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| ID | Type | Description | Link |
|---|---|---|---|
| 2025-A01077-42 | Other Identifier | ANSM |
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This non-interventional study is an observational cohort with parallel groups aiming primarily to describe the success of biosimilar of ustekinumab in Behçet's syndrome in whom conventional approaches have failed or are not suitable well tolerated, and then to compare with patients receiving apremilast within routine care.
Ustekinumab previously prescribed subcutaneously at 90 mg on Week 0, 4, 12 and 20 within the standard of care.
Following non-opposition to participate, patients data will be collected, which will comprise data of the 3-month interval medical visits, except for the first month of treatment, in which the short-term tolerance of treatments is usually assessed (ie, baseline visit, then week 4, 12, 24, 36 and 52). Clinical examination, biological tests and relevant clinical scores (BDCAF, BSAS and PhGA) data that were performed within routine care. No changes to patients' usual care will be made (no additional visits, additional examinations or questionnaires), their safety and well-being remaining therefore unchanged. Data will be collected from the participant's medical record (containing medical reports and examinations, biological tests, nursing records, etc.), for the period of participation in the research, with the only purpose of meeting the objectives of the research. Data will be collected using an electronic 'eCRF observation book on the REDCap platform. The following data will be collected: demographic data (age, sex, weight, height); clinical data (history of the disease, pathology diagnosed, activity of the pathology), treatments, biological data, adverse events. No genetic data will be collected as part of the study. No data will be transferred abroad. No additional questionnaires, examinations or visits will be added by the research. Activity indexes for BS will be calculated as part of routine care. The number, duration and intensity of oral ulcers of patients will be verified by the nurse or the site investigator according to the routine care.
Patients' data with active mucocutaneous Behçet's manifestations with an indication of starting apremilast within standard of care (according to AMM and PNDS) will be retrospectively collected in order to establish a retrospective cohort for comparative purposes.
This non-interventional study is an observational and comparative cohort aiming to describe the success of biosimilar of ustekinumab in Behçet's syndrome in whom conventional approaches have failed or are not suitable well tolerated. Data on 208 patients is expected to be identified as follows:
Ustekinumab is usually prescribed subcutaneously at 90 mg on Week 0, 4, 12 and 20 within the standard of care.
Following non-opposition to participate, patients data will be collected retrospectively according to local clinical practices and usual care, which usually comprises 3-month interval medical visits, except for the first month of treatment, in which the short-term tolerance of treatments is usually assessed (ie, baseline visit, then week 4, 12, 24, 36 and 52). Available data on clinical examination, biological tests and relevant clinical scores (BDCAF, BSAS and PhGA) that are performed within routine care will be retrieved. No changes to patients' usual care will be made (no additional visits, additional examinations or questionnaires), their safety and well-being remaining therefore unchanged. Data will be collected from the participant's medical record (containing medical reports and examinations, biological tests, nursing records, etc.), for the period of participation in the research, with the only purpose of meeting the objectives of the research. Data will be collected using an electronic eCRF observation book on the REDCap platform. The following data will be collected: demographic data (age, sex, weight, height); clinical data (history of the disease, pathology diagnosed, activity of the pathology), treatments, biological data, adverse events. No genetic data will be collected as part of the study. No data will be transferred abroad. No additional questionnaires, examinations or visits will be added by the research. Activity indexes for BS are usually calculated as part of routine care. The number, duration and intensity of oral ulcers of patients might be verified by the nurse or the site investigator according to the routine care.
