Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy
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|ClinicalTrials.gov Identifier: NCT00920309|
Recruitment Status : Terminated (This study was stopped after larger studies published in the NEJM failed to show a benefit in treating ADPKD)
First Posted : June 15, 2009
Results First Posted : April 14, 2014
Last Update Posted : April 14, 2014
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.
|Condition or disease||Intervention/treatment||Phase|
|Autosomal Dominant Polycystic Kidney Disease||Drug: Rapamycin Other: Standard of Care-Placebo||Phase 2 Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||21 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||Rapamycin as Treatment for ADPKD: The Role of Biomarkers in Predicting a Response to Therapy|
|Study Start Date :||June 2009|
|Actual Primary Completion Date :||July 2010|
|Actual Study Completion Date :||July 2010|
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|
- Total Kidney Volume (mL) [ Time Frame: 2 years ]
- Glomerular Filtration Rate (Kidney Function) [ Time Frame: 2 years ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00920309
|United States, Connecticut|
|Yale Center for Clinical Investigation|
|New Haven, Connecticut, United States, 06520|
|Principal Investigator:||Neera K Dahl, MD, PhD||Yale University|