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

NCT05822102 | Unknown

• 1 other identifier: HS-3978
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 2

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.

Conditions

Cystic Fibrosis

Keywords

trikafta; elexacaftor/tezacaftor/ivacaftor (ETI); ETI; cystic fibrosis

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (1)

People with Cystic Fibrosis who are taking ETI that has reached a steady state dosing level.

Blood samples will be collected from people with CF who are taking ETI at a continuous dosing regimen for a minimum of 14 days.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Up to 100 people with CF who attend the Adult CF Program of Colorado at National Jewish Health and are actively taking ETI hat has reached steady state dosing levels of ETI (defined as a continuous dosing regimen for a minimum of 14 days) will be recruited and enrolled.

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• National Jewish Health: lead

Study Sites (2)

National Jewish Health

Denver, Colorado, 80206, United States

Location

St. Joseph Hospital

Denver, Colorado, 80206, United States

Location

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Jerry Nick, MD

  National Jewish Health

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 7, 2023
• First Posted: April 20, 2023
• Study Start: February 9, 2023
• Primary Completion: September 1, 2023
• Study Completion: September 1, 2023
• Last Updated: July 24, 2023

Record last verified: 2023-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)