Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy - Tabular View

Tracking Information

First Received Date ^ICMJE

June 12, 2009

Last Updated Date

June 12, 2009

Start Date ^ICMJE

June 2009

Estimated Primary Completion Date

June 2012 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

combined kidney volume [ Time Frame: 2 years ] [ Designated as safety issue: No ]

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

No Changes Posted

Current Secondary Outcome Measures ^ICMJE

kidney function [ Time Frame: 2 years ] [ Designated as safety issue: No ]
change in biomarkers [ Time Frame: 2 years ] [ Designated as safety issue: No ]

Original Secondary Outcome Measures ^ICMJE

Same as current

Descriptive Information

Brief Title ^ICMJE

Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy

Official Title ^ICMJE

Rapamycin as Treatment for ADPKD: The Role of Biomarkers in Predicting a Response to Therapy

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.

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 Phase

Phase II, Phase III

Study Type ^ICMJE

Interventional

Study Design ^ICMJE

Treatment, Randomized, Open Label, Parallel Assignment, Safety/Efficacy Study

Condition ^ICMJE

Autosomal Dominant Polycystic Kidney Disease

Intervention ^ICMJE

Drug: Rapamycin

Study Arms / Comparison Groups

Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

Estimated Completion Date

June 2012

Estimated Primary Completion Date

June 2012 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

Inclusion Criteria:

adult ADPKD patients aged 18-70
combined kidney volume >1500 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

Gender

Both

Ages

18 Years to 70 Years

Accepts Healthy Volunteers

Yes

Contacts ^ICMJE

Location Countries ^ICMJE

United States

Administrative Information

NCT ID ^ICMJE

NCT00920309

Responsible Party

Dr. Neera Dahl, Yale University School of Medicine, Section of Nephrology

Study ID Numbers ^ICMJE

HIC#0903004934

Study Sponsor ^ICMJE

Yale University

Collaborators ^ICMJE

Investigators ^ICMJE

Principal Investigator:

Neera K Dahl, MD, PhD

Yale University

Information Provided By

Yale University

Verification Date

June 2009

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP