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Genetically Engineered Cells (BAFFR-CAR T Cells) for the Treatment of Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia and B-cell Lymphoblastic Lymphoma

2026年8月6日 更新者:City of Hope Medical Center

A Phase 1 Study to Evaluate BAFFR-Targeting CAR T Cells for Patients With Relapsed or Refractory B-Cell Acute Lymphoblastic Leukemia

This phase I trial tests the safety and side effects of B-cell activating factor receptor (BAFFR) chimeric antigen receptor (CAR) T cells and how well they work in treating patients with B-cell acute lymphoblastic leukemia (B-ALL) and B-cell lymphoblastic lymphoma that has come back after a period of improvement (relapsed) or that has not responded to previous treatment (refractory). CAR T-cell therapy, such as BAFFR-CAR T cells, is a type of treatment in which a patient's T cells (a type of immune system cell) are changed in the laboratory so they will attack cancer cells. T cells are taken from a patient's blood. Then the gene for a special receptor that binds to a certain protein on the patient's cancer cells is added to the T cells in the laboratory. The special receptor is called a CAR. Large numbers of the CAR T cells are grown in the laboratory and given to the patient by infusion for treatment of certain cancers. Giving BAFFR-CAR T cells may be safe, tolerable and/or effective in treating patients with relapsed or refractory B-cell ALL and B-cell lymphoblastic lymphoma.

調査の概要

詳細な説明

PRIMARY OBJECTIVE:

I. Assess the safety of administering autologous BAFFR-targeting CAR T cells (BAFFR[EQ]BBζ/EGFRt T cells) (also known as [aka], BAFFR-CAR T cells).

SECONDARY OBJECTIVES:

I. Evaluate the ability of BAFFR-CAR T cells to mediate clinical response in participants with B-ALL.

II. Evaluate the level of residual disease in participants who achieve remission after BAFFR-CAR T cell treatment.

III. Evaluate the duration of B cell aplasia as a surrogate for BAFFR-CAR T cell activity.

IV. Evaluate the rate and severity of graft-versus-host disease (GVHD) in recipients of prior allogeneic hematopoietic stem cell transplantation (allogeneic hematopoietic stem cell transplantation [allo-HCT]).

V. Evaluate progression-free survival (PFS) and overall survival (OS).

EXPLORATORY OBJECTIVES:

I. Measure expansion and persistence of BAFFR-CAR T cells in the peripheral blood (PB), bone marrow (BM), and cerebrospinal fluid (CSF), when available, and explore the correlation with BAFFR-CAR T cell efficacy.

II. Measure BAFF-R expression on leukemic cells, as well as other disease markers, before and after BAFFR-CAR T cell treatment and explore association with response and relapse, when feasible.

III. Measure cytokine levels in PB and CSF, when available, and explore association with response.

OUTLINE:

Patients undergo leukapheresis and may receive bridging therapy per treating physician discretion. Patients then receive lymphodepletion therapy with cyclophosphamide intravenously (IV) and fludarabine IV on days -5 to -3 and BAFFR-CAR T cells IV over 10-15 minutes on day 0. Patients also undergo blood sample collection, bone marrow aspiration and biopsy, chest x-ray, and positron emission tomography (PET)/computed tomography (CT) or CT throughout the study. Additionally, patients may also undergo liver ultrasonographic elastography at screening at discretion of investigator and brain magnetic resonance imaging (MRI) or CT, CSF specimen collection, and echocardiography (ECHO) or multigated acquisition scan (MUGA) throughout the study

After completion of study treatment, patients are followed up within 18-24 hours, at least every 2 days for 14 days, at 14, 21, 28, 60, and 100 days, at 4, 6, 7, and 12 months, then yearly for up to a total of 15 years.

研究の種類

介入

入学 (推定)

16

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。

研究場所

    • California
      • Duarte、California、アメリカ、91010
        • City of Hope Medical Center
        • 主任研究者:
          • Ibrahim Aldoss
        • コンタクト:
      • Irvine、California、アメリカ、92618
        • City of Hope at Irvine Lennar
        • 主任研究者:
          • Ibrahim Aldoss
        • コンタクト:

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
  • 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 ≥ 18 years
  • Eastern Cooperative Oncology Group (ECOG) ≤ 2
  • Life expectancy ≥ 16 weeks
  • Histologically confirmed B-ALL or B-cell lymphoblastic lymphoma
  • Relapsed/refractory disease. Minimal residual disease (MRD) relapse is allowed
  • Evidence of tumor expressing BAFF-R at any level either by flow or immunohistochemistry
  • Recovered to ≤ grade 1 from the acute toxic effects (except alopecia and peripheral neuropathy) of prior anti-cancer therapy
  • No known contraindications to leukapheresis, steroids or tocilizumab
  • Ineligible for or failed prior CD19-targeted immunotherapy (e.g., blinatumomab or CD19-CAR T cells)

    • For participants who had prior CD19-CAR T cell therapy:

