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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

2026년 8월 6일 업데이트: 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.

연구 개요

상세 설명

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.

연구 유형

중재적

등록 (추정된)

16

단계

  • 1단계

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
        • 수석 연구원:
          • Ibrahim Aldoss
        • 연락하다:
      • Irvine, California, 미국, 92618
        • City of Hope at Irvine Lennar
        • 수석 연구원:
          • Ibrahim Aldoss
        • 연락하다:

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
  • 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)

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 (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.
주어진 IV
다른 이름들:
  • 사이톡산
  • CTX
  • (-)-시클로포스파미드
  • 2H-1,3,2-옥사자포스포린, 2-[비스(2-클로로에틸)아미노]테트라하이드로-, 2-옥사이드, 일수화물
  • 칼록산
  • 시클로포스파미다
  • 시클로포스파마이드
  • 시클록살
  • 클라펜
  • CP 일수화물
  • CYCLO 셀
  • 사이클로블라스틴
  • 사이클로포스팜
  • 사이클로포스파미드 일수화물
  • 사이클로포스파미둠
  • 시클로포스판
  • 사이클로포스판
  • 시클로포스파늄
  • 사이클로스틴
  • 사이토포스판
  • 포스파세론
  • 제녹살
  • 제눅살
  • 레독시나
  • 미톡산
  • 네오사르
  • 리바이뮨
  • 실클로포스파미드
  • WR-138719
  • 아스타 B 518
  • B-518
  • B518
  • WR 138719
  • WR138719
  • 프린도빅스
주어진 IV
다른 이름들:
  • 플루라도사
백혈구 성분채집술을 받다
다른 이름들:
  • 백혈구 분리술
  • 치료 백혈구성분채집술
  • 백혈구 흡착 성분채집술
  • 백혈구 감소 성분채집술
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
PET/CT를 받다
다른 이름들:
  • 의료 영상, 양전자 방출 단층 촬영
  • 애완 동물
  • PET 스캔
  • 양전자 방출 단층 촬영 스캔
  • 양전자 방출 단층 촬영
  • PT
  • 양전자방출단층촬영(시술)
흉부 엑스레이를 찍다
다른 이름들:
  • 흉부 엑스레이
CT 또는 PET/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
뇌 MRI를 받다
다른 이름들:
  • MRI
  • 자기 공명
  • 자기공명영상 스캔
  • 의료영상, 자기공명 / 핵자기공명
  • 씨
  • MR 이미징
  • MRI 검사
  • NMR 이미징
  • NMRI
  • 핵자기공명영상
  • 자기공명영상(MRI)
  • SMRI
  • 자기공명영상(시술)
  • 구조적 MRI
골수 흡인 및 생검 실시
골수 흡인 및 생검 실시
다른 이름들:
  • 골수 생검
  • 생검, 골수
주어진 IV
다른 이름들:
  • 자가 BAFFR-CAR T 세포
  • 자가 BAFFR-CAR 발현 T 세포
에코를받습니다
다른 이름들:
  • 심초음파
  • EC
Undergo blood and CSF specimen collection
다른 이름들:
  • 생물학적 샘플 수집
  • 생체 표본 수집
  • 표본 수집
  • 샘플 수집
Receive bridging therapy
다른 이름들:
  • Holding Therapy
Undergo liver ultrasonographic elastography
다른 이름들:
  • 초음파 탄성학

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Incidence of adverse events
기간: 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
기간: 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

2차 결과 측정

결과 측정
측정값 설명
기간
Disease response rate
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

수사관

  • 수석 연구원: Ibrahim Aldoss, 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 17일

기본 완료 (추정된)

2028년 7월 26일

연구 완료 (추정된)

2028년 7월 26일

연구 등록 날짜

최초 제출

2026년 8월 6일

QC 기준을 충족하는 최초 제출

2026년 8월 6일

처음 게시됨 (실제)

2026년 8월 11일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 8월 11일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 8월 6일

마지막으로 확인됨

2026년 8월 1일

추가 정보

이 연구와 관련된 용어

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

예

미국 FDA 규제 기기 제품 연구

아니

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. .

구독하다