A Phase IIb, Multicentre, Randomised, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy and Safety of Dirocaftor/Posenacaftor/Nesolicaftor in Subjects with Cystic Fibrosis Aged 18 Years or Older (CHOICES)
This research study, called CHOICES, is testing new treatments for adults aged 18 and over who have Cystic Fibrosis. The treatments are three medications: Dirocaftor, Posenacaftor, and Nesolicaftor. We want to understand how effective these medications are at improving lung function, which is often a big concern for people with Cystic Fibrosis. We're also checking carefully to make sure they are safe and don't cause too many side effects. Some participants will receive the actual medications, while others will receive a dummy treatment (placebo) to help us compare the results fairly. The study will involve different groups taking the medicines at different times to help us get a clear picture of their effects.
At a glance
What is this study about?
This study, known as Project CHOICES, is for adults aged 18 and older who have Cystic Fibrosis. Cystic Fibrosis is a condition that mainly affects the lungs, making it harder to breathe over time. We are investigating a new combination of medicines: Dirocaftor, Posenacaftor, and Nesolicaftor. These medications are still in the testing phase, meaning they are not yet available to everyone. The main goal of this study is to see if this new combination of drugs can help improve how well people with Cystic Fibrosis breathe.
To do this, we will measure lung function, specifically something called 'forced expiratory volume in 1 second' (that's how much air you can blow out in one second). We'll look at this measure over several weeks. We will also be using a dummy treatment, called a placebo, for some participants. This is important for comparing the effects of the new medicines against not taking the active drugs, helping us understand if any improvements are genuinely due to the study medications.
Beyond just looking at breathing, we'll also be checking other important health markers. This includes measuring sweat chloride, which is often high in people with Cystic Fibrosis, checking body weight, and looking at how participants feel about their symptoms using a special questionnaire. Importantly, safety is a top priority, so we'll be closely monitoring for any unwanted side effects and regularly carrying out health checks like blood tests and heart recordings.
Key takeaways
- This study is testing new medicines for adults with Cystic Fibrosis.
- The aim is to see if the medicines improve breathing and are safe.
- Some participants will receive active medicine, others a dummy treatment (placebo).
- Your health will be closely monitored with regular check-ups and tests.
- Participation is voluntary, and you can withdraw at any time.
- Being 18 or older is a key requirement for joining.
Who may be eligible?
To be considered for this study, you must be 18 years old or older. There is no upper age limit. Both men and women are welcome to take part.
Having Cystic Fibrosis is the main requirement. The study team will check your medical history to make sure the study treatments are suitable and safe for you.
If you are interested, the study team will go through all the details with you and answer any questions to determine if this study is a good match for your health and circumstances.
- Are you 18 years old or older?
- Do you have Cystic Fibrosis?
- Are you open to potentially taking a dummy treatment (placebo) as part of the study?
- Are you able to attend regular clinic appointments for checks and tests?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you join this study, your participation will involve various visits to the study clinic over several weeks. During these visits, you will undergo different health assessments to monitor your well-being and the effects of the study medications. These assessments generally include breathing tests to check your lung function, blood and urine tests, body weight measurements, physical examinations, and heart recordings (ECG). You will also be asked to complete questionnaires about your symptoms and how you are feeling.
The study involves taking either the new medications (Dirocaftor, Posenacaftor, and Nesolicaftor) or a placebo (a dummy treatment). This is done in a 'crossover' way, which means you might receive the actual medication at one point and the placebo at another, depending on which group you are in. You won't know whether you are getting the active medication or the placebo. The total time you'll be participating in the study will be explained in detail by the study team, along with a schedule of all your appointments.
Potential risks and benefits
Locations (9)
- —France
- —Belgium
- —Spain
- —Sweden
- —Czechia
- —Portugal
- —Netherlands
- —Germany
- —Italy
Common questions
What is a 'placebo'?
A placebo is a dummy pill or treatment that looks exactly like the real study medication but contains no active ingredients. It helps us compare the effects of the new drug against not having the active drug.
What does 'double-blind' mean?
Double-blind means that neither you, the participant, nor the study doctors will know whether you are receiving the active study medication or the placebo. This helps ensure the study results are fair and unbiased.
What is lung function and why is it important in this study?
Lung function refers to how well your lungs are working, specifically how much air you can breathe in and out. It's very important in Cystic Fibrosis, so we measure it to see if the new medications help improve breathing.
Will I have to pay to join this study?
No, you will not have to pay to participate in this research study. All study-related medications and appointments will be covered.
What if I experience side effects?
Your safety is a priority. The study team will regularly check for any side effects and will be available to address any concerns you have throughout the study. Don't hesitate to report anything unusual.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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