      • At least 90-days has elapsed since participant received last CD19-CAR T cell therapy AND
      • Persistence of prior CD19-CAR T cells must be evaluated and found to be < 5% prior to leukapheresis procedure
      • Note: Participants who have undergone stem cell transplantation (at least 100 days prior to enrollment) after the last CD19-CAR T cell therapy, are considered eligible and do not need to meet the above criteria
  • Participants with central nervous system (CNS) involvement by leukemia (CNS2 and asymptomatic CNS3) may be considered eligible after discussions with the study team
  • Total serum bilirubin ≤ upper limit of normal (ULN) (unless has Gilbert's disease or related to liver involvement by leukemia, then ≤ 3.0)
  • Aspartate aminotransferase (AST) ≤ ULN, unless related to liver involvement by ALL, then ≤ 3.0
  • Alanine aminotransferase (ALT) ≤ ULN, unless related to liver involvement by ALL, then ≤ 3.0
  • Creatinine clearance of ≥ 40 mL/min per 24-hour urine test or the Cockcroft-Gault formula
  • Left ventricular ejection fraction (LVEF) ≥ 50%
  • Oxygen (O2) saturation ≥ 92% on room air
  • Seronegative for HIV quantitative polymerase chain reaction (qPCR), hepatitis C virus (HCV), and active hepatitis B virus (HBV) (surface antigen negative), and syphilis (rapid plasma reagin [RPR])

    • If positive, hepatitis C ribonucleic acid (RNA) quantitation must be performed OR
    • If seropositive for HIV, HCV or HBV, nucleic acid quantitation must be performed. The viral load must be undetectable
  • Meets other institutional and federal requirements for infectious disease titer requirements

    • Note Infectious disease testing to be performed within 28 days prior to start of protocol therapy
  • Women of childbearing potential (WOCBP): Negative urine or serum pregnancy test

    • If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required
  • QuantiFERON-tuberculosis (TB) Gold or equivalent

    • Results do not impact patient eligibility; however, the test must be initiated prior to enrollment
  • 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 3 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)
  • A complete liver evaluation (which includes ultrasound elastography, MRI of the liver and a hepatology consult) may be done if needed based on PI's recommendation

Exclusion Criteria:

  • Autologous/allogeneic stem cell transplant within 100 days at the time of enrollment
  • Immunosuppressant medications within 1 month prior to protocol enrollment
  • Concurrent use of systemic steroids or chronic use of immunosuppressant medications. Recent or current use of inhaled steroids is not exclusionary. Physiologic replacement of steroids (prednisone ≤ 7.5 mg /day or equivalent) is allowed
  • Auto-immune disease or active graft-versus-host disease (GvHD) within 3 months prior to protocol enrollment requiring systemic immunosuppressant therapy
  • Class III/IV cardiovascular disability according to the New York Heart Association (NYHA) Classification
  • Subjects with clinically significant arrhythmia or arrhythmias not stable on medical management within 2 weeks of enrollment
  • Any abnormal liver enzyme levels (as defined by grade 1 elevation from ULN in ALT, AST, and bilirubin levels) at time of enrollment, unless they are abnormal due to liver involvement by leukemia per the treating physician's discretion
  • Subjects with a known history or prior diagnosis of uncontrolled central nervous system (CNS) disorders such as optic neuritis or other immunologic or inflammatory disease affecting the CNS, including uncontrolled seizure disorder
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to study agent
  • Known significant bleeding disorders (e.g., severe von Willebrand's disease) or hemophilia
  • History of venous occlusive disease (VOD), or GvHD

    • Subjects with a history of the following GvHD may still be included in the study:

