Study on the Effects of Leniolisib in Children Aged 1 to 6 with Activated PI3K Delta Syndrome (APDS)

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What is this study about?

This clinical trial is focused on studying a rare condition known as Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS), which affects the immune system. The study is testing a treatment called leniolisib, which is a medication designed to help manage this condition. The trial is specifically for young children aged 1 to 6 years who have been diagnosed with APDS. The purpose of the study is to evaluate the safety and effectiveness of leniolisib in these children.

Participants in the study will receive leniolisib, which comes in the form of film-coated granules that are taken orally. The study will be conducted in two parts. The first part will focus on assessing how well children tolerate the medication and its immediate effects. The second part will look at the long-term safety and tolerability of leniolisib. Throughout the study, various health checks will be performed, including monitoring vital signs, conducting physical examinations, and using imaging techniques like MRI or computed tomography (CT) to observe changes in the body.

The study aims to gather information on how leniolisib affects the body, including its impact on the immune system and any potential side effects. This will involve regular health assessments and laboratory tests to track changes in blood chemistry and other health indicators. The ultimate goal is to determine if leniolisib can provide a safe and effective treatment option for young children with APDS, potentially improving their quality of life by reducing symptoms and complications associated with the condition.

1 initial treatment phase

The study begins with an open-label treatment phase where the medication leniolisib is administered. This phase is designed to evaluate the safety and effectiveness of the medication in children aged 1 to 6 years with a condition known as Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS).

During this phase, the medication is given orally in the form of film-coated granules contained in a single-dose container. The specific dosage and frequency of administration will be determined by the study team based on individual patient needs.

2 monitoring and assessment

Throughout the initial treatment phase, regular monitoring is conducted to assess the patient’s response to the medication. This includes checking vital signs, conducting physical examinations, and performing laboratory tests such as blood tests and urinalysis.

Imaging tests like MRI or low-dose CT scans are used to measure changes in lymphoproliferation, which is a characteristic of APDS. These assessments help determine the effectiveness of the treatment.

3 long-term extension phase

Following the initial treatment phase, patients may enter a long-term extension phase. This phase continues to evaluate the long-term safety and tolerability of leniolisib in pediatric patients with APDS.

The same monitoring and assessment procedures are maintained to ensure ongoing evaluation of the patient’s health and response to the medication.

4 completion and follow-up

Upon completion of the study, a final assessment is conducted to gather comprehensive data on the patient’s health status and the overall impact of the treatment.

Patients may be required to attend follow-up visits to monitor any long-term effects of the medication and to ensure their continued well-being.

Who Can Join the Study?

The patient must be a boy or girl between 1 to 6 years old at the time of the first study procedure.
The patient must weigh between 8 and 37 kilograms at the start of the study.
The patient must have a confirmed genetic change, called a mutation, in either the PIK3CD (APDS1) or PIK3R1 (APDS2) gene.
The patient must have at least one measurable swollen lymph node, which can be seen on a special scan like an MRI or CT scan, within 6 months before starting the study.
The patient must have swelling of lymph nodes or other tissues and symptoms that match APDS, such as a history of repeated ear, nose, and lung infections or problems with organs.
The patient must be able to swallow the study medication without any changes, according to the doctor’s opinion.
During the screening, the patient’s vital signs, like body temperature, blood pressure, and heart rate, must be checked while sitting (or lying down for infants) after resting for at least 3 minutes. The blood pressure should be less than the 95th percentile for their sex, age, and height. The heart rate should be:
- For children under 2 years: 100 to 190 beats per minute.
- For children 2 to 6 years: 60 to 140 beats per minute.
Written permission, called informed consent, must be obtained from the patient and/or their parent or legal guardian before any study-related procedures. This must be approved by a review board or ethics committee.
The patient’s parent or legal guardian must agree that the patient will not join any other study that involves treatment while they are in this study.
The patient’s parent or legal guardian must be willing and able to complete the consent process and follow the study procedures and visit schedule.

Who Cannot Join the Study?

Patients who are not between the ages of 1 to 6 years old.
Patients who do not have Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS), which is a specific medical condition.
Patients who are not able to safely take the study medication, leniolisib.
Patients who have other medical conditions that might interfere with the study.
Patients who are taking other medications that might interfere with the study medication.
Patients who have had a recent serious illness or surgery.
Patients who are not able to follow the study procedures.
Patients who have participated in another clinical trial recently.

Where you can join this trial?

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Other Sites

Site Name	City	Country	Status
Virgen del Rocío University Hospital	Sevilla	Spain
Cyaumd Hvedczessb E Uzduaarxpwdgd Db Clautkq Elnwce	Coimbra	Portugal

Trial status

Country	Status	Recruitment Start
Portugal	Not recruiting	22.07.2024
Spain	Not recruiting	22.07.2024

Trial locations

Leniolisib is a medication being studied for its safety, how it moves through the body, how it affects the body, and its effectiveness in treating young children with a condition called Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS). This condition affects the immune system, and the study aims to see if leniolisib can help manage the symptoms in children aged 1 to 6 years. The study is open-label, meaning both the researchers and participants know what treatment is being given, and it includes a long-term extension to observe the effects over a longer period.

Activated Phosphoinositide 3-Kinase Delta Syndrome – This is a rare genetic disorder that affects the immune system, leading to problems with immune cell function. It is caused by mutations in the PIK3CD gene, which results in the overactivation of a specific enzyme pathway. Individuals with this syndrome often experience recurrent infections, particularly in the respiratory tract, due to impaired immune responses. Over time, the condition can lead to lymphoproliferation, where there is an abnormal increase in the number of lymphocytes, a type of white blood cell. This can cause enlargement of lymph nodes, spleen, and other organs. The syndrome may also affect the development and function of B cells, which are crucial for producing antibodies.

Trial ID:

2022-502180-38-00

Protocol code:

LE 3302

NCT ID:

NCT05693129

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Effects of Leniolisib in Children Aged 1 to 6 with Activated PI3K Delta Syndrome (APDS)

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?