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

Sponsor

National Jewish Health (Other)

Overall Status

Enrolling by invitation

CT.gov ID

NCT05822102

Collaborator

(none)

Enrollment

Locations

4.7

Anticipated Duration (Months)

Patients Per Site

3.2

Patients Per Site Per Month

Study Details

Study Description

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 as prescribed.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis

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 Design

Study Type:

Observational

Anticipated Enrollment :

30 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

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

Actual Study Start Date :

Feb 9, 2023

Anticipated Primary Completion Date :

Jul 1, 2023

Anticipated Study Completion Date :

Jul 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
People with Cystic Fibrosis who are taking ETI as prescribed. Blood samples will be collected to measure steady state blood levels for participants who are being treated with ETI and taking as prescribed.

Outcome Measures

Primary Outcome Measures

Patient expected reference range [6 hours]
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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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 not on a full dose of ETI
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