Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy

Rapamycin as Treatment for ADPKD: The Role of Biomarkers in Predicting a Response to Therapy

Currently the only approved use for rapamycin (sirolimus) is for immunosuppression after renal transplantation.

This trial is designed to determine whether rapamycin is safe and effective treatment for patients with polycystic kidney disease (ADPKD). Patients will be followed by volumetric magnetic resonance imaging (MRI) to observe for change in kidney (and cyst) size. Blood and urine samples will also be collected to evaluate for change in biomarkers with treatment.

This is a 2 year, open label trial to evaluate the role of rapamycin as treatment for ADPKD. Patients will be randomized 2:1 to the rapamycin arm or to standard therapy. The dose of rapamycin will be adjusted so that patients obtain 24 trough levels of 4-6ng/ml. There will be a volumetric MRI measurement at the start and end of the treatment period. Patients will be monitored every 4 months throughout the study.

Inclusion Criteria:

• adult ADPKD patients aged 18-70
• combined kidney volume >1200 ml
• estimated creatinine clearance >60 ml/min
• absence of implanted ferromagnetic objects

Exclusion Criteria:

• Age >70
• uncontrolled hyperlipidemia
• Proteinuria >500 mg/day
• unstable cerebral aneurysm
• active coronary artery disease
• diagnosis of another systemic condition affecting kidney function (ie: diabetes, hepatitis B or C, HIV)
• diagnosis of cancer other than skin cancer
• pregnancy or lactation
• presence of implanted ferromagnetic objects