Upadacitinib for Refractory Behcet's Syndrome

NCT07080346 | Completed Phase 2

• 1 other identifier: PKU People's Hospital UPA
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 1

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.

Conditions

Behcet Syndrome

Keywords

Behcet syndrome; Upadacitinib

Outcome Measures

Primary Outcomes (1)

• Patients getting improved condition

  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.

  Week 24

Secondary Outcomes (4)

• Changes of Behcet's Disease Current Activity Form (BDCAF) score of patients

  Week 24 and week 48

• Changes of C-reactive protein

  Week 24 and week 48

• Changes of erythrocyte sedimentation rate

  Week 24 and week 48

• Changes of dosage of glucocorticoids from baseline

  Week 24 and week 48

Study Arms (1)

Treated with upadacitinib

EXPERIMENTAL

Refractory BS patients were treated with upadacitinib.

Drug: Upadacitinib 15 MG

Interventions

Upadacitinib 15 MG DRUG

All the BS patients discontinued other biologic agents and received oral upadacitinib treatment at a dose of 15mg per day with background glucocorticoids and immunosuppressants for 48 weeks. All the patients will be followed up prospectively for 48 weeks.

Treated with upadacitinib

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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≥2).
• Resistant to glucocorticoids, at least two traditional immunosuppressants (one of which must have been cyclophosphamide or mycophenolate mofetil) or biological agents (one of which must have been a TNF-α inhibitor or a JAK inhibitor) 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Liu Tian: lead

Study Sites (1)

Department of Rheumatology and Immunology, Peking University People's Hospital

Beijing, Beijing Municipality, 100044, China

Location

MeSH Terms

Conditions

Behcet Syndrome

Interventions

upadacitinib

Condition Hierarchy (Ancestors)

Mouth Diseases; Stomatognathic Diseases; Uveitis, Anterior; Panuveitis; Uveitis; Uveal Diseases; Eye Diseases; Vasculitis; Vascular Diseases; Cardiovascular Diseases; Hereditary Autoinflammatory Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vascular

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: associate professor

Study Record Dates

• First Submitted: July 15, 2025
• First Posted: July 23, 2025
• Study Start: January 25, 2024
• Primary Completion: December 20, 2024
• Study Completion: July 8, 2025
• Last Updated: December 2, 2025

Record last verified: 2025-07

Locations

CN (1)