Table 1 Subject inclusion and exclusion criteria of the Vienna RAP Study

Inclusion criteria

• ADPKD, as confirmed by history, ultrasound, computed tomography or magnetic resonance imaging

• 18 years of age, or older

• Baseline eGFR (4-variable MDRD equation) below 60 mL/min per 1.73 m²

• Negative serum pregnancy test prior to administration of sirolimus and agreement to use contraception throughout the study and for three months after

• Written informed consent

Exclusion criteria

• Need for renal replacement therapy

• Pregnancy or lactation

• Plans to become pregnant in the near future

• Refusal to use sufficient contraception

• Proteinuria as defined as protein:creatinine ratio >1,000 or >1 g/d, respectively

• History of life-threatening complications of ADPKD

• Evidence of active systemic or localized major infection

• Evidence of infiltrate or consolidation on chest X-ray

• Use of any investigational drug or treatment up to four weeks prior to enrolment and during the study

• Known allergy or hypersensitivity to sirolimus and its derivatives

• Medication that will interfere with the CYP3A4/CYP3A5 system

• Total white blood cell count below or equal to 3,000/mm³

• Platelet count below or equal to 100,000/mm³

• Fasting triglycerides above or equal to 400 mg/dL

• Fasting total cholesterol above or equal to 300 mg/dL

• Concomitant glomerular diseases

• Psychiatric disorders and any condition that might prevent full comprehension of the purposes and risks of the study

• History of malignancy, with the exception of adequately treated basal cell and squamous cell carcinoma of the skin

• HIV positivity