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

Randomized, Controlled Pilot Study of Niacinamide in Polycystic Kidney Disease

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

Study Overview

• Status: Completed
• Conditions: Polycystic Kidney Disease
• Intervention / Treatment: Dietary supplement: Niacinamide; Other: Placebo

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.

• Study Type: Interventional
• Enrollment (Actual): 36
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • University of Kansas Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 60 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Basic Science
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Niacinamide; Participants will be asked to take niacinamide at a dose of 30 mg/kg/d orally.	Dietary supplement: Niacinamide; Other Names: Vitamin B3; Nicotinamide
Placebo Comparator: Placebo; Participants will be asked to take a placebo pill at a dose of 30 mg/kg/d orally.	Other: Placebo; Placebo pill that matches niacinamide pill is size, shape and color

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in acetylated/total p53 ratio measured by ELISA in peripheral blood mononuclear cells (PBMC)	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.	Change from Baseline to Month 12

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: University of Kansas Medical Center
• Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
• Investigators: Principal Investigator: Alan Yu, M.B., B.Chir, University of Kansas Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): September 22, 2015
• Primary Completion (Actual): December 18, 2017
• Study Completion (Actual): December 18, 2017

Study Registration Dates

• First Submitted: September 21, 2015
• First Submitted That Met QC Criteria: September 22, 2015
• First Posted (Estimate): September 24, 2015

Study Record Updates

• Last Update Posted (Actual): February 11, 2019
• Last Update Submitted That Met QC Criteria: February 8, 2019
• Last Verified: February 1, 2019

More Information

Terms related to this study

• Keywords: Kidney disease; PKD; Vitamin B3
• Additional Relevant MeSH Terms: Urologic Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Abnormalities, Multiple; Kidney Diseases, Cystic; Ciliopathies; Kidney Diseases; Polycystic Kidney Diseases; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Vasodilator Agents; Antimetabolites; Micronutrients; Hypolipidemic Agents; Lipid Regulating Agents; Vitamins; Vitamin B Complex; Nicotinic Acids; Niacinamide; Niacin
• Other Study ID Numbers: STUDY00002827; R21DK104086 (U.S. NIH Grant/Contract)