Ustekinumab for Intestinal Behçet's Syndrome With Myelodysplastic Syndrome
Efficacy and Safety of Ustekinumab for Intestinal Behçet's Syndrome Complicated by Myelodysplastic Syndrome
Descripción general del estudio
Estado
Intervención / Tratamiento
Descripción detallada
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.
Tipo de estudio
Inscripción (Actual)
Fase
- Fase 2
Contactos y Ubicaciones
Ubicaciones de estudio
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Beijing, Porcelana, 100044
- Department of Rheumatology and Immunology, Peking University People's Hospital
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
- Adulto
- Adulto Mayor
Acepta Voluntarios Saludables
Descripción
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.
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: N / A
- Modelo Intervencionista: Asignación de un solo grupo
- Enmascaramiento: Ninguno (etiqueta abierta)
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
|---|---|
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Experimental: Treated with ustekinumab
Eight patients with intestinal Behçet's syndrome complicated by myelodysplastic syndrome were treated with ustekinumab.
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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).
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¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Patients achieving complete remission, marked improvement, and improvement
Periodo de tiempo: Week 24
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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 (%).
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Week 24
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Changes of Behcet's Disease Current Activity Form (BDCAF) score of patients
Periodo de tiempo: Week 24
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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.
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Week 24
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Changes of Disease Activity Index for Intestinal Behcet's Disease (DAIBD) of patients
Periodo de tiempo: Week 24
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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.
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Week 24
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Changes of C-reactive protein
Periodo de tiempo: Week 24
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Blood samples were collected from all patients and the concentration of C-reactive protein (mg/L) were recorded.
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Week 24
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Changes of erythrocyte sedimentation rate
Periodo de tiempo: Week 24
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Blood samples were collected from all patients and the erythrocyte sedimentation rates (mm/h) were recorded.
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Week 24
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Changes of dosage of glucocorticoids from baseline
Periodo de tiempo: Week 24
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The dosage of glucocorticoids (mg/day) of all patients were recorded during the follow-up.
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Week 24
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Colaboradores e Investigadores
Patrocinador
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Actual)
Finalización del estudio (Actual)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Términos MeSH relevantes adicionales
- Enfermedades de la Boca
- Enfermedades Estomatognáticas
- Enfermedades Vasculares
- Enfermedades cardiovasculares
- Enfermedades Genéticas Congénitas
- Enfermedades de los ojos
- Enfermedades hematológicas
- Enfermedades de la piel
- Enfermedades de la médula ósea
- Enfermedades De La Piel Vasculares
- Enfermedades De La Piel Genéticas
- Enfermedades uveales
- Vasculitis
- Panuveítis
- Uveítis Anterior
- Uveítis
- Enfermedades autoinflamatorias hereditarias
- Enfermedades y anomalías congénitas, hereditarias y neonatales
- Enfermedades de la piel y del tejido conectivo
- Enfermedades hemic y linfáticas
- Síndromes mielodisplásicos
- Síndrome de Behçet
- Aminoácidos, péptidos y proteínas
- Proteínas
- Anticuerpos, monoclonales, humanizados
- Anticuerpos, monoclonal
- Anticuerpos
- Inmunoglobulinas
- Inmunoproteínas
- Proteínas de la sangre
- Globulinas séricas
- Globulinas
- Ustekinumab
Otros números de identificación del estudio
- Peking University PH
- PAYJ-023 (Otro número de subvención/financiamiento: Bethune-Puai Medical Research Fund)
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
producto fabricado y exportado desde los EE. UU.
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