- ICH GCP
- Registro de ensayos clínicos de EE. UU.
- Ensayo clínico NCT07582172
Total Marrow and Lymphoid Irradiation in Combination With Fludarabine and Melphalan as Conditioning for Allogeneic Peripheral Blood Stem Cell Hematopoietic Cell Transplant in Older Patients With Refractory and Relapsed Acute Myeloid Leukemia and High-risk Myelodysplastic Syndrome
Phase 2 Trial of Total Marrow and Lymphoid Irradiation (TMLI) as Conditioning for Allogeneic Peripheral Blood Stem Cell Hematopoietic Cell Transplantation (PBSC-HCT) From a Match Donor With Fludarabine and Melphalan in Older Patients With Refractory Acute Myeloid Leukemia and MDS
Descripción general del estudio
Estado
Condiciones
- Síndrome mielodisplásico
- Leucemia mieloide aguda recurrente
- Síndrome mielodisplásico recurrente
- Leucemia mieloide aguda refractaria
- Síndrome mielodisplásico refractario
- Leucemia mieloide aguda secundaria recurrente
- Leucemia mieloide aguda secundaria refractaria
- Acute Myeloid Leukemia With Complex Karyotype
- Myelodysplastic Chronic Myelomonocytic Leukemia
- Recurrent Myelodysplastic Chronic Myelomonocytic Leukemia
- Refractory Myelodysplastic Chronic Myelomonocytic Leukemia
Intervención / Tratamiento
- Otro: Administración del Cuestionario
- Procedimiento: Tomografía computarizada
- Droga: Melfalán
- Procedimiento: Escaneo de adquisición multipuerta
- Droga: Fosfato de fludarabina
- Droga: Tacrolimus
- Biológico: Palifermin
- Procedimiento: Biopsia de Médula Ósea
- Radiación: Irradiación linfoide y de médula total
- Droga: Sirolimus
- Procedimiento: Colección de muestras biológicas
- Procedimiento: Prueba de ecocardiografía
- Procedimiento: Peripheral Blood Stem Cell Transplantation
Descripción detallada
PRIMARY OBJECTIVE:
I. Assess the efficacy of the total marrow and lymphoid irradiation (TMLI)-based therapy at the recommended phase 2 dose (RP2D) of 1600 cGy prior to hematopoietic cell transplant (HCT) for older patients (≥ 50 years of age) with refractory and relapsed acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) undergoing peripheral blood stem cell (PBSC) HCT from matched related/unrelated donor, as measured by 2-years leukemia-free survival (LFS).
SECONDARY OBJECTIVES:
I. Further evaluation of safety of the TMLI-based conditioning regimen, by assessing the following: type, frequency, severity, attribution, time course and duration of adverse events, including acute/chronic GVHD, infection and delayed engraftment.
II. Estimate overall survival (OS: at 1 and 2 years post-HCT), cumulative incidence (CI) of relapse/progression (at 1 and 2 years post-HCT), and non-relapse mortality (NRM) at 100 days, 1 year and 2 years post-HCT.
III. Estimate the cumulative incidence and severity of acute graft-versus-host disease (GVHD) by day 180 using Malignant Germ Cell International Consortium (MAGIC) grading and chronic GVHD by 1 and 2 years post-HCT using National Institutes of Health (NIH) consensus criteria.
IV. Estimate the cumulative incidence of GVHD-free and relapse-free survival (GRFS) at 1-year post-HCT.
EXPLORATORY OBJECTIVES:
I. Collect longitudinal blood samples for immune analysis II. Collect longitudinal blood samples to assess presence and levels of GVHD biomarkers and inflammatory cytokines III. Collect longitudinal bone marrow samples to assess changes in the bone marrow environment after TMLI.
IV. Collect longitudinal blood samples for circulating tumor DNA (ctDNA) profiling.
V. Collect longitudinal stool samples to explore the potential effects of lower gastrointestinal (GI) tract radiation exposure on microbiome composition and HCT outcomes.
VI. In patients ≥ 50 years old, evaluate physical function and quality of life and cognitive impairment using Cancer Health Assessments Reaching Many (CHARM) assessments at baseline then frailty assessments, Patient Reported Outcomes Measurement Information Systems (PROMIS) Physical Function and Montreal cognitive assessment on day 100 and 180-, and 1-year post-HCT.
