FH-WT1-E50 TCR T Cells With Azacitidine for the Treatment of Minimal Residual Disease Positive Acute Myeloid Leukemia
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
調査の概要
状態
条件
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
入学 (推定)
段階
- フェーズ 1
連絡先と場所
研究連絡先
- 名前:Fred Hutch Immunotherapy Intake
- 電話番号:206-606-4668
- メール:immunotherapy@fredhutch.org
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:
- Fred Hutch Immunotherapy Intake
- 電話番号:206-606-4668
- メール:immunotherapy@fredhutch.org
-
主任研究者:
- Francesco Mazziotta, MD, PhD
-
-
参加基準
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
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
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:
採血を受ける
他の名前:
PETスキャンを受ける
他の名前:
CTスキャンを受ける
他の名前:
骨髄吸引を受ける
胸部レントゲンを受ける
他の名前:
与えられた IV
他の名前:
骨髄生検を受ける
他の名前:
心エコー検査を受けます
他の名前:
Given IV
他の名前:
Given MUGA scan
他の名前:
|
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
捜査官
- 主任研究者:Francesco Mazziotta, MD, PhD、Fred Hutch/University of Washington Cancer Consortium
研究記録日
主要日程の研究
研究開始 (推定)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- RG1126017
- 2P01CA018029 (米国 NIH グラント/契約)
- 21042 (その他の識別子:Fred Hutch/University of Washington Cancer Consortium)
- NCI-2026-02251 (レジストリ識別子:CTRP (Clinical Trial Reporting Program))
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?
IPD プランの説明
IPD 共有時間枠
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
米国FDA規制機器製品の研究
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