Evasayil TM: A placebo-controlled trial to evaluate the efficacy and safety of spesolimab in the treatment of patients with Netherton syndrome
This research study is looking into a new medicine called spesolimab (also known by its trial code BI 655130) for people with Netherton syndrome. Netherton syndrome is a rare genetic skin condition. The study aims to find out if spesolimab is effective at treating the symptoms of Netherton syndrome and if it is safe to use. To do this, some participants will receive the active medicine, while others will receive a 'dummy' treatment called a placebo, which looks just like the real medicine but contains no active drug. Researchers will compare the groups to understand the medicine's effects. The study will measure improvements in skin symptoms, as well as how participants feel in terms of pain, itch, and their daily lives. It's an important step in finding better ways to manage Netherton syndrome.
At a glance
What is this study about?
This study is about a new medicine called spesolimab (also known as BI 655130) for a rare skin condition called Netherton syndrome. Netherton syndrome is a lifelong condition that causes very dry, red, and flaky skin, often with severe itching and other problems. Because it's a genetic condition, people are born with it.
The main goal of this study is to see if spesolimab can make a real difference to the skin symptoms of people living with Netherton syndrome and if it is safe to use. Researchers want to find out if the medicine can reduce the severity of the skin problems by at least half. They will also be looking at other important things, like how much pain and itching people experience, how much the condition affects their daily lives, and if there are any side effects from the treatment.
To make sure the results are reliable, some participants will receive the active medicine, spesolimab, while others will get a 'placebo.' A placebo is a treatment that looks exactly like the real medicine but doesn't contain any active drug. This helps scientists compare the effects of the actual medicine against no treatment, so they can be sure any improvements are due to spesolimab and not just chance. Finding new, effective, and safe treatments could significantly improve the lives of people with Netherton syndrome.
Key takeaways
- This study is testing a new medicine (spesolimab) for Netherton syndrome.
- It aims to see if the medicine is effective and safe for people with the condition.
- Some participants will receive the active medicine; others will receive a placebo.
- The study measures improvements in skin symptoms, pain, itch, and quality of life.
- It's open to adults aged 18 and over.
- Participation involves regular clinic visits and monitoring.
Who may be eligible?
To join this study, you need to be an adult, aged 18 years or older. The study is open to both men and women.
There will be other, more detailed requirements that you'd need to discuss with your doctor or the study team. These might include things like your current health, any other medicines you're taking, or how stable your Netherton syndrome is right now. These rules are in place to help make sure the study is as safe as possible for everyone involved and that the results are clear and accurate.
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.
- Are you 18 years old or older?
- Do you have a diagnosis of Netherton syndrome?
- Are you able to travel to study appointments?
- Are you willing to potentially receive a placebo?
- Are you currently in a stable health condition (not including your Netherton syndrome)?
What does participation involve?
Taking part in this study means you would receive either the study medicine (spesolimab) or a placebo (dummy treatment), given as an injection or infusion. You would have regular visits to the clinic so the study team can monitor your health, check your skin, and see how you're responding to the treatment. These visits will involve various tests and assessments, such as looking at your skin, asking about your pain and itch, and how it impacts your daily life. The study will last for a period of time, which the study team will explain to you in detail. There will also be follow-up appointments after you finish taking the study medicine to make sure you're doing well.
Potential risks and benefits
Locations (11)
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- —UnverifiedNetherlands
- —UnverifiedFrance
Common questions
What is Netherton syndrome?
Netherton syndrome is a rare genetic skin condition that causes chronic red, scaly skin, severe itching, and hair abnormalities.
What is spesolimab?
Spesolimab is a new medicine being tested to see if it can help improve the skin symptoms of Netherton syndrome.
What is a 'placebo'?
A placebo is a 'dummy' treatment that looks like the real medicine but contains no active drug. It helps researchers compare the effects of the actual medicine.
Will I know if I'm getting the real medicine or the placebo?
Usually, in this type of study, neither you nor your doctor will know if you're getting the active medicine or the placebo until the study is over. This is called 'blinding' and helps ensure fair results.
How long does the study last?
The full duration of the study, including treatment and follow-up, will be explained in detail by the study team if you are interested in participating.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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