Effect of Statin Treatment on Urinary AQP2 (uAQP2/01)

NCT ID: NCT02523001

Last Updated: 2015-08-14

Study Results

Results pending

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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Recruitment Status

COMPLETED

Total Enrollment

37 participants

Study Classification

OBSERVATIONAL

Study Start Date

2013-10-31

Study Completion Date

2015-07-31

Brief Summary

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The purpose of this study is to test the hypothesis that the function and/or regulation of urinary aquaporin 2 in hypercholesterolemic humans is affected by standard statin therapy, as compared with diet alone

Detailed Description

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Statins are the first-line recommended pharmacological therapy in patients with dyslipidemias and play a key role in both primary and secondary prevention of coronary heart disease. By decreasing plasma total and low-density lipoprotein cholesterol (LDL-C) concentrations, statins decrease the risks for atherosclerotic cardiovascular disease and associated morbidity and mortality. Statins occupy part of the active binding site of 3-hydroxy-3-methylglutaryl-coenzyme A (HMG-CoA) and inhibit its enzymatic activity in the liver, a key step leading to the reduction of cellular sterol pool. Statins also have beneficial effects on the vascular wall by stabilizing the atherosclerotic plaques, ameliorating impaired endothelial function, and reducing vascular inflammation.

Besides the well-known metabolic and cardiovascular effects, it has been recently shown that statins increase the plasma membrane expression of the renal water channels Aquaporin 2 (AQP2). Water reabsorption in the kidney connecting tubule and collecting duct is regulated by the antidiuretic hormone arginine vasopressin (AVP), which promotes plasma membrane expression of the water channe aquaporin 2 (AQP2), the rate-limiting step controlling reabsorption of water, thus urine concentration, in this segment of the nephron. The investigators reported a number of evidences showing that statins accumulate AQP2 at the apical membrane of collecting duct cells by a AVP-independent mechanism. The effect of statins on AQP2 is independent of classical cholesterol homeostasis but rather depends on depletion of mevalonate-derived intermediates of cholesterol synthetic pathways, i.e. isoprenoid intermediates, including farnesylpyrophosphate (FPP) and geranylgeranylpyrophosphate (GGPP).

Water balance disorders are often associated with defects of AQP2 trafficking. Nephrogenic Diabetes Insipidus (NDI) is characterized by the inability of the kidney to respond to AVP stimulation and is caused by either mutations in AQP2 or vasopressin type-2 receptor (AVPR2) genes. Mutations in the AVPR2 gene lead to X-linked NDI (X-NDI). This cause of 90% of all diagnosed congenital NDI cases.

Conventional treatment of X-NDI patients consists in low-sodium, low-protein diet and the administration on thiazide diuretics sometimes in combination with indomethacin or amiloride. Although these drugs cause some relief of X-NDI symptoms, they most often do not eliminate them. Due to the partial beneficial effect of conventional treatments, much effort has been spent in the past years to uncover new and alternative methods to induce antidiuresis in X-NDI patients.

In this regard, the investigators recently reported that statins, in particular fluvastatin, accumulate AQP2 at the apical membrane of collecting duct cells by a AVP-independent mechanism and increase water reabsorption in both wild type and X-NDI mice.

The effect of statins on AQP2 trafficking in humans, however, deserves further investigation, also considering the potential efficacy of statins in patients with X-NDI. This was the reason to embark on the present study in which the investigators monitored the time-dependent effects of statin therapy on the urine excretion of AQP2, diuresis and urine osmolality in a cohort of hypercholesterolemic subjects initiating simvastatin therapy for three months. Two other groups of patients serve as controls: patients already on statin treatment and patients choosing to undergo an initial program with a standard hypolipidemic "mediterranean" style diet.

Conditions

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Hypercholesterolemia Diabetes Insipidus

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Naïve-S

Patients with hypercholesterolemia starting statin treatment for primary or secondary prevention of cardiovascular diseases.

