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

University of Aarhus

Status: Enrolling

Conditions: Cystic Fibrosis (CF); CFTR Gene Mutation

Treatments: Other: 60 hours ETI pause; Other: 36 hours ETI pause; Other: 12 hours ETI pause

Study type: Interventional

Funder types: Other

Identifiers

NCT05818319

CFPT29092022

Details and patient eligibility

About

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.

Enrollment: 30 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Diagnostic

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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30 participants in 3 patient groups

12 hours ETI pause

Other group

Treatment:

Other: 12 hours ETI pause

36 hours ETI pause

Other group

Treatment:

Other: 36 hours ETI pause

60 hours ETI pause

Other group

Treatment:

Other: 60 hours ETI pause