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FH-WT1-E50 TCR T Cells With Azacitidine for the Treatment of Minimal Residual Disease Positive Acute Myeloid Leukemia

2026年9月15日 更新者:Fred Hutchinson Cancer Center

Phase I Study of Autologous CD4+ and CD8+ T Cells That Have Been Transduced to Express a WT1-Specific T Cell Receptor for Treatment of MRD-Positive AML

This phase I trial tests the safety, side effects and best dose of FH-WT1-E50 TCR T cells with azacitidine for the treatment of minimal residual disease (MRD) positive acute myeloid leukemia (AML). T cells are infection fighting blood cells that can kill tumor cells. The T cells given in this study will come from the patient and will have a new gene put in them that makes them able to recognize WT1, a protein on the surface of cancer cells. These WT1-specific T cells may help the body's immune system identify and kill WT1 cancer cells. Azacitidine is in a class of medications called antimetabolites. It works by stopping or slowing the growth of cancer cells. Giving FH-WT1-E50 TCR T Cells with azacitidine may be safe and/or effective for the treatment of MRD positive AML.

調査の概要

詳細な説明

OUTLINE:

Patients undergo leukapheresis. 4 weeks later patients receive azacitidine intravenously (IV). At least 4 weeks after the first azacitidine infusion, patients receive azacitidine IV followed by Autologous HLA-A*02:01-restricted CD4+/CD8+ Anti-WT1 TCR/CD8ab-expressing T-cells (FHWT1-E50 TCR T) IV. If additional cells are available patients may receive a second infusion of azacitidine IV followed by FH-WT1-E50 TCR T cells IV, starting at 28 days, up to 1 year. Treatment is given in the absence of disease progression or unacceptable toxicity. Patients undergo multigated acquisition (MUGA) scan/echocardiography and chest x-ray during screening and bone marrow biopsy and aspiration and blood sample collection throughout the study. Patients may undergo lumbar puncture on study and computed tomography (CT) scan and/or positron emission tomography (PET) scan throughout the study.

After completion of study treatment, patients are followed up at day 1, 3, 7, 14, 21, 28, 56, 84, 168, 252 and 336 then every 6 months for years 1-5 the yearly until year 15.

研究の種類

介入

入学 (推定)

9

段階

  • フェーズ 1

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。

研究連絡先

研究場所

    • Washington
      • Seattle、Washington、アメリカ、98109
        • 募集
        • Fred Hutch/University of Washington Cancer Consortium
        • コンタクト:
        • 主任研究者:
          • Francesco Mazziotta, MD, PhD

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 for Leukapheresis:

  • LEUKAPHERESIS: Age 18 years or older at the time of enrollment
  • LEUKAPHERESIS: Confirmed diagnosis of AML that is not M3 subtype (acute promyelocytic leukemia [APL])
  • LEUKAPHERESIS: Human leukocyte antigen (HLA) type HLA-A*02:01 confirmed through HLA typing
  • LEUKAPHERESIS: Tissue confirmation of WT1 expression by immunohistochemistry. Confirmation of diagnosis must be or have been performed by internal pathology review of archival biopsy material or other pathologic material at Fred Hutch/University of Washington Medical Center (UWMC)
  • LEUKAPHERESIS: Capable of understanding and willing to provide informed consent
  • LEUKAPHERESIS: Fertile male and female participants must be willing to use an effective contraceptive method before, during, and for at least 4 months after the last FH-WT1-E50 TCR T infusion
  • LEUKAPHERESIS: Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 or Karnofsky Performance Status (KPS) ≥ 60%
  • LEUKAPHERESIS: No immediate plan for allogeneic stem cell transplantation: patients must not have a planned allogeneic hematopoietic stem cell transplant (HCT) within 8 weeks of leukapheresis. Patients may still be considered for HCT in the future but must not have a scheduled transplant at the time of enrollment due to factors including, but not limited to, donor availability, performance status, comorbidities, or patient preference. Patients who subsequently become candidates for HCT (e.g., donor identified or improvement in performance status) may proceed to transplant at the discretion of the treating physician without a mandated waiting period related to study participation
  • LEUKAPHERESIS: Creatinine clearance ≥ 30 ml/min by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) or 24-hour urine clearance
  • LEUKAPHERESIS: Total bilirubin < 3.0 mg/dL. Participants with suspected Gilbert syndrome may be included if total bilirubin (tBili) > 3mg/dL but no other evidence of hepatic dysfunction
  • LEUKAPHERESIS: Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) < 5 x upper limit of normal (ULN)
  • LEUKAPHERESIS: Participants 60 years of age or older are required to have left ventricular ejection fraction (LVEF) evaluation performed within 60 days prior to enrollment. LVEF may be established with echocardiogram or MUGA scan, and left ejection fraction must be ≥ 35%. Cardiac evaluation for other participants is at the discretion of the treating physician

