Ivarmacitinib for Refractory Behcet's Syndrome

Efficacy and Safety of the Novel, Highly Selective JAK Inhibitor Ivarmacitinib in the Treatment of Behçet's Disease: A Multicenter Prospective Study

Behcet's syndrome (BS) is a multisystem autoimmune vasculitis. Current clinical treatment primarily includes glucocorticoids, immunosuppressants, and molecularly targeted drugs such as TNF-α and IL-6 inhibitors; however, some patients still respond poorly to existing treatment regimens. The JAK-STAT signaling pathway serves as a common downstream signaling pathway for various cytokines involved in the pathogenesis of Behcet's syndrome. Upon binding to their receptors, cytokines activate JAK kinases, which in turn phosphorylate STAT proteins to regulate the expression of inflammation-related genes. Therefore, blocking the JAK-STAT pathway can simultaneously inhibit the signal transduction of multiple pathogenic cytokines, thereby exerting anti-inflammatory effects. Consequently, Ivarmacitinib-a highly selective JAK1 inhibitor-holds promise for improving the prognosis and quality of life of patients with refractory Behçet's syndrome.

This is a multi-center, single-arm trial conducted to evaluate the safety and efficacy of Ivarmacitinib in Behçet's syndrome (BS) . Patients with refractory Behçet's syndrome were enrolled . Patients received Ivarmacitinib for up to 24 weeks, which was added to the glucocorticoid and immunosuppressants. The clinical manifestations, inflammatory indicators, imaging and treatment of patients were recorded by investigators during the follow up.

Study Overview

• Status: Not yet recruiting
• Conditions: Behcet's Syndrome
• Intervention / Treatment: Drug: Ivarmacitinib

• Study Type: Interventional
• Enrollment (Estimated): 30
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Tian Liu; Phone Number: +8613661345637; Email: Liutian03973@pku.edu.cn
• Study Contact Backup: Name: Yirui Lian; Phone Number: +86 15037924173

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China
      • Department of Rheumatology and Immunology, Peking University People's Hospital
      • Contact: Yirui Lian; Phone Number: +86 15037924173
      • Contact: Tian Department of Rheumatology and Immunology, Peking University P; Phone Number: • +86 136 6134 5637; Email: Liutian03973@pku.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• ① Meets the 1990 ISG diagnostic criteria for Behçet's syndrome [9];

  • Aged 18-70 years; ③ Has shown an inadequate response to treatment after at least 6 months of therapy with glucocorticoids, at least two immunosuppressants, and/or biologics (including TNF-α inhibitors and IL-6 inhibitors);

    • Agrees to receive treatment with a new targeted therapy

Exclusion Criteria:

• ① Patients with one or more other autoimmune diseases;

  • Patients who have undergone major surgery within 4 weeks prior to enrollment, or who are scheduled to undergo elective surgery during the study; ③ Patients with a confirmed acute or chronic active infection (e.g., bacterial, viral [such as EBV, CMV, HIV, or active hepatitis virus]) within 4 weeks prior to enrollment; ④ Patients with a current or past history of any malignancy;

    • Female patients who are pregnant or within 6 months postpartum;

      • Patients with severe liver failure (Child-Pugh Class C) or end-stage renal disease (dialysis-dependent).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ivarmacitinib for the Treatment of Refractory Behçet's Disease	Drug: Ivarmacitinib; The specific regimen was 4 mg of Ivarmacitinib administered daily for 24 weeks. All patients will undergo 24 weeks of prospective follow-up.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Patients achieving complete remission and partial remission	The primary endpoint was defined as the proportion(percent) of patients in the whole cohort achieving complete remission and partial remission by week 24.	Week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes of C-reactive protein	Blood samples were collected from all patients and the concentration of C-reactive protein (mg/L) were recorded.	Week 24
Changes of erythrocyte sedimentation rate	Blood samples were collected from all patients and the erythrocyte sedimentation rates (mm/h) were recorded.	week24
Changes of dosage of glucocorticoids from baseline.	The dosage of glucocorticoids (mg/day) of all patients were recorded during the follow-up.	week 24

Collaborators and Investigators

• Sponsor: Liu Tian

Study record dates

Study Major Dates

• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): May 31, 2027
• Study Completion (Estimated): May 31, 2027

Study Registration Dates

• First Submitted: May 31, 2026
• First Submitted That Met QC Criteria: June 7, 2026
• First Posted (Actual): June 9, 2026

Study Record Updates

• Last Update Posted (Actual): June 9, 2026
• Last Update Submitted That Met QC Criteria: June 7, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Behçet's syndrome、Ivarmacitinib
• Additional Relevant MeSH Terms: Mouth Diseases; Stomatognathic Diseases; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, Inborn; Eye Diseases; Skin Diseases; Skin Diseases, Vascular; Skin Diseases, Genetic; Uveal Diseases; Vasculitis; Panuveitis; Uveitis, Anterior; Uveitis; Hereditary Autoinflammatory Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin and Connective Tissue Diseases; Behcet Syndrome; ivarmacitinib
• Other Study ID Numbers: PKU People's Hospital SHR0302

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No