      • Resolved grade 2 or less steroid-sensitive acute skin GvHD
      • Grade 1 gastrointestinal (GI)-GvHD developed within 100 days post prior alloHCT
      • Limited chronic GVHD
  • History of stroke or intracranial hemorrhage within 6 months of enrollment
  • History of other malignancies, except for malignancy surgically resected (or treated with other modalities) with curative intent, basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin; non-muscle invasive bladder cancer; malignancy treated with curative intent with no known active disease present for ≥ 2 years
  • Clinically significant uncontrolled illness
  • Active systemic uncontrolled infection
  • Known history of immunodeficiency virus (HIV) or hepatitis B or hepatitis C infection
  • Females only: Pregnant or breastfeeding
  • Any other condition that would, in the investigator's judgment, contraindicate the subject'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 (BAFFR-CAR T cells)
Patients undergo leukapheresis and may receive bridging therapy per treating physician discretion. Patients then receive lymphodepletion therapy with cyclophosphamide IV and fludarabine IV on days -5 to -3 and BAFFR-CAR T cells IV over 10-15 minutes on day 0. Patients also undergo blood sample collection, bone marrow aspiration and biopsy, chest x-ray, and PET/CT or CT throughout the study. Additionally, patients may also undergo liver ultrasonographic elastography at screening at discretion of investigator and brain MRI or CT, CSF specimen collection, and ECHO or MUGA throughout the study.
与えられた IV
他の名前:
  • シトキサン
  • CTX
  • (-)-シクロホスファミド
  • 2H-1,3,2-オキサザホスホリン、2-[ビス(2-クロロエチル)アミノ]テトラヒドロ-、2-オキシド、一水和物
  • カルロキサン
  • シクロホスファミダ
  • シクロホスファミド
  • シクロキサール
  • クラフェン
  • CP一水和物
  • サイクロセル
  • シクロスブラスチン
  • シクロブラスチン
  • シクロホスファム
  • シクロホスファミド一水和物
  • シクロホスファミダム
  • シクロホスファン
  • シクロホスファナム
  • シクロスチン
  • サイトホスファン
  • シトホスファン
  • フォスファセロン
  • ジェノクサル
  • ジェヌサル
  • レドキシン
  • ミトキサン
  • ネオサー
  • レビミューン
  • WR-138719
  • アスタB518
  • B-518
  • B518
  • WR 138719
  • WR138719
  • フリンドビックス
与えられた IV
他の名前:
  • フルラドサ
白血球除去療法を受ける
他の名前:
  • 白血球除去法
  • 治療的白血球除去療法
  • 白血球吸着アフェレーシス
  • 白血球減少アフェレーシス
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
PET/CT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)
胸部レントゲンを受ける
他の名前:
  • 胸部X線
CTまたはPET/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
脳MRIを受ける
他の名前:
  • MRI
  • 磁気共鳴
  • 磁気共鳴画像スキャン
  • 医用画像、磁気共鳴 / 核磁気共鳴
  • 氏
  • MRイメージング
  • MRI スキャン
  • NMRイメージング
  • NMRI
  • 核磁気共鳴イメージング
  • 磁気共鳴画像法 (MRI)
  • sMRI
  • 磁気共鳴画像法(手順)
  • 構造MRI
骨髄穿刺と生検を受ける
骨髄穿刺と生検を受ける
他の名前:
  • 骨髄の生検
  • 生検、骨髄
ギヴンIV
他の名前:
  • 自家BAFFR-CAR T細胞
  • 自家BAFFR-CAR発現T細胞
エコーを受ける
他の名前:
  • 心エコー検査
  • EC
Undergo blood and CSF specimen collection
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
  • サンプル収集
Receive bridging therapy
他の名前:
  • Holding Therapy
Undergo liver ultrasonographic elastography
他の名前:
  • 超音波エラストグラフィ

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
Incidence of adverse events
時間枠:Up to 28 days after chimeric antigen receptor (CAR) T cells
Will be graded using Common Terminology Criteria for Adverse Events version 5.0, American Society for Transplantation and Cellular Therapy Consensus Criteria on Cytokine Release Syndrome/Neurotoxicity, graft-versus-host disease (GVHD) criteria, and Immune Effector Cell-Associated Hemophagocytic Lymphohistiocytosis-Like Syndrome identification and grading. Will be summarized by organ involved, severity, time of onset, and attribution.
Up to 28 days after chimeric antigen receptor (CAR) T cells
Dose-limiting toxicity
時間枠:From the start of CAR T infusion up to 28 days
Will be described individually.
From the start of CAR T 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
Disease response rate
時間枠:At 4 weeks
Will be defined as complete response (CR) or CR with incomplete blood count recovery or CR with partial hematological recovery. Will be evaluated using European LeukemiaNet criteria. Rates and associated 95% binomial exact confidence limits will be estimated.
At 4 weeks
Minimal residual disease negative rate
時間枠:At 4 weeks
Will be defined by malignant cells < 0.01% by flow cytometry or clonoSEQ. Rates and associated 95% binomial exact confidence limits will be estimated.
At 4 weeks
Duration of B-cell aplasia
時間枠:Up to 15 years
Will be measured by serum immunoglobulin G level. Will be summarized by descriptive statistics.
Up to 15 years
Severity of GVHD in recipients of prior allogeneic hematopoietic stem cell transplantation
時間枠:Within 8 weeks after T cell infusion
Will be defined per Keystone criteria for acute GVHD and revised National Institutes of Health consensus on grading of chronic GVHD. Competing risk method will be used to estimate.
Within 8 weeks after T cell infusion
Progression-free survival
時間枠:From T cell infusion to the first observation of disease relapse/progression or death from any cause, whichever occurs first, assessed up to 15 years
Kaplan-Meier product limit method with log-log transformation for the confidence interval will be used to estimate.
From T cell infusion to the first observation of disease relapse/progression or death from any cause, whichever occurs first, assessed up to 15 years
Overall survival
時間枠:From T cell infusion to death from any cause, assessed up to 15 years
Kaplan-Meier product limit method with log-log transformation for the confidence interval will be used to estimate.
From T cell infusion to death from any cause, assessed up to 15 years

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:Ibrahim Aldoss、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17日

一次修了 (推定)

2028年7月26日

研究の完了 (推定)

2028年7月26日

試験登録日

最初に提出

2026年8月6日

QC基準を満たした最初の提出物

2026年8月6日

最初の投稿 (実際)

2026年8月11日

学習記録の更新

投稿された最後の更新 (実際)

2026年8月11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6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

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

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