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

Overview

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.

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: February 2016

Detailed Description

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.

Interventions

  • Dietary Supplement: Niacinamide

Arms, Groups and Cohorts

  • Experimental: Niacinamide
    • All subjects in this study will take niacinamide at a dose of 30 mg per kilogram of body weight by mouth daily, in two divided daily doses, for 12 months.

Clinical Trial Outcome Measures

Primary Measures

  • 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))

Secondary Measures

  • 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.

Participating in This Clinical Trial

Inclusion Criteria

  • Confirmed diagnosis of autosomal dominant polycystic kidney disease – eGFR > 90ml/min/1.73m2 as determined from the serum creatinine by the CKD-EPI equation – Ability to give informed consent in English Exclusion Criteria:

  • History of liver disease or abnormal liver function test – Heavy alcohol intake – Chronic diarrhea or malabsorption syndrome – Thrombocytopenia – Hypophosphatemia – Pregnancy or lactation or plan to become pregnant during the study – Treatment with anti-epileptic drugs – Treatment with tolvaptan, current or within 2 months prior to screening – Participation in another interventional trial currently or within 30 days prior to screening – Partial or total nephrectomy or renal cyst reduction (including aspiration) done <1 year ago – Cardiac pacemaker – Presence of magnetic resonance-incompatible metallic clips (e.g. clipped cerebral aneurysm) – Body weight >159 kg (350 lbs) or untreatable claustrophobia

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: 50 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Alan Yu, MB, BChir
  • Provider of Information About this Clinical Study
    • Sponsor-Investigator: Alan Yu, MB, BChir, Professor and Director, Kidney Institute – University of Kansas Medical Center
  • Overall Official(s)
    • Alan S Yu, MB, BChir, Principal Investigator, University of Kansas Medical Center

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