No interventions assigned to this group

ONC-S

Patients with hypercholesterolemia who had been treated with statin for at least one year and continued their previous therapy during the study period

No interventions assigned to this group

Naïve-MC

Patients with mild hypercholesterolemia either refusing initial statin treatment or intolerant to standard statin treatment and starting with monacolin K

No interventions assigned to this group

Naïve-Diet

Patients refusing an initial pharmacologic treatments and choosing the hypolipidic diet

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

* patients on primary prevention requiring moderate reduction of LDL-cholesterol according to the ACC/AHA Guidelines
* age 40 to 75 years
* estimated 10-year ASCVD risk ≥7.5%

Exclusion Criteria

* concomitant type 1 or type 2 diabetes
* blood hypertension
* concomitant use of diuretics or other drugs interfering with simvastatin
Minimum Eligible Age

40 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University of Bari

OTHER

Sponsor Role lead

Responsible Party

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piero portincasa

Professor of Medicine, PhD

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Piero Portincasa

Role: PRINCIPAL_INVESTIGATOR

University of Bari Medical School

Locations

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Department of Biomedical Sciences Human Oncology - Clinica Medica "A. Murri"

Bari, , Italy

Site Status

Countries

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Italy

References

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Reference Type BACKGROUND
PMID: 16639429 (View on PubMed)

Pisitkun T, Shen RF, Knepper MA. Identification and proteomic profiling of exosomes in human urine. Proc Natl Acad Sci U S A. 2004 Sep 7;101(36):13368-73. doi: 10.1073/pnas.0403453101. Epub 2004 Aug 23.

Reference Type BACKGROUND
PMID: 15326289 (View on PubMed)

Valenti G, Laera A, Pace G, Aceto G, Lospalluti ML, Penza R, Selvaggi FP, Chiozza ML, Svelto M. Urinary aquaporin 2 and calciuria correlate with the severity of enuresis in children. J Am Soc Nephrol. 2000 Oct;11(10):1873-1881. doi: 10.1681/ASN.V11101873.

Reference Type BACKGROUND
PMID: 11004218 (View on PubMed)

Kanno K, Sasaki S, Hirata Y, Ishikawa S, Fushimi K, Nakanishi S, Bichet DG, Marumo F. Urinary excretion of aquaporin-2 in patients with diabetes insipidus. N Engl J Med. 1995 Jun 8;332(23):1540-5. doi: 10.1056/NEJM199506083322303.

Reference Type BACKGROUND
PMID: 7537863 (View on PubMed)

Valenti G, Laera A, Gouraud S, Pace G, Aceto G, Penza R, Selvaggi FP, Svelto M. Low-calcium diet in hypercalciuric enuretic children restores AQP2 excretion and improves clinical symptoms. Am J Physiol Renal Physiol. 2002 Nov;283(5):F895-903. doi: 10.1152/ajprenal.00354.2001.

Reference Type BACKGROUND
PMID: 12372764 (View on PubMed)

Wade JB. Statins affect AQP2 traffic. Am J Physiol Renal Physiol. 2011 Aug;301(2):F308. doi: 10.1152/ajprenal.00248.2011. Epub 2011 May 11. No abstract available.

Reference Type BACKGROUND
PMID: 21561996 (View on PubMed)

Li W, Zhang Y, Bouley R, Chen Y, Matsuzaki T, Nunes P, Hasler U, Brown D, Lu HA. Simvastatin enhances aquaporin-2 surface expression and urinary concentration in vasopressin-deficient Brattleboro rats through modulation of Rho GTPase. Am J Physiol Renal Physiol. 2011 Aug;301(2):F309-18. doi: 10.1152/ajprenal.00001.2011. Epub 2011 Apr 20.

Reference Type BACKGROUND
PMID: 21511701 (View on PubMed)

Grunfeld JP, Rossier BC. Lithium nephrotoxicity revisited. Nat Rev Nephrol. 2009 May;5(5):270-6. doi: 10.1038/nrneph.2009.43.

Reference Type BACKGROUND
PMID: 19384328 (View on PubMed)

Frokiaer J, Marples D, Knepper MA, Nielsen S. Bilateral ureteral obstruction downregulates expression of vasopressin-sensitive AQP-2 water channel in rat kidney. Am J Physiol. 1996 Apr;270(4 Pt 2):F657-68. doi: 10.1152/ajprenal.1996.270.4.F657.

Reference Type BACKGROUND
PMID: 8967344 (View on PubMed)

Marples D, Frokiaer J, Dorup J, Knepper MA, Nielsen S. Hypokalemia-induced downregulation of aquaporin-2 water channel expression in rat kidney medulla and cortex. J Clin Invest. 1996 Apr 15;97(8):1960-8. doi: 10.1172/JCI118628.

