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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

2026년 5월 6일 업데이트: 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.

연구 개요

상세 설명

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.

연구 유형

중재적

등록 (추정된)

35

단계

  • 2 단계

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 장소

    • California
      • Duarte, California, 미국, 91010
        • City of Hope Medical Center
        • 연락하다:
        • 수석 연구원:
          • Monzr M. Al Malki

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

  • 성인
  • 고령자

건강한 자원 봉사자를 받아들입니다

아니

설명

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)

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 해당 없음
  • 중재 모델: 단일 그룹 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: 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.

보조 연구
CT를 받다
다른 이름들:
  • CT
  • 고양이
  • 고양이 스캔
  • 컴퓨터 축 단층 촬영
  • 전산화 단층 촬영
  • CT 스캔
  • 단층 촬영
  • 컴퓨터 축 단층 촬영(시술)
  • 컴퓨터 단층촬영(CT) 스캔
  • 진단 CAT 스캔
  • 진단 CAT 스캔 서비스 유형
주어진 IV
다른 이름들:
  • CB-3025
  • 엘팜
  • L-사르콜리신
  • 알라닌 질소 머스타드
  • L-페닐알라닌 머스타드
  • L-사콜리신 페닐알라닌 머스타드
  • L-사르콜라이신
  • 멜파라눔
  • 페닐알라닌 머스타드
  • 페닐알라닌 질소 머스타드
  • 사코클로린
  • 사르콜리신
  • WR-19813
  • 간 주사 전달 시스템용 Melphalan
무가를 겪다
다른 이름들:
  • 혈액 풀 스캔
  • 평형 방사성핵종 혈관조영술
  • 게이티드 혈액 풀 이미징
  • 무가
  • 방사성핵종 뇌실조영술
  • RNVG
  • SYMA 스캐닝
  • 동기화된 다중 게이트 수집 스캐닝
  • 무가 스캔
  • 다중 게이트 획득 스캔
  • 방사성핵종 뇌실도 스캔
  • 게이트 하트 풀 스캔
  • RNV 스캔
주어진 IV
다른 이름들:
  • 2-F-ara-AMP
  • 베네플러
  • 플루다라
  • 9H-퓨린-6-아민, 2-플루오로-9-(5-O-포스포노-.베타.-D-아라비노푸라노실)-
  • SH T 586
주어진 IV 또는 PO
다른 이름들:
  • 프로그라프
  • FK506
  • 헤코리아
  • FK 506
  • 후지마이신
  • 프로토픽
  • FK-506
  • 탁포리우스
주어진 IV
다른 이름들:
  • 성장인자, 재조합 인간 케라티노사이트
  • 케피밴스
  • 케라티노사이트 성장 인자, 재조합 인간
  • 재조합 인간 케라티노사이트 성장 인자
  • rhKGF
  • rhu 케라티노사이트 성장 인자
골수 생검을 받다
다른 이름들:
  • 골수 생검
  • 생검, 골수
TMLI를 받다
다른 이름들:
  • TMLI
시롤리무스 제공
다른 이름들:
  • 라파마이신
  • 라파뮨
  • AY 22989
  • 라파
  • 실라 9268A
  • WY-090217
  • AY-22989
  • AY22989
  • SILA-9268A
  • SILA9268A
  • WY 090217
  • WY090217
소변 및 혈액 샘플 수집을 수행합니다.
다른 이름들:
  • 생물학적 샘플 수집
  • 생체 표본 수집
  • 표본 수집
심 초음파 검사를받습니다
다른 이름들:
  • 심초음파
  • EC
Undergo allogeneic PBSC HCT
다른 이름들:
  • PBPC 이식
  • 말초 혈액 전구 세포 이식
  • 말초 줄기 세포 지원
  • 말초 줄기 세포 이식
  • PBSCT
  • 말초혈액 줄기세포 이식
  • 말초 혈액

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Leukemia-free survival
기간: 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)

2차 결과 측정

결과 측정
측정값 설명
기간
Incidence of adverse events (AEs)
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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)
기간: 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
기간: 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
기간: 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

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

수사관

  • 수석 연구원: Monzr M Al Malki, City of Hope Medical Center

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (추정된)

2027년 4월 5일

기본 완료 (추정된)

2029년 4월 5일

연구 완료 (추정된)

2029년 4월 5일

연구 등록 날짜

최초 제출

2026년 5월 6일

QC 기준을 충족하는 최초 제출

2026년 5월 6일

처음 게시됨 (실제)

2026년 5월 12일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 5월 12일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 5월 6일

마지막으로 확인됨

2026년 5월 1일

추가 정보

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

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설문지 관리에 대한 임상 시험

구독하다