Monitoring Kidney Function in Adults with Cystic Fibrosis During a Short Pause of Elexacaftortezacaftorivacaftor Treatment

What this Trial Is About

Researchers are studying cystic fibrosis CF, a condition caused by mutations in the CFTR gene which affects kidney function, specifically the kidneys ability to regulate bicarbonate HCO3- excretion. The study focuses on measuring changes in urine bicarbonate excretion as a biomarker of CFTR function. It aims to evaluate how the triple CFTR modulator therapy Elexacaftortezacaftorivacaftor ETI affects this kidney function before, during, and after a short pause in treatment. Participants with CF who are currently treated with ETI will be randomly assigned to pause their therapy for 12, 36, or 60 hours. Researchers will monitor changes in urine bicarbonate excretion and ETI plasma concentrations at baseline, during the pause, and after therapy is resumed. This randomized trial does not include blinding and compares different durations of ETI pause to assess its impact. During the study, participants will undergo repeated urine and blood tests to measure urine bicarbonate excretion, ETI plasma levels, acid-base balance, and electrolyte changes at multiple time points including 12, 36, and 60 hours after pausing ETI and after restarting the therapy. The total study duration varies per participant, and safety monitoring will be conducted. This research is sponsored by the University of Aarhus to better understand kidney effects of ETI treatment in CF patients.

Cystic Fibrosis in the Kidney: Monitoring the Effectiveness of Elexacaftor/tezacaftor/ivacaftor in Urine After a Short Pause of Therapy