A phase I/II, multicenter, randomized, double-blind, placebo within-patient controlled, first-in-human (FIH) Proof of Concept (PoC) study to evaluate the safety and efficacy of topically applied SXR1096 cream in patients with Netherton syndrome (NS)
This research is a trial into a new cream, SXR1096, for people who have Netherton syndrome, a condition that affects the skin. It's the very first time this cream will be tried in humans. The main goals are to carefully check if the cream is safe to use and if it helps make the skin condition better. We'll be comparing the new cream to a 'dummy' cream (placebo) which doesn't contain any medicine, to see if the new cream works better. We'll also be looking at how the cream affects symptoms like itching, redness, and flaking of the skin. This will help us understand if SXR1096 could be a helpful new treatment option.
At a glance
What is this study about?
This study is about a new cream, called SXR1096, that we hope might help people with Netherton syndrome. Netherton syndrome is a rare genetic skin condition that can cause red, flaky skin, severe itching, and hair problems. We are testing this cream for the very first time in people, so it's an important early step in seeing if it could become a new treatment.
Our main aims are to find out two things: first, how safe the cream is to use, and second, if it actually improves the skin symptoms caused by Netherton syndrome. To do this, some people in the study will get the new SXR1096 cream, and others will get a 'dummy' cream, which looks and feels the same but doesn't have any active medicine. This helps us understand if any improvements are genuinely due to the new cream.
We will be carefully monitoring participants for any side effects or changes in their health. We'll also be looking closely at their skin using special assessment tools to see how much their skin redness, flaking, and itching improve. We'll also measure things like how much water their skin loses and the skin's pH balance. This detailed information will help us understand if SXR1096 cream could be a safe and effective treatment for Netherton syndrome in the future.
Key takeaways
- This study is testing a new cream, SXR1096, for Netherton syndrome.
- It's the first time this cream will be used in people.
- The main goals are to check for safety and see if skin symptoms improve.
- Participants will apply two different creams, one with medicine, one without.
- Regular clinic visits will monitor your health and skin condition.
- You can withdraw from the study at any time without affecting your care.
Who may be eligible?
To be able to take part in this study, you need to be at least 18 years old. There's no upper age limit, so adults of any age are welcome to inquire. The study is open to both men and women.
The most important requirement is that you have a diagnosis of Netherton syndrome. The research team will also check other health factors to make sure that taking part in the study is safe for you. They will explain these in more detail if you are interested.
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 attend clinic appointments regularly?
- Are you willing to apply creams to your skin as instructed?
What does participation involve?
If you decide to take part, you'll be given two different creams to apply to different areas of your skin. One will contain the new medicine, and the other will be a 'dummy' cream with no medicine. Neither you nor the study team will know which cream is which, to ensure the results are as fair as possible. You'll use these creams for a set period of time.
Throughout the study, you'll have several visits to the clinic. At these visits, doctors and nurses will check your overall health, measure your vital signs (like blood pressure and temperature), and take blood samples. They will also carefully examine your skin, measure things like redness, flaking, and itching, and ask you about any side effects you might be having. These appointments will involve applying cream and undergoing skin assessments. The total length of your participation, including follow-up after stopping the cream, will be fully explained to you before you agree to join.
Potential risks and benefits
Locations (4)
- —UnverifiedGermany
- —UnverifiedFrance
- —UnverifiedAustria
- —UnverifiedSweden
Common questions
What is Netherton syndrome?
Netherton syndrome is a rare inherited skin condition that causes widespread red, flaky, and itchy skin, along with hair problems, from birth.
What does 'first-in-human' mean?
It means this is the first time the new cream, SXR1096, is being tested in people. It has been tested in labs and on animals before this stage.
What is a 'placebo'?
A placebo is a 'dummy' treatment that looks exactly like the real medicine but contains no active ingredients. It helps us see if the new medicine really works better than no treatment.
Will I know if I'm getting the real cream or the placebo?
No, this is a 'double-blind' study, meaning neither you nor the doctors will know who is getting the active cream and who is getting the placebo until after the study has finished. This helps make the results fair.
How long will the study last if I join?
The team will explain the exact duration, including how long you'll use the cream and any follow-up visits, during your first appointment. It typically involves several weeks or months.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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