Patients data with active mucocutaneous Behçet's manifestations with an indication of starting apremilast within standard of care will be also retrospectively collected in order to establish a comparative group, using the same parameters above mentioned
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ustekinumab group | patients who have received the Ustekinumab | ||
| Apremilast group | patients who have received the Apremilast |
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| Measure | Description | Time Frame |
|---|---|---|
| Success at Week 24 will be defined as the absence of active mucocutaneous manifestations | Success at Week 24 will be defined as the absence of active mucocutaneous manifestations at week 24 without treatment discontinuation due to inefficacy or toxicity. | Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Area under the curve (AUC) for the cumulative number of oral ulcers | Area under the curve (AUC) for the cumulative number of oral ulcers along the study period at W24. | Week 24 |
| Duration (days) of oral and genital ulcers |
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Inclusion Criteria:
Due to its fluctuant characteristics over time, active oral ulcers are defined as two or more oral ulcers over the month preceding inclusion and must have occurred at least three times in the previous 12-month period, despite the previous use of colchicine
Exclusion Criteria:
Platelets < 50 x 103/mm3 Neutrophils < 1000/mm3 Hemoglobin < 8 g/Dl
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Two groups will be established in this observational cohort:
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| DAVID SAADOUN, Professor | Contact | +33142178042 | david.saadoun@aphp.fr |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| CHU BORDEAUX Hôpital Saint-André - Service de médecine interne | Not yet recruiting | Bordeaux | France |
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| ID | Term |
|---|---|
| D001528 | Behcet Syndrome |
| ID | Term |
|---|---|
| D009059 | Mouth Diseases |
| D009057 | Stomatognathic Diseases |
| D014606 | Uveitis, Anterior |
| D015864 | Panuveitis |
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Duration (days) of oral and genital ulcers up to W24
| Week 24 |
| Number of oral and genital ulcers | Number of oral and genital ulcers up to week 24 | Week 24 |
| Number of painful and swollen joints | Number of painful and swollen joints up to W24 | Week 24 |
| Rate of patients with complete response (ie, no mucocutaneous manifestations), partial response (>50% reduction in overall mucocutaneous manifestations), and no response | Rate of patients with complete response (ie, no mucocutaneous manifestations), partial response (>50% reduction in overall mucocutaneous manifestations), and no response (ie, persistence of mucocutaneous manifestations not fulfilling complete or partial response definitions) at W12 and W24 | Week 12 and Week 24 |
| Rate of treatment discontinuations for safety reasons | Rate of treatment discontinuations for safety reasons up to W24 | Week 24 |
| Rate of treatment failures | Rate of treatment failures (i.e., persistence of active mucocutaneous manifestations that require a change in immunomodulatory therapy) up to W24 | Week 24 |
| Change in BDCAF scores | The score is between 0 and 12. Higher scores indicate a worse evaluation | Week 24 |
| Frequency and severity of adverse events | Frequency and severity of adverse events (CTCAE grade) along the study period. | 48 months |
| Safety of patients receiving the Apremilast | frequency and severity of adverse events in patients receiveing apremilast. | 48 months |
| pain intensity (0-10 EVA score) for genital and oral ulcers | pain intensity (0-10 EVA score) for genital and oral ulcers up to W24 | WEEK24 |
| Change in BSAS scores | The score is between 0 and 100. Higher scores indicate a worse evaluation | week24 |
| Change PhGA scores | The score is between 0 and 10. Higher scores indicate a worse evaluation | week 24 |
| Service de médecine interne-APHP - Henri Mondor | Not yet recruiting | Créteil | 94000 | France |
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| HOPITAL CROIX-ROUSSE - HCL - service de médecine interne | Not yet recruiting | Lyon | France |
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| Hospices civils de Lyon | Not yet recruiting | Lyon | France |
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| GHSIF Melun | Recruiting | Melun | 77000 | France |
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| AP-HP-Hôpital COCHIN | Not yet recruiting | Paris | France |
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| service de dermatologie - APHP - St Louis | Not yet recruiting | Paris | France |
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| CHU Rouen_Hôpital Charles Nicolle | Not yet recruiting | Rouen | 76000 | France |
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| CHU de Rouen - service dermatologie | Not yet recruiting | Rouen | France |
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| APHP_Hopital Lariboisière | Active, not recruiting | Paris | Île-de-France Region | 75010 | France |
| APHP_ Hôpital Pitié-Salpêtrière | Recruiting | Paris | Île-de-France Region | 75013 | France |
|
| D014605 |
| Uveitis |
| D014603 | Uveal Diseases |
| D005128 | Eye Diseases |
| D014657 | Vasculitis |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D056660 | Hereditary Autoinflammatory Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012873 | Skin Diseases, Genetic |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D017445 | Skin Diseases, Vascular |