OUTLINE:
Patients receive palifermin intravenously (IV) on days -11, -10, -9, 0, 1 and 2, fludarabine IV on days -4 to -2 and melphalan IV on day -2 and undergo TMLI twice daily (BID) for 8 fractions on days -8 to -5. Patients receive allogeneic PBSC-HCT on day 0. Starting on day -1, patients also receive tacrolimus IV or orally (PO) once daily (QD) and sirolimus QD per standard of care.
Additionally, patients undergo echocardiography or multigated acquisition scan (MUGA), computed tomography (CT), urine and blood sample collection, and bone marrow biopsy throughout the study.
After completion of study treatment, patients are followed up at 30, 60, 100 and 180 days and at 1 and 2 years.
Tipo de estudio
Inscripción (Estimado)
Fase
- Fase 2
Contactos y Ubicaciones
Ubicaciones de estudio
-
-
California
-
Duarte, California, Estados Unidos, 91010
- Reclutamiento
- City of Hope Medical Center
-
Contacto:
- Monzr M. Al Malki
- Número de teléfono: 626-218-2405
- Correo electrónico: malamalki@coh.org
-
Investigador principal:
- Monzr M. Al Malki
-
-
Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
- Adulto
- Adulto Mayor
Acepta Voluntarios Saludables
Descripción
Inclusion Criteria:
Documented informed consent of the participant and/or legally authorized representative
- Assent, when appropriate, will be obtained per institutional guidelines
Agreement to allow the use of archival tissue from diagnostic tumor biopsies
- If unavailable, exceptions may be granted with study principal investigator (PI) approval
Age: ≥ 50 years (no upper age limit)
- Note: Patients ≥ 18 years and < 50 years are also included if they are not candidates for myeloablative conditioning regimens due to comorbidities or active disease
- Karnofsky or Lansky performance status ≥ 70
Eligible patients will have a histopathological confirmed diagnosis of hematologic malignancy in one of the following categories:
Acute myeloid leukemia (AML):
- Patients with de novo or secondary disease in unfavorable risk group including poor risk cytogenetics according to National Comprehensive Cancer Network (NCCN) guidelines for AML i.e., monosomal karyotype, -5,5q-,-7,7q-,11q23-non t(9;11), inv (3), t(3;3), t(6;9), t(9;22) and complex karyotypes (≥ 3 unrelated abnormalities), or all patient in intermediate risk groups
Patients with active disease:
- Morphologically
- Minimal residual disease (MRD) testing (MRD+ through flow cytometry, cytogenetics, or molecular assays)
- Myelodysplastic syndrome/chronic myelomonocytic leukemia (CMML) (MDS) with ≥ 10% blast
- Patients must have an human leukocyte antigen (HLA) (A, B, C, and DRB1) identical sibling or a 8/8 (A, B, C, and DR) allele matched unrelated donor who is willing to donate primed blood stem cells
- Serum direct bilirubin ≤ 2.0 x upper limit of normal (ULN) (unless has Gilbert's disease) (within 35 days prior to day 1 of protocol therapy unless otherwise stated)
- Aspartate aminotransferase (AST) ≤ 2.5 x ULN (within 35 days prior to day 1 of protocol therapy unless otherwise stated)
- Alanine aminotransferase (ALT) ≤ 2.5 x ULN (within 35 days prior to day 1 of protocol therapy unless otherwise stated)
- Creatinine clearance of ≥ 60 mL/min per 24 hour urine test or the Cockcroft-Gault formula (within 35 days prior to day 1 of protocol therapy unless otherwise stated)
Left ventricular ejection fraction (LVEF) ≥ 50%
- Note: To be performed within 35 days prior to day 1 of protocol therapy
If able to perform pulmonary function tests: Forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC) and DLCO (diffusion capacity) ≥ 50% of predicted (corrected for hemoglobin)
- Note: To be performed within 35 days prior to day 1 of protocol therapy
If unable to perform pulmonary function tests: Oxygen (O2) saturation > 92% on room air
- Note: To be performed within 35 days prior to day 1 of protocol therapy
Seronegative for HIV antigen (Ag)/antibody (Ab) combo, hepatitis C virus (HCV), active hepatitis B virus (HBV) (surface antigen negative) (within 35 days prior to day 1 of protocol therapy unless otherwise stated)
- If seropositive for HIV, HCV or HBV, nucleic acid quantitation must be performed. Viral load must be undetectable
- HIV-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial
Meets institutional and federal requirements for infectious disease titer requirements
- Note: Infectious disease testing to be performed within 28 days prior to day 1 of protocol therapy
Women of childbearing potential (WOCBP): Negative urine or serum pregnancy test (within 35 days prior to day 1 of protocol therapy unless otherwise stated)
- If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required
