Active not recruitingPHASE2INTERVENTIONAL

Leniolisib for Immune Dysregulation in CVID

This study is investigating a new medication called leniolisib for people with Common Variable Immunodeficiency (CVID). CVID is a condition where the immune system doesn't work properly. The study will involve about 20 patients who will take leniolisib tablets twice a day for six months. They'll start with a low dose, then move to a medium and finally a higher dose. The main goal is to check how safe leniolisib is and if people can tolerate it well. Researchers will also look to see if the medicine can help reduce some of the immune-related problems that CVID patients experience, such as swollen spleen or lymph glands, or issues with blood cells. This is a Phase 2 study, meaning it's still early research into this treatment.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Pharming Technologies B.V.

Enrolment target

Start

12 Feb 2025

Estimated completion

01 Oct 2026

Common Variable Immunodeficiency (CVID)

What is this study about?

This study is designed to explore a new potential treatment, called leniolisib, for individuals living with Common Variable Immunodeficiency (CVID). CVID is a long-term health condition where the immune system, which usually fights off infections, doesn't produce enough protective proteins (antibodies). This can lead to frequent infections and other immune problems, such as swollen glands or issues with blood cells.

The main purpose of this study is to understand if leniolisib is safe for people with CVID and how well their bodies cope with taking the drug. The research team will also be carefully watching to see if leniolisib might help improve some of the immune-related issues linked to CVID. This is an important step in finding new ways to manage the condition and improve quality of life for patients.

About 20 CVID patients will take part in this study. Everyone involved will receive leniolisib, starting with a lower dose and gradually increasing it over 24 weeks (about 6 months). The study includes people with CVID, whether or not a specific genetic cause for their condition has been found. After the treatment period, participants will have a follow-up visit about a month later.

Key takeaways

This study is testing a new medicine, leniolisib, for CVID.
It aims to check the safety and potential benefits of the drug.
About 20 CVID patients will take part, with initial UK participants being 18-75 years old.
The treatment involves taking tablets twice daily for 6 months, with increasing doses.
All participants will receive the active study drug.
It's a Phase 2 study, meaning it's still early research into this new treatment.

Who may be eligible?

To join this study, people generally need to be between 12 and 75 years old. However, if you are in the UK, you must be between 18 and 75 years old. You also need to weigh at least 45 kilograms (about 7 stone 1 pound). Importantly, you must have an official diagnosis of CVID, with your doctor confirming low levels of certain protective proteins (antibodies) in your blood.

Additionally, your CVID symptoms should include lymphoproliferation, which means you have an enlarged spleen (over 10 cm) or swollen lymph glands (over 1.5 cm). You also need to be experiencing at least one other immune-related problem due to your CVID, such as symptoms from your enlarged spleen or lymph glands that affect your daily life, or specific issues with your blood cells.

Finally, for your CVID, either a specific genetic cause hasn't been found, or if one has been found, it must be from a list of particular genes that the researchers are studying. The research team will review all your medical information to confirm if you meet all the requirements for the study.

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 between 12 and 75 years old (18-75 in the UK)?
Do you weigh at least 45 kilograms?
Have you been officially diagnosed with CVID and have low antibody levels?
Do you have an enlarged spleen or swollen lymph nodes?
Are you experiencing other immune-related problems due to your CVID?
Have you discussed your CVID diagnosis and any known genetic causes with your doctor?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you will receive leniolisib tablets to take twice a day. The treatment plan starts with a lower dose (10mg twice daily) for the first four weeks. Then, the dose will increase to a medium level (30mg twice daily) for another four weeks. Finally, you will take a higher dose (70mg twice daily) for 16 more weeks. This means you will be taking the study drug for a total of 24 weeks (about 6 months).

Throughout the study, you will have regular visits with the study team. These visits will involve check-ups, blood tests, and other assessments to monitor your health, how you are responding to the medication, and to check for any side effects. After you finish taking the leniolisib, there will be one final follow-up visit about 28 days later to see how you are doing. The doctors involved can adjust your dose if they think it's necessary for your well-being.

Potential risks and benefits

Taking part in a clinical trial means you might be among the first to receive a new treatment for CVID, which could potentially help manage some of your immune problems. However, like all medicines, leniolisib may have side effects, which the study team will monitor very closely. It's important to remember that this is a research study, and there's no guarantee the treatment will be effective for everyone. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (7)

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

Children's Hospital Colorado
Verified postcode
Aurora, United States
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
Texas Children's Hospital
Verified postcode
Houston, United States
IIS La Fe
Verified postcode
Valencia, Spain
Leeds Teaching Hospital NHS Trust
Verified postcode
Leeds, United Kingdom

Common questions

What is CVID?

CVID (Common Variable Immunodeficiency) is a condition where your immune system doesn't make enough antibodies, making it harder to fight off infections.

What is leniolisib?

Leniolisib is the study drug being tested. It's a new medicine that aims to help with the immune problems in CVID.

How long will the treatment last?

You will take the study drug for about 6 months, followed by a final check-up about a month later.

Will I get a placebo (dummy pill)?

No, in this study, all participants will receive the active study drug, leniolisib.

Can I leave the study at any time?

Yes, you can choose to stop participating in the study at any point, and it won't affect your ongoing medical care.