Recessive Dystrophic Epidermolysis BullosaEBUDENCT07684105
Treatment of Dysphagia in Patients With Dystrophic Epidermolysis Bullosa With Budesonide: a Retrospective Bicentric Study Nice - Paris St Louis / Necker (EBUDE Study)
A study testing The retrospective data collected will be entered into an eCRF and then analyzed centrally in people with Recessive Dystrophic Epidermolysis Bullosa and Dysphagia, run by Centre Hospitalier Universitaire de Nice at 3 sites in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Recessive Dystrophic Epidermolysis Bullosa, Dysphagia, Esophageal Stenosis
- What is being tested?
- The retrospective data collected will be entered into an eCRF and then analyzed centrally
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 3 sitesFrance
- Study dates
- 1 Jul 2026to 30 Jun 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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)
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
France3 sites
CHU de Nice
Nice, France, 06000
APHP Necker
Paris, France, 75000
Nathalia BELLON, Doctor, 0492034702, nathalia.bellon@aphp.fr
APHP St Louis
Paris, France, 75000
Pauline Bataille, Doctor, 0492034702, pauline.bataille@aphp.fr
About this study
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.
Read more
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.
Read the detailed description
Budesonide is a non-halogenated glucocorticoid that has an anti-inflammatory effect by binding to glucocorticoid receptors. In the treatment of eosinophilic esophagitis, whether as Jorveza or as a compounded formulation, it inhibits the antigen-induced secretion of various cytokines and pro-inflammatory molecules such as thymic stromal lymphopoietin, interleukin-13, and eotaxin-3 in the esophageal epithelium (1). This action leads to a significant reduction in inflammatory eosinophil infiltrates in the esophagus.
Dystrophic epidermolysis bullosa (DEB) is a rare genetic disease that causes fragile skin and mucous membranes, leading to blisters and wounds after trauma. Healing is abnormal and often associated with scarring and contractures.
The literature only reports two pediatric studies, with a total of eight DEB patients treated with a compounded formulation (CF) budesonide-based. These studies have shown good tolerance and satisfactory effectiveness on dysphagia (2, 3). To date, there is no data on the use of Jorveza in orally disintegrating tablets in adults with EBD, even though it is recommended for this indication in official guidelines (4). Our goal is to evaluate, within a cohort followed at the MAGEC reference center, the usefulness of budesonide in PM/orally disintegrating tablets, in patients of all ages with EBD.
- Being tested
- The retrospective data collected will be entered into an eCRF and then analyzed centrally
- Medical subject headings
- Epidermolysis Bullosa Dystrophica, Deglutition Disorders, Esophageal Stenosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
The retrospective data collected will be entered into an eCRF and then analyzed centrallyOther
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 group | Role | What they receive |
|---|---|---|
| recessive dystrophic epidermolysis bullosa with dysphagia treated with oral BUDESONIDE | Other | The retrospective data collected will be entered into an eCRF and then analyzed centrally Receives: Other: The retrospective data collected will be entered into an eCRF and then analyzed centrally |
What is being measured
The primary outcome is the main question the study is designed to answer.
overall patient satisfaction with the treatment
5-point Likert scale (4: complete satisfaction and 0: no change)
Measured: month 1
overall patient satisfaction with the treatment
5-point Likert scale (4: complete satisfaction and 0: no change)
Measured: month 3
Secondary outcomes (5)
The assessment of budesonide treatment tolerance in patients
Assessed based on the side effects and/or reasons for stopping the treatment recorded in the medical file.
Measured: Month 3
Reduce meal time
yes or no
Measured: Month 0
Reduce meal time
yes or no
Measured: Month 1
oesophagal expansion number
number of necessary expansions during the study
Measured: Month 0
oesophagal expansion number
number of necessary expansions during the study
Measured: Month 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jul 2026
- Study start1 Jul 2026 (estimated)
- Primary completion31 Dec 2026 (estimated)
- Study completion30 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Recessive Dystrophic Epidermolysis BullosaStudies of the same condition, open and under way.
