Conditions: Autosomal Dominant Polycystic Kidney Disease
Keywords: end-stage renal disease, ESRD, autosomal dominant polycystic kidney disease, ADPKD, everolimus
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 431
Healthy volunteers: no

Brief summary:
This study will assess whether everolimus (RAD001) is effective in preventing cyst and kidney expansion as well as worsening of renal function in patients with ADPKD and whether the application of 5 mg/day everolimus as monotherapy is safe and well tolerated.

Detailed description:

Arms:
arm 1: Patients in the everolimus group initially received 5 mg/day everolimus divided in 2 equal doses (i.e. 2.5 mg b.i.d.). Dose adjustments were performed to achieve a blood trough level of 3-8 ng/mL (maximum daily dose: 10 mg/day \[5 mg b.i.d.\]). arm 2: Placebo tablets equivalent to the dosage of everolimus 5 mg/day, divided in 2 equal doses.

Interventions: intervention 1: Placebo intervention 2: Everolimus
Intervention descriptions: intervention 1: placebo comparator intervention 2: experimental

Protocol document: