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

• Status: Terminated
• Trial end date: 2010-07-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Yale University
• Treatments: Everolimus; Sirolimus

Criteria

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