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:

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

History of Changes

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 Endpoint Classification: Safety/Efficacy Study 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

Drug Information available for: Sirolimus Everolimus Temsirolimus

U.S. FDA Resources

Further study details as provided by Yale University:

Primary Outcome Measures:

Total Kidney Volume (mL) [ Time Frame: 2 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Glomerular Filtration Rate (Kidney Function) [ Time Frame: 2 years ] [ Designated as safety issue: No ]


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
Genders Eligible for Study:	Both
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

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

No publications provided


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
Health Authority:	United States: Institutional Review Board

Keywords provided by Yale University:


Autosomal Dominant Polycystic Kidney Disease rapamycin ADPKD

Additional relevant MeSH terms:


Kidney Diseases Multicystic Dysplastic Kidney Polycystic Kidney Diseases Polycystic Kidney, Autosomal Dominant Congenital Abnormalities Kidney Diseases, Cystic Urogenital Abnormalities Urologic Diseases Everolimus Sirolimus	Anti-Bacterial Agents Anti-Infective Agents Antibiotics, Antineoplastic Antifungal Agents Antineoplastic Agents Immunologic Factors Immunosuppressive Agents Pharmacologic Actions Physiological Effects of Drugs Therapeutic Uses

ClinicalTrials.gov processed this record on March 03, 2015

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