A pilot multicentric open study assessing the efficacy and safety of NEMOlizumab in patients ≥12yo with Dystrophic Epidermolysis bullosa; NEMODE
Dystrophic epidermolysis bullosa (DEB) is a rare genetic condition that causes very fragile skin, blisters, pain, and severe itching. This clinical study is testing a drug called nemolizumab (Nemluvio) to see if it can relieve these symptoms. Participants receive the medicine as an injection using a pre-filled pen. Doctors will track how much itching decreases over the first four weeks using simple rating scales. Over six months, the study will also check whether the treatment eases pain, improves everyday quality of life, and helps with joint movement and swallowing problems caused by scarring. Because this is an early-stage study, researchers will also carefully monitor safety and side effects throughout the 24 weeks.
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¿De qué trata este estudio?
Dystrophic epidermolysis bullosa (DEB) is a rare condition that makes the skin tear and blister very easily. Alongside painful wounds, many people with DEB experience intense, non-stop itching. Constant scratching can cause further skin damage and slow down healing. Currently, there are limited treatments that directly target this severe itch.
This study is looking at whether a medicine called nemolizumab can help. Nemolizumab is designed to block specific signals in the body that trigger the feeling of itch. By calming these signals, researchers hope the treatment might break the cycle of itching and scratching, giving the skin a better chance to rest.
Researchers also want to understand if easing itch and inflammation can improve other parts of living with DEB. Over time, the condition can cause scarring that tightens the skin, making it hard to move joints, open the mouth wide, or swallow food comfortably. Finding a medicine that eases these daily burdens could make a meaningful difference to quality of life.
Puntos clave
- Tests a new injection called nemolizumab to treat severe itching in dystrophic epidermolysis bullosa.
- The primary focus is measuring itch relief during the first 4 weeks of treatment.
- The study also monitors pain, joint movement, swallowing, and quality of life over 24 weeks.
- Participants receive regular health checks, skin assessments, and safety monitoring.
- Participation is entirely voluntary and does not replace your standard EB care team.
¿Quién puede ser elegible?
This study is open to adults who have been diagnosed with dystrophic epidermolysis bullosa. Both men and women can take part. While the title notes the medicine may be studied in younger people, the current recruitment details list an age of 18 years and older.
To join, participants must experience significant itching related to their condition, as measuring changes in itch is the main goal of the trial. People should also be able to attend regular clinic visits for health checks and photographs of their skin.
Certain health issues may mean this study is not suitable. For instance, people with severe allergic reactions or other active medical conditions that could make the injections unsafe may not be able to participate. Your hospital doctor or the study team will review your full medical history to confirm if it is safe for you to take part.
¿Podría este estudio ser adecuado para ti?
Responde estas preguntas rápidas para ver si puedes ser elegible. Esto es solo una guía: el equipo de investigación toma la decisión final.
- I have a confirmed diagnosis of dystrophic epidermolysis bullosa (DEB).
- I am 18 years of age or older.
- I regularly experience troublesome skin itching.
- I am able and willing to attend hospital appointments over a 6-month period.
- I am comfortable receiving injections under the skin.
¿En qué consiste la participación?
Taking part in this study lasts for about 24 weeks (around 6 months). During this time, you will receive nemolizumab as an injection just under the skin, given with a pre-filled pen device.
You will need to attend hospital clinic visits at the start of the study, at week 4, and at week 24, along with routine check-ins. During these visits, doctors will ask you to score your itching and pain on simple number scales from 0 to 10. You will also fill out short questionnaires about your daily quality of life.
The research team will carry out physical checks, including measuring how easily you can open your mouth and move your elbow and knee joints. They will check for any swallowing difficulties and take photos of specific skin areas, such as your leg, to track changes over time.
Riesgos y beneficios potenciales
Ubicaciones (1)
- —No verificadoFrancia
Preguntas comunes
What is the main goal of this trial?
The main goal is to find out if nemolizumab can significantly reduce severe itching within the first 4 weeks of treatment.
How is the medicine given?
It is given as an injection under the skin using a pre-filled pen, similar to an insulin pen.
¿Cuánto tiempo estaré en el estudio?
The study lasts for 24 weeks (about 6 months), with key checks at week 4 and at the end of the trial.
Will taking part cure my DEB?
No, this medicine is not a cure for DEB. It is being tested to see if it can relieve symptoms like itch and skin discomfort.
¿Puedo abandonar el estudio si cambio de opinión?
Yes, you can leave the study at any point without giving a reason, and your normal medical care will continue as usual.
Cómo obtener más información
Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.
Discusión
Discusión comunitaria
Impulsado por nuestro foro en community.patient.info. Por favor, sea respetuoso — esto no es un consejo médico.