Polycystic Kidney Disease
Conditions
Keywords
PKD, Kidney disease, Vitamin B3
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.
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_SUPPLEMENTNiacinamide
OTHERPlacebo
Placebo pill that matches niacinamide pill is size, shape and color
Sponsors
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
University of Kansas Medical Center
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
BASIC_SCIENCE
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 60 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
United States
Outcome results
None listed