End Point Considerations for Clinical Trials in Enteric Hyperoxaluria.
Journal
Clinical journal of the American Society of Nephrology : CJASN
ISSN: 1555-905X
Titre abrégé: Clin J Am Soc Nephrol
Pays: United States
ID NLM: 101271570
Informations de publication
Date de publication:
01 Dec 2023
01 Dec 2023
Historique:
pubmed:
21
6
2023
medline:
21
6
2023
entrez:
21
6
2023
Statut:
ppublish
Résumé
Enteric hyperoxaluria is a medical condition characterized by elevated urinary oxalate excretion due to increased gastrointestinal oxalate absorption. Causative features include fat malabsorption and/or increased intestinal permeability to oxalate. Enteric hyperoxaluria has long been known to cause nephrolithiasis and nephrocalcinosis, and, more recently, an association with CKD and kidney failure has been shown. Currently, there are no US Food and Drug Administration-approved therapies for enteric hyperoxaluria, and it is unclear what end points should be used to evaluate the efficacy of new drugs and biologics for this condition. This study represents work of a multidisciplinary group convened by the Kidney Health Initiative to review the evidence supporting potential end points for clinical trials in enteric hyperoxaluria. A potential clinical outcome is symptomatic kidney stone events. Potential surrogate end points include ( 1 ) an irreversible loss of kidney function as a surrogate for progression to kidney failure, ( 2 ) asymptomatic kidney stone growth/new stone formation observed on imaging as a surrogate for symptomatic kidney stone events, ( 3 ) urinary oxalate and urinary calcium oxalate supersaturation as surrogates for the development of symptomatic kidney stone events, and ( 4) plasma oxalate as a surrogate for the development of the clinical manifestations of systemic oxalosis. Unfortunately, because of gaps in the data, this Kidney Health Initiative workgroup was unable to provide definitive recommendations. Work is underway to obtain robust information that can be used to inform trial design and medical product development in this space.
Identifiants
pubmed: 37342976
doi: 10.2215/CJN.0000000000000234
pii: 01277230-202312000-00021
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
1637-1644Subventions
Organisme : FDA HHS
ID : R18 FD005283
Pays : United States
Investigateurs
Dean Assimos
(D)
Juan Calle
(J)
Helena Cowley
(H)
Naomi Dahl
(N)
Andreas Grauer
(A)
Kim Hollander
(K)
Craig Langman
(C)
John Lieske
(J)
Lacy McClean
(L)
Dawn Milliner
(D)
Lama Nazzal
(L)
Marja Puurunen
(M)
Alistair Wheeler
(A)
Kyle Wood
(K)
Richard Yocum
(R)
Joumana Zeid
(J)
Informations de copyright
Copyright © 2023 by the American Society of Nephrology.
Références
Stauffer JQ. Hyperoxaluria and intestinal disease. The role of steatorrhea and dietary calcium in regulating intestinal oxalate absorption. Am J Dig Dis. 1977;22(10):921–928. doi: 10.1007/bf01076170
doi: 10.1007/bf01076170
Chadwick VS, Modha K, Dowling RH. Mechanism for hyperoxaluria in patients with ileal dysfunction. N Engl J Med. 1973;289(4):172–176. doi: 10.1056/nejm197307262890402
doi: 10.1056/nejm197307262890402
Witting C, Langman CB, Assimos D, et al. Pathophysiology and treatment of enteric hyperoxaluria. Clin J Am Soc Nephrol. 2021;16(3):487–495. doi: 10.2215/CJN.08000520
doi: 10.2215/CJN.08000520
Nazzal L, Puri S, Goldfarb DS. Enteric hyperoxaluria: an important cause of end-stage kidney disease. Nephrol Dial Transplant. 2016;31(3):375–382. doi: 10.1093/ndt/gfv005
doi: 10.1093/ndt/gfv005
Nelson WK, Houghton SG, Milliner DS, Lieske JC, Sarr MG. Enteric hyperoxaluria, nephrolithiasis, and oxalate nephropathy: potentially serious and unappreciated complications of Roux-en-Y gastric bypass. Surg Obes Relat Dis. 2005;1(5):481–485. doi: 10.1016/j.soard.2005.07.002
doi: 10.1016/j.soard.2005.07.002
Lieske JC, Mehta RA, Milliner DS, Rule AD, Bergstralh EJ, Sarr MG. Kidney stones are common after bariatric surgery. Kidney Int. 2015;87(4):839–845. doi: 10.1038/ki.2014.352
doi: 10.1038/ki.2014.352
