Chidamide Monotherapy for Intermediate-to-High-Risk Myelodysplastic Syndromes
A Single-Arm, Open-Label Phase II Clinical Trial Evaluating the Efficacy and Safety of Chidamide Monotherapy in Participants With Intermediate-to-High-Risk Myelodysplastic Syndromes
Myelodysplastic syndromes (MDS) are a group of bone marrow disorders that can cause low blood cell counts and may progress to acute leukemia. Treatment options for patients with intermediate-to-high-risk MDS are limited, especially for older patients or those who are not suitable for intensive chemotherapy or hypomethylating agents.
Chidamide is an oral histone deacetylase inhibitor that has shown antitumor activity in several hematologic malignancies. This study aims to evaluate the effectiveness and safety of chidamide used alone in patients with intermediate-to-high-risk MDS.
This is a prospective, single-arm, open-label phase II study conducted at a single center. Eligible participants will receive oral chidamide twice weekly and will be followed for treatment response and side effects. The results of this study may help determine whether chidamide could be a potential treatment option for patients with intermediate-to-high-risk MDS who have limited therapeutic choices.
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Descripción detallada
This is a prospective, open-label, single-arm phase II study designed to evaluate the efficacy and safety of chidamide monotherapy in participants with intermediate-to-high-risk myelodysplastic syndromes (MDS) who have limited therapeutic options or are not suitable for intensive chemotherapy or hypomethylating agent therapy.
Eligible participants will receive oral chidamide at an initial dose of 20 mg twice weekly. Each dose consists of four 5-mg tablets taken approximately 30 minutes after meals. Treatment may continue for up to 3 months, with a planned follow-up period of 6 months.
The primary objective is to evaluate the overall response rate (ORR) after chidamide treatment. Treatment response will be assessed according to hematologic response and bone marrow evaluation as specified in the study protocol. Secondary objectives include assessment of safety, tolerability, dose modification, treatment discontinuation, and changes in blood cell counts.
Participants will be followed for treatment response, adverse events, laboratory abnormalities, and overall clinical status for up to 6 months after initiation of study treatment.
Tipo de estudio
Inscripción (Estimado)
Fase
- Fase 2
Contactos y Ubicaciones
Estudio Contacto
- Nombre: Zheng Wei
- Número de teléfono: +86-0592-3569860
- Correo electrónico: wei.zheng@zs-hospital.sh.cn
Ubicaciones de estudio
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Fujian
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Xiamen, Fujian, Porcelana, 361015
- Zhongshan Hospital (Xiamen), Fudan University
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
- Adulto
- Adulto Mayor
Acepta Voluntarios Saludables
Descripción
Inclusion Criteria:
- Aged 60 years or older at the time of informed consent.
- Diagnosis of myelodysplastic syndromes (MDS) according to the World Health Organization (WHO) classification.
- Intermediate-to-high-risk MDS, defined as at least one of the following:
- International Prognostic Scoring System (IPSS) risk category of Intermediate-2 or High, with bone marrow blasts <15%.
- Revised International Prognostic Scoring System (IPSS-R) risk category of Intermediate, High, or Very High, with bone marrow blasts <15%.
- Intermediate-1 risk MDS with grade 1 to 3 anemia and not suitable for hypomethylating agent therapy.
- Evidence of persistent cytopenia affecting one or more hematopoietic lineages for at least 4 months, unless MDS-associated cytogenetic abnormalities or increased blasts are present.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
- Adequate organ function as assessed by laboratory tests within 7 days before enrollment, including liver, renal, and cardiac function, in the opinion of the investigator.
- Ability to understand and willingness to sign a written informed consent form.
Exclusion Criteria:
- Bone marrow blasts >=15% at screening.
- Prior treatment with chidamide.
- Concurrent diagnosis of acute myeloid leukemia (AML) or other active hematologic malignancy.
- Receipt of intensive chemotherapy, hypomethylating agents, or other investigational agents within 4 weeks before enrollment.
- Uncontrolled active infection or severe concurrent medical condition that, in the investigator's judgment, would interfere with study participation.
- Clinically significant cardiac disease, including uncontrolled arrhythmia or clinically relevant QT interval prolongation.
- Known hypersensitivity to chidamide or any of its excipients.
- Participation in another interventional clinical trial at the time of enrollment.
- Any condition that, in the investigator's judgment, would make the participant unsuitable for participation in this study.
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: Chidamide Monotherapy
Participants in this arm will receive oral chidamide monotherapy for the treatment of intermediate-to-high-risk myelodysplastic syndromes.
Chidamide will be administered twice weekly, with dose modification based on safety, tolerability, and hematologic response.
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Chidamide will be administered orally at an initial dose of 20 mg twice weekly. Each dose consists of four 5-mg tablets taken approximately 30 minutes after meals. Treatment may continue for up to 3 months, with a planned follow-up period of 6 months. Dose modifications are permitted based on hematologic and non-hematologic toxicities. In the event of grade 3 or higher hematologic toxicity or clinically significant non-hematologic toxicity, chidamide will be temporarily withheld and may be resumed at a reduced dose after recovery, according to the investigator's judgment. Treatment will be discontinued if unacceptable toxicity persists despite dose interruption or dose reduction.
Otros nombres:
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¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Overall Response Rate Assessed According to Study Protocol-Defined Response Criteria
Periodo de tiempo: Up to 6 months after initiation of study treatment
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Overall response rate is defined as the proportion of participants who achieve a protocol-defined treatment response after chidamide treatment.
Response assessment will be based on hematologic parameters and bone marrow evaluation, as specified in the study protocol.
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Up to 6 months after initiation of study treatment
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Incidence of Treatment-Emergent Adverse Events
Periodo de tiempo: Up to 6 months after initiation of study treatment
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Treatment-emergent adverse events will be assessed based on clinical evaluation, laboratory testing, and investigator assessment during the study period.
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Up to 6 months after initiation of study treatment
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Proportion of Participants With Dose Interruption, Dose Reduction, or Treatment Discontinuation Due to Adverse Events
Periodo de tiempo: Up to 6 months after initiation of study treatment
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This outcome will assess the proportion of participants who require dose interruption, dose reduction, or permanent discontinuation of chidamide due to adverse events.
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Up to 6 months after initiation of study treatment
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Proportion of Participants Achieving Hematologic Improvement
Periodo de tiempo: Up to 6 months after initiation of study treatment
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Hematologic improvement will be assessed based on changes in peripheral blood counts and transfusion requirements according to the study protocol.
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Up to 6 months after initiation of study treatment
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Colaboradores e Investigadores
Patrocinador
Investigadores
- Investigador principal: Zheng Wei, Zhongshan Hospital (Xiamen), Fudan University
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Estimado)
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
Otros números de identificación del estudio
- B2025-040R
Plan de datos de participantes individuales (IPD)
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Información sobre medicamentos y dispositivos, documentos del estudio
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