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

NCT ID: NCT05818319

Last Updated: 2025-02-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

NA

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-06-01

Study Completion Date

2025-12-31

Brief Summary

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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.

Detailed Description

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Conditions

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Cystic Fibrosis (CF) CFTR Gene Mutation

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

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.
Primary Study Purpose

DIAGNOSTIC

Blinding Strategy

NONE

Study Groups

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12 hours ETI pause

Group Type OTHER

12 hours ETI pause

Intervention Type OTHER

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

36 hours ETI pause

Group Type OTHER

36 hours ETI pause

Intervention Type OTHER

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

60 hours ETI pause

Group Type OTHER

60 hours ETI pause

Intervention Type OTHER

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

Interventions

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12 hours ETI pause

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

Intervention Type OTHER

36 hours ETI pause

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

Intervention Type OTHER

60 hours ETI pause

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

Intervention Type OTHER

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University of Aarhus

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Jens G. Leipziger

Role: PRINCIPAL_INVESTIGATOR

Department of Biomedicine, Aarhus University, Denmark

Majbritt Jeppesen

Role: PRINCIPAL_INVESTIGATOR

Department of Infectious Diseases, Aarhus University Hospital, Denmark

Locations

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Department of Infectious Diseases, Aarhus University Hospital

Aarhus C, Central Jutland, Denmark

Site Status RECRUITING

Countries

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Denmark

Central Contacts

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Amalie Q. Rousing, BM

Role: CONTACT

Facility Contacts

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Amalie Rousing

Role: primary

References

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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.

Reference Type BACKGROUND
PMID: 32703846 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 36315944 (View on PubMed)

Other Identifiers

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CFPT29092022

Identifier Type: -

Identifier Source: org_study_id

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