A Randomised, Double-Blind, Placebo Controlled, Two-Part Study to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of a Repeat Dose of Inhaled ETD001 in People with Cystic Fibrosis
This research is looking into a new inhaled medication called ETD001 for adults with cystic fibrosis. The study is split into two parts: one to mainly check safety and how the body processes the medication, and another to see how well it helps with breathing, compared to a dummy treatment (placebo). Participants won't know if they're receiving the active medicine or the placebo. Researchers will compare changes in lung function, other health markers, and any side effects to understand if ETD001 is a promising new treatment option. The goal is to find out if it's both safe and helpful for improving lung health.
At a glance
What is this study about?
This study is about a new inhaled medicine called ETD001, designed for adults who have cystic fibrosis. Cystic fibrosis is a condition that mainly affects the lungs, making it hard to breathe over time. Researchers are hoping this new medicine could help manage the condition better.
This study is a special type called a ‘Phase II’ trial. This means it's still relatively early in the development of the medicine. The main goals are to carefully check if the medicine is safe to use and to get an idea of how well it might work. They will also look at how your body handles the medicine once you breathe it in.
To do this, the study is set up in a particular way. Some people will receive the actual medicine, ETD001, while others will receive a 'placebo' – this looks exactly like the medicine but doesn't contain any active ingredients. This helps researchers compare the effects fairly. Participants won't know if they're getting the medicine or the placebo, and neither will their doctor during the study. This helps ensure the results are as accurate as possible.
Key takeaways
- This study is testing a new inhaled medicine (ETD001) for cystic fibrosis.
- It aims to check both safety and how well the medicine works on people's lungs.
- Some participants will get the study medicine, others a dummy treatment (placebo).
- Regular health checks, including breathing tests and blood tests, will be part of the study.
- Participation is for adults aged 18 and over.
- You can leave the study at any time without affecting your normal care.
Who may be eligible?
To join this study, you need to be an adult, aged 18 or older. The study is open to both men and women.
There will be other health checks to make sure the study is a good fit for you. For example, the study team will need to review your overall health, any other medications you take, and your specific cystic fibrosis symptoms to confirm you meet all the requirements for safe participation.
It's important that you discuss your interest with your doctor, as they know your medical history best and can help determine if this type of study is suitable for you.
- Are you 18 years old or older?
- Do you have a diagnosis of cystic fibrosis?
- Are you able to attend regular clinic appointments for checks?
- Are you happy for your health to be monitored closely during the study?
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, your involvement will be split into two main sections. For both parts, you will receive either the study medicine (ETD001) or a placebo, which looks identical but contains no active drug. You won't know which you are receiving, and neither will your study doctor.
During your time in the study, you'll have several visits to the clinic. At these visits, doctors and nurses will closely monitor your health. This will include checks like measuring your breathing (spirometry), taking blood tests, checking your heart with an ECG, and general physical examinations. They will also ask you questions about how you are feeling and any changes you notice. These tests help the researchers understand how the medicine is working and if there are any side effects. The total duration of your participation, including follow-up appointments, will be clearly explained by the study team.
Potential risks and benefits
Locations (3)
- —Germany
- —France
- —Italy
Common questions
What is cystic fibrosis?
Cystic fibrosis is a genetic condition that mainly affects the lungs, making mucus thick and sticky, which can lead to breathing problems and infections.
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 compare effects fairly.
Will I know if I'm getting the real medicine?
No, this is a 'blinded' study, meaning neither you nor your doctors will know if you're taking the active medicine or the placebo until after the study is complete.
What is an 'inhaled' medicine?
An inhaled medicine is taken by breathing it in, often through a device directly into your lungs.
What does a 'Phase II' study mean?
A Phase II study is an early stage of testing a new medicine that mainly looks at whether it's safe and starts to get an idea of how well it works.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.