- ICH GCP
- Registro de ensaios clínicos dos EUA
- Ensaio Clínico NCT07588594
Thiotepa-Containing Conditioning Regimen for Allogeneic HSCT in Chronic Myelomonocytic Leukemia
Prospective Single-Arm Clinical Study of Thiotepa-Containing Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation in Chronic Myelomonocytic Leukemia
This is a prospective, single-arm clinical study. It aims to evaluate the effectiveness and safety of a conditioning regimen containing thiotepa (in combination with busulfan and fludarabine, with or without ATG) of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with chronic myelomonocytic leukemia (CMML) who have an intermediate-2 or high-risk prognosis.
The main goal is to evaluate 1 year RFS and OS. Other goals include assessing engraftment, overall survival, transplant-related complications, and side effects. A total of 31 participants will be enrolled.
Visão geral do estudo
Status
Condições
Intervenção / Tratamento
Tipo de estudo
Inscrição (Estimado)
Estágio
- Não aplicável
Contactos e Locais
Contato de estudo
- Nome: Yuqian Sun, MD
- Número de telefone: +86 88326666
- E-mail: sunyuqian83@hotmail.com
Estude backup de contato
- Nome: Xueyi Luo, MD
- Número de telefone: +86 88326666
- E-mail: lll_xxx_yyy@126.com
Locais de estudo
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Beijing, China, 100044
- Recrutamento
- Peking University People's Hospital
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Contato:
- Yuqian Sun, MD
- Número de telefone: +86 010-88326666
- E-mail: sunyuqian83@hotmail.com
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Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
- Adulto
- Adulto mais velho
Aceita Voluntários Saudáveis
Descrição
Inclusion Criteria:
- Age ≥ 18 years, any sex/gender.
- Confirmed diagnosis of chronic myelomonocytic leukemia (CMML) according to the 2022 WHO classification.
- Intermediate-2 or high-risk CMML based on CPSS or CPSS-mol score, and planned to receive allo-HSCT.
- Has a suitable hematopoietic stem cell donor:
- For haploidentical donor: at least 5/10 HLA match at HLA-A, -B, -C, -DQB1, and -DRB1.
- For unrelated donor: at least 9/10 HLA match at the same five loci.
- For matched sibling donor: 10/10 HLA match at the same five loci.
- Hematopoietic cell transplantation comorbidity index (HCT-CI) ≤ 2, with generally good health and no significant organ abnormalities or major comorbidities.
- Adequate organ function as defined below:
- Left ventricular ejection fraction (LVEF) ≥ 50%, and no uncontrolled tachycardia or bradycardia-tachycardia syndrome.
- Total bilirubin ≤ 1.5 × upper limit of normal (ULN); ALT ≤ 2 × ULN; AST ≤ 2 × ULN.
- Serum creatinine ≤ 1.5 × ULN.
- Baseline oxygen saturation > 92%.
- Pulmonary function: DLCO (corrected for hemoglobin) ≥ 40%, FEV1 ≥ 50%.
- Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.
- Agrees not to participate in any other interventional study during the treatment period.
- Willing and able to provide written informed consent, understand the nature, purpose, and procedures of the study, and voluntarily comply with study requirements.
Exclusion Criteria:
- Previous allogeneic HSCT for CMML that later relapsed.
- Unwilling or unable to receive the study treatment regimen.
- Active hepatitis B or C, or chronic active hepatitis; known human immunodeficiency virus (HIV) infection.
- Active uncontrolled infection, including: hemodynamic instability related to infection, new or worsening signs/symptoms of infection, new infection lesions on imaging, or persistent fever without explanation despite no symptoms/signs.
- History of stroke or intracranial hemorrhage within 6 months before enrollment.
- Known pregnancy (positive urine pregnancy test), or currently breastfeeding.
- Diagnosis of another malignancy within the past 2 years, except for localized skin cancer, superficial bladder cancer, carcinoma in situ of the cervix, breast cancer, or localized prostate cancer (Gleason score ≤ 6) that has been treated with curative intent.
- Any other condition that, in the investigator's judgment, makes the patient unsuitable for study participation.
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: Single Arm: Thiotepa + Busulfan + Fludarabine ± ATG
Patients with intermediate-2 or high-risk chronic myelomonocytic leukemia (CMML) per CPSS/CPSS-mol criteria, who are scheduled to receive allogeneic hematopoietic stem cell transplantation (allo-HSCT) with a thiotepa-containing conditioning regimen.
