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

ConditionsCystic Fibrosis (CF)CFTR Gene Mutation
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age18+
SponsorUniversity of Aarhus

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

Adult (age >17 years) CF patients.

Normal kidney function estimated by eGFR>90.

Adults capable of understanding and voluntarily consenting.

Disqualifiers

Critical acute illness.

Severe lung disease (ppFEV1<40%).

Adults not capable of understanding and voluntarily consenting.

Trial design

Design model

Parallel

Treatments tested in this trial

  • 12 hours ETI pause

    Other intervention

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

  • 36 hours ETI pause

    Other intervention

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

  • 60 hours ETI pause

    Other intervention

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

Treatment groups

30 Participants
are divided into 3 treatment groups
Group A: 12 hours ETI pauseOther 1 intervention
Group B: 36 hours ETI pauseOther 1 intervention
Group C: 60 hours ETI pauseOther 1 intervention

Trial outcomes

Primary outcomes

1

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

Challenged urine HCO3- test: Quantification of urine bicarbonate excretion after an acute oral NaHCO3 challenge before, under, and after ETI pause.

Time frame
At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.
2

Link between changes in ETI plasma concentration and changes in urine bicarbonate excretion.

Venous blood sampling: ETI plasma concentration measurement. Challenged urine HCO3- test: Quantification of urine bicarbonate excretion

Time frame
At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.

Secondary outcomes

1

Link between plasma acid-base status and urine acid-base excretion.

Venous blood sampling: Venous acid-base measurements.

Time frame
At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.
2

Changes in plasma concentration of ETI during the trial.

Venous blood sampling: ETI plasma concentration measurement.

Time frame
At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.
3

Changes in acid-base and fluid status during the trial.

Venous blood sampling: Venous acid-base and fluid measurements.

Time frame
At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.
4

Changes in electrolytes during the trial.

Venous blood sampling: Venous electrolyte measurements. Challenged HCO3- urine test: Urine electrolyte measurements.

Time frame
At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

University of Aarhus

Lead sponsor

Source ClinicalTrials.gov
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