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

Sponsor

University of Kansas Medical Center (Other)

Overall Status

Completed

CT.gov ID

NCT02558595

Collaborator

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) (NIH)

Enrollment

Location

Arms

26.9

Actual Duration (Months)

1.3

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Polycystic Kidney Disease	Dietary Supplement: Niacinamide Other: Placebo	Phase 2

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 Design

Study Type:

Interventional

Actual Enrollment :

36 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Triple (Participant, Care Provider, Investigator)

Primary Purpose:

Basic Science

Official Title:

Randomized, Controlled Pilot Study of Niacinamide in Polycystic Kidney Disease

Actual Study Start Date :

Sep 22, 2015

Actual Primary Completion Date :

Dec 18, 2017

Actual Study Completion Date :

Dec 18, 2017

Arms and Interventions

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: Nicotinamide Vitamin B3
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

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:

Nicotinamide

Vitamin B3

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

Outcome Measures

Primary Outcome Measures

Change in acetylated/total p53 ratio measured by ELISA in peripheral blood mononuclear cells (PBMC) [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 Outcome Measures

Change in height-adjusted total kidney volume [Change from Baseline to Month 12]
Measured by MRI
Change in score on pain questionnaire [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 [Change from Baseline to Month 12]
Change in estimated GFR [Change from Baseline to Month 12]
Determined from serum creatinine concentrations using CKD-Epi equation

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 60 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.