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A Study of Leniolisib Safety and Effectiveness for Immune System Problems in Patients with Common Variable Immunodeficiency

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What is this study about?

This study involves people with common variable immunodeficiency, which is a condition where the body’s immune system does not work properly and cannot produce enough antibodies to fight infections. People with this condition may also develop problems where the immune system becomes overactive in certain ways, leading to enlarged organs like the spleen, swollen lymph nodes, low blood cell counts, lung problems, or digestive system issues. The treatment being tested is leniolisib, which comes as a film coated tablet that is taken by mouth. Different doses of this medicine will be used in the study to find the right amount for each person.

The purpose of this study is to check if leniolisib is safe and well tolerated in people with this immune condition, and to see if it might help with the various problems caused by the overactive immune system. During the study, participants will receive leniolisib tablets for a period that can last up to several months. The study will monitor how the medicine affects the size of the spleen and lymph nodes, blood cell counts, lung function, and other signs of immune system problems. Regular check-ups will include physical examinations, blood tests to measure different types of immune cells and substances in the blood, imaging scans to look at organs and lymph nodes, and tests to check lung function if needed.

Throughout the study, doctors will carefully watch for any side effects or unwanted reactions to the medicine by checking vital signs like blood pressure and heart rate, doing laboratory tests, and asking about any symptoms or health changes. The study will also measure specific markers in the blood that show how active the immune system is, and will track changes in different types of white blood cells over time. Some participants may have additional tests depending on their specific symptoms, such as breathing tests for those with lung involvement or endoscopy procedures for those with digestive system problems.

1 Initial treatment period

Upon joining the study, you will begin taking the study medication called leniolisib, which contains the active substance leniolisib phosphate.

The medication is provided as film-coated tablets that you will take by mouth.

The specific dosage, frequency, and duration of treatment will be determined by the study doctor based on the study protocol.

2 Regular monitoring visits

Throughout the study, you will attend regular monitoring visits where various assessments will be performed.

During these visits, your vital signs will be checked, including blood pressure, pulse rate, and oxygen saturation levels.

You will undergo physical examinations to assess your overall health and any changes in your condition.

Blood samples will be collected for safety laboratory tests to monitor how your body is responding to the medication.

3 Imaging assessments

You will have CT scans performed to measure changes in your spleen size and any enlarged lymph nodes.

The spleen will be measured using both three-dimensional volume and two-dimensional size measurements.

Lymph nodes will be evaluated to track any changes in size over time.

If you have lung involvement, chest CT scans will be performed to assess changes in lung tissue using a specific scoring method.

4 Blood cell monitoring

Regular blood tests will be conducted to monitor your hemoglobin levels, which measure the oxygen-carrying capacity of your blood.

Your platelet count will be checked, as platelets are blood cells that help with clotting.

Your absolute neutrophil count will be measured, which refers to a type of white blood cell that fights infections.

Additional blood tests will evaluate different types of white blood cells, including specific lymphocyte populations and immune cell subsets.

5 Immune system assessments

Blood samples will be analyzed to measure specific immune cells, including naïve B cells and CD21low B cells, which are types of immune cells involved in antibody production.

The percentage of T regulatory cells will be measured, which are immune cells that help control immune responses.

Levels of certain proteins in your blood will be monitored, including CXCL13 and soluble IL-2Rα, which are markers of immune system activity.

6 Lung function testing

If applicable to your condition, you will undergo pulmonary function tests to assess how well your lungs are working.

These tests will measure several parameters including how much air you can forcefully exhale in one second, the total amount of air you can exhale, your total lung capacity, and how well oxygen moves from your lungs into your blood.

7 Safety monitoring

Throughout the study, any adverse events will be documented and monitored. This includes any unwanted or unexpected symptoms or health changes you experience.

You should report any new symptoms or changes in your health to the study doctor promptly.

The study team will continuously evaluate the safety and tolerability of the medication.

8 Medication level testing

Blood samples will be collected at specific times to measure the levels of leniolisib in your bloodstream.

These measurements help determine how your body processes the medication over time.

