NCT00920309

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

• Status: Terminated
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 21 (actual)
• Sponsor: Yale University · Academic / Other
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Accepted

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.

Detailed description

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.

Conditions

• Autosomal Dominant Polycystic Kidney Disease

Interventions

Type	Name	Description
DRUG	Rapamycin	The starting dose of rapamycin will be 1 mg daily. The dose will be increased as needed to achieve a 24 hour trough level of 4-6 ng/ml.
OTHER	Standard of Care-Placebo

Timeline

• Start date: 2009-06-01
• Primary completion: 2010-07-01
• Completion: 2010-07-01
• First posted: 2009-06-15
• Last updated: 2014-04-14
• Results posted: 2014-04-14

Locations

1 site across 1 country: United States