A Multicenter, Open-Label, Phase II Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Subcutaneous Anifrolumab in Pediatric Participants 5 to < 18 Years of Age with Systemic Lupus Erythematosus.
Systemic lupus erythematosus, usually just called lupus, is an autoimmune condition where the body's immune system mistakenly attacks healthy tissues, causing pain, swelling, and tiredness. Anifrolumab (brand name Saphnelo) is already approved for some adults with lupus, but doctors need to know if it works safely and properly in younger patients. This Phase II clinical trial is testing the medicine in children and teenagers aged 5 to 17. The medicine is given as a gentle injection under the skin using a pre-filled syringe. Doctors will carefully measure how much medicine stays in the blood over time, how it affects common signs of lupus inflammation, and whether it causes any unwanted side effects. The goal is to help find the right doses and see if anifrolumab could become a helpful future treatment option for young people living with lupus.
At a glance
What is this study about?
Lupus is a long-term condition that causes the body's immune system to attack healthy joints, skin, and organs. In people with lupus, parts of the immune system become overactive, leading to problems like joint pain, extreme tiredness, and skin rashes. While there are treatments available, managing lupus can be tricky, especially for children and young people who may need options that fit their growing bodies.
This study is testing a medicine called anifrolumab, also known as Saphnelo. It works by calming down specific proteins in the immune system that drive inflammation in lupus. Although doctors already know quite a bit about how this medicine works in adults, they need clear evidence on how it behaves in children and teenagers. Because young bodies process medicines differently, specific child-focused studies are essential.
During this trial, researchers want to see how the body absorbs, breaks down, and clears the medicine after an injection under the skin. They will also look at blood tests to see if the medicine reduces known markers of lupus activity. By learning how anifrolumab works in younger patients, doctors hope to understand whether it could safely offer another treatment choice for children dealing with this challenging condition.
Key takeaways
- The trial tests anifrolumab injections for children and teens aged 5 to 17 with lupus.
- Researchers want to find the safest and most effective dose for younger bodies.
- The study lasts about a year and involves regular clinic check-ups and blood tests.
- Everyone in this trial receives the real medicine; there is no dummy drug.
- Taking part is voluntary, and you can stop at any time without affecting your NHS care.
Who may be eligible?
This study is designed for children and young people between the ages of 5 and 17 who have been diagnosed with systemic lupus erythematosus (lupus). Both boys and girls can take part.
To join, participants need to have active lupus symptoms or blood markers that show ongoing inflammation. The study team will look closely at each child's health records to ensure it is safe for them to take part.
Children may not be able to join if they have other serious medical conditions, certain ongoing infections, or if they have recently started specific treatments that could interfere with the study medicine. A doctor from the study team will carefully check all health details before anyone signs up.
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.
- The patient is between 5 and 17 years old.
- The patient has a confirmed diagnosis of systemic lupus erythematosus (lupus).
- The patient is able to attend regular hospital appointments for check-ups and blood tests.
- The patient is willing to receive injections under the skin.
- The patient does not have a severe, active infection that would make taking part unsafe.
What does participation involve?
Taking part in this study involves receiving anifrolumab as an injection under the skin using a pre-filled syringe. Because this is an open-label study, you and the doctors will know that you are receiving the real medicine rather than a dummy drug (placebo).
Participants will need to visit the hospital clinic regularly over about a year (52 weeks). During these visits, the study team will carry out standard health checks. These include checking blood pressure, taking small blood and urine samples, and asking how the young person is feeling.
Special blood tests will be done at key points, such as after the first dose, at 12 weeks, and at 52 weeks, to measure medicine levels in the blood and see how the immune system responds. The study doctors will provide close support throughout the trial and will keep in touch between visits to check on your child's well-being.
Potential risks and benefits
Locations (3)
- —UnverifiedBelgium
- —UnverifiedPortugal
- —UnverifiedNetherlands
Common questions
What is anifrolumab?
Anifrolumab (brand name Saphnelo) is a medicine that blocks certain immune system signals that cause inflammation in people with lupus.
How is the medicine given?
It is given as an injection just under the skin (subcutaneous injection) using a pre-filled syringe.
Will my child definitely get the real medicine?
Yes. This is an open-label study, meaning every child who takes part receives the active medicine and there is no placebo.
How long does the study last?
Key tests and visits take place over at least 52 weeks (around one year) to see how the body handles the medicine over time.
Can we leave the study if we change our minds?
Yes. You can withdraw your child from the trial at any point, and their normal medical care will continue as usual.
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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