Not recruitingRare disease

Study on the Effects of Leniolisib in Patients with Activated Phosphoinositide 3-Kinase Delta Syndrome

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Activated Phosphoinositide 3-Kinase Delta Syndrome, a rare disorder that affects the immune system. The study will use a medication called CDZ173/Leniolisib, which is a small molecule designed to inhibit a specific part of the immune system. This medication is taken orally in the form of film-coated tablets or granules.

The purpose of the study is to compare how the body absorbs different doses of CDZ173/Leniolisib in healthy volunteers. Participants will receive single doses of the medication in various forms, including 10 mg, 30 mg, 40 mg, and 50 mg tablets, as well as 30 mg granules, and these will be compared to a 70 mg tablet. The study will also assess how well participants tolerate the medication after taking these single doses.

Throughout the study, participants will be monitored to ensure their safety and to observe any side effects that may occur. The trial aims to provide valuable information on the best way to administer CDZ173/Leniolisib for treating Activated Phosphoinositide 3-Kinase Delta Syndrome, helping to improve future treatment options for this condition.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Admission to clinical research center
Upon joining the study, you will be admitted to the clinical research center. This is where the trial will take place.
You will undergo a series of initial assessments to ensure you meet the study requirements. These assessments include a review of your medical history, a physical examination, and laboratory tests.
Step 2
Medication administration
You will receive a single dose of the study medication, leniolisib, in one of its forms: 10 mg, 30 mg, 40 mg, 50 mg film-coated tablets, 30 mg film-coated granules, or 70 mg film-coated tablet.
The medication is taken orally, meaning you will swallow it. The specific dosage and form you receive will be determined by the study protocol.
Step 3
Monitoring and observation
After taking the medication, you will be monitored closely to observe how your body responds. This includes measuring the concentration of the medication in your blood over time.
You will remain in the clinical research center for a specified period to ensure your safety and to collect necessary data.
Step 4
Follow-up assessments
You will undergo follow-up assessments to evaluate the effects of the medication. This includes additional blood tests and possibly other examinations.
These assessments help determine the medication's bioavailability, which is how much of the drug reaches your bloodstream and how quickly.
Step 5
Completion of trial participation
Once all necessary data is collected, your participation in the trial will be complete.
You will receive information about any further steps or follow-up that may be required after the trial ends.

Who can join the trial?

15 criteria

Must be a male or female between 18 and 60 years old.
Females must be of nonchildbearing potential, meaning they cannot become pregnant due to surgery, menopause (no periods for 12 months), or other reasons.
Must have a negative pregnancy test at the start and during the study.
Must be a non-smoker or have quit smoking more than 3 months ago.
Must stop all prescribed medications at least 30 days before starting the study.
Must stop all over-the-counter medications, vitamins, and herbal supplements (except paracetamol) at least 14 days before starting the study.
Must have normal liver function tests. This means certain liver enzymes and bilirubin levels must be within specific limits.
Must not drink alcohol for 48 hours before the study and during the study.
Must not consume caffeine (found in coffee, tea, cola, chocolate, energy drinks) or grapefruit products for 48 hours before the study and during the study.
Must understand the study and agree to participate by signing a written consent form.
Must have a Body Mass Index (BMI) between 18.5 and 30.0. BMI is a measure of body fat based on height and weight.
Must be in good health based on medical history, physical exam, and lab tests.
Must have a normal heart test (ECG) at the start of the study.
Must be willing to follow all study procedures and rules.
If male and fertile, must agree to use contraception (like condoms) during the study and for 3 months after the last dose. Female partners should use effective birth control methods.

Who cannot join the trial?

8 criteria

Patients with any other serious health conditions that might interfere with the study.
Patients who are pregnant or breastfeeding.
Patients who have participated in another clinical trial within the last 30 days.
Patients who have a known allergy to the study medication or its ingredients.
Patients who are unable to comply with the study procedures.
Patients with a history of drug or alcohol abuse within the past year.
Patients who have had a major surgery within the last 3 months.
Patients with any condition that the study doctor believes would make participation unsafe.

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CRS Clinical Research Services Management GmbH
Berlin, Germany

Trial locations

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Investigated drugs

Leniolisib is a medication being studied to understand how it is absorbed in the body when taken in different forms and amounts. In this trial, participants took leniolisib as either a tablet or granules. The main goal was to see how the body processes these different forms and to check if there are any differences in how well they are absorbed. The study also aimed to ensure that leniolisib is safe and well-tolerated by the participants when taken as a single dose in these various forms. This information helps researchers understand the best way to use leniolisib for future treatments.

Leniolisib Phosphate

What is already known about the treatment

Leniolisib – Leniolisib is administered orally in the form of film-coated tablets or granules. It is currently being studied in clinical trials to assess its bioavailability and tolerability in healthy volunteers. The main therapeutic indication for leniolisib is the treatment of Activated Phosphoinositide 3-Kinase Delta Syndrome, a rare immune disorder. At the molecular level, leniolisib works by inhibiting a specific enzyme involved in immune cell function, helping to regulate the immune response. It is classified pharmacologically as a selective phosphoinositide 3-kinase delta inhibitor.

Investigated diseases

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.