Tocilizumab and Tofacitinib in the Treatment of Vascular Behçet's Syndrome
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-65
SponsorPeking Union Medical College Hospital
About this trial
This project aims to evaluate the efficacy and safety of the combination of glucocorticoids with tocilizumab or tofacitinib, compared to the traditional combination of glucocorticoids with cyclophosphamide in the treatment of vascular Behçet's syndrome.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
None
Disqualifiers
Cardiovascular manifestations that cannot be distinguished from giant cell arteritis, Burger's disease, or atherosclerotic aneurysm; infectious aneurysm;
Other active organ involvement related to BS that requires intensified immunosuppressive treatment, including gastrointestinal ulcers, uveitis, and parenchymal neurological involvement;
Patients with severe aneurysms requiring emergency intervention surgery; patients with elective surgery indications require the consensus between rheumatologists and vascular surgeons to determine inclusion or exclusion.
Severe organ dysfunction, including ALT, AST, and TBIL exceeding the upper limit of normal by more than 2 times, serum creatinine ≥ 133 mmol/L, white blood cell count < 3×10^9/L, ANC < 2×10^9/L, hemoglobin < 80g/L, platelet count < 100×10^9/L;
Trial design
Design model
Parallel
Treatments tested in this trial
tocilizumab
DrugParticipants will receive intravenous infusion of tocilizumab 8mg/kg every 4 weeks for 24 weeks.
tofacitinib
DrugParticipants will receive tofacitinib 5mg twice a day for 24 weeks of treatment.
cyclophosphamide
DrugParticipants will receive intravenous infusion of cyclophosphamide 0.5g biweekly for 24 weeks.
Treatment groups
81 Participants
are divided into 3 treatment groupsGroup A: Tocilizumab+GCS for VBSExperimental treatment 1 intervention
Group B: Tofacitinib+GCS for VBSExperimental treatment 1 intervention
Group C: Cyclophosphamide+GCS for VBSExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
The primary endpoint is the complete response (CR) rate at week 12.
CR: the resolution of VBS-related symptoms and abnormal acute phase reactants including erythrocyte sedimentation rate (ESR) and high-sensitivity C reactive protein (hs-CRP), no progression or new occurrence of vascular lesions compared to baseline.
Time frame
Baseline to week 12Secondary outcomes
1
The secondary endpoints include CR rate at week 24, and partial response (PR) rate at week 12/24.
PR: improvement in clinical symptoms, \>50% decrease in inflammatory markers, no progression or new occurrence of vascular lesions.
Time frame
Baseline to week 242
Change From Baseline in Disease Activity as Measured by Behçet's Disease Current Activity Form (BDCAF).
The BDCAF consists of 3 component scores: the Behçet's Disease Current Activity Index (BDCAI) score, the Patient's Perception of Disease Activity, and the Clinician's Overall Perception of Disease Activity. The BDCAI consists of 12 questions regarding disease manifestations over the previous 4 weeks, including oral and genital disease activity, as well as other manifestations of BS involving the skin, joints, GI tract, eyes, nervous system, and vascular system. The BDCAI score is the sum score of 12 items and ranges from 0 to 12. The Patient's Perception of Disease Activity and the Clinician's Overall Perception of Disease Activity were assessed on a scale from 1 to 7. A higher score indicates a higher level of disease activity (worsening), and a negative change from baseline indicates improvement.
Time frame
BDCAF were assessed at weeks 0, 4, 8, 12, 18, and 243
Change From Baseline in the degree of vasculitis damage as Measured by Behçet's syndrome Overall Damage Index (BODI).
The BODI consists of 4 overarching principles and 34 items with 12 subitems, categorized into 9 organ/system domains: mucocutaneous, musculoskeletal, ocular, vascular, cardiovascular, neuropsychiatric, gastrointestinal, reproductive system, and miscellaneous. Each item and subitem score 1 point. The total score ranges from 0 to 46. The higher the score, the greater the degree of damage.
Time frame
BODI were assessed at weeks 0 and 244
4.Change From Baseline in the degree of vasculitis damage as Measured by Vasculitis Damage Index (VDI).
The VDI consists of 64 items, categorized into 11 organ/system domains: musculoskeletal, skin, ear, nose, and throat, pulmonary, cardiovascular, renal, gastrointestinal, peripheral vascular, ocular, neuropsychiatric, and other damage. Each item scores 1 point. The total score ranges from 0 to 64. The higher the score, the greater the degree of damage.
Time frame
Time Frame: VDI were assessed at weeks 0, 12, and 24Sponsors and contacts
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Source ClinicalTrials.gov