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

Overview

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

Full Title of Study: “Randomized, Controlled Pilot Study of Niacinamide in Polycystic Kidney Disease”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Basic Science
    • Masking: Triple (Participant, Care Provider, Investigator)
  • Study Primary Completion Date: December 18, 2017

Detailed Description

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.

Interventions

  • Dietary Supplement: Niacinamide
  • Other: Placebo
    • Placebo pill that matches niacinamide pill is size, shape and color

Arms, Groups and Cohorts

  • Experimental: Niacinamide
    • Participants will be asked to take niacinamide at a dose of 30 mg/kg/d orally.
  • Placebo Comparator: Placebo
    • Participants will be asked to take a placebo pill at a dose of 30 mg/kg/d orally.

Clinical Trial Outcome Measures

Primary Measures

  • 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.

Secondary Measures

  • 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

Participating in This Clinical Trial

Inclusion Criteria

  • Confirmed diagnosis of autosomal dominant polycystic kidney disease (ADPKD). – Estimated glomerular filtration rate (eGFR) > 50 ml/min/1.73m2 as determined from the serum creatinine by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation – Provide Informed consent 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 2 months prior to screening. Exclusions specific to magnetic resonance (MR) imaging acquisition and measurement: – Partial or total nephrectomy or renal cyst reduction (including aspiration) done <1 year ago – Cardiac pacemaker. – Presence of MR 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: 60 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • University of Kansas Medical Center
  • Collaborator
    • National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
  • Provider of Information About this Clinical Study
    • Principal Investigator: Alan Yu, MB, BChir, Professor of Medicine – University of Kansas Medical Center
  • Overall Official(s)
    • Alan Yu, M.B., B.Chir, Principal Investigator, University of Kansas Medical Center

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