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Recruiting NCT05818319

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

No phase Interventional Cystic Fibrosis (CF) CFTR Gene Mutation

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: 12 hours ETI pause, 36 hours ETI pause, 60 hours ETI pause.
Who it may be relevant to
Registry conditions: Cystic Fibrosis (CF), CFTR Gene Mutation. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Denmark
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Overview

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.

Interventions

  • 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.

Primary outcome measures

  • Difference in cumulative urine bicarbonate excretion before, during, and after ETI pause. [Time frame: At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.]
  • Link between changes in ETI plasma concentration and changes in urine bicarbonate excretion. [Time frame: At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.]
Secondary outcome measures (4)
  • Link between plasma acid-base status and urine acid-base excretion. [Time frame: At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.]
  • Changes in plasma concentration of ETI during the trial. [Time frame: 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. [Time frame: At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.]
  • Changes in electrolytes during the trial. [Time frame: At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Open label
Primary purpose
Diagnostic

Study locations

Denmark · 1 center
  • Department of Infectious Diseases, Aarhus University Hospital — Aarhus C

Publications

  • Berg P, Svendsen SL, Sorensen MV, Larsen CK, Andersen JF, Jensen-Fangel S, Jeppesen M, Schreiber R, Cabrita I, Kunzelmann K, Leipziger J. Impaired Renal HCO3- Excretion in Cystic Fibrosis. J Am Soc Nephrol. 2020 Aug;31(8):1711-1727. doi: 10.1681/ASN.2020010053. Epub 2020 Jul 23. PMID 32703846
  • Berg P, Sorensen MV, Rousing AQ, Vebert Olesen H, Jensen-Fangel S, Jeppesen M, Leipziger J. Challenged Urine Bicarbonate Excretion as a Measure of Cystic Fibrosis Transmembrane Conductance Regulator Function in Cystic Fibrosis. Ann Intern Med. 2022 Nov;175(11):1543-1551. doi: 10.7326/M22-1741. Epub 2022 Nov 1. PMID 36315944

Identifiers

NCT: NCT05818319 · CFPT29092022

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