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Study of elexacaftor, tezacaftor and ivacaftor combination therapy to evaluate blood levels in patients with Cystic Fibrosis

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on patients with Cystic Fibrosis, a genetic condition that affects multiple organs, particularly the lungs and digestive system. The study will evaluate how the body processes a combination of three medications: elexacaftor, tezacaftor, and ivacaftor, which are taken as tablets by mouth. These medications work together to help improve the function of a defective protein that causes Cystic Fibrosis.

The purpose of this study is to measure the levels of these medications in the blood of patients who are already taking this drug combination. The medications are available as Kaftrio (which contains all three drugs) and Kalydeco (which contains ivacaftor alone). The study will look at how these medications circulate in the body after patients have been taking them regularly.

During the study, participants will continue their regular treatment with these medications, and blood samples will be collected to measure drug levels. The study will also track how well patients respond to the treatment and monitor any side effects that may occur. This research will help better understand how these medications work in people with Cystic Fibrosis.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial medication phase
You will receive Kaftrio (containing elexacaftor, tezacaftor, and ivacaftor) and Kalydeco (containing ivacaftor) tablets for oral use
The medication must be taken for at least 8 days to reach the required steady state in your body
Step 2
Blood sampling
Blood samples will be collected to measure the levels of medications in your blood
The samples will help determine how the medications are processed by your body
Step 3
Health monitoring
Your lung function will be measured using a test called ppFEV1 (a breathing test that shows how well your lungs are working)
Your chloride levels will be checked
Any side effects you experience will be recorded
Step 4
Response evaluation
Your response to the medication will be evaluated based on the test results
You will be classified into one of three groups: responders, non-responders, or unresolved responders
The relationship between medication levels in your blood and how well the treatment works will be analyzed
Step 5
Study duration
The study period runs from August 2025 to July 2027
Your individual participation time will be determined by your healthcare provider

Who can join the trial?

5 criteria

You must have Cystic Fibrosis with at least one F508del mutation (a specific genetic change associated with Cystic Fibrosis)
You must be currently receiving treatment with ETI combination therapy (a combination of medications: elexacaftor, tezacaftor, and ivacaftor) for at least 8 days to ensure stable levels in your body
You can be either male or female
You must be 6 years of age or older
You must be able to provide informed consent, which means you understand and agree to participate in the study and allow the processing of your personal data. For children, this consent must be given by a parent or legal guardian

Who cannot join the trial?

13 criteria

History of organ or hematopoietic stem cell transplant (transplant of blood-forming cells)
Moderate or severe liver problems
Abnormal liver function tests showing values more than 3 times the normal upper limit
Abnormal kidney function with creatinine clearance below 50 mL/min (a measure of how well kidneys filter blood)
History of alcohol or drug abuse within the past year
Use of strong CYP3A inhibitors (medications that can affect how other drugs are processed in the body)
Pregnant or nursing mothers
Participation in another clinical trial within 28 days before this study
Known allergic reactions to the study medications or their components
Any medical condition that could interfere with study procedures or participant safety
Unable to swallow tablets
Mental health conditions that could affect ability to follow study procedures
Acute illness within 14 days before the first study visit

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Verified sites

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Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Milan, Italy
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Istituto Di Ricovero E Cura A Carattere Scientifico Materno Infantile Burlo Garofolo
Trieste, Italy

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Investigated drugs

Elexacaftor is a medication used to treat cystic fibrosis. It works by helping abnormal proteins function better in people with specific genetic mutations that cause cystic fibrosis. This medication helps improve lung function and breathing. Tezacaftor is another medication used in treating cystic fibrosis. It works together with other medications to help the proteins in the body function more normally. This helps thin the mucus in the lungs of people with cystic fibrosis. Ivacaftor is a medication that helps improve the function of a specific protein in people with cystic fibrosis. It helps improve breathing and lung function by helping to maintain proper salt and water balance in the lungs. These three medications are used together as a combination therapy to provide better treatment outcomes for people with cystic fibrosis. The combination helps improve lung function and breathing more effectively than using any of these medications alone.

What is already known about the treatment

Elexacaftor/Tezacaftor/Ivacaftor – A combination medication administered orally for the treatment of Cystic Fibrosis, specifically designed to improve lung function in patients with certain genetic mutations. This drug combination works by helping defective CFTR proteins (which regulate salt and water balance in the body) function more effectively, leading to better mucus clearance in the airways. As a CFTR modulator therapy, it represents one of the most advanced treatments available for Cystic Fibrosis, and clinical studies have shown significant improvements in patients' respiratory function and quality of life.

Investigated diseases

Cystic Fibrosis - A genetic disorder that affects cells producing mucus, sweat, and digestive fluids. The condition causes these fluids to become thick and sticky instead of thin and slippery. This buildup of thick, sticky secretions can clog various tubes and ducts throughout the body, particularly in the lungs and pancreas. The disease primarily affects the respiratory and digestive systems, causing recurring lung infections and difficulty absorbing nutrients from food. People with cystic fibrosis inherit a defective gene from both parents that affects a protein regulating salt and water movement in cells.