Agrawal V, Wilfong JB, Rich CE, Gibson PC. Reversal of gastric bypass resolves hyperoxaluria and improves oxalate nephropathy secondary to Roux-en-Y gastric bypass. Case Rep Nephrol Dial. 2016;6(3):114–119. doi: 10.1159/000449128
doi: 10.1159/000449128
Dhar NB, Grundfest S, Jones JS, Streem SB. Jejunoileal bypass reversal: effect on renal function, metabolic parameters and stone formation. J Urol. 2005;174(5):1844–1846; discussion 1846. doi: 10.1097/01.ju.0000177079.56949.1a
doi: 10.1097/01.ju.0000177079.56949.1a
Nasr SH, D'Agati VD, Said SM, et al. Oxalate nephropathy complicating Roux-en-Y gastric bypass: an underrecognized cause of irreversible renal failure. Clin J Am Soc Nephrol. 2008;3(6):1676–1683. doi: 10.2215/CJN.02940608
doi: 10.2215/CJN.02940608
Cartery C, Faguer S, Karras A, et al. Oxalate nephropathy associated with chronic pancreatitis. Clin J Am Soc Nephrol. 2011;6(8):1895–1902. doi: 10.2215/CJN.00010111
doi: 10.2215/CJN.00010111
Troxell ML, Houghton DC, Hawkey M, Batiuk TD, Bennett WM. Enteric oxalate nephropathy in the renal allograft: an underrecognized complication of bariatric surgery. Am J Transplant. 2013;13(2):501–509. doi: 10.1111/ajt.12029
doi: 10.1111/ajt.12029
Karaolanis G, Lionaki S, Moris D, Palla VV, Vernadakis S. Secondary hyperoxaluria: a risk factor for kidney stone formation and renal failure in native kidneys and renal grafts. Transplant Rev. 2014;28(4):182–187. doi: 10.1016/j.trre.2014.05.004
doi: 10.1016/j.trre.2014.05.004
Lumlertgul N, Siribamrungwong M, Jaber BL, Susantitaphong P. Secondary oxalate nephropathy: a systematic review. Kidney Int Rep. 2018;3(6):1363–1372. doi: 10.1016/j.ekir.2018.07.020
doi: 10.1016/j.ekir.2018.07.020
Wharton R, D'Agati V, Magun AM, Whitlock R, Kunis CL, Appel GB. Acute deterioration of renal function associated with enteric hyperoxaluria. Clin Nephrol. 1990;34(3):116–121.
Williams HE. Oxalic acid and the hyperoxaluric syndromes. Kidney Int. 1978;13(5):410–417. doi: 10.1038/ki.1978.59
doi: 10.1038/ki.1978.59
Earnest DL. Enteric hyperoxaluria. Adv Intern Med. 1979;24:407–427.
D'Costa MR, Kausz AT, Carroll KJ, et al. Subsequent urinary stone events are predicted by the magnitude of urinary oxalate excretion in enteric hyperoxaluria. Nephrol Dial Transplant. 2021;36(12):2208–2215. doi: 10.1093/ndt/gfaa281
doi: 10.1093/ndt/gfaa281
Milliner DS, McGregor TL, Thompson A, et al. End points for clinical trials in primary hyperoxaluria. Clin J Am Soc Nephrol. 2020;15(7):1056–1065. doi: 10.2215/CJN.13821119
doi: 10.2215/CJN.13821119
Agyeman AS, Siegel JN, Leptak C. Establishing a public resource for acceptable surrogate endpoints to support FDA marketing applications. Front Med (Lausanne). 2022;9:820990. doi: 10.3389/fmed.2022.820990
doi: 10.3389/fmed.2022.820990
21st Century Cures Act. Subtitle B—Advancing New Drug Therapies. Sec 507. 2016. Accessed October 4, 2023. https://www.congress.gov/114/plaws/publ255/PLAW-114publ255.pdf
U.S. Department of Health and Human Services. Guidance for Industry Expedited Programs for Serious Conditions – Drugs and Biologics. U.S. Department of Health and Human Services Food and Drug Administration Center for Drug Evaluation and Research (CDER) Center for Biologics Evaluation and Research (CBER); 2014. Accessed October 4, 2023. https://www.fda.gov/media/86377/download
Park S, Pearle MS. Pathophysiology and management of calcium stones. Urol Clin North Am. 2007;34(3):323–334. doi: 10.1016/j.ucl.2007.04.009
doi: 10.1016/j.ucl.2007.04.009
Sakhaee K, Maalouf NM, Sinnott B. Clinical review. Kidney stones 2012: pathogenesis, diagnosis, and management. J Clin Endocrinol Metab. 2012;97(6):1847–1860. doi: 10.1210/jc.2011-3492
doi: 10.1210/jc.2011-3492
Dhondup T, Kittanamongkolchai W, Vaughan LE, et al. Risk of ESRD and mortality in kidney and bladder stone formers. Am J Kidney Dis. 2018;72(6):790–797. doi: 10.1053/j.ajkd.2018.06.012
doi: 10.1053/j.ajkd.2018.06.012
Alexander RT, Hemmelgarn BR, Wiebe N, et al. Kidney stones and kidney function loss: a cohort study. BMJ. 2012;345:e5287. doi: 10.1136/bmj.e5287
doi: 10.1136/bmj.e5287
