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- Registro de ensayos clínicos de EE. UU.
- Ensayo 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.
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Tipo de estudio
Inscripción (Estimado)
Fase
- No aplica
Contactos y Ubicaciones
Estudio Contacto
- Nombre: Yuqian Sun, MD
- Número de teléfono: +86 88326666
- Correo electrónico: sunyuqian83@hotmail.com
Copia de seguridad de contactos de estudio
- Nombre: Xueyi Luo, MD
- Número de teléfono: +86 88326666
- Correo electrónico: lll_xxx_yyy@126.com
Ubicaciones de estudio
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Beijing, Porcelana, 100044
- Reclutamiento
- Peking University People's Hospital
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Contacto:
- Yuqian Sun, MD
- Número de teléfono: +86 010-88326666
- Correo electrónico: sunyuqian83@hotmail.com
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
- Adulto
- Adulto Mayor
Acepta Voluntarios Saludables
Descripción
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.
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: 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.
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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One-year relapse-free survival (RFS) after allogeneic hematopoietic stem cell transplantation
Periodo de tiempo: 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 resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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One-year overall survival (OS)
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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
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
Palabras clave
Términos MeSH relevantes adicionales
- Procesos Patológicos
- Neoplasias
- Enfermedad crónica
- Atributos de la enfermedad
- Neoplasias por tipo histológico
- Enfermedades hematológicas
- Leucemia Mieloide
- Enfermedades mielodisplásicas-mieloproliferativas
- Enfermedades de la médula ósea
- Leucemia
- Condiciones Patológicas, Signos y Síntomas
- Enfermedades hemic y linfáticas
- Leucemia Mielomonocítica Crónica
- Compuestos de azufre
- Químicos orgánicos
- Compuestos heterocíclicos, 1 anillo
- Compuestos heterocíclicos
- Hidrocarburos, acíclico
- Hidrocarburos
- Alcanos
- Alcoholes
- Butilenglicoles
- Glicolos
- Mesilatos
- Alcanosulfonatos
- Ácidos alcanosulfónicos
- Ácidos sulfónicos
- Ácidos de azufre
- Fosforamidas
- Compuestos organofosforados
- Trietilenefosforamida
- Aziridinas
- Azirines
- Busulfán
- Tiotepa
- fludarabina
Otros números de identificación del estudio
- 2025PHD048-001
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
Descripción del plan IPD
Información sobre medicamentos y dispositivos, documentos del estudio
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Estudia un producto de dispositivo regulado por la FDA de EE. UU.
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