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

NCT05818319 · tracked via the Priya Life Science Denmark tracker

Sponsor

University of Aarhus

Phase

Not applicable

Started

2023-06-01

Last updated

2025-02-19

Condition(s) studied

Cystic Fibrosis (CF)CFTR Gene Mutation

Investigational drug(s) / intervention(s)

12 hours ETI pause36 hours ETI pause60 hours ETI pause

12 hours ETI pause: Patients with CF are randomly allocated to ETI pause lasting 12 hours.

36 hours ETI pause: Patients with CF are randomly allocated to ETI pause lasting either 36 hours.

60 hours ETI pause: Patients with CF are randomly allocated to ETI pause lasting either 60 hours.

Study 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.

Eligibility

Sex

ALL

Min age

18 Years

Max age

—

Healthy volunteers

Inclusion Criteria: * Adult (age \>17 years) CF patients. * Normal kidney function estimated by eGFR\>90. * Adults capable of understanding and voluntarily consenting. Exclusion Criteria: * Critical acute illness. * Severe lung disease (ppFEV1\<40%). * Adults not capable of understanding and voluntarily consenting.

Primary outcome measure(s)

Difference in cumulative urine bicarbonate excretion before, during, and after ETI pause. — 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. — 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

Trial sites (1)

Facility	City	Region	Status
Department of Infectious Diseases, Aarhus University Hospital	Aarhus C	Central Jutland	Recruiting

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05818319 on ClinicalTrials.gov ↗ ← All trials in Denmark