Cystic Fibrosis (CF)NCT05818319
Cystic Fibrosis in the Kidney: Monitoring the Effectiveness of Elexacaftor/tezacaftor/ivacaftor in Urine After a Short Pause of Therapy
A study testing 12 hours ETI pause, 36 hours ETI pause and 60 hours ETI pause in people with Cystic Fibrosis (CF) and CFTR Gene Mutation, run by University of Aarhus at 1 site in Denmark.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis (CF), CFTR Gene Mutation
- What is being tested?
- 12 hours ETI pause, 36 hours ETI pause and 60 hours ETI pause
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteDenmark
- Study dates
- 1 Jun 2023to Dec 2025
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult (age >17 years) CF patients.
- Normal kidney function estimated by eGFR>90.
- Adults capable of understanding and voluntarily consenting.
Who cannot join
- Critical acute illness.
- Severe lung disease (ppFEV1<40%).
- Adults not capable of understanding and voluntarily consenting.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Amalie Q. Rousing, BM contact
Denmark1 site, 1 recruiting
- Recruiting
Department of Infectious Diseases, Aarhus University Hospital
Aarhus C, Central Jutland, 8000
Amalie Rousing
About this study
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
- Being tested
- 12 hours ETI pause, 36 hours ETI pause, 60 hours ETI pause
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
12 hours ETI pauseOther
Patients with CF are randomly allocated to ETI pause lasting 12 hours.
36 hours ETI pauseOther
Patients with CF are randomly allocated to ETI pause lasting either 36 hours.
60 hours ETI pauseOther
Patients with CF are randomly allocated to ETI pause lasting either 60 hours.
| Study group | Role | What they receive |
|---|---|---|
| 12 hours ETI pause | Other | Not described Receives: Other: 12 hours ETI pause |
| 36 hours ETI pause | Other | Not described Receives: Other: 36 hours ETI pause |
| 60 hours ETI pause | Other | Not described Receives: Other: 60 hours ETI pause |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Venous blood sampling: ETI plasma concentration measurement. Challenged urine HCO3- test: Quantification of urine bicarbonate excretion
Measured: At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.
Secondary outcomes (4)
Link between plasma acid-base status and urine acid-base excretion.
Venous blood sampling: Venous acid-base measurements.
Measured: At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.
Changes in plasma concentration of ETI during the trial.
Venous blood sampling: ETI plasma concentration measurement.
Measured: 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.
Venous blood sampling: Venous acid-base and fluid measurements.
Measured: At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.
Changes in electrolytes during the trial.
Venous blood sampling: Venous electrolyte measurements. Challenged HCO3- urine test: Urine electrolyte measurements.
Measured: At baseline, after 12/36/60 hours of therapy pause and after therapy is resumed.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Apr 2023
- Study start1 Jun 2023
- Primary completionDec 2025 (estimated)
- Study completionDec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Cystic Fibrosis (CF)Studies of the same condition, open and under way.
- More trials run by University of AarhusThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- CF patients will perform a challenged urine bicarbonate test three times during this trial; 1) before, 2) during, and 3) after ETI therapy pause. Each test performance takes 90 min. and is accompanied by baseline blood sampling. First, a baseline urine sample is collected. Then the test person ingests 79 mg. NaHCO3/kg body weight dissolved in tap water (2,25 mL/kg body weight) together with the same amount of clean tap water. After 90 min. the test person delivers the second urine sample, and the test is completed. The test should preferably be performed between breakfast and lunch, at least one hour after intake of food. There should be at least three days between each test. CF patients will be randomly allocated to an ETI treatment pause for either 12, 36, or 60 hours. Treatment will be resumed immediately after the pause is finished.
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Aarhus
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jens G. Leipziger, Principal investigator, Department of Biomedicine, Aarhus University, DenmarkMajbritt Jeppesen, Principal investigator, Department of Infectious Diseases, Aarhus University Hospital, Denmark
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05818319
- Sponsor's protocol ID
- CFPT29092022
- Status last verified
- Feb 2025
- First submitted
- 28 Mar 2023
- Last updated
- 19 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
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.
PubMed 32703846Berg 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.
PubMed 36315944
Study at a glance
From the registry record, last updated by the sponsor on 19 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cystic Fibrosis in the Kidney: Monitoring the Effectiveness of Elexacaftor/tezacaftor/ivacaftor in Urine After a Short Pause of Therapy
- ClinicalTrials.gov ID
- NCT05818319
- Acronym / sponsor ID
- CFPT29092022
- Conditions
- Cystic Fibrosis (CF), CFTR Gene Mutation
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- University of AarhusUniversity, hospital or other
- Study start
- 1 Jun 2023 (actual)
- Primary completion
- Dec 2025 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 18 Apr 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Diagnosis
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- CF patients will perform a challenged urine bicarbonate test three times during this trial; 1) before, 2) during, and 3) after ETI therapy pause. Each test performance takes 90 min. and is accompanied by baseline blood sampling. First, a baseline urine sample is collected. Then the test person ingests 79 mg. NaHCO3/kg body weight dissolved in tap water (2,25 mL/kg body weight) together with the same amount of clean tap water. After 90 min. the test person delivers the second urine sample, and the test is completed. The test should preferably be performed between breakfast and lunch, at least one hour after intake of food. There should be at least three days between each test. CF patients will be randomly allocated to an ETI treatment pause for either 12, 36, or 60 hours. Treatment will be resumed immediately after the pause is finished.
| Arm | Type | Receives |
|---|---|---|
| 12 hours ETI pause | Other | Other: 12 hours ETI pause |
| 36 hours ETI pause | Other | Other: 36 hours ETI pause |
| 60 hours ETI pause | Other | Other: 60 hours ETI pause |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Adult (age >17 years) CF patients.
- Normal kidney function estimated by eGFR>90.
- Adults capable of understanding and voluntarily consenting.
Key exclusion criteria (3 listed)
- Critical acute illness.
- Severe lung disease (ppFEV1<40%).
- Adults not capable of understanding and voluntarily consenting.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 12 hours ETI pausePatients with CF are randomly allocated to ETI pause lasting 12 hours. | Other | 12 hours ETI pause |
| 36 hours ETI pausePatients with CF are randomly allocated to ETI pause lasting either 36 hours. | Other | 36 hours ETI pause |
| 60 hours ETI pausePatients with CF are randomly allocated to ETI pause lasting either 60 hours. | Other | 60 hours ETI pause |
Primary outcome
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.
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
4 secondary outcomes
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Feb 2025.
- Background: PubMed 32703846Berg 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.
- Background: PubMed 36315944Berg 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Amalie Q. Rousing, BM contact
Jens G. Leipziger principal investigator
Department of Biomedicine, Aarhus University, Denmark
Majbritt Jeppesen principal investigator
Department of Infectious Diseases, Aarhus University Hospital, Denmark
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing 12 hours ETI pause, 36 hours ETI pause and 60 hours ETI pause in people with Cystic Fibrosis (CF) and CFTR Gene Mutation, run by University of Aarhus at 1 site in Denmark.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 19 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Amalie Q. Rousing, BM (arousing@biomed.au.dk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05818319, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Feb 2025.