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Intravenous thiotepa 5 mg/kg/d on days -11,-10; busulfan 3.2 mg/kg/d on days -8,-7,-6; fludarabine 30 mg/m²/d on days -6 to -2; ATG per donor type (haplo/unrelated: 2.5 mg/kg/d days -5 to -2; MSD: 1.125 mg/kg/d days -5 to -2).
Allogeneic stem cells infused on day 0. GVHD prophylaxis: CsA, MMF, MTX per protocol.
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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One-year relapse-free survival (RFS) after allogeneic hematopoietic stem cell transplantation
Prazo: At 12 months after allogeneic hematopoietic stem cell transplantation.
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Time from stem cell infusion to hematologic/extramedullary relapse or death, censored at 12 months for event-free participants.
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At 12 months after allogeneic hematopoietic stem cell transplantation.
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Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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One-year overall survival (OS)
Prazo: At 12 months after allogeneic hematopoietic stem cell transplantation.
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Overall survival is defined as the time from stem cell infusion to death from any cause.
Participants alive at 12 months are censored at the last follow-up.
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At 12 months after allogeneic hematopoietic stem cell transplantation.
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One-year cumulative incidence of relapse (RR)
Prazo: At 12 months after allogeneic hematopoietic stem cell transplantation.
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Relapse is defined as recurrence of CMML .
Non-relapse mortality is treated as a competing risk.
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At 12 months after allogeneic hematopoietic stem cell transplantation.
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One-year non-relapse mortality (NRM)
Prazo: At 12 months after allogeneic hematopoietic stem cell transplantation.
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Non-relapse mortality is defined as death from any cause other than disease relapse or progression.
Relapse is treated as a competing risk.
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At 12 months after allogeneic hematopoietic stem cell transplantation.
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Regimen toxicity at day +30
Prazo: Up to day +30 post-transplant
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Toxicity is graded according to NCI CTCAE v5.0.
Any grade 3-5 non-hematologic toxicity occurring within 30 days after stem cell infusion is reported.
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Up to day +30 post-transplant
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Incidence and severity of adverse events (AEs)
Prazo: At 12 months after allogeneic hematopoietic stem cell transplantation.
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AEs are coded using MedDRA and graded per NCI CTCAE v5.0.
All AEs, serious AEs (SAEs), and AEs leading to treatment discontinuation are summarized.
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At 12 months after allogeneic hematopoietic stem cell transplantation.
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Incidence and severity of acute graft-versus-host disease (aGVHD)
Prazo: Up to day +100 post-transplant
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Acute GVHD is diagnosed and graded according to the modified Glucksberg criteria or MAGIC criteria.
Both overall incidence and grade II-IV/III-IV severity are reported.
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Up to day +100 post-transplant
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Incidence and severity of chronic graft-versus-host disease (cGVHD)
Prazo: From day +100 up to 1 year post-transplant
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Chronic GVHD is diagnosed and graded according to the NIH consensus criteria (mild, moderate, severe).
Overall incidence and severity distribution are reported.
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From day +100 up to 1 year post-transplant
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Colaboradores e Investigadores
Patrocinador
Colaboradores
Investigadores
- Investigador principal: Yuqian Sun, MD, Peking University People's Hospital
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Real)
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
Palavras-chave
Termos MeSH relevantes adicionais
- Processos Patológicos
- Neoplasias
- Doença crônica
- Atributos da doença
- Neoplasias por Tipo Histológico
- Doenças Hematológicas
- Leucemia Mieloide
- Doenças mielodisplásicas-mieloproliferativas
- Doenças da Medula Óssea
- Leucemia
- Condições Patológicas, Sinais e Sintomas
- Doenças hemic e linfáticas
- Leucemia Mielomonocítica Crônica
- Compostos de enxofre
- Produtos químicos orgânicos
- Compostos heterocíclicos, 1 anel
- Compostos heterocíclicos
- Hidrocarbonetos, acíclico
- Hidrocarbonetos
- Alcans
- Álcoons
- Butileno glicols
- Glicols
- Mesilatos
- Alkanesulfonatos
- Ácidos alcanesulfônicos
- Ácidos sulfônicos
- Ácidos de enxofre
- Fosforamidas
- Compostos organofosforos
- Trietilenefosforamida
- Aziridinas
- Azirines
- Bussulfano
- Tiotepa
- fludarabina
Outros números de identificação do estudo
- 2025PHD048-001
Plano para dados de participantes individuais (IPD)
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Descrição do plano IPD
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