Ustekinumab for Intestinal Behçet's Syndrome With Myelodysplastic Syndrome

NCT07582991 | Completed Phase 2 DMC

• 2 other identifiers: Peking University PHPAYJ-023
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Behcet's Syndrome, Intestinal Type; Myelodysplastic Syndrome

Keywords

Behcet's Syndrome, Intestinal Type; Myelodysplastic Syndrome; Ustekinumab

Outcome Measures

Primary Outcomes (1)

• Patients achieving complete remission, marked improvement, and improvement

  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 (%).

  Week 24

Secondary Outcomes (5)

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

  Week 24

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

  Week 24

• Changes of C-reactive protein

  Week 24

• Changes of erythrocyte sedimentation rate

  Week 24

• Changes of dosage of glucocorticoids from baseline

  Week 24

Study Arms (1)

Treated with ustekinumab

EXPERIMENTAL

Eight patients with intestinal Behçet's syndrome complicated by myelodysplastic syndrome were treated with ustekinumab.

Drug: Ustekinumab 90 mg

Interventions

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).

Treated with ustekinumab

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Liu Tian: lead

Study Sites (1)

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

Beijing, 100044, China

Location

MeSH Terms

Conditions

Behcet Syndrome; Myelodysplastic Syndromes

Interventions

Ustekinumab

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; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

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: Professor

Model Details: Eight patients with intestinal Behçet's syndrome complicated by myelodysplastic syndrome

Study Record Dates

• First Submitted: April 23, 2026
• First Posted: May 13, 2026
• Study Start: March 24, 2024
• Primary Completion: February 23, 2026
• Study Completion: February 23, 2026
• Last Updated: May 13, 2026

Record last verified: 2026-03

Locations

CN (1)