Treatment of Dysphagia in Patients With Dystrophic Epidermolysis Bullosa With Budesonide: a Retrospective Bicentric Study Nice - Paris St Louis / Necker (EBUDE Study)

Sponsor
Centre Hospitalier Universitaire de Nice
Study ID
NCT07684105
Status
Not Yet Recruiting

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Conditions

  • Dysphagia
  • Esophageal Stenosis
  • Recessive Dystrophic Epidermolysis Bullosa

Eligibility Criteria

Sex
ALL
Age
1 Year - N/A
Healthy Volunteers
Not accepted

Interventions

  • The retrospective data collected will be entered into an eCRF and then analyzed centrally — OTHER
    there is no treatment for the study but the patient take a treatment out of MMA and the retrospectives datas collected will be entered into an eCRF and then analyzed centrally by the sponsor

Study Details

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.

Key Dates

First listed
Jul 6, 2026
Start date
Jul 1, 2026
Status verified
Jul 2026
Primary completion
Dec 31, 2026
Completion
Jun 30, 2027

Study Design

Enrollment
15 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Other: recessive dystrophic epidermolysis bullosa with dysphagia treated with oral BUDESONIDE
    The retrospective data collected will be entered into an eCRF and then analyzed centrally

Primary Outcome Measure

overall patient satisfaction with the treatment [ Time Frame: month 1 ]

Central Contacts

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