- More trials run by Centre Hospitalier Universitaire de NiceThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Retrospective study of a patient with recessive dystrophic epidermolysis bullosa and dysphagia treated with oral BUDESONIDE
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire de Nice
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07684105
- Sponsor's protocol ID
- 26-PP-03
- Other IDs
- IDRCB
- Acronym
- EBUDE
- Status last verified
- Jul 2026
- First submitted
- 16 Jun 2026
- Last updated
- 6 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Dysphagia in Patients With Dystrophic Epidermolysis Bullosa With Budesonide: a Retrospective Bicentric Study Nice - Paris St Louis / Necker (EBUDE Study)
- ClinicalTrials.gov ID
- NCT07684105
- Acronym / sponsor ID
- EBUDE / 26-PP-03
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Centre Hospitalier Universitaire de NiceUniversity, hospital or other
- Study start
- 1 Jul 2026 (estimated)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 30 Jun 2027 (estimated)
- First posted
- 6 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Budesonide is a non-halogenated glucocorticoid that has an anti-inflammatory effect by binding to glucocorticoid receptors. In the treatment of eosinophilic esophagitis, whether as Jorveza or as a compounded formulation, it inhibits the antigen-induced secretion of various cytokines and pro-inflammatory molecules such as thymic stromal lymphopoietin, interleukin-13, and eotaxin-3 in the esophageal epithelium (1). This action leads to a significant reduction in inflammatory eosinophil infiltrates in the esophagus.
Dystrophic epidermolysis bullosa (DEB) is a rare genetic disease that causes fragile skin and mucous membranes, leading to blisters and wounds after trauma. Healing is abnormal and often associated with scarring and contractures.
The literature only reports two pediatric studies, with a total of eight DEB patients treated with a compounded formulation (CF) budesonide-based. These studies have shown good tolerance and satisfactory effectiveness on dysphagia (2, 3). To date, there is no data on the use of Jorveza in orally disintegrating tablets in adults with EBD, even though it is recommended for this indication in official guidelines (4). Our goal is to evaluate, within a cohort followed at the MAGEC reference center, the usefulness of budesonide in PM/orally disintegrating tablets, in patients of all ages with EBD.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Retrospective study of a patient with recessive dystrophic epidermolysis bullosa and dysphagia treated with oral BUDESONIDE
| Arm | Type | Receives |
|---|---|---|
| recessive dystrophic epidermolysis bullosa with dysphagia treated with oral BUDESONIDE | Other | Other: The retrospective data collected will be entered into an eCRF and then analyzed centrallyThe retrospective data collected will be entered into an eCRF and then analyzed centrally |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (1 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| The retrospective data collected will be entered into an eCRF and then analyzed centrallythere 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 | Other | recessive dystrophic epidermolysis bullosa with dysphagia treated with oral BUDESONIDE |
Primary outcome
overall patient satisfaction with the treatment
Time frame: month 1
5-point Likert scale (4: complete satisfaction and 0: no change)
overall patient satisfaction with the treatment
Time frame: month 3
5-point Likert scale (4: complete satisfaction and 0: no change)
5 secondary outcomes
The assessment of budesonide treatment tolerance in patients
Time frame: Month 3
Reduce meal time
Time frame: Month 0
Reduce meal time
Time frame: Month 1
oesophagal expansion number
Time frame: Month 0
oesophagal expansion number
Time frame: Month 1
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jul 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing The retrospective data collected will be entered into an eCRF and then analyzed centrally in people with Recessive Dystrophic Epidermolysis Bullosa and Dysphagia, run by Centre Hospitalier Universitaire de Nice at 3 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 6 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Christine CHIAVERINI, Doctor (0492034702, chiaverini.c@chu-nice.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
overall patient satisfaction with the treatment (time frame: month 1). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jul 2026.