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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

2026年8月18日 更新者:City of Hope Medical Center

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

This phase II trial tests the effect of total marrow and lymphoid irradiation (TMLI) in combination with fludarabine and melphalan as conditioning regimen in older patients with acute myeloid leukemia or high-risk myelodysplastic syndrome that has not responded to previous treatment (refractory) and that has come back after a period of improvement (relapsed) and are undergoing a donor (allogeneic) peripheral blood stem cell (PBSC) hematopoietic cell transplant (HCT) from a matched related or unrelated donor. HCT is the only curative treatment for high-risk patients, but the side effects related to the current conditioning treatments limit the use to younger and more fit patients. TMLI is a targeted form of total body radiation that uses intensity-modulated radiation therapy to target marrow, lymph node chains, and the spleen. It is designed to reduce radiation-associated side effects and maximize the radiation therapeutic effect. Fludarabine blocks cells from making deoxyribonucleic acid (DNA) and may kill cancer cells. It is a type of purine antagonist and a type of ribonucleotide reductase inhibitor. Melphalan is in a class of medications called alkylating agents. It may kill cancer cells by damaging their DNA and stopping them from dividing. Giving chemotherapy, such as fludarabine and melphalan, and TMLI before an allogeneic transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells to grow. When healthy stem cells from a related or unrelated donor, such as PBSC HCT, that closely match the patient's blood, are infused into a patient, they may help the patient's bone marrow make more healthy cells and platelets, an may help destroy any remaining cancer cells. Giving TMLI in combination with fludarabine and melphalan as conditioning treatment for an allogeneic PBSC HCT from a matched related or unrelated donor may be safe, tolerable, and/or effective in treating high-risk older patients with relapsed and refractory acute myeloid leukemia or high-risk myelodysplastic syndrome.

研究概览

详细说明

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.

研究类型

介入性

注册 (估计的)

35

阶段

  • 阶段2

联系人和位置

本节提供了进行研究的人员的详细联系信息,以及有关进行该研究的地点的信息。

学习地点

    • California
      • Duarte、California、美国、91010
        • 招聘中
        • City of Hope Medical Center
        • 接触:
        • 首席研究员:
          • Monzr M. Al Malki

参与标准

研究人员寻找符合特定描述的人,称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

  • 成人
  • 年长者

接受健康志愿者

不

描述

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)

学习计划

本节提供研究计划的详细信息,包括研究的设计方式和研究的衡量标准。

研究是如何设计的?

设计细节

  • 主要用途:治疗
  • 分配:不适用
  • 介入模型:单组作业
  • 屏蔽:无(打开标签)

武器和干预

参与者组/臂
干预/治疗
实验性的: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.

辅助研究
接受CT
其他名称:
  • CT
  • 猫
  • 电脑扫描
  • 计算机轴向断层扫描
  • 计算机断层扫描
  • CT扫描
  • 断层扫描
  • 计算机轴向断层扫描(程序)
  • 计算机断层扫描 (CT) 扫描
  • 诊断猫扫描
  • 诊断猫扫描服务类型
鉴于IV
其他名称:
  • CB-3025
  • 左旋聚丙烯酰胺
  • L-肌溶酶
  • 丙氨酸氮芥
  • L-苯丙氨酸芥末
  • L-肌溶酶苯丙氨酸芥
  • L-肌赖氨酸
  • 苯丙胺
  • 苯丙氨酸芥末
  • 苯丙氨酸氮芥
  • 肌氯蛋白
  • 溶血酶
  • WR-19813
  • 注射用美法仑-肝输送系统
接受MUGA
其他名称:
  • 血池扫描
  • 平衡放射性核素血管造影
  • 门控血池成像
  • 穆加
  • 放射性核素脑室造影
  • 导航导航仪
  • 司马扫描
  • 同步多门采集扫描
  • 穆加扫描
  • 多门控采集扫描
  • 放射性核素脑室造影扫描
  • 门控心池扫描
  • RNV扫描
鉴于IV
其他名称:
  • 2-F-ara-AMP
  • 益氟乐
  • 氟达拉
  • 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-
  • SH T 586
给予 IV 或 PO
其他名称:
  • 程序
  • FK506
  • 希科里亚
  • FK 506
  • 富士霉素
  • 原主题
  • FK-506
  • 塔克福留斯
鉴于IV
其他名称:
  • 生长因子,重组人角质形成细胞
  • 凯普万斯
  • 角质形成细胞生长因子,重组人
  • 重组人角化细胞生长因子
  • rhKGF
  • rhu 角质形成细胞生长因子
进行骨髓活检
其他名称:
  • 骨髓活检
  • 活组织检查,骨髓
接受TMLI
其他名称:
  • TMLI
给予西罗莫司
其他名称:
  • 雷帕霉素
  • 拉帕蒙
  • AY 22989
  • 拉帕
  • 西拉9268A
  • WY-090217
  • AY-22989
  • AY22989
  • SILA-9268A
  • SILA9268A
  • 威云090217
  • WY090217
进行尿液和血液样本采集
其他名称:
  • 生物样本采集
  • 收集的生物样本
  • 标本采集
经历超声心动图
其他名称:
  • 超声心动图
  • 欧共体
Undergo allogeneic PBSC HCT
其他名称:
  • PBPC移植
  • 外周血祖细胞移植
  • 外周干细胞支持
  • 外周血干细胞移植
  • PBSCT
  • 外周血

研究衡量的是什么?

主要结果指标

结果测量
措施说明
大体时间
Leukemia-free survival
大体时间: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)

次要结果测量

结果测量
措施说明
大体时间
Incidence of adverse events (AEs)
大体时间: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
大体时间: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
大体时间: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
大体时间: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
大体时间: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)
大体时间: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
大体时间: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
大体时间: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

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

调查人员

  • 首席研究员:Monzr M Al Malki、City of Hope Medical Center

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查,以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (估计的)

2027年4月5日

初级完成 (估计的)

2029年4月5日

研究完成 (估计的)

2029年4月5日

研究注册日期

首次提交

2026年5月6日

首先提交符合 QC 标准的

2026年5月6日

首次发布 (实际的)

2026年5月12日

研究记录更新

最后更新发布 (实际的)

2026年8月20日

上次提交的符合 QC 标准的更新

2026年8月18日

最后验证

2026年8月1日

更多信息

此信息直接从 clinicaltrials.gov 网站检索,没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求,请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改,我们的网站上也会自动更新.

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