Ongoing Clinical Trials for Activated PI3 Kinase Delta Syndrome
There are currently 3 ongoing clinical trials studying treatments for Activated PI3 Kinase Delta Syndrome, a rare genetic disorder affecting the immune system. These trials are investigating leniolisib, an investigational medication designed to help manage symptoms by inhibiting a specific part of the immune system. The trials are taking place across several European countries including Germany, Hungary, Portugal, and Spain, and involve participants ranging from young children to adults.
Clinical trial locations
- Germany
- Hungary
- Portugal
- Spain
Study on Leniolisib for Patients with Activated Phosphoinositide 3-Kinase Delta Syndrome and Impaired Liver Function
This trial is examining how leniolisib behaves in the body when liver function is impaired. The study aims to understand whether liver problems affect how the medication is absorbed and processed in the bloodstream.
Who can participate: This study is recruiting men and women aged 18 to 79 years who cannot have children. Participants must weigh at least 50 kg and have a Body Mass Index between 18.0 and 34.9 kg/m². Those with liver impairment must meet specific criteria according to the Child-Pugh Classification system, which measures the severity of liver disease. Their liver condition must be stable with no significant changes in the past 30 days. Participants with normal liver function are also needed for comparison purposes. Light smokers or non-smokers are eligible, and all participants must be willing to attend study visits and follow the required procedures.
Who cannot participate: Interestingly, patients who actually have the syndrome cannot participate in this particular trial. The study is focused on understanding how the liver affects leniolisib processing, so it enrolls people with various liver function levels but excludes those with the actual condition being treated.
Study focus: Participants will receive a single dose of leniolisib in tablet form and will be monitored closely afterward. Blood samples will be collected at multiple time points (1, 2, 3, 24, 36, and 48 hours after taking the medication) to measure drug concentration levels. The study involves regular health checks including physical examinations, blood pressure monitoring, heart activity tests, and laboratory assessments. Follow-up continues for up to 120 hours after medication administration.
Investigational medication: Leniolisib is taken orally as a film-coated tablet. It works by selectively inhibiting an enzyme called phosphoinositide 3-kinase delta, which plays a key role in immune system function.
Study on the Effects of Leniolisib in Children Aged 1 to 6 with Activated PI3K Delta Syndrome (APDS)
This trial specifically focuses on young children with the syndrome, evaluating both the safety and effectiveness of leniolisib in this age group.
Who can participate: Boys and girls between 1 and 6 years old who weigh between 8 and 37 kilograms can join this study. Children must have a confirmed genetic mutation in either the PIK3CD gene or PIK3R1 gene. They need to have at least one measurable swollen lymph node visible on an MRI or CT scan within six months before starting the trial. Participants should also have symptoms consistent with the syndrome, such as repeated ear, nose, and lung infections or organ problems. Children must be able to swallow the study medication without modifications. Parents or legal guardians must provide written informed consent and agree that the child will not participate in other treatment studies during this trial.
Who cannot participate: Children outside the 1 to 6 year age range cannot participate. Those who cannot safely take leniolisib or who have other medical conditions or are taking medications that might interfere with the study are excluded. Children who have had recent serious illness or surgery, or who cannot follow study procedures, are also excluded.
Study focus: The trial is conducted in two parts. The first part assesses immediate effects and how well children tolerate the medication. The second part is a long-term extension evaluating safety over an extended period. Throughout the study, children undergo regular monitoring including vital sign checks, physical examinations, laboratory tests, and imaging studies like MRI or CT scans to measure changes in lymphoproliferation. The study tracks changes in blood chemistry and other health indicators to understand how leniolisib affects the immune system and to identify any side effects.
Investigational medication: Leniolisib is given as film-coated granules in single-dose containers. The dosage and frequency are determined individually based on each child’s needs. This formulation is specifically designed for young children who may have difficulty swallowing tablets.
Study on the Effects of Leniolisib in Patients with Activated Phosphoinositide 3-Kinase Delta Syndrome
This trial compares how the body absorbs different doses and forms of leniolisib in healthy volunteers, helping researchers understand the best way to administer the medication.
Who can participate: Healthy male and female volunteers aged 18 to 60 years can join this study. Women must be unable to become pregnant due to surgery, menopause, or other reasons, and must have negative pregnancy tests. Participants must be non-smokers or have quit smoking more than three months prior. They need to stop all prescription medications at least 30 days before the study and all over-the-counter medications, vitamins, and supplements (except paracetamol) at least 14 days beforehand. Normal liver function tests are required. Participants must have a Body Mass Index between 18.5 and 30.0 and be in good general health. They must avoid alcohol for 48 hours before and during the study, and avoid caffeine and grapefruit products during the same period. Male participants who are fertile must agree to use contraception during the study and for three months afterward.
Who cannot participate: People with serious health conditions that might interfere with the study cannot participate. Pregnant or breastfeeding women are excluded. Those who have participated in another clinical trial within the last 30 days, have known allergies to the study medication or its ingredients, or cannot comply with study procedures are not eligible. People with a history of drug or alcohol abuse within the past year, those who have had major surgery within the last three months, or anyone the study doctor believes would be unsafe to participate are also excluded.
Study focus: Participants receive a single dose of leniolisib in various forms including 10 mg, 30 mg, 40 mg, 50 mg tablets, 30 mg granules, or 70 mg tablets. These different formulations are compared to understand how each is absorbed by the body. After taking the medication, participants are monitored closely to measure drug concentration in the blood over time. They remain at the clinical research center for observation to ensure safety and collect necessary data. Follow-up assessments include additional blood tests and examinations to determine the medication’s bioavailability—how much of the drug reaches the bloodstream and how quickly.
Investigational medication: Leniolisib is administered orally in film-coated tablets or granules. The study aims to understand how different doses and forms are processed by the body and to ensure the medication is safe and well-tolerated when taken as a single dose.
Summary
All three trials focus exclusively on leniolisib, a selective phosphoinositide 3-kinase delta inhibitor designed to regulate immune system function in people with this rare genetic disorder. The trials take a comprehensive approach, studying the medication across different age groups and in various clinical situations.
The research spans multiple European countries, with Germany participating in two trials and Portugal, Spain, and Hungary each hosting one trial. This geographic distribution helps ensure diverse patient populations and access to specialized centers experienced in treating rare immune disorders.
Notably, the trials address different aspects of leniolisib treatment. One focuses specifically on pediatric patients aged 1 to 6 years, filling an important gap in understanding how the medication works in young children. Another examines how liver function affects drug processing, which is crucial information for treating patients with liver complications. The third evaluates different formulations and doses in healthy volunteers to optimize how the medication should be administered.
Together, these trials represent a thorough investigation of leniolisib as a potential treatment option, examining safety, effectiveness, and optimal dosing strategies across different patient populations and clinical scenarios.
