Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (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.
Autosomal Dominant Polycystic Kidney Disease
Other: Standard of Care-Placebo
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Rapamycin as Treatment for ADPKD: The Role of Biomarkers in Predicting a Response to Therapy|
- Total Kidney Volume (mL) [ Time Frame: 2 years ] [ Designated as safety issue: No ]
- Glomerular Filtration Rate (Kidney Function) [ Time Frame: 2 years ] [ Designated as safety issue: No ]
|Study Start Date:||June 2009|
|Study Completion Date:||July 2010|
|Primary Completion Date:||July 2010 (Final data collection date for primary outcome measure)|
sirolimus 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.
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 Name: sirolimus
Placebo Comparator: Standard of Care-Placebo
Standard of Care
|Other: Standard of Care-Placebo|
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.
|United States, Connecticut|
|Yale Center for Clinical Investigation|
|New Haven, Connecticut, United States, 06520|
|Principal Investigator:||Neera K Dahl, MD, PhD||Yale University|