Ongoing, recruitingHuman Pharmacology (Phase I)- OtherInterventional

A six-way relative bioavailability study comparing 10, 30, 40 and 50 mg leniolisib film coated tablets and 30 mg film coated granules with 70 mg film coated tablet in healthy volunteers

This study is investigating a new medicine called leniolisib, which is being developed to treat a rare genetic condition known as Activated Phosphoinositide 3-Kinase Delta Syndrome. Researchers are testing various forms and doses of leniolisib – including different strengths of tablets and granules – in healthy adult volunteers. The main goal is to understand how the body absorbs and processes the medicine from these different preparations. This helps find the best way to deliver the treatment effectively and safely. They will also be monitoring for any side effects to ensure the safety of these different drug formulations. This is an early-stage study, meaning it's one of the first times this drug is being tested in humans.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- Other

Sponsor

Pharming Technologies B.V.

Enrolment target

Start

28 Feb 2024

Activated Phosphoinositide 3-Kinase Delta Syndrome

What is this study about?

This study is looking into a medicine called leniolisib, which is being developed for a rare condition known as Activated Phosphoinositide 3-Kinase Delta Syndrome. This condition can cause problems with the body's immune system.

The main purpose of this study is to compare several different forms and strengths of the leniolisib medicine. Think of it like comparing different brands or types of a vitamin – some might be tablets, some might be capsules, and they might come in different strengths. Researchers want to see how well the body takes in and uses the leniolisib from these different versions. This is important because it helps them figure out the best way to make the medicine so it works effectively for patients.

They are doing this by giving different versions of the medicine to healthy adult volunteers and then carefully measuring how much of the medicine gets into their bloodstream and how long it stays there. They will also be watching closely for any side effects to make sure the medicine is safe. This type of study is an early step in developing a new medicine, helping scientists understand how it works in the human body before it's given to people with the actual condition.

Key takeaways

This study is testing different types and strengths of a new medicine called leniolisib.
It aims to find the best way for the body to absorb and use this medicine.
Healthy adult volunteers are needed for this early-stage research.
The findings will help develop a treatment for a rare immune system condition.
Safety and how the medicine moves through the body are the main focus.

Who may be eligible?

To take part in this study, you would need to be an adult, 18 years old or older. Both men and women are welcome to participate.

Because this study is looking at how the body handles the medicine in healthy people, you would need to be generally fit and well, without any long-term health problems that might affect how your body deals with new medication.

The doctors and nurses running the study will check if you meet all the specific requirements to ensure it's safe for you to take part.

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?
Are you generally healthy with no major ongoing health problems?
Are you able to attend several clinic visits, possibly including overnight stays?
Are you comfortable having regular blood tests?
Are you willing to discuss your full medical history with the study team?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would receive different versions of the leniolisib medication at different times. This would likely involve several visits to a clinic over a period of time, perhaps including an overnight stay for some of the medication doses.

During your visits, you would have regular blood tests to measure the levels of the medicine in your body. You would also have general health checks, like measuring your blood pressure and heart rate, and answering questions about how you are feeling. This is to make sure you remain safe and well throughout the study. The total duration of your involvement could range from several weeks to a few months, depending on the number of different medication doses you receive and the follow-up required after each dose. All study procedures would be fully explained before you agree to participate.

Potential risks and benefits

Taking part in any study has potential benefits and risks. While this study is in healthy volunteers, it helps in the development of a potential new medicine for a serious condition, which could ultimately benefit future patients. You would also receive health checks and access to medical professionals. However, there are risks, including potential side effects from the medication, which will be carefully monitored, and discomfort from procedures like blood tests. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

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

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Unverified
Germany

Common questions

What is Activated Phosphoinositide 3-Kinase Delta Syndrome?

It's a rare genetic condition that affects the immune system, making people more prone to infections and other health issues.

Why are healthy people taking part in a study for a disease?

Early studies like this use healthy volunteers to understand how a new medicine is absorbed and processed by the body in general, before it's tested in people who actually have the condition.

What do 'film-coated tablets' or 'granules' mean?

These are just different ways the medicine is made. Tablets are solid pills, and granules are like tiny beads or powder that can be mixed into food or liquid. The 'film-coated' means it has a thin protective layer.

Will I know if I'm getting the active medicine or a dummy pill?

In this specific study, all participants will receive different versions of the active leniolisib medicine. There is no dummy pill (placebo) involved in this comparison.

Will I get paid for taking part?

Sometimes studies offer payment or reimbursement for your time, travel, and inconvenience. This would be clearly explained to you before you agree to take part.