Agreement by females and males of childbearing potential to use an effective method of birth control or abstain from heterosexual activity for the course of the study through at least 6 months after the last dose of protocol therapy
- Childbearing potential defined as not being surgically sterilized (men and women) or have not been free from menses for > 1 year (women only)
Exclusion Criteria:
- Allogeneic stem cell transplant or autologous HCT within 1 year prior to day 1 of protocol therapy
Chemotherapy, radiation therapy, biological therapy, immunotherapy within 14 days of day 1 of protocol therapy
- Note: Low dose chemotherapy or maintenance chemotherapy given within 7 days of planned study enrollment is permitted. These include hydroxyurea, 6-meraptopurine, oral methotrexate, vincristine, oral etoposide, and tyrosine kinase inhibitors (TKIs). TKIs can also be given up to 3-5 days before conditioning regimen
- More than three previous lines of intensive chemotherapy, where the regimen intent was to induce remission
- Co-enrollment in other clinical trials involving post-HCT maintenance interventions or any study with potential to affect disease-free survival is not allowed
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to study agent
- Clinically significant uncontrolled illness
- Active infection not responding to antibiotics
- Other active malignancy. Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial
- Females only: Pregnant or breastfeeding
- Any other condition that would, in the investigator's judgment, contraindicate the patient's participation in the clinical study due to safety concerns with clinical study procedures
- Prospective participants who, in the opinion of the investigator, may not be able to comply with all study procedures (including compliance issues related to feasibility/logistics)
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 |
|---|---|
|
Experimental: Treatment (TMLI, fludarabine, melphalan, allogeneic PBSC-HCT)
Patients receive palifermin IV on days -11, -10, -9, 0, 1 and 2, fludarabine IV on days -4 to -2 and melphalan IV on day -2 and undergo TMLI BID for 8 fractions on days -8 to -5. Patients receive allogeneic PBSC-HCT on day 0. Starting on day -1, patients also receive tacrolimus IV or PO QD and sirolimus QD per standard of care. Additionally, patients undergo echocardiography or MUGA, CT, urine and blood sample collection, and bone marrow biopsy throughout the study. |
Estudios complementarios
Someterse a una tomografía computarizada
Otros nombres:
Dado IV
Otros nombres:
Someterse a MUGA
Otros nombres:
Dado IV
Otros nombres:
Dado IV o PO
Otros nombres:
Dado IV
Otros nombres:
Someterse a una biopsia de médula ósea
Otros nombres:
Someterse a TMLI
Otros nombres:
Se le administró sirolimus
Otros nombres:
Someterse a una recolección de muestras de orina y sangre.
Otros nombres:
Sufrir ecocardiografía
Otros nombres:
Undergo allogeneic PBSC HCT
Otros nombres:
|
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Leukemia-free survival
Periodo de tiempo: From the date of stem cell infusion to the date of first observation of relapse/progression, or date of death, whichever comes first, assessed at 2 years post-hematopoietic cell transplantation (HCT)
|
Will be calculated using the Kaplan-Meier method.
Point estimates and 95% confidence intervals will be provided.
|
From the date of stem cell infusion to the date of first observation of relapse/progression, or date of death, whichever comes first, assessed at 2 years post-hematopoietic cell transplantation (HCT)
|
Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Incidence of adverse events (AEs)
Periodo de tiempo: From day 1 of protocol therapy up to day 30 post-HCT
|
Will be scored on both the Bearman Scale and National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version (v) 5.0.
Will be summarized by type, attribution, grade and duration.
|
From day 1 of protocol therapy up to day 30 post-HCT
|
|
Incidence of highest grades of AEs
Periodo de tiempo: From day 31 up to day 100 post-HCT
|
Will be scored on both the Bearman Scale and NCI CTCAE v 5.0.
Will be summarized by type, attribution, grade and duration.
|
From day 31 up to day 100 post-HCT
|
|
Overall survival
Periodo de tiempo: From date of stem cell infusion t the date of death, assessed at 1 and 2 years post-HCT
|
Will be calculated using the Kaplan-Meier method.
Point estimates and 95% confidence intervals will be provided.
|
From date of stem cell infusion t the date of death, assessed at 1 and 2 years post-HCT
|
|
Relapse
Periodo de tiempo: From date of stem cell infusion to first observation of relapse/progression, assessed at 1 and 2 years post-HCT
|
The cumulative incidence of will be calculated using the competing risk method as described by Gooley et al. (1999).
