Pilot Study of Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD)
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ClinicalTrials.gov Identifier: NCT00286156 |
Recruitment Status :
Completed
First Posted : February 3, 2006
Results First Posted : April 6, 2015
Last Update Posted : April 6, 2015
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This study is a prospective, randomized, open-label, pilot clinical trial designed to compare the effects of an agent that has antiproliferative (1,2), antiangiogenesis (3),and tumor-progression blocking capabilities (4), namely, rapamycin (Rapamune®), in the treatment of autosomal-dominant polycystic kidney disease (ADPKD).
Up to this time, only generic renal disease treatments for ADPKD have been in use, such as the treatment of hypertension, urinary tract infections, renal stones, renal call carcinomas, and replacement therapy with dialysis and/or renal transplantation. The fundamental aberrations in ADPKD are proliferation of cyst-forming tubuloepithelial cells, secretion of cytokine-rich fluid into those cysts, and progressive cyst expansion and release of inflammatory mediators that injure surrounding normal renal tissue. Consequently, therapy directed specifically at blocking the proliferation of tubuloepithelial cells and their tendency to malignant transformation, as well as impeding their blood supply, should have obvious merit.
General Procedures:
In Group I participants will have an iothalamate glomerular filtration rate (GFR) equal to or greater than 60 ml/min/1.73 m2, and in Group II participants will have a GFR less than 25-59 ml/min/1.73 m2. Both males and females with ADPKD who volunteer and qualify, will be randomly and prospectively assigned to treatment with rapamycin at either a high or low trough blood level or to standard care (each 1/3 of enrolled patients) for one year. The two treatment groups will receive rapamycin doses aimed at maintaining the 20- to 24-hour trough blood levels at either 2 to 5 ng/mL (low-dose), or greater than 5 to 8 ng/mL (high-dose). These trough levels are in the lower range of levels used when treating renal transplant recipients in whom trough levels are typically maintained between 5 and 15 ng/mL.
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Polycystic Kidney Diseases | Drug: Rapamune | Phase 1 Phase 2 |
This study is a prospective, randomized,open label, pilot clinical trial designed to compare the effects of an agent that has antiproliferative (1,2), antiangiogenesis (3),and tumor-progression blocking capabilities (4), namely, rapamycin (Rapamune®), in the treatment of autosomal-dominant polycystic kidney disease (ADPKD).
Up to this time, only generic renal disease treatments for ADPKD have been in use, such as the treatment of hypertension, urinary tract infections, renal stones, renal call carcinomas, and replacement therapy with dialysis and/or renal transplantation. The fundamental aberrations in ADPKD are proliferation of cyst-forming tubuloepithelial cells, secretion of cytokine-rich fluid into those cysts, and progressive cyst expansion and release of inflammatory mediators that injure surrounding normal renal tissue. Consequently, therapy directed specifically at blocking the proliferation of tubuloepithelial cells and their tendency to malignant transformation, as well as impeding their blood supply, should have obvious merit.
General Procedures:
In Group I participants will have an iothalamate glomerular filtration rate (GFR) equal to or greater than 60 ml/min/1.73 m2, and in Group II participants will have a GFR less than 25-59 ml/min/1.73 m2. Both males and females with ADPKD who volunteer and qualify, will be randomly and prospectively assigned to treatment with rapamycin at either a high or low trough blood level or to standard care (each 1/3 of enrolled patients) for one year. The two treatment groups will receive rapamycin doses aimed at maintaining the 20- to 24-hour trough blood levels at either 2 to 5 ng/mL (low-dose), or greater than 5 to 8 ng/mL (high-dose). These trough levels are in the lower range of levels used when treating renal transplant recipients in whom trough levels are typically maintained between 5 and 15 ng/mL.
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 30 participants |
Allocation: | Randomized |
Intervention Model: | Factorial Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Pilot Study of Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease |
Study Start Date : | October 2006 |
Actual Primary Completion Date : | March 2012 |
Actual Study Completion Date : | December 2014 |

Arm | Intervention/treatment |
---|---|
Experimental: 1
Arm 1 Rapamune dose 2-6mg aimed at maintaining trough levels 5-8 ng/ml
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Drug: Rapamune
Group 1- doses of Rapamune aimed at maintaining trough levels 5-8ng/ml Group 2 - doses of Rapamune aimed at maintaining trough levels 2-5ng/ml Group 3- Standard Care
Other Name: Rapamycin |
Experimental: 2
Arm 2 Rapamune dose 2-6 mg aimed at maintaining trough levels of 2-5ng/ml
|
Drug: Rapamune
Group 1- doses of Rapamune aimed at maintaining trough levels 5-8ng/ml Group 2 - doses of Rapamune aimed at maintaining trough levels 2-5ng/ml Group 3- Standard Care
Other Name: Rapamycin |
No Intervention: 3
Standard Care
|
- Change in GFR From Baseline to 12 Months [ Time Frame: From baseline to 12 months ]GFR (glomerular filtration rate) was measured by iothalamate. GFR is a key indicator of renal function.
- Change in Total Kidney Volume as Measured by 3D-CT From Baseline to 12 Months [ Time Frame: From baseline to 12 months ]Total kidney volume measured by CT from baseline to 12 months

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Ages Eligible for Study: | 18 Years to 75 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- ADPKD
- > 18 y.o. GFR greater than or equal to 25. Willingness to be randomized to any treatment group Willingness to follow protocol requirements-frequent testing and follow-up required at Cleveland Clinic(Cleveland, OH) signed informed consent Willingness to use birth control(male and female)
Exclusion Criteria:
- Pregnancy
- post partum
- lactating
- system illness with renal involvement

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): NCT00286156
United States, Ohio | |
The Cleveland Clinic- main campus | |
Cleveland, Ohio, United States, 44195 |
Principal Investigator: | William E. Braun, MD | The Cleveland Clinic |
Responsible Party: | The Cleveland Clinic |
ClinicalTrials.gov Identifier: | NCT00286156 |
Other Study ID Numbers: |
7736 |
First Posted: | February 3, 2006 Key Record Dates |
Results First Posted: | April 6, 2015 |
Last Update Posted: | April 6, 2015 |
Last Verified: | March 2014 |
ADPKD |
Kidney Diseases Polycystic Kidney Diseases Polycystic Kidney, Autosomal Dominant Urologic Diseases Kidney Diseases, Cystic Abnormalities, Multiple Congenital Abnormalities 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 |