Who Can Join the Study?

  • You must be between 12 and 75 years old
  • You must weigh at least 45 kilograms, which is about 99 pounds
  • You must have a diagnosis of common variable immunodeficiency, which is a condition where your immune system does not produce enough antibodies to fight infections. This diagnosis should be supported by low levels of IgG, which is a type of antibody, measured on at least two separate occasions at least 3 weeks apart, unless you only had one test done before starting antibody replacement therapy, which is a treatment where you receive antibodies through infusions or injections
  • If the low IgG levels cannot be documented, you must have at least one of the following: absent or low response to certain blood type markers or vaccines, very low levels of a specific type of immune cells called class-switched memory B cells (less than 2 percent), and low levels of other antibodies called IgA or IgM, and no other identified cause for your low antibody levels
  • You must have completed genetic testing for inborn errors of immunity, which are inherited problems with the immune system, and either have no identified genetic cause found, or have a genetic change in specific genes that are known to be associated with this condition
  • You must have lymphoproliferation, which means abnormal growth of lymph tissue, shown on a CT scan, which is a special type of imaging test. This includes an enlarged spleen measuring more than 10 centimeters from top to bottom, or enlarged lymph nodes with at least one measurable node larger than 1.5 centimeters in its longest dimension
  • You must have at least one of the following symptoms: physical symptoms from your enlarged spleen or lymph nodes that affect your daily activities or cause ongoing pain, breathing difficulty, or functional limitations; low blood cell counts including low hemoglobin (less than 10 grams per deciliter, which measures red blood cells), low platelet count (less than 100,000 per microliter, which are cells that help blood clot), or low neutrophil count (less than 1,000 per microliter, which are white blood cells that fight infection); previous confirmation of interstitial lung disease, which is scarring or inflammation of the lungs, that is visible on a baseline CT scan; or intestinal disease related to your condition with ongoing abdominal pain or diarrhea at least 3 days per week for at least 4 weeks, or need for supplemental feeding through a tube or vein, along with specific test results and biopsy findings
  • Your vital signs must be within acceptable ranges after resting for at least 3 minutes. This includes blood pressure between 80-159 over 50-109 millimeters of mercury, pulse rate between 50-110 beats per minute, and oxygen saturation between 93-100 percent without oxygen support
  • You or your legal representative must be able to communicate with the study doctor, understand and follow study requirements, and provide written informed consent, which is your agreement to participate after understanding the study details

Who Cannot Join the Study?

  • The source data does not provide specific exclusion criteria, which are the reasons why a patient cannot participate in the study
  • Without detailed information about what would prevent someone from joining this clinical trial, it is not possible to list the specific conditions or situations that would make a patient ineligible
  • The trial is studying a medicine called leniolisib in patients with common variable immunodeficiency, which is a condition where the body’s immune system does not work properly and cannot fight infections well
  • The study includes both male and female participants of different age groups

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Hospital Universitario Y Politecnico La Fe Valencia Spain

Other Sites

No sites found in this category

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Spain Spain
Not recruiting
01.03.2025

Trial locations

Leniolisib is a medication being tested in this study for people with common variable immunodeficiency (CVID), which is a condition where the immune system does not work properly. This medicine is being studied to see if it is safe and well-tolerated by patients, and to explore whether it can help with immune system problems associated with CVID.

Common Variable Immunodeficiency – Common variable immunodeficiency is a disorder of the immune system where the body cannot produce enough antibodies to fight infections. People with this condition have low levels of protective proteins called immunoglobulins in their blood. The disease often leads to frequent and recurring infections, particularly in the lungs, sinuses, and ears. Over time, patients may develop enlarged organs such as the spleen and liver, as well as swollen lymph nodes throughout the body. The condition can also cause abnormal collections of immune cells in various organs, leading to inflammation. Some individuals may experience low blood cell counts, affecting red blood cells, white blood cells, or platelets.

Trial ID:
2024-517725-93-00
Protocol code:
LE 8201
Trial Phase:
Therapeutic exploratory (Phase II)

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