Point estimates and 95% confidence intervals will be provided for each outcome measure.
|
From date of stem cell infusion to first observation of relapse/progression, assessed at 1 and 2 years post-HCT
|
|
Non-relapse mortality
Periodo de tiempo: From date of stem cell infusion until non-disease related death, assessed at 100 days, and at 1 and 2 years post-HCT
|
The cumulative incidence of will be calculated using the competing risk method as described by Gooley et al. (1999).
Point estimates and 95% confidence intervals will be provided for each outcome measure.
|
From date of stem cell infusion until non-disease related death, assessed at 100 days, and at 1 and 2 years post-HCT
|
|
Acute graft-versus-host disease (GVHD)
Periodo de tiempo: From date of stem cell infusion to document/biopsy proven acute GVHD onset date, assessed up to 180 days post-transplant
|
Will be graded according to the 1994 Keystone Consensus Grading.
The cumulative incidence of will be calculated using the competing risk method as described by Gooley et al. (1999).
Point estimates and 95% confidence intervals will be provided for each outcome measure.
|
From date of stem cell infusion to document/biopsy proven acute GVHD onset date, assessed up to 180 days post-transplant
|
|
Chronic GVHD
Periodo de tiempo: From approximately 80-100 days post-transplant to the documented/biopsy proven chronic GVHD onset date, assessed at 1 and 2 years
|
Will be scored according to Jagasia et al.
The cumulative incidence of will be calculated using the competing risk method as described by Gooley et al. (1999).
Point estimates and 95% confidence intervals will be provided for each outcome measure.
|
From approximately 80-100 days post-transplant to the documented/biopsy proven chronic GVHD onset date, assessed at 1 and 2 years
|
|
GVHD-free and relapse-free survival
Periodo de tiempo: From the date of stem cell infusion to garde 3-4 acute GVHD, chronic GVHD requiring systemic treatment, relapse/progression, whichever comes first, assessed at 1 year post-HCT
|
Will be calculated using the Kaplan-Meier method.
Point estimates and 95% confidence intervals will be provided.
|
From the date of stem cell infusion to garde 3-4 acute GVHD, chronic GVHD requiring systemic treatment, relapse/progression, whichever comes first, assessed at 1 year post-HCT
|
Colaboradores e Investigadores
Patrocinador
Colaboradores
Investigadores
- Investigador principal: Monzr M Al Malki, City of Hope Medical Center
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
- Neoplasias
- Neoplasias por tipo histológico
- Enfermedades hematológicas
- Leucemia Mieloide
- Enfermedades de la médula ósea
- Leucemia
- Enfermedades hemic y linfáticas
- Leucemia Mieloide Aguda
- Síndromes mielodisplásicos
- Péptidos
- Aminoácidos, péptidos y proteínas
- Proteínas
- Químicos orgánicos
- Técnicas de investigación
- Terapéutica
- Técnicas de laboratorio clínico
- Técnicas y procedimientos de diagnóstico
- Diagnóstico
- Procedimientos quirúrgicos, operativo
- Técnicas citológicas
- Citodiagnóstico
- Hidrocarburos
- Factores biológicos
- Trasplante
- Aminoácidos
- Técnicas de diagnóstico, quirúrgico
- Macrólidos
- Lactonas
- Compuestos de mostaza de nitrógeno
- Compuestos de mostaza
- Hidrocarburos, halogenados
- Péptidos de señalización intercelular y proteínas
- Radioterapia
- Trasplante de células
- Terapia basada en células y tejidos
- Terapia biológica
- Fenilalanina
- Aminoácidos, aromáticos
- Aminoácidos, cíclico
- Trasplante de células madre
- Factores de crecimiento de fibroblastos
- Trasplante de células madre hematopoyéticas
- Sirolimus
- Melfalán
- Tacrolimús
- Biopsia
- Manejo de muestras
- fosfato de fludarabina
- Factor de crecimiento de fibroblastos 7
- Trasplante de células madre de sangre periférica
- Irradiación Linfática
Otros números de identificación del estudio
- 25663 (Otro identificador: City of Hope Medical Center)
- P30CA033572 (Subvención/contrato del NIH de EE. UU.)
- NCI-2026-03208 (Identificador de registro: CTRP (Clinical Trial Reporting Program))
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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