Table 2.

Human studies in autosomal dominant polycystic kidney disease.

Drug	Location	Type of study	Primary end point
Octreotide	Mario Negri Institute, Italy	Randomized, single-blind, placebo-controlled	Kidney volume on MRI
Octreotide	Mayo Clinic, Novartis	Randomized, double-blind, placebo-controlled, crossover	Liver volume on MRI
Lanreotide	Radboud University, The Netherlands	Randomized, placebo-controlled	Liver volume on CT
Tolvaptan	Otsuka Pharm, Multicenter, USA	Open-label	Long-term safety
Tolvaptan	Otsuka Pharm, Multicenter, International	Double-blind, placebo-controlled	Kidney volume on MRI
Everolimus	Novartis Pharm, Germany	Randomized, placebo-controlled	Kidney volume on MRI
Rapamycin	University of Colorado, USA	Pilot study of rapamycin vs non-rapamycin immunosuppression in kidney transplant patients with ADPKD	Kidney and liver volume on MRI
Rapamycin	Mario Negri Institute, Bergamo, Italy	Randomized, placebo-controlled	Kidney volume on CT scan
Rapamycin	University Hospital, Zurich, Switzerland	Randomized, placebo-controlled	Kidney volume on MRI
Rapamycin	Cleveland Clinic, Cleveland, USA	Randomized, placebo-controlled	Iothalamate GFR
Lisinopril/termistan vs lisinopril/placebo and low BP vs standard BP	Multicenter, USA	Randomized, double-blind, placebo-controlled, 2 × 2 factorial assignment	Kidney volume on MRI
Lisinopril/termistan vs lisinopril/placebo	Multicenter, USA	Randomized, double-blind, placebo-controlled	Time to 50% decrease in eGFR, ESRD, or death
Pravastatin	University of Colorado, USA	Randomized, double-blind, placebo-controlled	Kidney volume, LVMI, urine albumin, endothelial-dependent vasodilation

BP: Blood pressure; eGFR: Estimated glomerular filtration rate; ESRD: End-state renal disease; LVMI: Left ventricular myocardial infarction.