Back to resultsRapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy
Primary Purpose
Autosomal Dominant Polycystic Kidney Disease
Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Rapamycin
Standard of Care-Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Autosomal Dominant Polycystic Kidney Disease focused on measuring Autosomal Dominant Polycystic Kidney Disease, rapamycin, ADPKD
Eligibility Criteria
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
Sites / Locations
- Yale Center for Clinical Investigation
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Rapamycin
Standard of Care-Placebo
Arm Description
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.
Standard of Care
Outcomes
Primary Outcome Measures
Total Kidney Volume (mL)
Secondary Outcome Measures
Glomerular Filtration Rate (Kidney Function)
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00920309
Brief Title
Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy
Official Title
Rapamycin as Treatment for ADPKD: The Role of Biomarkers in Predicting a Response to Therapy
Study Type
Interventional
2. Study Status
Record Verification Date
March 2014
Overall Recruitment Status
Terminated
Why Stopped
This study was stopped after larger studies published in the NEJM failed to show a benefit in treating ADPKD
Study Start Date
June 2009 (undefined)
Primary Completion Date
July 2010 (Actual)
Study Completion Date
July 2010 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Yale University
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Autosomal Dominant Polycystic Kidney Disease
Keywords
Autosomal Dominant Polycystic Kidney Disease, rapamycin, ADPKD
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
21 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Rapamycin
Arm Type
Experimental
Arm Description
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.
Arm Title
Standard of Care-Placebo
Arm Type
Placebo Comparator
Arm Description
Standard of Care
Intervention Type
Drug
Intervention Name(s)
Rapamycin
Other Intervention Name(s)
sirolimus
Intervention Description
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 Type
Other
Intervention Name(s)
Standard of Care-Placebo
Primary Outcome Measure Information:
Title
Total Kidney Volume (mL)
Time Frame
2 years
Secondary Outcome Measure Information:
Title
Glomerular Filtration Rate (Kidney Function)
Time Frame
2 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Neera K Dahl, MD, PhD
Organizational Affiliation
Yale University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Yale Center for Clinical Investigation
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06520
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy
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