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

• Conditions: Cystic Fibrosis

• Registration Number: NCT05822102
• Lead Sponsor: National Jewish Health

Brief Summary

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

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.

Study Timeline

• Study Start: 2023-02-09
• Study First Submitted: 2023-04-07
• Study First Submitted QC: 2023-04-07
• Study First Posted: 2023-04-20
• Status Verified: 2023-07
• Last Update Submitted: 2023-07-21
• Last Update Posted: 2023-07-24
• Primary Completion: 2023-09
• Study Completion: 2023-09

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 100

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of patients with ETI in expected reference range	3 hours, 4.5 hours and 6 hours after taking the medication	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.

Trial Locations

Locations (2)

National Jewish Health; 🇺🇸 Denver, Colorado, United States

St. Joseph Hospital; 🇺🇸 Denver, Colorado, United States