Upadacitinib for Refractory Behcet's Syndrome

Not Applicable

Completed

• Conditions: Behcet Syndrome
• Interventions: Drug: Upadacitinib 15 MG

• Registration Number: NCT07080346
• Lead Sponsor: Liu Tian

Brief Summary

Behçet's syndrome (BS) is a systemic autoimmune vasculitis that can affect multiple organs, including the skin, eyes, and vascular system. Refractory BS poses significant treatment challenges, necessitating novel therapeutic approaches. Upadacitinib, a selective JAK1 inhibitor within the JAK-STAT pathway, has shown promise in modulating immune responses. This study aims to evaluate the efficacy and safety of upadacitinib in patients with refractory BS.

Detailed Description

This multicenter, single-arm study investigates the efficacy and safety of upadacitinib (15 mg once daily) in refractory Behçet's syndrome (BS) patients. Adult patients had active BS with inadequate response to glucocorticoids and at least two conventional immunosuppressants or biologics over six months. Prior biologics were discontinued, and upadacitinib was added to ongoing glucocorticoids and immunosuppressants for 48 weeks. Clinical symptoms (oral/genital ulcers, skin lesions, uveitis), inflammatory markers (CRP, ESR), and medication usage were monitored. Adverse events were recorded to assess safety.

Study Timeline

• Study Start: 2024-01-25
• Primary Completion: 2024-12-20
• Status Verified: 2025-07
• Study Completion: 2025-07-08
• Study First Submitted: 2025-07-15
• Study First Submitted QC: 2025-07-15
• Last Update Submitted: 2025-07-15
• Study First Posted: 2025-07-23
• Last Update Posted: 2025-07-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 27

Inclusion Criteria

• • Male or female aged 18-70 years at time of screening.

  • Diagnosis of Behcet's syndrome (according to the International Criteria for Behçet's Disease) for ≥3 months before screening.
  • Active Behcet's syndrome at time of screening (BDCAF≥1).
  • Resistant to glucocorticoids, at least two traditional immunosuppressants or biological agents for at least 6 months.
  • Given their written informed consent to participate in the trial and expected to be able to adhere to the study visit schedule and other protocol requirements.

Exclusion Criteria

• • BS-related active major organ involvement requiring immunosuppressive therapy, e.g., pulmonary (e.g., pulmonary artery aneurysm), vascular (e.g., thrombophlebitis, recurrent malignant aneurysms), gastrointestinal (e.g., gastrointestinal ulcers), and central nervous system (e.g., meningoencephalitis).

  • High-dose glucocorticoid (>1mg/kg/d) usage within 1 month.
  • Severe comorbidities: including heart failure (≥ grade III NYHA), renal insufficiency (creatinine clearance ≤30 ml/min), hepatic insufficiency (serum ALT or AST >3 times the ULN, or total bilirubin >ULN for the central laboratory conducting the test). Other severe, progressive or uncontrolled hematologic, gastrointestinal, endocrine, pulmonary, cardiac, neurologic, or cerebral disease (including demyelinating diseases such as multiple sclerosis).
  • Known allergies, hypersensitivity, or intolerance to Baricitinib or its excipients.
  • Had a severe infection (including, but not limited to hepatitis, pneumonia, sepsis, or pyelonephritis); had been hospitalized for an infection; or had been treated with IV antibiotics for an infection, within 2 months prior to the first administration of study agent.
  • Chest radiograph within 3 months prior to the first administration of study agent that showed an abnormality suggestive of a malignancy or current active infection, including tuberculosis.
  • Infected with HIV (positive serology for HIV antibody) or hepatitis C (positive serology for Hep C antibody). If seropositive, consultation with a physician with expertise in the treatment of HIV or hepatitis C virus infection was recommended.
  • Infected with hepatitis B virus. For patients who were not eligible for this study due to hepatitis B virus test results, consultation with a physician with expertise in the treatment of hepatitis B virus infection was recommended.
  • Had any known malignancy or has a history of malignancy within the previous 5 years (with the exception of a nonmelanoma skin cancer that had been treated with no evidence of recurrence for ≥3 months before the first study agent administration or cervical neoplasia with surgical cure).
  • Had uncontrolled psychiatric or emotional disorder, including a history of drug and alcohol abuse within the past 3 years that might prevent the successful completion of the study.
  • Received, or was expected to receive, any live virus or bacterial vaccination within 3 months before the first administration of study agent, during the study, or within 4 months after the last administration of study agent. Had a BCG vaccination within 12 months of screening.
  • Pregnancy, lactation or women of child-bearing potential (WCBP) unwilling to use medically approved contraception whilst receiving treatment and for 12 months after treatment has finished.
  • Men whose partners are of child-bearing potential but who are unwilling to use appropriate medically approved contraception whilst receiving treatment and for 12 months after treatment has finished.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Treated with upadacitinib	Upadacitinib 15 MG	Refractory BS patients were treated with upadacitinib.

Primary Outcome Measures

Name	Time	Method
Patients getting improved condition	Week 24	The primary endpoint was defined as the proportion(percent) of patients in the whole cohort getting improved condition by week 24. Improved condition was defined as BS-related manifestations resolved and no newly onset imaging/endoscopic findings observed.

Secondary Outcome Measures

Name	Time	Method
Changes of Behcet's Disease Current Activity Form (BDCAF) score of patients	Week 24 and week 48	The clinical manifestation of patients were recorded during the follow-up. The disease activity of patients was accessed by Behcet's Disease Current Activity Form (BDCAF) score and the BDCAF scores (Range: 0\~12, higher scores mean higher disease activity) at week 24, week 48 and the baseline scores were compared.
Changes of erythrocyte sedimentation rate	Week 24 and week 48	Blood samples were collected from all patients and the erythrocyte sedimentation rates (mm/h) were recorded. Erythrocyte sedimentation rates at 24 weeks, 48 weeks and the baseline were compared.
Changes of dosage of glucocorticoids from baseline	Week 24 and week 48	The dosage of glucocorticoids (mg/day) of all patients were recorded during the follow-up. The dosage of glucocorticoids at 24 weeks, 48 weeks and the baseline were compared.
Changes of C-reactive protein	Week 24 and week 48	Blood samples were collected from all patients and the concentration of C-reactive protein (mg/L) were recorded. C-reactive protein at 24 weeks, 48 weeks and the baseline were compared.

Trial Locations

Locations (1)

Department of Rheumatology and Immunology, Peking University People's Hospital; 🇨🇳 Beijing, Beijing, China