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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Niacinamide | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Niacinamide | Dietary Supplement |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| 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)) | Change from Baseline to 12 Months |
| Measure | Description | Time Frame |
|---|---|---|
| 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)) | Change from Baseline to 6 Months |
| Estimated Glomerular Filtration Rate (eGFR) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Alan S Yu, MB, BChir | University of Kansas Medical Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
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| ID | Term |
|---|---|
| D007690 | Polycystic Kidney Diseases |
| ID | Term |
|---|---|
| D052177 | Kidney Diseases, Cystic |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
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| ID | Term |
|---|---|
| D009536 | Niacinamide |
| ID | Term |
|---|---|
| D009539 | Nicotinic Acids |
| D000147 | Acids, Heterocyclic |
| D006571 | Heterocyclic Compounds |
| D011725 | Pyridines |
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Measurements at each visit using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation |
| 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. | 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. | 12 Months |
| Subject pain | Subject feelings collected via abbreviated pain questionnaire. Questionnaire will be used to calculate pain score. | Change from Baseline to 12 Months |
| D005261 |
| Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000072661 | Ciliopathies |
| D030342 | Genetic Diseases, Inborn |
| D006573 |
| Heterocyclic Compounds, 1-Ring |