Cystic Fibrosis CFTR Modulator Study
Investigating Cystic Fibrosis Transmembrane Conductance Regulator Modulator Concentrations and Adverse Events in Cystic Fibrosis
Emma Tillman
Primary Investigator
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
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Age: 12 years or above
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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.
Pre-Screener
Interested in the study?
Select a study center that’s convenient for you, and get in touch with the study team.
Connect with the Study TeamPlease choose between Voice or SMS based delivery of verification code
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