Randomized, Controlled Pilot Study of Niacinamide in Polycystic Kidney Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 36
- 试验地点
- 2
- 主要终点
- Change in acetylated/total p53 ratio measured by ELISA in peripheral blood mononuclear cells (PBMC)
研究概览
简要总结
The purpose of this study is to observe the effects of niacinamide on markers of kidney injury, inflammation, kidney cyst growth and kidney function.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Basic Science
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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.
研究组 & 干预措施
Placebo
Participants will be asked to take a placebo pill at a dose of 30 mg/kg/d orally.
干预措施: Placebo (Other)
Niacinamide
Participants will be asked to take niacinamide at a dose of 30 mg/kg/d orally.
干预措施: Niacinamide (Dietary Supplement)
结局指标
主要结局
Change in acetylated/total p53 ratio measured by ELISA in peripheral blood mononuclear cells (PBMC)
时间窗: 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.
次要结局
- Change in height-adjusted total kidney volume(Change from Baseline to Month 12)
- Change in score on pain questionnaire(Change from Baseline to Month 12)
- Change in urinary concentration of MCP-1(Change from Baseline to Month 12)
- Change in estimated GFR(Change from Baseline to Month 12)
研究者
Alan Yu, MB, BChir
Professor of Medicine
University of Kansas Medical Center