Inclusion Criteria at Start of Treatment:

  • START OF TREATMENT: Presence of Measurable Residual Disease (MRD) after induction therapy at the time of screening. Patients must have achieved a morphologic remission (marrow that is at least 10% cellular with < 5% blasts on morphologic review) with detectable MRD, regardless of incomplete recovery of neutrophil counts/less than 1,000/mm3 (CRi) or platelet counts less than 100,000/mm3 (CRp). Eligible remission-induction regimens include, but are not limited to, conventional induction chemotherapy (e.g., 7+3 or similar anthracycline/cytarabine-based regimens) and hypomethylating agent-based combinations (e.g., azacitidine/venetoclax).

    • MRD definition:

      • MRD by flow cytometry: defined by any abnormal myeloid blasts identified by flow cytometric analysis. In addition, for patients with NPM1-mutated disease, we will incorporate a clinically validated quantitative NPM1 MRD assay performed at Fred Hutch. For patients with FLT3-ITD-mutated disease, MRD assessment will include a clinically validated FLT3-ITD assay performed at Invivoscribe.
  • START OF TREATMENT: Participants must be willing to undergo serial tumor biopsies and/or aspirates for research purposes if safe and feasible at baseline (prior to first T cell infusion), 2-3 weeks after the first T cell infusion, and approximately 2 weeks +/- 1 week after the second infusion (if applicable) (these windows may vary due to manufacturing or clinical reasons). Should there be no marrow tissue that is accessible, participants will still be considered for participation, at the discretion of the investigator. Similarly, should an investigator determine that a marrow assessment cannot be performed safely for clinical reasons, those may be cancelled or rescheduled. Participants must be at least two weeks or five half lives (for small molecules) from last systemic treatment: At least 2 weeks must have passed since any: immunotherapy (for example, T cell infusions, immunomodulatory agents, interleukins, vaccines), or chemotherapy cancer treatment. At least five half-lives must have passed from treatment with small molecules or other investigational agents
  • START OF TREATMENT: ECOG performance status of 0, 1, or 2 or Karnofsky Performance Status (KPS) ≥ 60%
  • START OF TREATMENT: Creatinine clearance ≥ 30 ml/min by CKD-EPI or 24-hour urine clearance
  • START OF TREATMENT: Total bilirubin < 3.0 mg/dL. Participants with suspected Gilbert syndrome may be included if tBili > 3mg/dL but no other evidence of hepatic dysfunction.
  • START OF TREATMENT: Participants 60 years of age or older are required to have left ventricular ejection fraction (LVEF) evaluation performed within 60 days prior to enrollment. LVEF may be established with echocardiogram or MUGA scan, and left ejection fraction must be ≥ 35%. Cardiac evaluation for other participants is at the discretion of the treating physician
  • START OF TREATMENT: Participants with a history of chronic obstructive pulmonary disease (COPD), emphysema, or greater than 30 pack year smoking history should undergo pulmonary function tests (PFTs) and meet the following criteria:

    • Forced expiratory volume in 1 second (FEV1) ≥ 50% of predicted and carbon monoxide diffusing capacity (DLCO) (corrected) ≥ 40% of predicted will be eligible

