A Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX‑828/Deutivacaftor With and Without Tezacaftor in Subjects Aged 18 Years and Older With Cystic Fibrosis
This research is a Phase 2 study investigating new drug combinations for adults aged 18 and over who have cystic fibrosis (CF). The main goal is to find out if a new medicine called VX-828/Deutivacaftor, sometimes with another drug called Tezacaftor, is safe to use and whether it helps improve the health of people with CF. Researchers will carefully check for any side effects and measure how the new treatment affects important health markers, such as sweat chloride levels, which are often high in CF. They will also compare these new treatments to existing medications or inactive 'dummy' pills (placebos) to understand their effects. This early stage study is crucial for learning more about potential new ways to treat cystic fibrosis.
At a glance
What is this study about?
This study is trying to find new and better ways to treat cystic fibrosis (CF). CF is a genetic condition that mainly affects the lungs, making it hard to breathe, but it can also impact other parts of the body like the digestive system. Current treatments have come a long way, but researchers are always looking for even more effective options.
In this study, doctors are looking at a new combination of medicines, primarily VX-828 and Deutivacaftor. Sometimes, another medicine called Tezacaftor might also be part of the treatment. The researchers want to understand if these new combinations are safe for people with CF and if they can help improve their symptoms or overall health. They will compare these new drugs to existing treatments or inactive dummy pills (called placebos) to see the difference they make.
The main things they will be checking are how safe the drugs are – by looking for any side effects – and how well they work to reduce the amount of chloride in sweat, which is a common way to measure how CF treatments are working. They will also look at how these drugs affect breathing and how people with CF feel in their daily lives. This is an important step in developing new medicines that could potentially help many people living with cystic fibrosis.
Key takeaways
- This study is testing new drug combinations for adults with cystic fibrosis (CF).
- It aims to find out if the new treatments are safe and help improve health markers like sweat chloride levels and breathing.
- Participants will receive either a new drug, an existing drug, or a placebo, and will be closely monitored.
- The study is 'double-blind', meaning neither you nor the doctors will know which treatment you are getting.
- This is an important step in finding better treatments for CF.
Who may be eligible?
This study is particularly looking for adults aged 18 years and older who have been diagnosed with cystic fibrosis. Both men and women are welcome to take part.
To join, you must meet specific health criteria that the study doctors will check. These criteria are put in place to make sure the study is safe for you and that the results are clear and reliable. For example, your overall health and any other medications you are taking will be carefully reviewed.
It's very important to discuss your full medical history with the study team. They will explain all the requirements in detail and help you understand if this study might be a good fit for you.
- Are you 18 years old or older?
- Have you been diagnosed with cystic fibrosis (CF)?
- Are you able to attend regular clinic visits?
- Are you willing to potentially receive a placebo?
- Are you comfortable having regular health checks like blood tests and ECGs?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part in this study, you will receive either the new study medication (VX-828/Deutivacaftor, possibly with Tezacaftor), another existing CF medication, or a placebo (an inactive dummy pill that looks like the real medicine). You won't know which one you are receiving, nor will your study doctors, until the study is over. This helps ensure the results are fair.
You would need to attend several visits at a clinic or hospital where the research team will monitor your health very closely. These visits will involve various tests, including blood and urine tests, heart checks (ECGs), and measurements of your breathing. You might also be asked to complete questionnaires about how you are feeling and any symptoms you experience. The study will last for a specific period, but the detailed schedule and total duration will be explained by the study team if you are interested in participating.
Potential risks and benefits
Locations (11)
- —Spain
- —Sweden
- —Germany
- —Italy
- —Belgium
- —Czechia
- —Ireland
- —Portugal
- —Netherlands
- —France
- —Denmark
Common questions
What is a Phase 2 study?
A Phase 2 study is an early stage of clinical research where doctors look into the safety of a new medicine and how well it might work, usually in a small group of people. It helps confirm if a drug has potential before larger studies begin.
What is a placebo?
A placebo is a substance that looks exactly like the real medicine but contains no active drug. It's used to compare against the actual treatment to see if the medicine really makes a difference or if the effects are due to other factors.
What is sweat chloride, and why is it measured?
Sweat chloride is a measure of the amount of salt in your sweat. In cystic fibrosis, sweat chloride levels are often higher than normal. Measuring it helps doctors know how well a CF treatment is working to correct the underlying problems of the disease.
Will I know which treatment I'm getting?
No, in this study, neither you nor the doctors will know which treatment you are receiving (this is called 'double-blind'). This helps make the study results as fair and unbiased as possible.
What are 'adverse events'?
Adverse events are any unwanted or unexpected health problems that happen during the study, whether they are related to the study medicine or not. Researchers carefully track all of these to understand the safety of new treatments.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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