Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

An Open-Label, Single Arm Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Leniolisib in Pediatric Patients (Aged 4 to 11 Years) With APDS (Activated Phosphoinositide 3-Kinase Delta Syndrome) Followed by an Open-Label Long-Term Extension

This study is testing a medicine called leniolisib for children aged 4 to 11 who have a rare immune condition called Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS). APDS can cause ongoingfections and swollen glands. The main goals are to see how safe the medicine is, how it moves through the body, and if it helps children feel better. Researchers will monitor for any side effects, check blood tests, and measure any changes in swollen glands using scans. This research hopes to find a new way to help young patients manage their APDS.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Pharming Technologies B.V.

Enrolment target

Start

01 Oct 2024

Activated Phosphoinositide 3-Kinase Delta Syndrome

What is this study about?

This study is all about a new medicine called leniolisib, which is being tested in children aged 4 to 11 years old who have a condition called Activated Phosphoinositide 3-Kinase Delta Syndrome, often shortened to APDS. APDS is a rare condition that affects the body's immune system, which is what normally fights off infections. Children with APDS can get infections more often and may have swollen glands like their tonsils or lymph nodes, which don't go away.

The main aim of this study is to find out if leniolisib is a safe and helpful treatment for children with APDS. Researchers will be carefully checking for any side effects the medicine might cause. They also want to see if the medicine helps reduce the swollen glands and improves the way the children's immune system works. This is a "Phase III" study, which means it's one of the final steps in testing a new medicine before it might be made more widely available.

By taking part, children will help doctors learn a lot more about APDS and hopefully find a better way to treat it. The study will look at many aspects of their health, including how their body handles the medicine, changes in their immune cells, and their overall growth and development. It's a long-term study, so they'll continue to be monitored for a longer period after the initial treatment.

Key takeaways

This study is testing a new medicine, leniolisib, for children aged 4-11 with APDS.
It aims to check if the medicine is safe and if it helps reduce infections and swollen glands.
Participants will have regular clinic visits for checks, blood tests, and scans.
The study includes an initial 12-week period followed by a long-term extension.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To be considered for this study, your child must be between 4 and 11 years old and have been diagnosed with Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS).

There may be other specific health requirements for taking part, which the study doctors will discuss with you. These are put in place to ensure your child's safety and to make sure the study results are as clear and useful as possible. Everyone who joins the study will be carefully checked to make sure it's suitable for them.

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.

Is your child between 4 and 11 years old?
Has your child been diagnosed with Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS)?
Are you able to attend regular clinic visits?
Are you comfortable with your child having blood tests and scans?

Answer every question to see your result.

What does participation involve?

If your child takes part in this study, they will take the medicine leniolisib. The study has two main parts. In the first part, which lasts for 12 weeks, your child will have regular visits to the clinic. During these visits, doctors will perform physical examinations, check their vital signs (like heart rate and blood pressure), and take blood and urine samples. They will also have heart scans (ECGs) and some imaging scans like MRI or CT scans to check for swollen glands. Throughout this time, researchers will closely monitor for any side effects and how your child is growing and developing.

After the first 12 weeks, if your child is still responding well and it is safe, they can continue into a long-term extension part of the study. This means they will keep taking the medicine and continue to have regular check-ups, though perhaps less often than in the first part. These longer-term check-ups will continue to monitor their safety, their overall health, and how well the medicine is working, including how their glands and immune system are doing over a longer period. The total duration of participation will be discussed with you by the study team.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. Your child might benefit from receiving a new medicine that could help improve their APDS symptoms, such as reducing infections and swollen glands. However, as with any medicine, there's a possibility of side effects, which the doctors will explain in detail and monitor closely. You are free to withdraw your child from the study at any time, for any reason, without it affecting their regular 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
France

Common questions

What is APDS?

APDS is a rare genetic condition that weakens the immune system, making children more prone to infections and causing swollen glands.

What does 'open-label' mean?

Open-label means that both you and the study doctors will know that your child is receiving the study medicine, leniolisib.

Will my child have to stay in hospital?

The study involves regular clinic visits and check-ups, but typically, children do not need to stay in hospital for the entire duration of the study.

What are 'lymphoproliferation' and 'immunophenotype normalization'?

Lymphoproliferation refers to the swollen glands. The study aims to reduce these. Immunophenotype normalization means trying to get the immune cells working more normally.

How long will my child be in the study?

The initial part of the study is 12 weeks, and then there's an option for a longer-term extension. The exact total time will be discussed by the study team.