Exclusion Criteria for Leukapheresis:

  • LEUKAPHERESIS: Prior solid organ transplant or allogeneic hematopoietic stem cell transplant. Kidney transplant participants will be considered on a case-by-case basis requiring discussion with principal investigator (PI). If the participant has had a kidney transplant, participant must have dialysis access, dialysis plan, supportive nephrologist, willingness to stop transplant immunosuppression, and express understanding that rejection is possible outcome. Dialysis or costs related to transplant kidney will not be supported by the study. Participants having had any other solid organ transplants will be excluded, as will those with any history of allogeneic hematopoietic stem cell transplant

Exclusion Criteria at Start of Treatment:

  • START OF TREATMENT: Evidence of TET2, ASXL1 or DNMT3a mutations as sole evidence of MRD
  • START OF TREATMENT: Pregnancy, breastfeeding, or expecting to conceive or father children for the duration of the trial through 4 months after last T cell infusion. Participants of childbearing potential must have a negative serum pregnancy test within the 2 weeks (14 days) preceding T cell infusion. Childbearing potential is defined as women who have not been surgically sterilized and who are not post-menopausal (free of menses for at least 1 year)
  • START OF TREATMENT: Active autoimmune disease: Participants with active autoimmune disease requiring immunosuppressive therapy are excluded. Case-by-case exemptions are possible with approval by PI
  • START OF TREATMENT: Unable to generate FH-WT1-E50 TCR T cells for infusions. However, if a lower than planned number of cells is available, the participant will have the option to receive the generated WT1-specific T cells
  • START OF TREATMENT: Corticosteroid therapy at a systemic dose equivalent of > 0.5 mg/kg of prednisone-equivalent per day. The following treatments are permitted: intranasal, inhaled, topical, or local steroid applications; systemic corticosteroids at physiologic doses equivalent to no more than 10 mg/day prednisone; steroids as premedication for contrast dye allergy
  • START OF TREATMENT: Concurrent use of other investigational anti-cancer agents
  • START OF TREATMENT: Active uncontrolled infection: HIV positive participants on highly active antiretroviral therapy (HAART) with a CD4 count > 500 cells/mm3 are considered controlled, as are individuals with a history of hepatitis C who have successfully completed antiviral therapy with an undetectable viral load, and those with hepatitis B who have, per standard practice, hepatitis well-controlled on medication
  • START OF TREATMENT: Uncontrolled concurrent illness: Participants may not have uncontrolled or concurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
  • START OF TREATMENT: Known allergic reactions to any of the components of study treatments
  • START OF TREATMENT: Other medical, social, or psychiatric factors that interfere with medical appropriateness and/or ability to comply with study, as determined by the PI

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 (FH-WT1-E50 TCR T cells, azacitidine)
Patients undergo leukapheresis. 4 weeks later patients receive azacitidine IV. At least 4 weeks after the first azacitidine infusion, patients receive azacitidine IV followed by FH-WT1-E50 TCR T cells IV. If additional cells are available patients may receive a second infusion of azacitidine IV followed by FH-WT1-E50 TCR T cells IV, starting at 28 days, up to 1 year. Treatment is given in the absence of disease progression or unacceptable toxicity. Patients undergo MUGA scan/echocardiography and chest x-ray during screening and bone marrow biopsy and aspiration and blood sample collection throughout the study. Patients may undergo lumbar puncture on study and CT scan and/or PET scan throughout the study.
腰椎穿刺を受ける
他の名前:
  • LP
  • スパイナルタップ
採血を受ける
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
PETスキャンを受ける
他の名前:
  • 医用画像、陽電子放出断層撮影
  • ペット
  • PETスキャン
  • 陽電子放出断層撮影スキャン
  • 陽電子放出断層撮影
  • PT
  • 陽電子放出断層撮影(手順)
CTスキャンを受ける
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
骨髄吸引を受ける
胸部レントゲンを受ける
他の名前:
  • 胸部X線
与えられた IV
他の名前:
  • 5AZC
  • 5-AC
  • 5-アザシチジン
  • 5-AZC
  • アザシチジン
  • アザシチジン、5-
  • ラダカマイシン
  • ミロサー
  • U-18496
  • ヴィダーザ
骨髄生検を受ける
他の名前:
  • 骨髄の生検
  • 生検、骨髄
心エコー検査を受けます
他の名前:
  • 心エコー検査
  • EC
Given IV
他の名前:
  • Autologous HLA-A*02:01-restricted WT1-E50 TCR-CD8ab CD4+/CD8+ T Cells
  • Autologous WT1-specific HLA-A*02:01-restricted TCR/CD8ab CD4-/CD8-positive T-cells
  • FH-WT1-E50 TCR T
Given MUGA scan
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

