Uncontrolled, Open Label, Pilot and Feasibility Study of Niacinamide in Polycystic Kidney Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 10
- 试验地点
- 2
- 主要终点
- Sirtuin deacetylase activity
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 50 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
- •eGFR > 90ml/min/1.73m2 as determined from the serum creatinine by the CKD-EPI equation
- •Ability to give informed consent in English
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 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
结局指标
主要结局
Sirtuin deacetylase activity
时间窗: 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))
次要结局
- Sirtuin deacetylase activity(Change from Baseline to 6 Months)
- Biomarker levels(12 Months)
- Height-adjusted total kidney volumes (htTKV)(Change from Baseline to 12 Months)
- Estimated Glomerular Filtration Rate (eGFR)(12 Months)
- Subject pain(Change from Baseline to 12 Months)
研究者
Alan Yu, MB, BChir
Professor and Director, Kidney Institute
University of Kansas Medical Center