Combining tocilizumab and tofacitinib for treating vascular Behçet's syndrome

Efficacy and Safety of Tocilizumab and Tofacitinib in the Treatment of Patients With Vascular Behçet's Syndrome

This study is testing if combining two newer medications, tocilizumab and tofacitinib, with steroids can help people with vascular Behçet's syndrome feel better compared to the usual treatment.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 81 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Peking Union Medical College Hospital (Academic / other)
• Drugs / interventions: infliximab, adalimumab, golimumab, ustekinumab, secukinumab, tocilizumab, baricitinib, tofacitinib, Cyclophosphamide
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT05845723 on ClinicalTrials.gov

What this trial studies

This project evaluates the efficacy and safety of combining glucocorticoids with either tocilizumab or tofacitinib compared to the traditional treatment of glucocorticoids with cyclophosphamide for vascular Behçet's syndrome. The study is a Phase IIb, multi-center, randomized, open-label trial where patients will be assigned to one of three treatment groups. The primary endpoint is the complete remission rate at 24 weeks, with follow-ups every 4 to 6 weeks. The study also aims to identify biomarkers related to the response to treatment, enhancing precision in diagnosis and therapy.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18-65 with confirmed vascular Behçet's syndrome and aneurysms.

Not a fit: Patients with severe aneurysms requiring emergency surgery or other active organ involvement may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could significantly improve treatment outcomes for patients with vascular Behçet's syndrome.

How similar studies have performed: While this approach is novel for vascular Behçet's syndrome, similar studies in other conditions have shown promising results with biologic agents.

Eligibility criteria

Inclusion Criteria:

1． Understand and voluntarily sign an informed consent form prior to any study-related assessments/procedures being conducted.

2\. Male and female subjects aged 18-65 years.

3． Fulfill the 2013 International Classification Criteria for Behcet's Disease (ICBD).

4． Patients with aneurysmal dilatation/aneurysm of the descending aorta and/or peripheral arteries confirmed by ultrasonography and/or computed tomography angiography (CTA).

5\. Elevated acute phase reactants ESR and hs-CRP.

Exclusion Criteria:

1. Cardiovascular manifestations that cannot be distinguished from giant cell arteritis, Burger's disease, or atherosclerotic aneurysm; infectious aneurysm;
2. Other active organ involvement related to BS that requires intensified immunosuppressive treatment, including gastrointestinal ulcers, uveitis, and parenchymal neurological involvement;
3. 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.
4. 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;
5. Active infection such as active tuberculosis, hepatitis B or C, syphilis, chronic EBV infection, persistent or severe bacterial or viral infection;
6. Primary or secondary immunodeficiency;
7. Malignant tumor;
8. Use of immunosuppressants such as Cyclosporin A (CsA), Azathioprine (AZA), Tacrolimus (TAC), Mycophenolate Mofetil (MMF), or Cyclophosphamide (CTX) within 1 month;
9. Use of biologics/small molecule drugs within 5 half-lives (baricitinib within 10 days; etanercept within 4 weeks; infliximab within 8 weeks; adalimumab, golimumab, ustekinumab, and abatacept within 10 weeks, secukinumab within 6 months, and previously use of tocilizumab and tofacitinib);
10. Pregnant, lactating, or planning a recent pregnancy;
11. Subjects who do not agree to or are unable to comply with regular visits.

Where this trial is running

Beijing, Beijing Municipality

• Peking Union Medical College Hospital — Beijing, Beijing Municipality, China (Recruiting)

Study contacts

• Study coordinator: Jinjing Liu, M.D.

Conditions Aneurysm; Behcet Syndrome, Vascular Type; Tocilizumab; Tofacitinib; Vascular Behçet's Syndrome