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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

6. august 2026 opdateret af: 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.

Studieoversigt

Status

Ikke rekrutterer endnu

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

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.

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

16

Fase

  • Fase 1

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiesteder

    • California
      • Duarte, California, Forenede Stater, 91010
        • City of Hope Medical Center
        • Ledende efterforsker:
          • Ibrahim Aldoss
        • Kontakt:
      • Irvine, California, Forenede Stater, 92618
        • City of Hope at Irvine Lennar
        • Ledende efterforsker:
          • Ibrahim Aldoss
        • Kontakt:

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ingen

Beskrivelse

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)

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Interventionel model: Enkelt gruppeopgave
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: 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.
Givet IV
Andre navne:
  • Cytoxan
  • CTX
  • (-)-Cyclophosphamid
  • 2H-1,3,2-Oxazaphosphorin, 2-[bis(2-chlorethyl)amino]tetrahydro-, 2-oxid, monohydrat
  • Carloxan
  • Ciclofosfamida
  • Ciclofosfamid
  • Cicloxal
  • Clafen
  • Claphene
  • CP monohydrat
  • CYCLO-celle
  • Cykloblastin
  • Cycloblastin
  • Cyclofosfam
  • Cyclophosphamid monohydrat
  • Cyclophosphamid Monohydrat
  • Cyclophospamidum
  • Cyclofosfan
  • Cyclophosphanum
  • Cyclostin
  • Cytophosphan
  • Cytofosfan
  • Fosfaseron
  • Genoxal
  • Genuxal
  • Ledoxina
  • Mitoxan
  • Neosar
  • Revimune
  • Syklofosfamid
  • WR- 138719
  • Asta B 518
  • B-518
  • WR-138719
  • B 518
  • B518
  • WR 138719
  • WR138719
  • Frindovyx
Givet IV
Andre navne:
  • Fluradosa
Gennemgå leukaferese
Andre navne:
  • Leukocytoferese
  • Terapeutisk leukoferese
  • Leukocytadsorptiv aferese
  • Aferese til reduktion af hvide blodlegemer
Gennemgå MUGA
Andre navne:
  • Blood Pool Scan
  • Ligevægtsradionuklidangiografi
  • Gated Blood Pool Imaging
  • MUGA
  • Radionuklid ventrikulografi
  • RNVG
  • SYMA-scanning
  • Synkroniseret Multigated Acquisition Scanning
  • MUGA Scan
  • Multi-Gated Acquisition Scan
  • Radionuklid Ventrikulogram Scan
  • Gated Heart Pool Scan
  • RNV Scan
Gennemgå PET/CT
Andre navne:
  • Medicinsk billeddannelse, Positron Emission Tomografi
  • KÆLEDYR
  • PET-scanning
  • Positron Emission Tomografi Scan
  • Positron-emissionstomografi
  • PT
  • Positron emissionstomografi (procedure)
Gennemgå røntgen af ​​thorax
Andre navne:
  • Røntgen af ​​thorax
Gennemgå CT eller PET/CT
Andre navne:
  • CT
  • KAT
  • CAT-scanning
  • Beregnet aksial tomografi
  • Computerstyret aksial tomografi
  • Computerstyret tomografi
  • CT-scanning
  • tomografi
  • Computerstyret aksial tomografi (procedure)
  • Computerstyret tomografi (CT) scanning
  • Diagnostisk CAT -scanning
  • Diagnostic CAT Scan Service Type
Gennemgå hjerne MR
Andre navne:
  • MR
  • Magnetisk resonans
  • Magnetisk resonansbilledscanning
  • Medicinsk billeddannelse, magnetisk resonans / kernemagnetisk resonans
  • HR
  • MR billeddannelse
  • MR-scanning
  • NMR billeddannelse
  • NMRI
  • Kernemagnetisk resonansbilleddannelse
  • Magnetisk resonansbilleddannelse (MRI)
  • sMRI
  • Magnetisk resonansbilleddannelse (procedure)
  • MRI'er
  • Strukturel MR
Gennemgå knoglemarvsaspiration og biopsi
Gennemgå knoglemarvsaspiration og biopsi
Andre navne:
  • Biopsi af knoglemarv
  • Biopsi, knoglemarv
Givet IV
Andre navne:
  • Autologe BAFFR-CAR T-celler
  • Autologe BAFFR-CAR-udtrykkende T-celler
Gennemgå Echo
Andre navne:
  • Ekkokardiografi
  • EC
Undergo blood and CSF specimen collection
Andre navne:
  • Biologisk prøvesamling
  • Bioprøve indsamlet
  • Prøvesamling
  • Prøvekollektion
Receive bridging therapy
Andre navne:
  • Holding Therapy
Undergo liver ultrasonographic elastography
Andre navne:
  • Ultralyd Elastografi

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Incidence of adverse events
Tidsramme: 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
Tidsramme: 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

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Disease response rate
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Samarbejdspartnere

Efterforskere

  • Ledende efterforsker: Ibrahim Aldoss, City of Hope Medical Center

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Anslået)

17. april 2027

Primær færdiggørelse (Anslået)

26. juli 2028

Studieafslutning (Anslået)

26. juli 2028

Datoer for studieregistrering

Først indsendt

6. august 2026

Først indsendt, der opfyldte QC-kriterier

6. august 2026

Først opslået (Faktiske)

11. august 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

11. august 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

6. august 2026

Sidst verificeret

1. august 2026

Mere information

Begreber relateret til denne undersøgelse

Andre undersøgelses-id-numre

  • 260193 (Anden identifikator: City of Hope Medical Center)
  • P30CA033572 (U.S. NIH-bevilling/kontrakt)
  • NCI-2026-05498 (Registry Identifier: CTRP (Clinical Trial Reporting Program))

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .