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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
Sponsor:
Information provided by (Responsible Party):
Yale University

Tracking Information
First Submitted Date  ICMJE June 12, 2009
First Posted Date  ICMJE June 15, 2009
Results First Submitted Date  ICMJE August 29, 2013
Results First Posted Date  ICMJE April 14, 2014
Last Update Posted Date April 14, 2014
Study Start Date  ICMJE June 2009
Actual Primary Completion Date July 2010   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 7, 2014)
Total Kidney Volume (mL) [ Time Frame: 2 years ]
Original Primary Outcome Measures  ICMJE
 (submitted: June 12, 2009)
combined kidney volume [ Time Frame: 2 years ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 7, 2014)
Glomerular Filtration Rate (Kidney Function) [ Time Frame: 2 years ]
Original Secondary Outcome Measures  ICMJE
 (submitted: June 12, 2009)
  • kidney function [ Time Frame: 2 years ]
  • change in biomarkers [ Time Frame: 2 years ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy
Official Title  ICMJE Rapamycin as Treatment for ADPKD: The Role of Biomarkers in Predicting a Response to Therapy
Brief 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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Autosomal Dominant Polycystic Kidney Disease
Intervention  ICMJE
  • 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
  • Other: Standard of Care-Placebo
Study Arms  ICMJE
  • Experimental: Rapamycin

    Drug: Rapamycin

    Other Names:

    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.

    Intervention: Drug: Rapamycin
  • Placebo Comparator: Standard of Care-Placebo
    Standard of Care
    Intervention: Other: Standard of Care-Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: March 7, 2014)
21
Original Estimated Enrollment  ICMJE
 (submitted: June 12, 2009)
45
Actual Study Completion Date  ICMJE July 2010
Actual Primary Completion Date July 2010   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 70 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00920309
Other Study ID Numbers  ICMJE HIC#0903004934
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Yale University
Study Sponsor  ICMJE Yale University
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Neera K Dahl, MD, PhD Yale University
PRS Account Yale University
Verification Date March 2014

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