Treatment of Dysphagia in Patients With Dystrophic Epidermolysis Bullosa With Budesonide: a Retro… (NCT07684105) | Clinical Trial Compass
Not Yet RecruitingNot Applicable
Treatment of Dysphagia in Patients With Dystrophic Epidermolysis Bullosa With Budesonide: a Retrospective Bicentric Study Nice - Paris St Louis / Necker (EBUDE Study)
France15 participantsStarted 2026-07-01
Plain-language summary
Budesonide is an anti-inflammatory medication that works by decreasing the immune system's overreaction. In the treatment of eosinophilic esophagitis, especially with Jorveza or a preparation made in a pharmacy, it reduces the production of substances responsible for inflammation in the esophagus. This action reduces the accumulation of certain inflammatory cells (eosinophils) and thus improves the symptoms and lesions of the esophagus.
Dystrophic epidermolysis bullosa (EBD) is a rare genetic disease that makes the skin and mucous membranes very fragile. Blisters, sores and wounds can appear as a result of even minor trauma. Scarring is often abnormal and can cause tissue to shrink or shrink.
To date, only two studies involving a total of eight children with EBD have evaluated a budesonide-based preparation. The results show an improvement in swallowing difficulties (dysphagia) as well as good tolerance of the treatment. However, there are currently no published data regarding the use of Jorveza in the form of an orally disintegrating tablet in adults with EBD.
Through the follow-up of patients treated at the MAGEC reference center, specialized in rare genetic diseases of the skin and mucous membranes, we aim to evaluate the interest and effects of budesonide treatment in individuals with EBD presenting with dysphagia.
Who can participate
Age range
1 Year
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
Patients of both sexes, of any age, with EBD in dominant or recessive form, treated for at least one month with budesonide for dysphagia resistant to usual treatments.
A description of the clinical form must be available in the file. The patient must assess the benefit of treatment at 1 and/or 3 months of Budesonide.
Patients must be affiliated with social security. For adults, consent of non-opposition is required, and for minors, non-opposition from one of the parents or the person holding parental authority must be collected.
Exclusion Criteria:
No precise clinical or biological diagnosis. Treatment with Budesonide \< 1 month. No evaluation criteria in the file / Objection to the use of data (withdrawal of non-opposition)
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.