ClinicalTrials.gov record NCT05845723
Tocilizumab and Tofacitinib in the Treatment of Vascular Behçet's Syndrome
Efficacy and Safety of Tocilizumab and Tofacitinib in the Treatment of Patients With Vascular Behçet's Syndrome
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase IIb randomized study compares three drug combinations for people with vascular Behçet's syndrome and aneurysms: prednisone plus tocilizumab, prednisone plus tofacitinib, or prednisone plus cyclophosphamide. The main outcome is complete remission at week 12; participants are followed for 24 weeks. Assessments include blood inflammatory markers, imaging, disease activity and damage scores, quality-of-life questionnaires, and safety monitoring.
What it is studying. The study is testing whether combining glucocorticoids (prednisone) with tocilizumab or with tofacitinib leads to different rates of complete remission at 12 weeks compared with glucocorticoids plus cyclophosphamide in people with vascular Behçet's syndrome and aneurysms. It also aims to compare safety and other clinical outcomes through 24 weeks and to identify biomarkers related to treatment response.
What the researchers hope to learn. To evaluate the efficacy and safety of glucocorticoids combined with tocilizumab or tofacitinib versus glucocorticoids combined with cyclophosphamide for vascular Behçet's aneurysms; to screen for biomarkers associated with response; and to improve assessment and develop more precise diagnosis and treatment strategies for vascular Behçet's.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- tocilizumab
- tofacitinib
- cyclophosphamide
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 1． Understand and voluntarily sign an informed consent form prior to any study-related assessments/procedures being conducted.
- 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).
- Elevated acute phase reactants ESR and hs-CRP.
Exclusion criteria
- 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;
- Active infection such as active tuberculosis, hepatitis B or C, syphilis, chronic EBV infection, persistent or severe bacterial or viral infection;
- Primary or secondary immunodeficiency;
- Malignant tumor;
- Use of immunosuppressants such as Cyclosporin A (CsA), Azathioprine (AZA), Tacrolimus (TAC), Mycophenolate Mofetil (MMF), or Cyclophosphamide (CTX) within 1 month;
- 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);
- Pregnant, lactating, or planning a recent pregnancy;
- Subjects who do not agree to or are unable to comply with regular visits.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Show the detailed description from the registry
Behçet's Syndrome (BS) is a recurrent and systemic vasculitis, with an incidence rate of about 14/100,000 in China, higher than that in Europe and America. Vascular Behcet's Syndrome (VBS) is an important subtype of BS characterized by multiple venous and artery lesions. It affects 20-25% of BS patients, predominantly young adult males, and is the leading cause of mortality in BS patients (67%), especially in patients with aneurysms. Therefore, exploring the diagnosis and treatment strategies for VBS is key to improving the prognosis of BS. This project aims to investigate the efficacy and safety of the combination of glucocorticoids (GCS) with the biologic agent tocilizumab or the targeted small molecule tofacitinib, compared to the combination of GCS with cyclophosphamide in the treatment of VBS aneurysms, as well as to screen for biomarkers related to the response of tocilizumab or tofacitinib, and to improve the assessment and treatment of VBS and establish a precision diagnosis and treatment strategy. This is a Phase IIb, multi-center, randomized, open-label, GCS plus cyclophosphamide-controlled, parallel design, prospective clinical study. Patients will be randomly assigned to three groups in a 1:1:1 ratio (tocilizumab + GCS vs tofacitinib + GCS vs cyclophosphamide + GCS, 27:27:27) after screening and recruitment. Patients will be followed up every 4 weeks from weeks 0-12, and every 6 weeks from weeks 12-24. The primary endpoint is the complete remission (CR) rate at the 12th-week follow-up. All participants will undergo their final visit at 24 weeks.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
You will be randomly assigned to one of three 24‑week treatment groups: prednisone (tapered to 10 mg/day by week 12) combined with either IV tocilizumab every 4 weeks (8 mg/kg), oral tofacitinib 5 mg twice daily, or IV cyclophosphamide 0.5 g every two weeks. Clinic visits are scheduled every 4 weeks from weeks 0–12 and every 6 weeks from weeks 12–24, with a final visit at week 24. At visits you will have blood tests including ESR and hs‑CRP (weeks 0, 4, 8, 12, 18, 24); the Behçet's Disease Current Activity Form (BDCAF) questionnaires (weeks 0, 4, 8, 12, 18, 24); imaging with vascular ultrasound and CT angiography (CTA) (weeks 0, 12, 24); damage assessments using BODI (weeks 0 and 24) and VDI (weeks 0, 12, 24); and SF‑36 quality‑of‑life surveys (weeks 0, 12, 24). Study staff will record any adverse events or serious adverse events at the scheduled visits (weeks 0, 4, 8, 12, 18, 24).
How the study is structured. Randomized, open-label, parallel Phase IIb trial with three treatment groups assigned 1:1:1. All groups receive prednisone tapered from 1 mg/kg/day to 10 mg/day by week 12 plus one of: intravenous tocilizumab (8 mg/kg every 4 weeks), oral tofacitinib (5 mg twice daily), or intravenous cyclophosphamide (0.5 g every 2 weeks). Visits occur every 4 weeks from week 0 to 12 and every 6 weeks from week 12 to 24, with a final visit at week 24. Primary endpoint is complete remission at week 12.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry lists this study under Aneurysm; Behcet Syndrome, Vascular Type. Does it apply to my diagnosis?
- The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Behçet's disease
This trial is also listed on this site under familial Mediterranean fever.
Important dates
- Study start
- Jun 1, 2023
- Primary completion
- Jun 1, 2026 (estimated)
- Study completion
- Jun 1, 2027 (estimated)
- Target enrollment
- 81 participants (estimated)
- Last updated on registry
- Aug 19, 2024
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Peking Union Medical College Hospital
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Jinjing Liu, M.D. 📞 8613581605769 ✉ wingsforjane@163.com
- Wenjie Zheng, M.D. 📞 8613661281939 ✉ wenjzheng@gmail.com
Participating sites (1)
- Peking Union Medical College HospitalRecruiting
Beijing, Beijing Municipality, 100730 · China
- Jinjing Liu
- Wenjie Zheng, M.D.
Terms used on this page · explanations by MedicalUpdates.ai
- glucocorticoids
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- informed consent form
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- computed tomography
- An X-ray scan that builds detailed cross-section images of the body. 'High-resolution' CT (HRCT) is used to look closely at the lungs.
- immunosuppressive
- A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- ANC
- The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
- 5 half-lives
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- biomarkers
- Something measurable in the body, such as a blood test result or an imaging finding, that indicates how a condition or treatment is behaving.
- randomized
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- 1:1:1
- How many participants go to each group. In a 2:1 ratio, two are assigned to the first group for every one assigned to the second.
- primary endpoint
- The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
- complete remission
- No sign of the condition can be found with the tests used in the study. It does not always mean the condition is gone permanently, and the definition depends on the condition and the study.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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