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

Sponsor:	Yale University
Information provided by:	Yale University
ClinicalTrials.gov Identifier:	NCT00920309

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	Phase II Phase III

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

Drug Information available for: Sirolimus Everolimus CCI 779

U.S. FDA Resources

Further study details as provided by Yale University:

Primary Outcome Measures:

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

Secondary Outcome Measures:

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

Estimated Enrollment:	45
Study Start Date:	June 2009
Estimated Study Completion Date:	June 2012
Estimated Primary Completion Date:	June 2012 (Final data collection date for primary outcome measure)

Intervention Details:

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

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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00920309

Locations

United States, Connecticut
Yale Center for Clinical Investigation	Recruiting
New Haven, Connecticut, United States, 06520
Contact: Neera K Dahl, MD, PhD 203-785-4184 neera.dahl@yale.edu
Contact: Tammy Stalmack, Study Coordinator 203-737-1575
Principal Investigator: Neera K Dahl, MD, PhD

Sponsors and Collaborators

Yale University

Investigators

Principal Investigator:

Neera K Dahl, MD, PhD

Yale University

More Information

No publications provided

Responsible Party:	Dr. Neera Dahl, Yale University School of Medicine, Section of Nephrology
ClinicalTrials.gov Identifier:	NCT00920309 History of Changes
Other Study ID Numbers:	HIC#0903004934
Study First Received:	June 12, 2009
Last Updated:	June 28, 2010
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
Polycystic Kidney Diseases
Polycystic Kidney, Autosomal Dominant
Urologic Diseases
Kidney Diseases, Cystic
Sirolimus
Everolimus
Antibiotics, Antineoplastic
Antineoplastic Agents

Therapeutic Uses
Pharmacologic Actions
Antifungal Agents
Anti-Infective Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Anti-Bacterial Agents

ClinicalTrials.gov processed this record on February 12, 2012