Bhojani N, Mandeville JA, Hameed TA, et al. Lithotripter outcomes in a community practice setting: comparison of the LithoGold LG-380 and Storz Modulith SLX. J Urol. 2015;193(3):875–879. doi: 10.1016/j.juro.2014.09.117
doi: 10.1016/j.juro.2014.09.117
Jungers P, Joly D, Barbey F, Choukroun G, Daudon M. ESRD caused by nephrolithiasis: prevalence, mechanisms, and prevention. Am J Kidney Dis. 2004;44(5):799–805. doi: 10.1016/s0272-6386(04)01131-x
doi: 10.1016/s0272-6386(04)01131-x
Rule AD, Krambeck AE, Lieske JC. Chronic kidney disease in kidney stone formers. Clin J Am Soc Nephrol. 2011;6(8):2069–2075. doi: 10.2215/CJN.10651110
doi: 10.2215/CJN.10651110
Mulay SR, Anders HJ. Crystal nephropathies: mechanisms of crystal-induced kidney injury. Nat Rev Nephrol. 2017;13(4):226–240. doi: 10.1038/nrneph.2017.10
doi: 10.1038/nrneph.2017.10
Knauf F, Asplin JR, Granja I, et al. NALP3-mediated inflammation is a principal cause of progressive renal failure in oxalate nephropathy. Kidney Int. 2013;84(5):895–901. doi: 10.1038/ki.2013.207
doi: 10.1038/ki.2013.207
Turgeon NA, Perez S, Mondestin M, et al. The impact of renal function on outcomes of bariatric surgery. J Am Soc Nephrol. 2012;23(5):885–894. doi: 10.1681/ASN.2011050476
doi: 10.1681/ASN.2011050476
Selby MG, Vrtiska TJ, Krambeck AE, et al. Quantification of asymptomatic kidney stone burden by computed tomography for predicting future symptomatic stone events. Urology. 2015;85(1):45–50. doi: 10.1016/j.urology.2014.08.031
doi: 10.1016/j.urology.2014.08.031
Burgher A, Beman M, Holtzman JL, Monga M. Progression of nephrolithiasis: long-term outcomes with observation of asymptomatic calculi. J Endourol. 2004;18(6):534–539. doi: 10.1089/end.2004.18.534
doi: 10.1089/end.2004.18.534
Li X, Zhu W, Lam W, Yue Y, Duan H, Zeng G. Outcomes of long-term follow-up of asymptomatic renal stones and prediction of stone-related events. BJU Int. 2019;123(3):485–492. doi: 10.1111/bju.14565
doi: 10.1111/bju.14565
Darrad MP, Yallappa S, Metcalfe J, Subramonian K. The natural history of asymptomatic calyceal stones. BJU Int. 2018;122(2):263–269. doi: 10.1111/bju.14354
doi: 10.1111/bju.14354
Werness PG, Bergert JH, Lee KE. Urinary crystal growth: effect of inhibitor mixtures. Clin Sci. 1981;61(4):487–491. doi: 10.1042/cs0610487
doi: 10.1042/cs0610487
Asplin JR, Coe FL. Hyperoxaluria in kidney stone formers treated with modern bariatric surgery. J Urol. 2007;177(2):565–569. doi: 10.1016/j.juro.2006.09.033
doi: 10.1016/j.juro.2006.09.033
Valezi AC, Fuganti PE, Junior JM, Delfino VD. Urinary evaluation after RYGBP: a lithogenic profile with early postoperative increase in the incidence of urolithiasis. Obes Surg. 2013;23(10):1575–1580. doi: 10.1007/s11695-013-0916-0
doi: 10.1007/s11695-013-0916-0
Canales BK, Hatch M. Kidney stone incidence and metabolic urinary changes after modern bariatric surgery: review of clinical studies, experimental models, and prevention strategies. Surg Obes Relat Dis. 2014;10(4):734–742. doi: 10.1016/j.soard.2014.03.026
doi: 10.1016/j.soard.2014.03.026
Siener R, Petzold J, Bitterlich N, Alteheld B, Metzner C. Determinants of urolithiasis in patients with intestinal fat malabsorption. Urology. 2013;81(1):17–24. doi: 10.1016/j.urology.2012.07.107
doi: 10.1016/j.urology.2012.07.107
Gregory JG, Park KY, Schoenberg HW. Oxalate stone disease after intestinal resection. J Urol. 1977;117(5):631–634. doi: 10.1016/s0022-5347(17)58564-x
doi: 10.1016/s0022-5347(17)58564-x
Prochaska M, Taylor E, Ferraro PM, Curhan G. Relative supersaturation of 24-hour urine and likelihood of kidney stones. J Urol. 2018;199(5):1262–1266. doi: 10.1016/j.juro.2017.10.046
doi: 10.1016/j.juro.2017.10.046
Perinpam M, Enders FT, Mara KC, et al. Plasma oxalate in relation to eGFR in patients with primary hyperoxaluria, enteric hyperoxaluria and urinary stone disease. Clin Biochem. 2017;50(18):1014–1019. doi: 10.1016/j.clinbiochem.2017.07.017
doi: 10.1016/j.clinbiochem.2017.07.017
Hueppelshaeuser R, von Unruh GE, Habbig S, et al. Enteric hyperoxaluria, recurrent urolithiasis, and systemic oxalosis in patients with Crohn's disease. Pediatr Nephrol. 2012;27(7):1103–1109. doi: 10.1007/s00467-012-2126-8
doi: 10.1007/s00467-012-2126-8