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
時間枠
Incidence of treatment related grade 3 or higher adverse events
時間枠:From first infusion up to 1 year
From first infusion up to 1 year
Incidence of dose limiting toxicity
時間枠:From first T cell infusion, up to 28 days
From first T cell infusion, up to 28 days

二次結果の測定

結果測定
メジャーの説明
時間枠
Ability to reproducibly generate and infuse T cells at the planned dose level (feasibility)
時間枠:From baseline up to 1 year after eligibility determination
No formal statistical considerations will be used to evaluate this endpoint beyond a simple estimate of the proportion along with its 95% confidence interval.
From baseline up to 1 year after eligibility determination
Relapse
時間枠:At 1 and 2 years after first infusion
Defined as with or without measurable tumor burden prior to T cell transfer, including patients who convert to minimal residual disease-negative status during consolidation.
At 1 and 2 years after first infusion

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。

捜査官

  • 主任研究者:Francesco Mazziotta, MD, PhD、Fred Hutch/University of Washington Cancer Consortium

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)

2026年10月15日

一次修了 (推定)

2030年7月19日

研究の完了 (推定)

2030年7月19日

試験登録日

最初に提出

2026年6月9日

QC基準を満たした最初の提出物

2026年6月9日

最初の投稿 (実際)

2026年6月12日

学習記録の更新

投稿された最後の更新 (実際)

2026年9月17日

QC基準を満たした最後の更新が送信されました

2026年9月15日

最終確認日

2026年8月1日

詳しくは

本研究に関する用語

個々の参加者データ (IPD) の計画

個々の参加者データ (IPD) を共有する予定はありますか?

はい

IPD プランの説明

Individual participant data (IPD) that will be shared include de-identified clinical and laboratory data from enrolled Acute Myeloid Leukemia (AML) participants, including baseline demographics, clinical outcomes, treatment-related variables, adverse events, and all data underlying published results. In addition, multi-omic datasets will be shared, including single-cell RNA sequencing data, spatial transcriptomics data, and flow cytometry data generated from participant biospecimens. All shared data will be de-identified in accordance with applicable privacy regulations and institutional policies.

IPD 共有時間枠

IPD and supporting documents will become available after primary manuscript publication or within 12 months of study completion, whichever occurs later. Data will remain available for a period of at least 5 years following initial release. Access may begin after publication-related data lock and completion of required data de-identification and curation processes.

IPD 共有アクセス基準

Access to IPD and supporting materials will be granted to qualified researchers whose proposed use is consistent with scientifically sound secondary analyses. Requests will require submission of a research proposal, institutional affiliation, and IRB or ethics approval or exemption where applicable. Data will be shared under a data use agreement that prohibits re-identification attempts and restricts use to non-commercial research. Requests will be reviewed by a study steering committee or designated data access committee.

Data will be shared via a controlled-access repository or secure data-sharing platform, with appropriate governance and security controls.

A dedicated controlled-access data repository will be established upon study completion. The URL will be provided when the repository becomes active.

IPD 共有サポート情報タイプ

  • STUDY_PROTOCOL
  • SAP
  • ICF

医薬品およびデバイス情報、研究文書

米国FDA規制医薬品の研究

はい

米国FDA規制機器製品の研究

いいえ

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。

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