Cystic Fibrosis
Conditions
Keywords
trikafta, elexacaftor/tezacaftor/ivacaftor (ETI), ETI, cystic fibrosis
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.
Interventions
None listed
Sponsors
National Jewish Health
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
United States
Outcome results
None listed