Active not recruitingPHASE2INTERVENTIONAL

Long-term Safety and Efficacy of Leniolisib in PIDs With Immune Dysregulation

This research is looking at a medicine called leniolisib for people with certain immune system problems, known as Primary Immunodeficiencies (PIDs), specifically those linked to a pathway called PI3K in their cells. This study is for people who have already taken part in an earlier study of leniolisib. It's designed to see how safe the medicine is over a longer period and whether it continues to help with their condition. Researchers will also be looking at how the medicine affects the immune system in various ways. The study welcomes both males and females aged between 12 and 75 years old who have previously used leniolisib in the earlier trial.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Pharming Technologies B.V.

Enrolment target

Start

29 Apr 2025

Estimated completion

12 Aug 2029

PIDs Linked to PI3K

What is this study about?

This study is focused on a group of health conditions called Primary Immunodeficiencies (PIDs). These are rare conditions where a part of the body's immune system doesn't work properly, making people more likely to get infections or have other immune-related problems. In this particular study, they are looking at PIDs that are connected to a specific pathway inside immune cells, often referred to as the PI3K pathway.

The research is testing a medicine called leniolisib. This isn't a brand-new medicine for everyone in this study; it's an extension of a previous trial. This means that people taking part have already received leniolisib in an earlier study. The main aim now is to understand how safe leniolisib is when used over a longer time, and if it continues to be helpful for people with these specific immune conditions.

Researchers will be carefully checking how the body reacts to the medicine and if it helps to manage the symptoms of the PIDs. This kind of research is important because it helps doctors and scientists learn more about new treatments, potentially improving the lives of people living with these challenging immune conditions.

Key takeaways

This study is for people who have already taken part in a previous leniolisib trial for specific immune conditions (PIDs).
Its main goal is to check the long-term safety and effectiveness of leniolisib.
Participants will continue to receive leniolisib and have regular check-ups.
Both males and females aged 12 to 75 can participate, provided they meet other criteria.
You can stop participating at any time if you wish.

Who may be eligible?

This study is specifically for people who have already taken part in a previous study of leniolisib, called LE 7201. Your doctor will need to agree that continuing to take leniolisib would be beneficial for you.

To be included, you must be able to understand what the study involves and agree to take part. If you're under 18, a parent or legal guardian will need to give permission for you. The study is open to both males and females aged between 12 and 75 years old.

There are also some reasons why you might not be able to join. For example, if you've had a bone marrow transplant, or if you're currently taking certain other medications that could interfere with the study drug, like strong immunosuppressants (medicines that reduce the activity of the immune system) or some other experimental treatments. If you have a known allergy to leniolisib or similar drugs, you also won't be able to participate. Your doctor will carefully review all your medications and medical history to see if this study is right for you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Did you take part in the previous LE 7201 leniolisib study?
Does your doctor think continuing leniolisib would be good for you?
Are you between 12 and 75 years old?
Are you able to understand the study and give your permission to take part (or have a guardian do so if you're under 18)?
Have you discussed all your current medications with your doctor to make sure none would stop you from taking part?
Have you *not* had a bone marrow transplant?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll be continuing to receive the medicine called leniolisib, which you started in the previous trial. The exact details of your visits and how often you'll see the study team will be explained to you fully by the researchers. These visits are important so that the doctors can monitor how you're responding to the medicine, check for any side effects, and generally look after your health.

During these visits, you can expect to have check-ups, which might include blood tests, physical examinations, and possibly other tests to see how your immune system is doing. You'll be asked about any symptoms you're experiencing and how you're feeling overall. The study team will make sure you understand how to take your medicine correctly and will be there to answer any questions you have. The total length of your participation will depend on the study plan and will be discussed with you.

Potential risks and benefits

Taking part in any medical study has potential benefits and potential risks. The potential benefit of continuing with leniolisib is that it might help manage your specific immune condition (PID) over a longer period, potentially improving your health and quality of life. However, there's no guarantee it will work for everyone, and it might not provide the benefits you hope for. Potential risks could include side effects from the medicine, which will be carefully monitored by the study team. These might be similar to any side effects you experienced in the previous study. You'll be given detailed information about these. It's very important to remember that joining this study is completely voluntary, and you have the right to withdraw at any time, for any reason, without it affecting your usual medical care.

Locations (5)

National Institute of Health
Verified postcode
Bethesda, United States
Lahey Hospital & Medical Center
Verified postcode
Burlington, United States
Mount Sinai Hospital
Verified postcode
New York, United States
IIS La Fe
Verified postcode
Valencia, Spain
St James Hospital
Verified postcode
Leeds, United Kingdom

Common questions

What is leniolisib?

Leniolisib is a medicine being tested for certain immune conditions (Primary Immunodeficiencies) that are linked to a specific problem with immune cells.

Why is this study needed if I've already taken leniolisib?

This study helps researchers understand the long-term safety and effects of leniolisib. It allows them to gather more information on how it works over a longer time.

Will I get paid to take part?

The study information doesn't mention payment. This is a good question to ask the study team directly.

What if I experience side effects?

The study team will monitor you closely for any side effects. You should always tell them about anything new or unusual you experience.

Can I leave the study at any time?

Yes, taking part is voluntary. You can decide to leave the study at any time, for any reason, without it affecting your other medical care.