Exploratory Study Into the Effect of Salt Supplementation in Gitelman Syndrome
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 21
- 试验地点
- 3
- 主要终点
- Change in symptoms measured by personalized symptom score sheet
研究概览
简要总结
The purpose of this study is to determine the effect of salt (NaCl) supplementation on serum potassium and clinical signs and symptoms in patients with Gitelman syndrome.
详细描述
Single subject multiple randomized double-blinded multi-crossover placebo-controlled trials (N-of-1 trials) will be performed. Participants will enroll the study after giving written informed consent. The individual trials will consist of 3 treatment blocks which each contain a 4-week treatment period of salt supplementation (12 grams of NaCl) and a 4-week treatment period of placebo, in a randomized, blinded order. After each period of 4 weeks, outcomes measures will be measured.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically-proven, symptomatic Gitelman syndrome
- •Written informed consent
排除标准
- •inability to discontinue potassium-sparing diuretics, mineralocorticoid antagonists and NSAIDs; this means inability to reach a potassium level of 2.5 mmol/L or higher with maximally tolerable potassium supplementation after discontinuation of potassium-sparing diuretics
- •pregnancy
研究组 & 干预措施
Salt (NaCl)
12 grams (12 capsules) of NaCl per day
Note: participants will be treated with active and placebo comparator (N-of-1 trial design)
干预措施: Salt (NaCl) (Dietary Supplement)
Placebo
12 capsules of placebo per day
Note: participants will be treated with active and placebo comparator (N-of-1 trial design)
干预措施: Placebo (Other)
结局指标
主要结局
Change in symptoms measured by personalized symptom score sheet
时间窗: Weekly (week 0 till week 24)
Symptoms will be scored via a personalized symptom score sheet, which will be composed with the individual participant. Symptoms will be scored at a range from 0 - 10
Change in serum Potassium
时间窗: Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Change in serum potassium
次要结局
- Change in serum bicarbonate(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in serum renin(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in fractional urinary excretion of potassium(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in fractional urinary excretion of sodium(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in fractional urinary excretion of magnesium(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in urinary excretion of aldosterone(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Gitelman symptom questionnaire(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Weight(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in serum chloride(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in fractional urinary excretion of chloride(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in urinary calcium/creatinine ratio(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in urinary excretion of renin(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in serum sodium(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in serum magnesium(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in serum calcium(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in serum creatinine(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in serum aldosterone(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Blood pressure (including orthostatic hypotension measurement)(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Muscle strength(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)