A Phase 2b, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate Nebulized Bacteriophage Treatment in Outpatient Adult Cystic Fibrosis (CF) Subjects with Chronic Pseudomonas aeruginosa (PsA) Pulmonary Infection
This research study is testing a new treatment called BX004 for adults with Cystic Fibrosis (CF). If you have CF, you might often get lung infections caused by a bacteria called Pseudomonas aeruginosa (PsA). This study uses a special mist (nebuliser) to deliver the new treatment directly to the lungs. It's a 'Phase 2b' study, which means researchers are checking if the treatment is safe and if it helps reduce the amount of PsA bacteria in your sputum (phlegm) after 8 weeks. Some participants will receive the actual treatment, and others will receive a placebo (a dummy treatment). We will also look at how it affects lung function and your quality of life. This is to see if BX004 could be a helpful new way to manage these common infections in CF.
At a glance
What is this study about?
This study is designed for adults living with Cystic Fibrosis (CF) who frequently experience lung infections caused by a specific type of bacteria called Pseudomonas aeruginosa (PsA). These infections can make breathing difficult and impact overall health. The research team is very interested in finding new and better ways to control these infections.
The study is testing a new treatment, named BX004, which is given as a mist that you breathe in using a nebuliser. This method delivers the medicine directly to your lungs where the infection is. The main goal is to see if BX004 can reduce the amount of PsA bacteria in your sputum (phlegm) after 8 weeks of treatment. This would suggest that the treatment is effectively tackling the infection.
Beyond simply counting bacteria, the researchers will also be looking at other important aspects. They will check how the treatment affects your lung function, which is often measured by how much air you can breathe out in one second. Your quality of life will also be assessed, using special questionnaires that ask about your symptoms and how you feel in general. Safety is a top priority, so the study will carefully monitor any side effects and changes in your health.
Key takeaways
- This study is testing a new inhaled treatment (BX004) for adults with CF and Pseudomonas aeruginosa lung infections.
- It aims to see if BX004 can reduce bacteria levels in the lungs.
- The study also monitors lung function and quality of life.
- You might receive the active treatment or a dummy treatment (placebo).
- Participation involves 8 weeks of treatment and regular clinic visits.
Who may be eligible?
To be part of this study, you need to be an adult, aged 18 years or older, and have been diagnosed with Cystic Fibrosis.
A key requirement is that you have an ongoing lung infection with a bacteria called Pseudomonas aeruginosa (often shortened to PsA). The research team will do tests to confirm you have this specific infection before you can join.
There might be other health conditions or medicines that mean you can't take part, as these could affect the study results or your safety. The study doctor will review your full medical history to confirm if this study is right for you.
- Are you 18 years old or older?
- Do you have a diagnosis of Cystic Fibrosis?
- Have you recently had a lung infection confirmed to be caused by Pseudomonas aeruginosa bacteria?
- Are you able to use a nebuliser to breathe in medicine?
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, you'll be assigned by chance to receive either the active study treatment (BX004) or a placebo (a dummy treatment that looks the same but contains no active medicine). Neither you nor your study doctor will know which one you are receiving during the study. You will administer the treatment yourself at home using a nebuliser.
The study treatment will last for 8 weeks. During this time, you'll need to attend several clinic visits so the doctors can monitor your health, check your lung function, and collect samples like sputum to see how the bacteria levels are changing. You'll also complete questionnaires about your quality of life. After the 8-week treatment period, there will be follow-up visits to ensure your safety and well-being. The total duration of your involvement in the study, including all follow-up, will be explained fully by the study team.
Potential risks and benefits
Locations (8)
- —Germany
- —Ireland
- —France
- —Czechia
- —Italy
- —Netherlands
- —Poland
- —Spain
Common questions
What is Cystic Fibrosis (CF)?
CF is a genetic condition that mainly affects the lungs, making mucus thick and sticky, which can lead to frequent infections and breathing problems.
What is Pseudomonas aeruginosa (PsA)?
PsA is a common type of bacteria that often causes persistent lung infections in people with Cystic Fibrosis.
What is a 'placebo'?
A placebo is a 'dummy' treatment that looks just like the real medicine but contains no active ingredients. It helps researchers understand if the new treatment actually works better than doing nothing.
Will I know if I'm getting the real medicine or the placebo?
No, this is a 'double-blind' study, meaning neither you nor your doctor will know whether you are receiving the active treatment or the placebo until the study is over.
How long will I be in the study?
The main treatment period is 8 weeks, but there will be additional visits before and after this, so the total time you spend in the study will be longer and will be explained by the study team.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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