A Prospective Study of Madecassoside Tablets in Sclerotic Chronic Graft-versus-Host Disease After Allo-HSCT
A Prospective, Multicenter, Single-Arm Phase 2 Study of Madecassoside Tablets Combined With Standard Therapy for Sclerotic Chronic Graft-versus-Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation
Sclerotic chronic graft-versus-host disease (scl-cGVHD) is a severe subtype of chronic GVHD after allogeneic hematopoietic stem cell transplantation (allo-HSCT), characterized by skin sclerosis, fascial involvement, and restricted joint mobility, leading to substantial functional impairment and reduced quality of life. Current standard therapies, including corticosteroids and other immunosuppressive agents, have limited efficacy in patients with refractory or steroid-dependent disease.
Madecassoside tablets, a standardized extract of Centella asiatica, have demonstrated anti-inflammatory and anti-fibrotic properties in previous studies and may provide therapeutic benefit in fibrotic diseases.
This study is a prospective, multicenter, single-arm phase 2 clinical trial designed to evaluate the efficacy and safety of madecassoside tablets combined with standard therapy in patients with scl-cGVHD after allo-HSCT. Eligible participants will receive oral madecassoside tablets (0.2 g three times daily) in addition to standard treatment for 6 months. Clinical assessments will be performed at baseline and at weeks 4, 8, 12, and 24.
The primary endpoint is the improvement rate of skin and/or joint/fascia NIH cGVHD scores at 6 months according to the 2014 NIH consensus criteria. Secondary endpoints include overall cGVHD response rate, failure-free survival, non-relapse mortality, corticosteroid dose reduction, patient-reported outcomes, and safety assessments. Peripheral blood samples will also be collected to explore changes in inflammatory cytokines and lymphocyte subsets during treatment.
Descripción general del estudio
Estado
Intervención / Tratamiento
Tipo de estudio
Inscripción (Estimado)
Fase
- Fase 2
Contactos y Ubicaciones
Estudio Contacto
- Nombre: wei MD, PhD
- Número de teléfono: +86 027-85726003
- Correo electrónico: shiwei076@hust.edu.cn
Ubicaciones de estudio
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Hubei
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Wuhan, Hubei, Porcelana, 430022
- Reclutamiento
- Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
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Contacto:
- Wei Shi, M.D.
- Número de teléfono: +86 027-85726003
- Correo electrónico: shiwei076@hust.edu.cn
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
- Niño
- Adulto
- Adulto Mayor
Acepta Voluntarios Saludables
Descripción
Inclusion Criteria:
- Ability to understand and willingness to sign written informed consent;
- Age 14 to 65 years;
- Diagnosis of sclerotic chronic graft-versus-host disease (scl-cGVHD) according to the 2014 NIH consensus criteria, including superficial or deep skin sclerosis, fasciitis, or restricted joint mobility;
Prior treatment history meeting at least one of the following:
- Received systemic corticosteroid therapy for ≥12 months and at least 1 additional systemic therapy; OR
- Received corticosteroid therapy and at least 2 additional systemic therapies;
- Karnofsky Performance Status (KPS) score ≥60;
- Absolute neutrophil count >1,000/μL;
- Platelet count ≥50,000/μL.
Exclusion Criteria:
- 1. Total bilirubin >1.5 times the upper limit of normal; 2. Creatinine clearance <30 mL/min; 3. Uncontrolled infection; 4. Uncontrolled cardiovascular or pulmonary disease; 5. Any clinical condition that, in the investigator's judgment, makes the participant unsuitable for study participation.
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: Experimental: Madecassoside Tablets Plus Standard Therapy
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Madecassoside tablets will be administered orally at a dose of 0.2 g (2 tablets) three times daily for 6 months in combination with standard therapy.
Standard therapy may include corticosteroids, calcineurin inhibitors, ruxolitinib, belumosudil, topical medications, and supportive care according to institutional guidelines and physician discretion.
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¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Change in Skin and/or Joint/Fascia NIH cGVHD Scores
Periodo de tiempo: 6 months
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The proportion of participants achieving complete response (CR), partial response (PR), stable disease (SD), or progressive disease (PD) in skin and/or joint/fascia manifestations according to the 2014 NIH chronic graft-versus-host disease response criteria after 6 months of treatment.
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6 months
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Overall cGVHD Response Rate
Periodo de tiempo: 6 months
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Overall response rate of chronic graft-versus-host disease based on the 2014 NIH response criteria.
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6 months
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Failure-Free Survival
Periodo de tiempo: 2 years
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Failure-free survival defined as survival without relapse, non-relapse mortality, or addition of new systemic therapy.
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2 years
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Non-Relapse Mortality
Periodo de tiempo: 2 years
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Incidence of death without relapse of the underlying disease during follow-up.
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2 years
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Corticosteroid Dose Reduction
Periodo de tiempo: 6 months
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Proportion of participants achieving at least 50% reduction or discontinuation of corticosteroid therapy.
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6 months
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Patient-Reported Outcomes
Periodo de tiempo: Baseline to 6 months
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Changes in patient-reported outcomes including Lee Symptom Scale (LSS), Scleroderma Health Assessment Questionnaire (SHAQ), and Photographic Range of Motion (P-ROM) scores.
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Baseline to 6 months
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Traditional Chinese Medicine Symptom Score
Periodo de tiempo: Baseline to 6 months
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Changes in Traditional Chinese Medicine symptom scores during treatment.
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Baseline to 6 months
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Safety and Adverse Events
Periodo de tiempo: Up to 2 years
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Incidence and severity of adverse events assessed according to CTCAE version 5.0.
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Up to 2 years
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Colaboradores e Investigadores
Patrocinador
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Estimado)
Finalización del estudio (Estimado)
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
- Organización de la neumonía
- Enfermedades del sistema inmunológico
- Enfermedades de las vías respiratorias
- Enfermedades pulmonares
- Enfermedades bronquiales
- Enfermedades Pulmonares Obstructivas
- Bronquiolitis Obliterante
- Bronquiolitis
- Bronquitis
- Síndrome de bronquiolitis obliterante
- Enfermedad de injerto contra huésped
- Administración de Servicios de Salud
- Calidad, acceso y evaluación de la atención médica
- Calidad de la atención médica
- Indicadores de calidad, atención médica
- Estándar de cuidado
- madecassoside
Otros números de identificación del estudio
- OMD-HSCT-20250817
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
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.
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