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NCT Number: NCT07582991

Ustekinumab for Intestinal Behçet's Syndrome With Myelodysplastic Syndrome

Behçet's syndrome is a systemic vasculitis. Gastrointestinal involvement in Behçet's syndrome complicated by myelodysplastic syndrome represents a rare and severe subtype for which no standardized treatment guidelines currently exist, posing significant challenges in clinical practice. Ustekinumab, a biologic agent targeting IL-12/IL-23, has demonstrated favorable efficacy in both gastrointestinal Behçet's syndrome and inflammatory bowel disease. This study aims to evaluate the efficacy and safety of ustekinumab in patients with intestinal Behçet's syndrome and coexisting myelodysplastic syndrome.

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Key information

Conditions

Behcet's Syndrome, Intestinal Type Behcet Syndrome Bone Marrow Diseases Cardiovascular Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Eye Diseases Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hereditary Autoinflammatory Diseases Mouth Diseases Myelodysplastic Syndrome Myelodysplastic Syndromes Panuveitis Skin Diseases Skin Diseases, Genetic Skin Diseases, Vascular Skin and Connective Tissue Diseases Stomatognathic Diseases Uveal Diseases Uveitis Uveitis, Anterior Vascular Diseases Vasculitis

Age range

18 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

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

Beijing, 100044, China

About this study

This is a multicenter, single-arm clinical trial designed to evaluate the efficacy and safety of ustekinumab in patients with intestinal Behçet's syndrome complicated by myelodysplastic syndrome (MDS). A total of 8 patients with intestinal Behçet's syndrome and concomitant MDS will be enrolled. Prior to enrollment, all patients will discontinue any previous biologic agents. Ustekinumab will be administered subcutaneously at a dose of 90 mg at weeks 0, 4, and 8, followed by a maintenance dose of 90 mg every 12 weeks (every 3 months). All patients will be followed for a total of 24 months.

Clinical manifestations, inflammatory biomarkers, and endoscopic findings will be documented throughout the study period. Concomitant medications will be recorded, and adverse events will be systematically monitored to evaluate the efficacy and safety of the treatment.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Meet the 2014 International Criteria for Behçet's Disease (ICBD) for the diagnosis of Behçet's syndrome; Have a confirmed diagnosis of myelodysplastic syndrome (MDS) by bone marrow aspiration and/or biopsy; Have confirmed intestinal ulcers on colonoscopy; Aged between 18 and 70 years; Have active disease at enrollment, defined as a Behçet's Disease Current Activity Form (BDCAF) score ≥ 1; Provide signed informed consent.

Exclusion criteria

Presence of one or more other autoimmune diseases; Involvement of other vital organs (e.g., cardiovascular, neurological) requiring treatment with other biologic agents or high-dose corticosteroids; Receipt of surgical treatment; Severe trilineage cytopenia attributed to myelodysplastic syndrome; Presence of acute or chronic active infection (e.g., bacterial, or viral infections such as EBV, CMV, HIV, or active hepatitis virus) within 4 weeks prior to enrollment; Current or prior history of any malignancy; Pregnancy or within 6 months postpartum; Presence of severe liver failure (Child-Pugh Class C) or end-stage renal disease requiring dialysis.

Treatment and study plan

ustekinumab 90 mg

Drug

Ustekinumab will be administered subcutaneously at a dose of 90 mg at weeks 0, 4, and 8, followed by a maintenance dose of 90 mg every 12 weeks (every 3 months).

Primary outcomes

  1. Patients achieving complete remission, marked improvement, and improvement

    Time frame: Week 24

    The primary endpoint was defined as the proportion of patients achieving each response category at week 24. The response categories included complete remission, marked improvement, and improvement, as defined by the Global Gastrointestinal Symptoms criteria, with the proportion of patients achieving each category expressed as a percentage (%).

Secondary outcomes

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

    Time frame: Week 24

    Clinical manifestations are recorded at enrollment and at week 24, and changes in the BDCAF score from baseline to week 24 are evaluated. The score of BDCAF ranges from 0 to 12, with higher scores indicating greater severity.

  2. Changes of Disease Activity Index for Intestinal Behcet's Disease (DAIBD) of patients

    Time frame: Week 24

    Intestinal-related clinical manifestations are documented at enrollment and at week 24. Changes in the DAIBD score from baseline to week 24 are evaluated. The score of DAIBD ranges from 0 to 295, with higher scores indicating greater severity.

  3. Changes of C-reactive protein

    Time frame: Week 24

    Blood samples were collected from all patients and the concentration of C-reactive protein (mg/L) were recorded.

  4. Changes of erythrocyte sedimentation rate

    Time frame: Week 24

    Blood samples were collected from all patients and the erythrocyte sedimentation rates (mm/h) were recorded.

  5. Changes of dosage of glucocorticoids from baseline

    Time frame: Week 24

    The dosage of glucocorticoids (mg/day) of all patients were recorded during the follow-up.

Sponsors and collaborators

Lead sponsor

Liu Tian

Other

Registry information

Official study title

Efficacy and Safety of Ustekinumab for Intestinal Behçet's Syndrome Complicated by Myelodysplastic Syndrome

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
May 13, 2026
Registry last updated
May 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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