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

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.

Condition or disease	Intervention/treatment	Phase
Autosomal Dominant Polycystic Kidney Disease	Drug: Rapamycin Other: Standard of Care-Placebo	Phase 2 Phase 3

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 Design

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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

Resource links provided by the National Library of Medicine

Genetics Home Reference related topics: Polycystic kidney disease

MedlinePlus related topics: Kidney Diseases

Drug Information available for: Sirolimus Everolimus Temsirolimus

U.S. FDA Resources

Arms and Interventions

Arm	Intervention/treatment
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

Outcome Measures

Primary Outcome Measures :

Total Kidney Volume (mL) [ Time Frame: 2 years ]

Secondary Outcome Measures :

Glomerular Filtration Rate (Kidney Function) [ Time Frame: 2 years ]

Eligibility Criteria

Information from the National Library of Medicine

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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

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

Information from the National Library of Medicine

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

Locations

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United States, Connecticut
Yale Center for Clinical Investigation
New Haven, Connecticut, United States, 06520

Sponsors and Collaborators

Yale University

Investigators

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Principal Investigator:	Neera K Dahl, MD, PhD	Yale University

More Information

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Responsible Party:	Yale University
ClinicalTrials.gov Identifier:	NCT00920309 History of Changes
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

Keywords provided by Yale University:


Autosomal Dominant Polycystic Kidney Disease rapamycin ADPKD

Additional relevant MeSH terms:

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Kidney Diseases Polycystic Kidney Diseases Polycystic Kidney, Autosomal Dominant Urologic Diseases Kidney Diseases, Cystic Abnormalities, Multiple Congenital Abnormalities Ciliopathies Genetic Diseases, Inborn Sirolimus	Everolimus Anti-Bacterial Agents Anti-Infective Agents Antibiotics, Antineoplastic Antineoplastic Agents Antifungal Agents Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs

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