Study on Leniolisib for Patients with Activated Phosphoinositide 3-Kinase Delta Syndrome and Impaired Liver Function

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What is this study about?

This clinical trial is focused on studying a condition known as Activated Phosphoinositide 3-Kinase Delta Syndrome, which is a rare disorder affecting the immune system. The study will use a medication called CDZ173/Leniolisib, which is taken as a film-coated tablet. Leniolisib is a small molecule that works by inhibiting a specific part of the immune system, known as p110δ, to help manage the symptoms of this syndrome.

The purpose of the study is to understand how the body processes leniolisib in people with different liver functions. Participants will include those with impaired liver function and those with normal liver function. The study will involve taking a single dose of leniolisib and then monitoring how it is absorbed and processed in the body. This will help researchers learn more about the medication’s behavior in the bloodstream, which is referred to as its pharmacokinetics.

Participants will be divided into groups based on their liver function, and the study will follow a parallel-group design, meaning that all groups will be observed simultaneously. The study will not involve any placebo treatments. Throughout the study, participants will have regular check-ups and tests to monitor their health and the effects of the medication. This research aims to provide valuable information that could improve treatment options for those with Activated Phosphoinositide 3-Kinase Delta Syndrome.

1 initial visit

Upon joining the study, you will attend an initial visit. During this visit, your medical history will be reviewed, and a physical examination will be conducted. This includes measuring your blood pressure and pulse rate, as well as performing an electrocardiogram (ECG) to check your heart’s activity.

Blood samples will be taken to ensure you meet the study’s health criteria. You will also be asked to provide a urine sample for testing. This visit ensures that you are eligible to participate in the study.

2 medication administration

You will receive a single dose of the study medication, leniolisib phosphate, in the form of a film-coated tablet. This medication is taken orally.

The purpose of this medication is to evaluate how it is processed in your body, particularly if you have impaired liver function.

3 monitoring period

After taking the medication, you will be monitored closely. Blood samples will be collected at various times to measure the concentration of the medication in your blood.

These samples will be taken at 1, 2, 3, 24, 36, and 48 hours after you have taken the medication. This helps to understand how the medication is absorbed, distributed, and eliminated by your body.

4 follow-up visits

You will have follow-up visits to check your health and any effects of the medication. These visits will include additional blood tests and health assessments.

The follow-up period lasts for up to 120 hours after taking the medication. During this time, any side effects or adverse reactions will be recorded.

5 end of study

At the end of the study, a final health check will be conducted. This includes a physical examination and any necessary tests to ensure your well-being.

You will have the opportunity to discuss any concerns or questions with the study team before concluding your participation.

Who Can Join the Study?

Participants must be men or women who cannot have children, aged between 18 and 79 years old.
Participants should not have any known or suspected liver problems, known as hepatic impairment.
Participants must be in good health, with no significant medical issues found during a medical history review, physical exam, heart test (ECG), or lab tests.
Participants must meet specific criteria for liver function, known as Class B or Class C in the Child-Pugh Classification, which is a system used to assess the severity of liver disease.
Participants with liver problems must have a stable condition, meaning no significant changes in their liver health in the last 30 days.
Participants must not have started a new medication or changed the dose of their current medication within seven days before the study begins.
Participants with a history of alcohol abuse can join if they test negative for alcohol at the start and agree to follow lifestyle guidelines.
Women who cannot have children must meet one of the following: be postmenopausal (no periods for at least 12 months), have had surgery to remove the uterus or ovaries, or have confirmed ovarian failure.
Men must agree to use a condom and a highly effective birth control method with their female partner if she can have children, from the start of the study until 90 days after the last dose.
Participants must have a Body Mass Index (BMI) between 18.0 and 34.9 kg/m² and weigh more than 50 kg.
Participants must sign a consent form indicating they understand the study details.
Participants must be willing and able to attend all study visits and follow the study procedures.
Participants can be light smokers (less than 10 cigarettes per day) or non-smokers.
Participants must have normal liver function tests, including specific levels of liver enzymes and proteins.
Participants must match certain demographic criteria, such as gender, weight, and age, with other participants in the study.

Who Cannot Join the Study?

Patients with Activated Phosphoinositide 3-Kinase Delta Syndrome cannot participate.
Patients with liver problems that affect how the body processes medication cannot participate.
Patients who are not within the specified age range for the study cannot participate.
Patients who are part of a vulnerable population, such as those unable to give consent, cannot participate.

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country	Status
CRS Clinical Research Services Management GmbH	Kiel	Germany
Cnc Hwwmmcq Knoe	Encs	Hungary

Trial status

Country	Status	Recruitment Start
Germany	Not recruiting	01.01.2024
Hungary	Not recruiting	01.01.2024

Trial locations

Leniolisib is a medication being studied to understand how it behaves in the body, especially in people with liver problems. The study aims to see how the liver affects the way leniolisib is absorbed and processed in the blood. This medication is taken by mouth, and researchers are comparing its effects in people with liver issues to those with normal liver function. The goal is to ensure that leniolisib is safe and effective for everyone, regardless of their liver health.

Investigated diseases:

Activated PI3 kinase delta syndrome

Activated Phosphoinositide 3-Kinase Delta Syndrome – This is a rare genetic disorder that affects the immune system. It is caused by mutations in the PIK3CD gene, which leads to an overactive immune response. Individuals with this syndrome often experience recurrent infections, particularly in the respiratory tract. Over time, the condition can lead to the development of autoimmune disorders, where the immune system mistakenly attacks the body’s own tissues. The syndrome can also cause lymphoproliferation, which is an abnormal increase in the number of white blood cells. This can result in enlarged lymph nodes and other lymphoid tissues.

Trial ID:

2023-508519-22-00

Protocol code:

LE 6101

Trial Phase:

Human Pharmacology (Phase I) – Other

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Study on Leniolisib for Patients with Activated Phosphoinositide 3-Kinase Delta Syndrome and Impaired Liver Function

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?