Pediatric Patients Aged 1 to 6 Years With APDS
This study is for young children, specifically those aged 1 to 6 years old, who have a rare genetic condition called APDS (Activated PI3K-delta Syndrome). The main goal is to find out if a medicine called leniolisib is safe and effective in treating APDS. Researchers will be observing how the medicine works in the body and if it helps reduce the symptoms of this condition. Participants will take the medicine based on their weight and will be closely monitored over about 15 months. The study hopes to understand the potential benefits and any side effects of leniolisib for these children.
At a glance
What is this study about?
This study is designed for young children, aged 1 to 6, who have a rare genetic condition called APDS. This condition can cause problems with the body's immune system, leading to repeated infections and swelling of lymph glands. The study wants to explore a new medicine, called leniolisib, which might help manage these symptoms.
Researchers will observe how the medicine affects the children's bodies, checking for both potential benefits and any side effects. This is a "Phase 3" study, which means the medicine has already been tested in adults and now needs to be further evaluated in children to confirm its safety and effectiveness specifically in this age group.
Understanding how leniolisib works in children with APDS is very important. The results of this study could help doctors better treat this condition in the future, potentially improving the health and quality of life for young patients.
Key takeaways
- The study is for children aged 1-6 with APDS.
- It tests a new medicine called leniolisib.
- The main goals are to check safety and how well the medicine works.
- Participation lasts about 15 months.
- Close medical monitoring will be provided.
- Your child can leave the study at any time.
Who may be eligible?
To join this study, children must be between 1 and 6 years old and have a confirmed diagnosis of APDS, which is identified through a specific genetic test. They also need to have certain signs of the condition, such as swollen lymph glands that can be seen on scans, or a history of frequent ear, nose, throat, or chest infections.
Children must be able to swallow the study medicine without difficulty. Their blood pressure and pulse rate will be checked to make sure they are within a healthy range for their age. Parents or legal guardians will need to give their full permission for their child 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.
- Is your child between 1 and 6 years old?
- Does your child have a confirmed genetic diagnosis of APDS?
- Does your child have signs of lymph gland swelling or a history of frequent infections?
- Can your child swallow medication by mouth without difficulty?
What does participation involve?
If your child joins the study, they will take the leniolisib medicine by mouth every day. The amount of medicine will be carefully chosen based on their weight. The study begins with an initial period of 12 weeks where your child's safety and how well the medicine works will be closely watched. This will involve regular check-ups, and likely some blood tests and scans, though the exact number of visits isn't specified in detail.
After these 12 weeks, your child will continue to take the medication and be monitored for a longer period of about one year. This longer period helps researchers understand the medicine's long-term safety and effects. The total duration of participation would therefore be around 15 months. You will be kept informed about your child's progress throughout the study.
Potential risks and benefits
Locations (9)
- University of California Los AngelesVerified postcodeLos Angeles, United States
- National Institutes of HealthVerified postcodeBethesda, United States
- Rainbow Childrens HospitalVerified postcodeShaker Heights, United States
- Texas Children's HospitalVerified postcodeHouston, United States
- Kyoto University HospitalVerified postcodeKyoto, Japan
- Institute of Science Tokyo HospitalVerified postcodeTokyo, Japan
- Hospital Pediátrico de Coimbra da ULS Coimbra UNIDADE LOCAL DE SAÚDE DE COIMBRAVerified postcodeCoimbra, Portugal
- Hospital Universitario Virgen del RocíoVerified postcodeSeville, Spain
- Great Ormond Street HospitalVerified postcodeLondon, United Kingdom
Common questions
What is APDS?
APDS is a rare genetic condition that affects the immune system, often causing repeated infections and swollen glands.
What is leniolisib?
Leniolisib is the new medicine being tested in this study to see if it can help children with APDS.
Will my child have to stay in hospital?
The information provided does not say children need to stay in hospital, but there will be regular visits for check-ups.
How long will the study last for my child?
Your child would participate for about 15 months in total, with more frequent checks in the first 12 weeks.
Are there any costs involved for me?
Clinical trials usually cover the cost of the study medicine and any specific tests related to the trial itself. You should ask the study team for details.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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