Uncontrolled, Open Label, Pilot and Feasibility Study of Niacinamide in Polycystic Kidney Disease
- Conditions
- Polycystic Kidney Disease
- Interventions
- Dietary Supplement: Niacinamide
- Registration Number
- NCT02140814
- Lead Sponsor
- Alan Yu, MB, BChir
- Brief Summary
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.
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 10
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Niacinamide 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.
- Primary Outcome Measures
Name Time Method Sirtuin deacetylase activity 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 Outcome Measures
Name Time Method Sirtuin deacetylase activity 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))
Biomarker levels 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.
Height-adjusted total kidney volumes (htTKV) 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.
Estimated Glomerular Filtration Rate (eGFR) 12 Months Measurements at each visit using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation
Subject pain Change from Baseline to 12 Months Subject feelings collected via abbreviated pain questionnaire. Questionnaire will be used to calculate pain score.
Trial Locations
- Locations (1)
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States