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

July 6, 2023 updated by: University of Aarhus

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

30

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Denmark
    • Midtjylland
      • Aarhus C, Midtjylland, Denmark, 8000
        • Recruiting
        • Department of Infectious Diseases, Aarhus University Hospital
        • Contact:
          • Amalie Rousing

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Diagnostic
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: 12 hours ETI pause
Other: 12 hours ETI pause
Patients with CF are randomly allocated to ETI pause lasting 12 hours.
Other: 36 hours ETI pause
Other: 36 hours ETI pause
Patients with CF are randomly allocated to ETI pause lasting either 36 hours.
Other: 60 hours ETI pause
Other: 60 hours ETI pause
Patients with CF are randomly allocated to ETI pause lasting either 60 hours.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Challenged urine HCO3- test: Quantification of urine bicarbonate excretion after an acute oral NaHCO3 challenge before, under, and after ETI pause.
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.
Venous blood sampling: ETI plasma concentration measurement. Challenged urine HCO3- test: Quantification of urine bicarbonate excretion
At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Venous blood sampling: Venous acid-base measurements.
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.
Venous blood sampling: ETI plasma concentration measurement.
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.
Venous blood sampling: Venous acid-base and fluid measurements.
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.
Venous blood sampling: Venous electrolyte measurements. Challenged HCO3- urine test: Urine electrolyte measurements.
At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Aarhus

Investigators

  • Principal Investigator: Jens G. Leipziger, Department of Biomedicine, Aarhus University, Denmark
  • Principal Investigator: Majbritt Jeppesen, Department of infectious diseases, Aarhus University Hospital, Denmark

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 1, 2023

Primary Completion (Estimated)

December 1, 2023

Study Completion (Estimated)

December 1, 2023

Study Registration Dates

First Submitted

March 28, 2023

First Submitted That Met QC Criteria

April 17, 2023

First Posted (Actual)

April 18, 2023

Study Record Updates

Last Update Posted (Actual)

July 10, 2023

Last Update Submitted That Met QC Criteria

July 6, 2023

Last Verified

February 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CFPT29092022

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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