Table 1.

Mouse models used in PKD pre-clinical trials.

Drug	Effect	Model	Clinical Trial
OPC-31260	Lowers cAMP (antagonist of Vasopressin 2 Receptor, VPV2R)	cpk mouse (non-ortholgous ARPKD model) [56] Mouse orthologous Pkd2 (Pkd2-/WS25) [57]. PCK rat (frameshit in exon 36 of Pkhd1 rat ortholog) [58] pcy mouse (missense mutation in the nephronophtisis gene Nph3) [58]
Tolvaptan (OPC-41061)	Lowers cAMP (antagonist of VPV2R); derived from OPC-31260	PCK rat [59]	TEMPO (1, 445 patients; slower rate of cyst growth in drug group: 5.5% per year versus 2.8% per year; P <0.001 and slower decline in kidney function (reciprocal of the serum creatinine level, −2.61 mg.ml−1 per year vs. −3.81 mg.ml−1 per year; P<0.001)) [4]
Octreotide (somatostatin analogue)	Lowers cAMP	PCK rat [60]	42 patients with polycystic liver and kidney disease (liver volume changed 4.95% ± 6.77% in the octreotide group compared to (0.92% ± 8.33%) in the placebo group (P = 0.048, rank-sum test); no difference in kidney function [61]
Pasireotide (somatostatin analogue)	Lowers cAMP	PCK rat and Pkd2-/WS25 mouse [23]
Sirulimus (Rapamycin)/Everolimus	mTOR inhibitor	Han:SPRD rat (non-orthologous ADPKD model) [62–64] Pkd1cond/cond:Nestincre mouse model (mosaic, random, kidney inactivation) [65] Tg737orpk/orpk; TgRsq (non-orthologous polaris mouse model) and bpk (non-orthologous Bicaudal-C mouse model) [22]	433 patients with ADPKD (reduction in the rate of increase in kidney volume: least-square mean differences between everolimus and placebo groups were 54 ml at 1 year (P=0.02) and 71 ml at 2 years (P=0.06); everolimus group had a greater decline in the estimated GFR (by 5.4 ml per minute) than placebo (with a decline of 3.2 ml per minute) (P=0.004) [20]. 100 ADPKD patients in early stages of chronic kidney disease (sirolimus group had no difference in rate of kidney growth [19])