Reference Type BACKGROUND
PMID: 8621781 (View on PubMed)

Bustamante M, Hasler U, Leroy V, de Seigneux S, Dimitrov M, Mordasini D, Rousselot M, Martin PY, Feraille E. Calcium-sensing receptor attenuates AVP-induced aquaporin-2 expression via a calmodulin-dependent mechanism. J Am Soc Nephrol. 2008 Jan;19(1):109-16. doi: 10.1681/ASN.2007010092. Epub 2007 Nov 21.

Reference Type BACKGROUND
PMID: 18032798 (View on PubMed)

Procino G, Carmosino M, Tamma G, Gouraud S, Laera A, Riccardi D, Svelto M, Valenti G. Extracellular calcium antagonizes forskolin-induced aquaporin 2 trafficking in collecting duct cells. Kidney Int. 2004 Dec;66(6):2245-55. doi: 10.1111/j.1523-1755.2004.66036.x.

Reference Type BACKGROUND
PMID: 15569313 (View on PubMed)

Moeller HB, Rittig S, Fenton RA. Nephrogenic diabetes insipidus: essential insights into the molecular background and potential therapies for treatment. Endocr Rev. 2013 Apr;34(2):278-301. doi: 10.1210/er.2012-1044. Epub 2013 Jan 29.

Reference Type BACKGROUND
PMID: 23360744 (View on PubMed)

Patrick L, Uzick M. Cardiovascular disease: C-reactive protein and the inflammatory disease paradigm: HMG-CoA reductase inhibitors, alpha-tocopherol, red yeast rice, and olive oil polyphenols. A review of the literature. Altern Med Rev. 2001 Jun;6(3):248-71.

Reference Type BACKGROUND
PMID: 11410071 (View on PubMed)

Bonfrate L, Procino G, Wang DQ, Svelto M, Portincasa P. A novel therapeutic effect of statins on nephrogenic diabetes insipidus. J Cell Mol Med. 2015 Feb;19(2):265-82. doi: 10.1111/jcmm.12422. Epub 2015 Jan 16.

Reference Type RESULT
PMID: 25594563 (View on PubMed)

Murer L, Addabbo F, Carmosino M, Procino G, Tamma G, Montini G, Rigamonti W, Zucchetta P, Della Vella M, Venturini A, Zacchello G, Svelto M, Valenti G. Selective decrease in urinary aquaporin 2 and increase in prostaglandin E2 excretion is associated with postobstructive polyuria in human congenital hydronephrosis. J Am Soc Nephrol. 2004 Oct;15(10):2705-12. doi: 10.1097/01.ASN.0000139689.94776.7A.

Reference Type RESULT
PMID: 15466275 (View on PubMed)

Procino G, Milano S, Carmosino M, Barbieri C, Nicoletti MC, Li JH, Wess J, Svelto M. Combination of secretin and fluvastatin ameliorates the polyuria associated with X-linked nephrogenic diabetes insipidus in mice. Kidney Int. 2014 Jul;86(1):127-38. doi: 10.1038/ki.2014.10. Epub 2014 Feb 12.

Reference Type RESULT
PMID: 24522493 (View on PubMed)

Procino G, Barbieri C, Carmosino M, Rizzo F, Valenti G, Svelto M. Lovastatin-induced cholesterol depletion affects both apical sorting and endocytosis of aquaporin-2 in renal cells. Am J Physiol Renal Physiol. 2010 Feb;298(2):F266-78. doi: 10.1152/ajprenal.00359.2009. Epub 2009 Nov 18.

Reference Type RESULT
PMID: 19923410 (View on PubMed)

Procino G. Fluvastatin Increases AQP2 Urine Excretion in a Dyslipidemic Patient with Nephrogenic Diabetes Insipidus: An In Vivo and In Vitro Study. Journal of Diabetes & Metabolism 2014; 5(7); 1000408

Reference Type RESULT

Other Identifiers

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MRAR08P011

Identifier Type: OTHER

Identifier Source: secondary_id

uAQP2/01

Identifier Type: -

Identifier Source: org_study_id

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