Pilot Study of RNA as a Biomarker for Autosomal Dominant Polycystic Kidney Disease

The aim of this pilot project is to assess the potential of urine micro-RNAs (miRNA) as biomarkers for characterizing patients with autosomal dominant polycystic kidney disease (ADPKD) compared with patients with other causes of chronic kidney disease.

Study Overview

• Status: Completed
• Conditions: Chronic Kidney Disease; Polycystic Kidney, Autosomal Dominant

Detailed Description

Proteins and small molecules in urine (biomarkers) have been used to probe for kidney and systemic diseases for hundreds of years. Urine reportedly contains a type of molecule called microRNA (miRNAs) that regulate a large number of biological processes. Impaired function of miRNAs is now recognized in an increasing number of disease processes. In the search for new biomarkers, the regulatory function of miRNAs and the relative simplicity and precision of characterizing miRNAs, are potential advantages when compared to traditional biomarkers.

The aim of this pilot project is to assess the potential of urine miRNAs as biomarkers for characterizing patients with autosomal dominant polycystic kidney disease (ADPKD), the most prevalent inherited cause of kidney failure. Individuals with other causes of chronic kidney disease (e.g., diabetes, glomerulonephritis), who are matched for key characteristics (e.g. age, sex, level of kidney function) will serve as the control population. A technique for isolation of miRNAs from urine samples will be tailored for the specific needs of this project. Biochemical and computational analysis of small RNAs from these samples will provide urine miRNA profiles and key variability statistics that will be use to design follow-up projects involving patients with kidney disease.

• Study Type: Observational
• Enrollment (Actual): 42

Contacts and Locations

Study Locations

• United States
  • New York
    • New York, New York, United States, 10021
      • The Rogosin Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

20 outpatients, 20 patients control

Description

Inclusion Criteria:

1. Male or female subject, 18 years of age or older, with diagnosis of ADPKD or non-PKD-CKD
2. If female, not pregnant.
3. Willing and able to understand and sign informed consent

Exclusion Criteria:

1. Presenting with any signs or symptoms of an infectious disease
2. Bacterial infection determined by urine culture
3. Use of systemic steroids within a week prior to screening
4. History, physical, or laboratory findings suggestive of any other medical or psychological condition that would, in the opinion of the Principal Investigator, make the candidate ineligible for the study.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
PKD; Patients with Autosomal Dominant Polycystic Kidney Disease
non-PKD CKD; Patients with non-Polycystic Chronic Kidney Disease

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Identification of micro-RNA Biomarkers Associated with Autosomal Dominant Chronic Kidney Disease	5 years

Collaborators and Investigators

• Sponsor: The Rogosin Institute
• Collaborators: Weill Medical College of Cornell University; Rockefeller University
• Investigators: Principal Investigator: Jon Blumenfeld, MD, The Rogosin Institute

Study record dates

Study Major Dates

• Study Start: April 1, 2010
• Primary Completion (Actual): April 1, 2012
• Study Completion (Actual): April 1, 2012

Study Registration Dates

• First Submitted: April 26, 2010
• First Submitted That Met QC Criteria: April 30, 2010
• First Posted (Estimate): May 3, 2010

Study Record Updates

• Last Update Posted (Actual): November 6, 2018
• Last Update Submitted That Met QC Criteria: November 2, 2018
• Last Verified: November 1, 2018

More Information

Terms related to this study

• Keywords: Chronic Kidney Disease (CKD); Autosomal Dominant Polycystic Kidney Disease (ADPKD)
• Additional Relevant MeSH Terms: Urologic Diseases; Congenital Abnormalities; Renal Insufficiency; Genetic Diseases, Inborn; Joint Diseases; Musculoskeletal Diseases; Muscular Diseases; Musculoskeletal Abnormalities; Abnormalities, Multiple; Kidney Diseases, Cystic; Ciliopathies; Kidney Diseases; Renal Insufficiency, Chronic; Polycystic Kidney Diseases; Polycystic Kidney, Autosomal Dominant; Arthrogryposis
• Other Study ID Numbers: 1003010924