Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A study to assess safety and tolerability, and explore efficacy of leniolisib for immune dysregulation in common variable immunodeficiency

This research study is investigating an experimental medicine called leniolisib for people who have Common Variable Immunodeficiency (CVID). CVID is a long-term condition that weakens the immune system, making people more prone to infections and other health problems. The main goal of this particular study is to find out if leniolisib is safe to use and if people can tolerate it well. Researchers will closely monitor participants for any unwanted side effects. Beyond safety, the study will also explore whether leniolisib could help with some common issues in CVID, such as swollen lymph nodes or an enlarged spleen. They will also look at whether it might improve lung problems or blood cell counts. This is a Phase 2 study, meaning it's an early step in testing a new medicine to see if it holds promise before larger studies are done.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Pharming Technologies B.V.

Enrolment target

Start

26 May 2025

Common variable immunodeficiency

What is this study about?

This study is exploring a new medicine named leniolisib for people living with Common Variable Immunodeficiency (CVID). CVID is a condition where your immune system doesn't make enough protective antibodies, leading to frequent infections and sometimes other health issues like swollen organs (for example, the spleen or lymph nodes) or lung problems. The main purpose of this study is to carefully check if leniolisib is safe to use and if people can take it without too many unwanted effects. Safety is always the top priority when testing a new medicine.

Besides looking at safety, the researchers also want to see if leniolisib might help improve some of the health problems linked to CVID. For example, they will measure if it can reduce the size of enlarged spleens or swollen lymph nodes. They will also check if it has any positive effects on lung health or on the levels of different types of blood cells.

This is a 'Phase 2' study, which means it's still in the early stages of testing this new medicine. It's designed to get a better understanding of how leniolisib works in the body and if it shows enough potential to be studied further in the future. The findings from this study will help decide if leniolisib could be a useful treatment option for CVID in the long run.

Key takeaways

The study is about a new medicine called leniolisib for CVID.
Its main goal is to check if leniolisib is safe and well-tolerated.
It will also explore if the medicine can help with CVID symptoms like swollen organs or lung issues.
This is an early-stage (Phase 2) study.
Participants must be aged 18 or over.

Who may be eligible?

To be considered for this study, you would need to be 18 years old or older. The study is open to both men and women.

The research team will have a specific list of health requirements that you would need to meet. For example, they might look at your overall health, any other medical conditions you have, and the current treatments you are receiving for CVID.

It's important to understand that not everyone with CVID will be able to join. The researchers need to make sure that the people taking part are suitable for the study and that it would be safe for them.

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.

Are you 18 years old or older?
Have you been diagnosed with Common Variable Immunodeficiency (CVID)?
Are you willing and able to attend regular study appointments?
Are you able to take an experimental medicine?
Are you comfortable with having various medical tests, including blood tests and scans?

Answer every question to see your result.

What does participation involve?

If you join this study, you would be given the experimental medicine, leniolisib. Throughout the study, you would attend regular appointments where the research team will closely monitor your health. These visits will involve various checks, such as measuring your vital signs (like blood pressure and heart rate), taking blood and urine samples for lab tests, and having physical examinations. You might also have scans to measure changes in your spleen or lymph nodes, and lung function tests to check your breathing.

The study team will carefully track any side effects you might experience. The total duration of your participation in the study, including the follow-up, will be explained to you by the research team if you are deemed eligible.

Potential risks and benefits

Participating in this study might offer potential benefits, such as receiving a new, experimental treatment for CVID and contributing to medical knowledge that could help others in the future. However, there are also potential risks, including side effects from the study medicine that are not yet fully known. The research team will explain all known side effects. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Spain

Common questions

What is CVID?

CVID stands for Common Variable Immunodeficiency. It's a long-term health condition where your immune system doesn't make enough antibodies to fight off infections properly.

What is leniolisib?

Leniolisib is an experimental medicine being tested in this study. It's not yet approved for general use, and researchers are studying how safe and effective it is.

What does 'Phase 2 study' mean?

A Phase 2 study is an early stage of research where doctors test a new medicine in a small group of people to see if it's safe and if it shows any promise for treating a condition.

Will I definitely get better if I join?

There's no guarantee that participating in the study will make you feel better. The main goal is to find out if the medicine is safe and if it might help. Some people may not experience any improvement.

What kind of tests will I have?

You might have blood tests, physical exams, vital sign checks, and possibly scans (like ultrasound or CT scans) to check your spleen and lymph nodes, and lung function tests.