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Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy
This study has been terminated.
(This study was stopped after larger studies published in the NEJM failed to show a benefit in treating ADPKD)
Sponsor:
Yale University
Information provided by (Responsible Party):
Yale University
ClinicalTrials.gov Identifier:
NCT00920309
First received: June 12, 2009
Last updated: March 7, 2014
Last verified: March 2014
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Purpose
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 | Intervention | Phase |
|---|---|---|
| Autosomal Dominant Polycystic Kidney Disease | Drug: Rapamycin Other: Standard of Care-Placebo | Phase 2 Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized 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 |
Resource links provided by NLM:
Genetics Home Reference related topics:
polycystic kidney disease
MedlinePlus related topics:
Kidney Diseases
U.S. FDA Resources
Further study details as provided by Yale University:
Primary Outcome Measures:
- Total Kidney Volume (mL) [ Time Frame: 2 years ]
Secondary Outcome Measures:
- Glomerular Filtration Rate (Kidney Function) [ Time Frame: 2 years ]
| Enrollment: | 21 |
| Study Start Date: | June 2009 |
| Study Completion Date: | July 2010 |
| Primary Completion Date: | July 2010 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
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. |
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
|
|
Placebo Comparator: Standard of Care-Placebo
Standard of Care
|
Other: Standard of Care-Placebo |
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.
Eligibility| Ages Eligible for Study: | 18 Years to 70 Years (Adult, Senior) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | Yes |
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
Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00920309
Please refer to this study by its ClinicalTrials.gov identifier: NCT00920309
Locations
| United States, Connecticut | |
| Yale Center for Clinical Investigation | |
| New Haven, Connecticut, United States, 06520 | |
Sponsors and Collaborators
Yale University
Investigators
| Principal Investigator: | Neera K Dahl, MD, PhD | Yale University |
More Information
| Responsible Party: | Yale University |
| ClinicalTrials.gov Identifier: | NCT00920309 History of Changes |
| Other Study ID Numbers: |
HIC#0903004934 |
| Study First Received: | June 12, 2009 |
| Results First Received: | August 29, 2013 |
| Last Updated: | March 7, 2014 |
Keywords provided by Yale University:
|
Autosomal Dominant Polycystic Kidney Disease rapamycin ADPKD |
Additional relevant MeSH terms:
|
Kidney Diseases Polycystic Kidney Diseases Polycystic Kidney, Autosomal Dominant Urologic Diseases Kidney Diseases, Cystic Sirolimus Everolimus Anti-Bacterial Agents |
Anti-Infective Agents Antibiotics, Antineoplastic Antineoplastic Agents Antifungal Agents Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs |
ClinicalTrials.gov processed this record on June 23, 2017