A seamless Phase I/II trial with an initial open-label dose escalation part and a subsequent randomised, double-blind, placebo-controlled expansion part to evaluate the safety, tolerability, and efficacy of a single dose of BI 3720931, an inhaled lentiviral vector gene therapy, in adult people with cystic fibrosis who are ineligible for CFTR modulators (LenticlairTM 1)
This research study, called LenticlairTM 1, is looking into a new inhaled gene therapy, BI 3720931, for adult patients with cystic fibrosis. It is for people who are not able to take existing CFTR modulator medicines. The study has two main parts. The first part will check for the best dose and how safe the treatment is. The second, larger part will compare the new treatment with a dummy treatment (placebo) to see if it helps improve lung function. Researchers will be carefully monitoring participants for any side effects and changes in their breathing over several weeks. The goal is to find out if this new gene therapy could be a safe and effective option for some people with cystic fibrosis.
At a glance
What is this study about?
This study is looking at a new type of treatment for cystic fibrosis called BI 3720931. It's a gene therapy given through an inhaler, which means you breathe it in. This treatment aims to help people with cystic fibrosis by targeting the underlying genetic problem that causes the condition, rather than just treating the symptoms. This particular study is for adults with cystic fibrosis who are unable to use other specific cystic fibrosis medications known as CFTR modulators.
The study is called a 'seamless Phase I/II trial'. This means it combines two stages of research. In the first stage (Phase I), a small group of participants will receive increasing doses of the treatment. This helps researchers find the safest dose and see how well people tolerate it. In the second stage (Phase II), more participants will join. Some will receive the new gene therapy, and others will receive a dummy treatment (placebo) – neither the participants nor the doctors will know who is getting what until the end of the study. This helps to fairly assess if the new treatment is actually working.
The main goals are twofold: in Phase I, to monitor for any side effects or unwanted reactions to the new treatment for up to 24 weeks; and in Phase II, to see if the treatment improves lung function, specifically how much air you can breathe out in one second (FEV1pp), after 8 weeks. Researchers will also be looking at overall safety, other measures of lung function, and any serious side effects over a longer period.
Key takeaways
- This study is testing a new inhaled gene therapy for cystic fibrosis.
- It's for adults with CF who can't use certain other treatments.
- The study will check for safety, tolerability, and effects on lung function.
- It involves regular clinic visits, tests, and possible exposure to a placebo.
- Participation is voluntary, and you can withdraw at any time.
Who may be eligible?
This study is specifically looking for adult patients who have cystic fibrosis. You need to be at least 18 years old to take part, and there's no upper age limit currently stated.
Critically, this study is designed for adults with cystic fibrosis who are currently *not* able to use or take other specific cystic fibrosis medications called CFTR modulators. Your doctor will be able to tell you if this applies to you.
There may be other health requirements or conditions that would prevent you from joining, but these will be discussed in detail by the study team if you express interest.
- Am I 18 years old or older?
- Do I have a diagnosis of cystic fibrosis?
- Am I currently NOT taking CFTR modulator medicines?
- Am I unable to take CFTR modulator medicines for my cystic fibrosis?
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 have regular visits to the study clinic over several months. You will receive the study treatment, BI 3720931, through an inhaler, or a placebo (dummy treatment) if you are in the second part of the study. You'll likely undergo regular physical exams, blood tests, and lung function tests (like spirometry to measure your FEV1pp). The study also involves a special type of scan using a substance called Gadovist, which helps doctors see inside your body. You will be closely monitored for any side effects or changes in your health throughout the study. The exact number of visits, tests, and total duration will be explained by the study team, but based on the endpoints, your participation will involve follow-up for at least 24 weeks after receiving the treatment.
Potential risks and benefits
Locations (4)
- —Italy
- —Spain
- —Netherlands
- —France
Common questions
What is gene therapy?
Gene therapy is a new type of treatment that aims to correct the genetic problem causing a disease, rather than just treating the symptoms.
What does FEV1pp mean?
FEV1pp is a measure of how much air you can forcefully breathe out of your lungs in one second. It's often used to check lung health in cystic fibrosis.
What is a placebo?
A placebo is a 'dummy' treatment that looks just like the real medicine but contains no active drug. It helps researchers fairly compare the new treatment.
Who cannot take part in this study?
Adults with cystic fibrosis who are currently using or are able to use CFTR modulator medicines are not eligible for this particular study.
Will I know if I'm getting the drug or placebo?
No, this is a 'double-blind' study, meaning neither you nor your study doctor will know whether you are receiving the active treatment or the placebo until the study ends.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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