Clinical Trial: Pilot Study of Niacinamide in Polycystic Kidney Disease (NIAC-PKD2)

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Randomized, Controlled Pilot Study of Niacinamide in Polycystic Kidney Disease

Brief Summary: The purpose of this study is to observe the effects of niacinamide on markers of kidney injury, inflammation, kidney cyst growth and kidney function.

Detailed Summary:

Polycystic Kidney Disease (PKD) is a genetic disease that affects about 1 in 500 people worldwide. It can lead to kidney failure which can lead to death. PKD causes the development of kidney cysts (fluid-filled balloons), which cause worsening kidney function. It is common for people with PKD to also have blood in the urine, kidney pain, high blood pressure, kidney stones, kidney infections, and cysts in the brain or other parts of the body.

There is currently no treatment known to stop cyst growth or a cure for the disease.

Participants in this study will be randomly assigned to one of two groups: niacinamide or placebo. Participants have an equal chance of being assigned to either of the groups. For people that qualify and decide to participate in this study, they will be asked to make five visits to the study clinic and complete two study related phone calls over the course of 12 months.


Sponsor: University of Kansas Medical Center

Current Primary Outcome: Change in acetylated/total p53 ratio measured by ELISA in peripheral blood mononuclear cells (PBMC) [ Time Frame: Change from Baseline to Month 12 ]

Sirtuin 1 deacetylates p53 protein, and niacinamide inhibits sirtuin 1. So an increase in acetylated/total p53 ratio in PBMC will be used as a marker of biological efficacy.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change in height-adjusted total kidney volume [ Time Frame: Change from Baseline to Month 12 ]
    Measured by MRI
  • Change in score on pain questionnaire [ Time Frame: Change from Baseline to Month 12 ]
    PKD-9 Pain Questionnaire: Range of scores from 13 (no pain or symptoms) to 70 (very severe symptoms almost every day)
  • Change in urinary concentration of MCP-1 [ Time Frame: Change from Baseline to Month 12 ]
  • Change in estimated GFR [ Time Frame: Change from Baseline to Month 12 ]
    Determined from serum creatinine concentrations using CKD-Epi equation


Original Secondary Outcome: Same as current

Information By: University of Kansas Medical Center

Dates:
Date Received: September 21, 2015
Date Started: September 2015
Date Completion: December 2018
Last Updated: November 30, 2016
Last Verified: November 2016