Clinical Trial: Uncontrolled, Open Label, Pilot and Feasibility Study of Niacinamide in Polycystic Kidney Disease

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Uncontrolled, Open Label, Pilot and Feasibility Study of Niacinamide in Polycystic Kidney Disease

Brief Summary: The goal of this pilot study is to evaluate the feasibility of administering niacinamide to patients with autosomal dominant polycystic kidney disease, to develop methods to assess the biological efficacy of niacinamide, and to perform a preliminary exploration of its clinical effect on kidney cyst growth and kidney function.

Detailed Summary:

Niacinamide is a form of vitamin B3. Vitamin B3 is found in many foods including yeast, meat, fish, milk, eggs, green vegetables, beans, and cereal grains. Recent studies in mice have shown that niacinamide, at high doses, may slow kidney cyst growth from polycystic kidney disease (PKD).

By doing this study, the researchers will determine if a larger, long-term study to test whether niacinamide slows progression of PKD is justified.


Sponsor: Alan Yu, MB, BChir

Current Primary Outcome: Sirtuin deacetylase activity [ Time Frame: Change from Baseline to 12 Months ]

Assessed by the level of post-translational modifications of two intracellular markers (1. Acetylated and total p53 protein concentrations, 2. Phosphorylated and total retinoblastoma protein (Rb))


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Sirtuin deacetylase activity [ Time Frame: Change from Baseline to 6 Months ]
    Assessed by the level of post-translational modifications of two intracellular markers (1. Acetylated and total p53 protein concentrations, 2. Phosphorylated and total retinoblastoma protein (Rb))
  • Estimated Glomerular Filtration Rate (eGFR) [ Time Frame: 12 Months ]
    Measurements at each visit using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation
  • Height-adjusted total kidney volumes (htTKV) [ Time Frame: Change from Baseline to 12 Months ]
    Measurements will be taken from MRI images, and the annual percent change in htTKV will be compared to historical values reported in a separate study of a similar study population.
  • Biomarker levels [ Time Frame: 12 Months ]
    Aliquots of the urine samples from visits to be tested for a panel of biomarkers related to cyst growth compared to baseline levels.
  • Subject pain [ Time Frame: Change from Baseline to 12 Months ]
    Subject feelings collected via abbreviated pain questionnaire. Questionnaire will be used to calculate pain score.


Original Secondary Outcome: Same as current

Information By: University of Kansas Medical Center

Dates:
Date Received: May 14, 2014
Date Started: May 2014
Date Completion:
Last Updated: November 30, 2016
Last Verified: November 2016