Polycystic Kidney Disease
Conditions
Keywords
PKD, niacinamide, Vitamin B3, Vitamin B
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.
Interventions
DIETARY_SUPPLEMENTNiacinamide
Sponsors
Alan Yu, MB, BChir
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 50 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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)) |
| Estimated Glomerular Filtration Rate (eGFR) | 12 Months | Measurements at each visit using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation |
| 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. |
| 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. |
| Subject pain | Change from Baseline to 12 Months | Subject feelings collected via abbreviated pain questionnaire. Questionnaire will be used to calculate pain score. |
Countries
United States
Outcome results
None listed