GENEPID: A 44-weeks monocentric open study assessing the efficacy and safety of Deucravacitinib in adults with Inflammatory EPidermal GENodermatoses (epidermolysis bullosa simplex and inflammatory congenital ichthyoses)
Living with rare, inherited skin conditions like severe epidermolysis bullosa simplex or congenital ichthyosis can cause fragile, painful, red, or peeling skin. Researchers are running a clinical trial to see if an oral medicine called deucravacitinib can help. Deucravacitinib works by calming down parts of the immune system that cause skin inflammation. In this trial, adult volunteers will take the medicine over 44 weeks. The research team will track how well the treatment works, check if it relieves distressing symptoms like itching and pain, and ensure it is safe to use. Because these conditions currently have very few effective treatments, finding a medicine that brings relief could make a meaningful difference to everyday life and overall well-being. Participation is completely voluntary.
Överblick
Vad handlar denna studie om?
Some skin conditions are inherited, meaning they are caused by changes in our genes. Conditions such as severe epidermolysis bullosa simplex (EBS) and congenital ichthyosis can make the skin fragile, blister easily, or become very dry, scaly, and inflamed. Daily life with these conditions can be difficult, often involving constant pain, intense itching, and long routines to manage the skin.
Currently, there are limited medical options to treat the underlying inflammation in these conditions. This clinical trial is looking at whether a newer medicine called deucravacitinib can help. The drug works by blocking specific signals inside the body that tell the immune system to create inflammation. By turning down these signals, researchers hope the skin will have a chance to calm down and heal.
Over the course of 44 weeks, the study team will closely monitor volunteers to see if the medicine reduces skin redness, blisters, and scaling. They will also measure how much it helps with everyday discomfort, sleep, and overall quality of life. The ultimate goal is to discover if this medication could become a helpful new treatment option in the future.
Viktiga punkter
- Testing a new medicine called deucravacitinib for rare genetic skin conditions.
- Aimed at adults aged 18 and older with severe EBS or inflammatory ichthyosis.
- The study lasts for 44 weeks and involves regular clinic appointments.
- There is no placebo; all participants will receive the active medication.
- Your safety, pain levels, itch, and quality of life will be closely tracked.
Vem kan vara berättigad?
This study is open to adults aged 18 and over who have been diagnosed with specific rare, genetic skin conditions. In particular, it is looking for people with severe epidermolysis bullosa simplex (EBS) or inflammatory forms of congenital ichthyosis.
Both men and women can join. Because this is a medical research study, the doctors will need to confirm your exact diagnosis and check your overall health before you can take part.
People who are pregnant, planning a pregnancy, or who have certain other serious medical conditions might not be able to join for safety reasons. The research team will review your full medical history and any medicines you currently take to make sure the trial is safe for you.
Kan denna studie passa dig?
Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.
- I am aged 18 or older.
- I have been diagnosed with severe epidermolysis bullosa simplex (EBS) or inflammatory congenital ichthyosis.
- I am able to attend regular hospital appointments over 44 weeks.
- I am willing to take the study medicine as instructed.
- I am happy to complete regular questionnaires about my pain, itch, and well-being.
Vad innebär deltagande?
Taking part in this study lasts for 44 weeks. During this time, you will take the study medicine, deucravacitinib, exactly as directed by the clinic team. Because this is an 'open' study, both you and the doctors will know that you are receiving the active medicine.
You will need to attend regular clinic visits at the hospital or study centre. During these visits, doctors and nurses will examine your skin, carry out routine health checks, and take blood samples to monitor your body's immune system and check for side effects. You will also be asked to complete short questionnaires about your symptoms, including your pain levels, itchiness, and how your skin condition affects your day-to-day life.
Potentiella risker och fördelar
Platser (1)
- —ObekräftadFrankrike
Vanliga frågor
What is the purpose of this clinical trial?
The study aims to see if the medicine deucravacitinib is safe and effective at reducing inflammation, pain, and itching in adults with rare, genetic skin conditions.
Kommer jag definitivt att få studiemedicinen?
Yes. This is an open study, which means there is no dummy pill (placebo). Every person who takes part receives the active medicine.
Hur länge kommer jag att vara involverad i studien?
The trial runs for 44 weeks, during which you will take the medicine and attend regular check-ups with the study team.
Vad händer om jag upplever biverkningar?
The study team will monitor your health very closely. If you develop any unwanted symptoms or feel unwell, doctors are on hand to support you, adjust your care, or stop the medicine if needed.
Kan jag lämna studien om jag ändrar mig?
Yes, you can leave at any point. Taking part is entirely voluntary, and leaving will not change the standard of healthcare you normally receive.
Hur du får reda på mer
Prata alltid med din läkare eller specialist innan du bestämmer dig för att delta i en studie.
Intresserad av att delta?
Diskussion
Gemenskapsdiskussion
Drivs av vårt forum på community.patient.info. Var vänlig och respektfull — detta är inte medicinsk rådgivning.