Cystic Fibrosis in the Kidney: Monitoring the Effectiveness of Elexacaftor/tezacaftor/ivacaftor in Urine After a Short Pause of Therapy
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 30
- 试验地点
- 2
- 主要终点
- Difference in cumulative urine bicarbonate excretion before, during, and after ETI pause.
研究概览
简要总结
In cystic fibrosis (CF) renal base excretion is impaired, due to mutations in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene, since CFTR function is crucial in regulation of the kidney's HCO3- excretion.
The investigators suggest that challenged urine HCO3- excretion is a biomarker of CFTR function, which can be used to evaluate the extent of CFTR dysfunction and the possible correcting effects of CFTR modulating therapy.
This study aims to evaluate changes in challenged urine HCO3- excretion in CF patients, who are currently in treatment with the triple CFTR modulator combination therapy, Elexacaftor/tezacaftor/ivacaftor (ETI), before, during, and after a short treatment pause.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult (age >17 years) CF patients.
- •Normal kidney function estimated by eGFR>
- •Adults capable of understanding and voluntarily consenting.
排除标准
- •Critical acute illness.
- •Severe lung disease (ppFEV1<40%).
- •Adults not capable of understanding and voluntarily consenting.
结局指标
主要结局
Difference in cumulative urine bicarbonate excretion before, during, and after ETI pause.
时间窗: At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.
Challenged urine HCO3- test: Quantification of urine bicarbonate excretion after an acute oral NaHCO3 challenge before, under, and after ETI pause.
Link between changes in ETI plasma concentration and changes in urine bicarbonate excretion.
时间窗: At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.
Venous blood sampling: ETI plasma concentration measurement. Challenged urine HCO3- test: Quantification of urine bicarbonate excretion
次要结局
- Changes in plasma concentration of ETI during the trial.(At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.)
- Changes in acid-base and fluid status during the trial.(At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.)
- Link between plasma acid-base status and urine acid-base excretion.(At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.)
- Changes in electrolytes during the trial.(At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.)