Simultaneous quanTification of Elexacaftor/tezAcaftor/Ivacaftor Via Reverse Phase High Performance liquiD chromatographY

Study Overview
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Brief Summary
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The purpose of this study is to support the clinical validation of a new assay to measure the levels of ivacaftor, tezacaftor, and elexacaftor (the components of Trikafta) in the bloodstream in order to achieve greater understanding of the effectiveness of this medication in all people with cystic fibrosis. Blood will be drawn at 3.0, 4.5, and 6.0 hours after taking the medication in the morning.

Detailed Description
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Blood samples will be collected to measure steady state blood levels for participants who are being treated with elexacaftor/tezacaftor/ivacaftor (ETI). This study is being conducted to evaluate drug dosing in people with cystic fibrosis. The aim of this study is to support clinical validation of pharmacokinetics launch of the elexacaftor/tezacaftor/ivacaftor blood serum level assay to demonstrate instrument capabilities to pass ADx validation criteria.

Official Title
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Simultaneous quanTification of Elexacaftor/tezAcaftor/Ivacaftor Via Reverse Phase High Performance liquiD chromatographY

Conditions
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Cystic Fibrosis

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Written informed consent obtained from subject Diagnosis of CF Aged 18 years and older Use of ETI that has reached steady state dosing levels of ETI, defined as a continuous dosing regimen for a minimum of 14 days. Exclusion Criteria: A person with CF (pwCF) that is either not taking ETI or dose not follow a continuous dosing regimen for a minimum of 14 days. Use of prohibited medications as outlined by package insert Pregnant or lactating people with CF Acute, severe deterioration in health requiring intensive care unit admission

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| People with Cystic Fibrosis who are taking ETI that has reached a steady state dosing level.<br>Blood samples will be collected from people with CF who are taking ETI at a continuous dosing regimen for a minimum of 14 days. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of patients with ETI in expected reference range | Patient expected reference range determined by initial U.S FDA (2019 approval documentation): Cmax for Elexacaftor, Tezacaftor, Ivacaftor are 8.7± 2.1, 6.8 ± 1.5, 1.2 ± 0.3 mcg/mL, respectively. Limit of quantification estimated to be within +/- 20% of lowest calibration point of 0.2 mcg/mL. | 3 hours, 4.5 hours and 6 hours after taking the medication | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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trikafta, elexacaftor/tezacaftor/ivacaftor (ETI), ETI, cystic fibrosis