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

Alan Yu, MB, BChir10 enrolled

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.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Polycystic Kidney Disease

Interventions

NiacinamideDIETARY_SUPPLEMENT

Eligibility

Sex: ALLMin age: 18 YearsMax age: 50 Years

Medical Language ↔ Plain English

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

Locations (1)

University of Kansas Medical Center

Kansas City, Kansas, United States

Outcomes

Primary Outcomes

Sirtuin deacetylase activity

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))

Time frame: Change from Baseline to 12 Months

Secondary Outcomes

Sirtuin deacetylase activity

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))

Time frame: Change from Baseline to 6 Months

Estimated Glomerular Filtration Rate (eGFR)

Measurements at each visit using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation

Time frame: 12 Months

Height-adjusted total kidney volumes (htTKV)

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.

Time frame: Change from Baseline to 12 Months

Biomarker levels

Aliquots of the urine samples from visits to be tested for a panel of biomarkers related to cyst growth compared to baseline levels.

Time frame: 12 Months

Subject pain

Subject feelings collected via abbreviated pain questionnaire. Questionnaire will be used to calculate pain score.

Time frame: Change from Baseline to 12 Months

Data from ClinicalTrials.gov

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