Urine Sample
Annually at Year 1, Year 2, Year 3, Year 4 or Year 5
Patients of any gender who have been diagnosed with Fabry disease
Emory University
Decatur, Georgia, 30033, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Najafian B, Tondel C, Svarstad E, Sokolovkiy A, Smith K, Mauer M. One Year of Enzyme Replacement Therapy Reduces Globotriaosylceramide Inclusions in Podocytes in Male Adult Patients with Fabry Disease. PLoS One. 2016 Apr 15;11(4):e0152812. doi: 10.1371/journal.pone.0152812. eCollection 2016.
Najafian B, Fogo AB, Lusco MA, Alpers CE. AJKD Atlas of Renal Pathology: Fabry nephropathy. Am J Kidney Dis. 2015 Nov;66(5):e35-6. doi: 10.1053/j.ajkd.2015.08.006. No abstract available.
Urine Sample
Behzad Najafian, M.D.
University of Washington Associate Professor, Pathology
First Submitted
December 5, 2016
First Posted
December 15, 2016
Study Start
September 2014
Date type not reportedThis date has not been verified as actual or estimated.
Primary Completion
August 2019
Treatment Protocol of Replagal for Patients With Fabry Disease
Contact the study team to confirm eligibility.
University of Washington
Seattle, Washington, 98195, United States
Wijburg FA, Benichou B, Bichet DG, Clarke LA, Dostalova G, Fainboim A, Fellgiebel A, Forcelini C, An Haack K, Hopkin RJ, Mauer M, Najafian B, Scott CR, Shankar SP, Thurberg BL, Tondel C, Tylki-Szymanska A, Ramaswami U. Characterization of early disease status in treatment-naive male paediatric patients with Fabry disease enrolled in a randomized clinical trial. PLoS One. 2015 May 8;10(5):e0124987. doi: 10.1371/journal.pone.0124987. eCollection 2015.
Mauer M, Glynn E, Svarstad E, Tondel C, Gubler MC, West M, Sokolovskiy A, Whitley C, Najafian B. Mosaicism of podocyte involvement is related to podocyte injury in females with Fabry disease. PLoS One. 2014 Nov 11;9(11):e112188. doi: 10.1371/journal.pone.0112188. eCollection 2014.
Najafian B, Mauer M, Hopkin RJ, Svarstad E. Renal complications of Fabry disease in children. Pediatr Nephrol. 2013 May;28(5):679-87. doi: 10.1007/s00467-012-2222-9. Epub 2012 Aug 17.
Najafian B, Mauer M. Quantitating glomerular endothelial fenestration: an unbiased stereological approach. Am J Nephrol. 2011;33 Suppl 1(Suppl 1):34-9. doi: 10.1159/000327075. Epub 2011 Jun 10.
Najafian B, Svarstad E, Bostad L, Gubler MC, Tondel C, Whitley C, Mauer M. Progressive podocyte injury and globotriaosylceramide (GL-3) accumulation in young patients with Fabry disease. Kidney Int. 2011 Mar;79(6):663-670. doi: 10.1038/ki.2010.484. Epub 2010 Dec 15.
Ramaswami U, Najafian B, Schieppati A, Mauer M, Bichet DG. Assessment of renal pathology and dysfunction in children with Fabry disease. Clin J Am Soc Nephrol. 2010 Feb;5(2):365-70. doi: 10.2215/CJN.08091109. Epub 2010 Jan 7.
Mauer M, Najafian B. Implications of early renal changes in Fabry disease. Clin Ther. 2008;30 Suppl B:S40. doi: 10.1016/s0149-2918(08)80034-3. No abstract available.
Hopkin RJ, Cabrera G, Charrow J, Lemay R, Martins AM, Mauer M, Ortiz A, Patel MR, Sims K, Waldek S, Warnock DG, Wilcox WR. Risk factors for severe clinical events in male and female patients with Fabry disease treated with agalsidase beta enzyme replacement therapy: Data from the Fabry Registry. Mol Genet Metab. 2016 Sep;119(1-2):151-9. doi: 10.1016/j.ymgme.2016.06.007. Epub 2016 Jun 13.
Hopkin RJ, Jefferies JL, Laney DA, Lawson VH, Mauer M, Taylor MR, Wilcox WR; Fabry Pediatric Expert Panel. The management and treatment of children with Fabry disease: A United States-based perspective. Mol Genet Metab. 2016 Feb;117(2):104-13. doi: 10.1016/j.ymgme.2015.10.007. Epub 2015 Oct 23.
Warnock DG, Mauer M. Fabry disease: dose matters. J Am Soc Nephrol. 2014 Apr;25(4):653-5. doi: 10.1681/ASN.2013121322. Epub 2014 Feb 20. No abstract available.
Fall B, Scott CR, Mauer M, Shankland S, Pippin J, Jefferson JA, Wallace E, Warnock D, Najafian B. Urinary Podocyte Loss Is Increased in Patients with Fabry Disease and Correlates with Clinical Severity of Fabry Nephropathy. PLoS One. 2016 Dec 16;11(12):e0168346. doi: 10.1371/journal.pone.0168346. eCollection 2016.
This estimate has passed. The registry has not confirmed an actual date for this milestone.
Study Completion
August 2019
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Last Updated
August 28, 2017
Record last verified: 2017-08
De-identified individual data is input to the NIH-funded Rare Diseases Clinical Research Network's Data Management \& Coordinating Center ("DMCC"). Eventually this data will become part of the database of Genotypes and Phenotypes ("dbGaP"), which is part of the National Center for Biotechnology Information, U.S. National Library of Medicine.
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Source: ClinicalTrials.gov.
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