Ongoing, recruitingHuman Pharmacology (Phase I)- OtherInterventional

An open-label, single-dose, parallel-group study to evaluate the plasma pharmacokinetics of leniolisib in subjects with impaired hepatic function and in subjects with normal hepatic function

This study is investigating a new medicine called leniolisib for a rare genetic condition known as Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS). APDS can cause ongoing infections and other health issues. In this study, we want to understand how leniolisib is processed by the body, especially focusing on how it behaves in people who have liver problems compared to those with healthy livers. This will help doctors decide on the best and safest dose for different patients in the future. It’s an early-stage study, meaning it’s one of the first times this medicine is being given to people to understand its basic movement through the body.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- Other

Sponsor

Pharming Technologies B.V.

Enrolment target

Start

10 Jan 2024

Activated Phosphoinositide 3-Kinase Delta Syndrome

What is this study about?

This research study is about a new medication called leniolisib. It's for a rare genetic condition known as Activated Phosphoinositide 3-Kinase Delta Syndrome, often shortened to APDS. People with APDS can experience problems with their immune system, leading to frequent infections, inflammation, and other health issues.

The main goal of this study is to understand how leniolisib is handled by your body after you take it. Specifically, we want to see if having liver problems changes how the medicine is absorbed, used, and cleared from your system. To do this, we will compare people with APDS who have some degree of liver impairment to those with APDS who have healthy liver function.

By understanding how the liver affects this medicine, doctors can work out the safest and most effective dose for patients, especially for those who might also have liver issues. This helps ensure that future treatments for APDS are tailored to individual needs, making them as safe and helpful as possible.

Key takeaways

Study for a new medicine called leniolisib for a rare condition: APDS.
Investigating how leniolisib works in the body, especially in people with liver problems.
Compares people with APDS and healthy livers to those with APDS and liver issues.
Helps determine the safest and most effective dose for different patients.
It's an early-stage study, focused on understanding the medicine's basic movement.
Open to adults aged 18 and over, both men and women, with APDS.

Who may be eligible?

This study is looking for adults aged 18 and older. It includes both men and women.

We are particularly interested in people with Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS).

Some people in the study will have normal liver function, while others will have varying degrees of liver problems. Your study doctor will perform tests to see if your liver function matches the needs of 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 18 years old or older?
Do you have a diagnosis of Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS)?
Are you a man or a woman?
Are you willing to discuss your liver health with the study team?

Answer every question to see your result.

What does participation involve?

If you decide to take part, this study involves a single dose of the medicine. The research team will explain everything in detail. You will have several visits to the study clinic where doctors and nurses will monitor you closely. This will involve blood tests to see how the medicine is moving through your body, and they will also check your overall health. The total time you will spend in the study will be relatively short, as it's a 'single-dose' study, focusing on what happens right after you take the medication. The research team will let you know the exact schedule of visits and what each visit will involve.

Potential risks and benefits

Taking part in any study has potential risks and benefits. A potential benefit could be helping researchers understand how best to use this new medicine for APDS, which could potentially help future patients. However, as with any new medicine, there might be side effects or risks that we don't fully know yet. The study team will explain all known risks to you before you decide to join. Remember, taking part is completely voluntary, and you have the right to withdraw from the study at any time without giving a reason, and this will not affect your future medical care.

Locations (2)

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

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Unverified
Germany
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Unverified
Hungary

Common questions

What is Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS)?

APDS is a rare genetic condition that affects the immune system, leading to frequent infections and other health problems.

What is leniolisib?

Leniolisib is a new medicine being studied to treat Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS).

What does 'pharmacokinetics' mean?

It means how the body handles a medicine – how it's absorbed, distributed, used, and removed.

Why are you looking at people with liver problems?

The liver processes many medicines, so we want to see if liver problems change how leniolisib works in the body to make sure it's used safely.

Is this a new medicine or one already used?

This is an early-stage study for a new medicine, meaning it's still being investigated and is not yet available for general use.