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
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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 |
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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 |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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 |

Arm | Intervention/treatment |
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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
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Other: Standard of Care-Placebo |
- Total Kidney Volume (mL) [ Time Frame: 2 years ]
- Glomerular Filtration Rate (Kidney Function) [ Time Frame: 2 years ]

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Ages Eligible for Study: | 18 Years to 70 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | Yes |
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

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 |
Responsible Party: | Yale University |
ClinicalTrials.gov Identifier: | NCT00920309 |
Other Study ID Numbers: |
HIC#0903004934 |
First Posted: | June 15, 2009 Key Record Dates |
Results First Posted: | April 14, 2014 |
Last Update Posted: | April 14, 2014 |
Last Verified: | March 2014 |
Autosomal Dominant Polycystic Kidney Disease rapamycin ADPKD |
Arthrogryposis Kidney Diseases Polycystic Kidney Diseases Polycystic Kidney, Autosomal Dominant Urologic Diseases Joint Diseases Musculoskeletal Diseases Muscular Diseases Musculoskeletal Abnormalities Congenital Abnormalities Kidney Diseases, Cystic Abnormalities, Multiple |
Ciliopathies Genetic Diseases, Inborn Sirolimus Anti-Bacterial Agents Anti-Infective Agents Antibiotics, Antineoplastic Antineoplastic Agents Antifungal Agents Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs |