NCT05818319

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

• Status: Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: University of Aarhus · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Cystic Fibrosis (CF)
• CFTR Gene Mutation

Interventions

Type	Name	Description
OTHER	12 hours ETI pause	Patients with CF are randomly allocated to ETI pause lasting 12 hours.
OTHER	36 hours ETI pause	Patients with CF are randomly allocated to ETI pause lasting either 36 hours.
OTHER	60 hours ETI pause	Patients with CF are randomly allocated to ETI pause lasting either 60 hours.

Timeline

• Start date: 2023-06-01
• Primary completion: 2025-12-01
• Completion: 2025-12-01
• First posted: 2023-04-18
• Last updated: 2025-02-19

Locations

1 site across 1 country: Denmark