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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified June 2010 by Yale University.
Recruitment status was  Recruiting
Sponsor:
Information provided by:
Yale University
ClinicalTrials.gov Identifier:
NCT00920309
First received: June 12, 2009
Last updated: June 28, 2010
Last verified: June 2010
History of Changes

June 12, 2009
June 28, 2010
June 2009
June 2012   (final data collection date for primary outcome measure)
combined kidney volume [ Time Frame: 2 years ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT00920309 on ClinicalTrials.gov Archive Site
  • kidney function [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  • change in biomarkers [ Time Frame: 2 years ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
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.
Interventional
Phase 2
Phase 3
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Autosomal Dominant Polycystic Kidney Disease
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 Name: sirolimus
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
45
June 2012
June 2012   (final data collection date for primary outcome measure)

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
Both
18 Years to 70 Years
Yes
Not Provided
United States
 
NCT00920309
HIC#0903004934
Yes
Dr. Neera Dahl, Yale University School of Medicine, Section of Nephrology
Yale University
Not Provided
Principal Investigator: Neera K Dahl, MD, PhD Yale University
Yale University
June 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP