Oral Galactose in Children With Steroid Resistant Nephrotic Syndrome

Summary

Focal Segmental Glomerulosclerosis (FSGS) is a devastating kidney disease which is difficult to treat and carries a poor prognosis, with 50% of affected children progressing to end stage renal disease (ESRD). The purpose of this study is to investigate oral galactose as a benign treatment for FSGS in children. The investigators hypothesize that galactose, a simple milk sugar thought to bind to the protein factor (FSPF) that causes FSGS thereby inactivating it and stopping it from damaging the kidney, resulting in a reduction in glomerular permeability to albumin and decrease in proteinuria in children with nephrotic syndrome secondary to FSGS.

Timeline

Start

2009-10

Primary completion

2013-03

Completion

2013-03

Publications

• Background Fine RN. Recurrence of nephrotic syndrome/focal segmental glomerulosclerosis following renal transplantation in children. Pediatr Nephrol. 2007 Apr;22(4):496-502. doi: 10.1007/s00467-006-0361-6. Epub 2006 Dec 21.
• Background Srivastava T, Simon SD, Alon US. High incidence of focal segmental glomerulosclerosis in nephrotic syndrome of childhood. Pediatr Nephrol. 1999 Jan;13(1):13-8. doi: 10.1007/s004670050555.
• Background Tryggvason K, Patrakka J, Wartiovaara J. Hereditary proteinuria syndromes and mechanisms of proteinuria. N Engl J Med. 2006 Mar 30;354(13):1387-401. doi: 10.1056/NEJMra052131. No abstract available.
• Background Trachtman H, Greenbaum LA, McCarthy ET, Sharma M, Gauthier BG, Frank R, Warady B, Savin VJ. Glomerular permeability activity: prevalence and prognostic value in pediatric patients with idiopathic nephrotic syndrome. Am J Kidney Dis. 2004 Oct;44(4):604-10.
• Background Burdmann EA, Andoh TF, Yu L, Bennett WM. Cyclosporine nephrotoxicity. Semin Nephrol. 2003 Sep;23(5):465-76. doi: 10.1016/s0270-9295(03)00090-1.
• Background Savin VJ, McCarthy ET, Sharma R, Charba D, Sharma M. Galactose binds to focal segmental glomerulosclerosis permeability factor and inhibits its activity. Transl Res. 2008 Jun;151(6):288-92. doi: 10.1016/j.trsl.2008.04.001. Epub 2008 May 2.
• Background Savin VJ, Sharma R, Sharma M, McCarthy ET, Swan SK, Ellis E, Lovell H, Warady B, Gunwar S, Chonko AM, Artero M, Vincenti F. Circulating factor associated with increased glomerular permeability to albumin in recurrent focal segmental glomerulosclerosis. N Engl J Med. 1996 Apr 4;334(14):878-83. doi: 10.1056/NEJM199604043341402.
• Background Shreeve WW, Shoop JD, Ott DG, McInteer BB. Test for alcoholic cirrhosis by conversion of [14C]- or [13C]galactose to expired CO2. Gastroenterology. 1976 Jul;71(1):98-101.
• Background Frustaci A, Chimenti C, Ricci R, Natale L, Russo MA, Pieroni M, Eng CM, Desnick RJ. Improvement in cardiac function in the cardiac variant of Fabry's disease with galactose-infusion therapy. N Engl J Med. 2001 Jul 5;345(1):25-32. doi: 10.1056/NEJM200107053450104. No abstract available.
• Background Genkinger JM, Hunter DJ, Spiegelman D, Anderson KE, Beeson WL, Buring JE, Colditz GA, Fraser GE, Freudenheim JL, Goldbohm RA, Hankinson SE, Koenig KL, Larsson SC, Leitzmann M, McCullough ML, Miller AB, Rodriguez C, Rohan TE, Ross JA, Schatzkin A, Schouten LJ, Smit E, Willett WC, Wolk A, Zeleniuch-Jacquotte A, Zhang SM, Smith-Warner SA. A pooled analysis of 12 cohort studies of dietary fat, cholesterol and egg intake and ovarian cancer. Cancer Causes Control. 2006 Apr;17(3):273-85. doi: 10.1007/s10552-005-0455-7.
• Background Qin LQ, Xu JY, Wang PY, Hashi A, Hoshi K, Sato A. Milk/dairy products consumption, galactose metabolism and ovarian cancer: meta-analysis of epidemiological studies. Eur J Cancer Prev. 2005 Feb;14(1):13-9. doi: 10.1097/00008469-200502000-00003.
• Background Schwartz GJ, Haycock GB, Edelmann CM Jr, Spitzer A. A simple estimate of glomerular filtration rate in children derived from body length and plasma creatinine. Pediatrics. 1976 Aug;58(2):259-63.
• Background De Smet E, Rioux JP, Ammann H, Deziel C, Querin S. FSGS permeability factor-associated nephrotic syndrome: remission after oral galactose therapy. Nephrol Dial Transplant. 2009 Sep;24(9):2938-40. doi: 10.1093/ndt/gfp278. Epub 2009 Jun 9.

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