Cystic Fibrosis CFTR Modulator Study

Investigating Cystic Fibrosis Transmembrane Conductance Regulator Modulator Concentrations and Adverse Events in Cystic Fibrosis

Primary Investigator
E
Emma Tillman

Primary Investigator

Cystic Fibrosis CFTR Modulator Study
Recruiting
12 years or above
All
Phase N/A
48 participants needed
1 Location

Brief description of study

We hope to generate data through the variability in medication concentrations in a persons blood. We do not know yet if drug concentrations correlate with side effects. This study will help determine if CFTR modulator blood concentrations correlate with medication side effects and determine the value of proceeding with a large-scale trial.

Detailed description of study

This is an observational study designed to observe persons with Cystic Fibrosis at normal response to CFTR modulator and those currently experiencing an adverse event or side effect (AE) at the time of a 12-hour study visit. 

Study visits will be performed at the University Hospital clinical research center (CRC). Study participants will be consented prior to the visit. Half of participants must be currently experiencing an AE at the time of the study visit.

During the study visit, the following assessments will be done:

  • A pre-dose sample and genetic test blood draw before you are provided with a standardized high-fat meal to take with your medication
  • Regular blood sampling over the course of 12 hours (5 draws)
  • A sweat chloride test will be performed
  • Detailed medical history collected (with help of electronic medical record, if available)
  • Participants will answer questionnaires during the visit

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Cystic Fibrosis, CF, Riley
  • Age: 12 years or above
  • Gender: All

Inclusion Criteria

  • Participants ≥12 years old
  • Currently taking one of the medication below
    • elexacaftor/tezacaftor/ivacaftor (ETI) also known as Trikafta and Kaftrio
    • vanacaftor/tezacaftor/deutivacaftor (VTD) also known as Alyftrek
  • Currently either
    • Experiencing medication related side effects
    • Tolerating their medication with no side effects

Exclusion Criteria

  • Taking certain medications (short or long term) that may interact with your current medications
  • Principal Investigator decision

This study investigates the relationship between CFTR modulator blood concentrations and adverse events in individuals with cystic fibrosis (CF). CFTR modulators are medications that help manage CF, a genetic disorder affecting the lungs and digestive system. The study focuses on understanding if different levels of these medications in the blood are linked to side effects, known as adverse events (AEs).

Participants will undergo a 12-hour observational study visit where various tests will be conducted. These include blood sampling at multiple intervals to measure drug concentrations, a sweat chloride test to assess CF impact, and genetic testing to understand drug metabolism. Participants will also complete questionnaires to evaluate mental health and quality of life, and provide detailed medication histories to identify potential drug interactions.

  • Who can participate: Participants must be 12 years or older, currently taking CFTR modulators, and either tolerating the medication or experiencing adverse events. Those taking medications interacting with CFTR modulators are excluded.
  • Study details: Participants will receive a standardized high-fat meal with their medication and have blood samples taken to measure drug levels. They will also complete mental health and quality of life questionnaires.
  • Study visits: The study requires 1 visit.
Updated on 11 Aug 2026. Study ID: CPHR-IIR-CFTR, 25694

Pre-Screener

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