このページは自動翻訳されたものであり、翻訳の正確性は保証されていません。を参照してください。 英語版 ソーステキスト用。

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.

調査の概要

詳細な説明

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.

研究の種類

介入

入学 (推定)

35

段階

  • フェーズ2

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究場所

    • California
      • Duarte、California、アメリカ、91010
        • 募集
        • City of Hope Medical Center
        • コンタクト:
        • 主任研究者:
          • Monzr M. Al Malki

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

適格基準

就学可能な年齢

  • 大人
  • 高齢者

健康ボランティアの受け入れ

いいえ

説明

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)

研究計画

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

研究はどのように設計されていますか?

デザインの詳細

  • 主な目的:処理
  • 割り当て:なし
  • 介入モデル:単一グループの割り当て
  • マスキング:なし(オープンラベル)

武器と介入

参加者グループ / アーム
介入・治療
実験的: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.

補助研究
CTを受ける
他の名前:
  • CT
  • 猫
  • CATスキャン
  • コンピューター断層撮影
  • コンピュータ化されたアキシャルトモグラフィー
  • CTスキャン
  • トモグラフィー
  • コンピューター断層撮影 (手順)
  • コンピューター断層撮影 (CT) スキャン
  • 診断CATスキャン
  • 診断CATスキャンサービスタイプ
与えられた IV
他の名前:
  • CB-3025
  • L-PAM
  • L-サルコリシン
  • アラニン窒素マスタード
  • L-フェニルアラニンマスタード
  • L-サルコリシン フェニルアラニン マスタード
  • L-サルコリジン
  • メルパラナム
  • フェニルアラニンマスタード
  • フェニルアラニン窒素マスタード
  • サルコクロリン
  • サルコリシン
  • WR-19813
  • メルファラン注射肝臓送達システム
MUGAを受ける
他の名前:
  • 血液プールスキャン
  • 平衡放射性核種血管造影
  • ゲーテッド血液プールイメージング
  • ムガ
  • 放射性核種心室造影
  • RNVG
  • SYMAスキャン
  • 同期マルチゲート取得スキャン
  • ムガスキャン
  • マルチゲート取得スキャン
  • 放射性核種脳室造影スキャン
  • ゲーテッドハートプールスキャン
  • RNVスキャン
与えられた IV
他の名前:
  • 2-F-ara-AMP
  • ベネフラー
  • フルダーラ
  • 9H-プリン-6-アミン、2-フルオロ-9-(5-O-ホスホノ-β-D-アラビノフラノシル)-
  • SH T 586
与えられた IV または PO
他の名前:
  • プログラフ
  • FK506
  • ヘコリア
  • フジマイシン
  • プロトピック
  • FK-506
  • タクフォリウス
与えられた IV
他の名前:
  • 成長因子、組換えヒトケラチノサイト
  • ケピヴァンス
  • ケラチノサイト増殖因子、組換えヒト
  • 組換えヒトケラチノサイト増殖因子
  • rhKGF
  • rhuケラチノサイト成長因子
骨髄生検を受ける
他の名前:
  • 骨髄の生検
  • 生検、骨髄
TMLIを受ける
他の名前:
  • TMLI
シロリムスの投与
他の名前:
  • ラパマイシン
  • ラパミューン
  • 22989 年
  • ラパ
  • サイラ 9268A
  • WY-090217
  • AY-22989
  • SILA-9268A
  • SILA9268A
  • ワイオミング州 090217
  • WY090217
尿と血液サンプルの採取を受けます
他の名前:
  • 生物学的サンプルの収集
  • 採取された生体試料
  • 標本収集
心エコー検査を受けます
他の名前:
  • 心エコー検査
  • EC
Undergo allogeneic PBSC HCT
他の名前:
  • PBPC移植
  • 末梢血前駆細胞移植
  • 末梢幹細胞サポート
  • 末梢幹細胞移植
  • PBSCT
  • 末梢血幹細胞移植
  • 末梢血

この研究は何を測定していますか?

主要な結果の測定

結果測定
メジャーの説明
時間枠
Leukemia-free survival
時間枠: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)

二次結果の測定

結果測定
メジャーの説明
時間枠
Incidence of adverse events (AEs)
時間枠: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
時間枠: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
時間枠: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
時間枠: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
時間枠: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)
時間枠: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
時間枠: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
時間枠: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

協力者と研究者

ここでは、この調査に関係する人々や組織を見つけることができます。

スポンサー

捜査官

  • 主任研究者:Monzr M Al Malki、City of Hope Medical Center

研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始 (推定)

2027年4月5日

一次修了 (推定)

2029年4月5日

研究の完了 (推定)

2029年4月5日

試験登録日

最初に提出

2026年5月6日

QC基準を満たした最初の提出物

2026年5月6日

最初の投稿 (実際)

2026年5月12日

学習記録の更新

投稿された最後の更新 (実際)

2026年8月20日

QC基準を満たした最後の更新が送信されました

2026年8月18日

最終確認日

2026年8月1日

詳しくは

この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。

購読する