Ustekinumab in BEhçet's Syndrome STudy
ConditionBehçet's Syndrome (BS)
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorGroupe français d'étude des Maladies Inflammatoires de loeil
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age ≥ 18 years old;
Non-opposition to study;
Use of contraceptive measures;
Fulfillment of the international classification criteria for Behçet's disease, revised in 2013;
Disqualifiers
Pregnancy;
Previous treatment failure to ustekinumab;
Active chronic infections (e.g., active tuberculosis, replicative hepatitis B, HIV, etc.) or malignancies;
Live vaccins in the past 3 months;
Trial population
Two groups will be established in this observational cohort: 1. Patients with active mucocutaneous manifestations of Behçet's syndrome that are either relapsing, refractory or intolerant to conventional treatments, that have received an indication of ustekinumab according to the standard of care of tertiary centres. 2. Patients with active mucocutaneous manifestations of Behçet's syndrome treated by apremilast within standard of care will be also retrospectively collected in order to establish a comparative group.
Trial design
Design model
Case-control
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
208 Participants
are grouped into 2 trial groupsGroup A: Ustekinumab group
Group B: Apremilast group
Trial outcomes
Primary outcomes
1
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.
Time frame
Week 24Secondary outcomes
1
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.
Time frame
Week 242
Duration (days) of oral and genital ulcers
Duration (days) of oral and genital ulcers up to W24
Time frame
Week 243
Number of oral and genital ulcers
Number of oral and genital ulcers up to week 24
Time frame
Week 244
Number of painful and swollen joints
Number of painful and swollen joints up to W24
Time frame
Week 24Sponsors and contacts
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Source ClinicalTrials.gov