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.
Visão geral do estudo
Status
Condições
Intervenção / Tratamento
Descrição detalhada
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 estudo
Inscrição (Estimado)
Estágio
- Fase 2
Contactos e Locais
Contato de estudo
- Nome: Zheng Wei
- Número de telefone: +86-0592-3569860
- E-mail: wei.zheng@zs-hospital.sh.cn
Locais de estudo
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Fujian
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Xiamen, Fujian, China, 361015
- Zhongshan Hospital (Xiamen), Fudan University
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Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
- Adulto
- Adulto mais velho
Aceita Voluntários Saudáveis
Descrição
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.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Alocação: N / D
- Modelo Intervencional: Atribuição de grupo único
- Mascaramento: Nenhum (rótulo aberto)
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
|---|---|
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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.
|
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.
Outros nomes:
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O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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Overall Response Rate Assessed According to Study Protocol-Defined Response Criteria
Prazo: 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 resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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Incidence of Treatment-Emergent Adverse Events
Prazo: 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
Prazo: 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
Prazo: 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
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Estimado)
Conclusão Primária (Estimado)
Conclusão do estudo (Estimado)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
- B2025-040R
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