Genetically Engineered Cells (BAFFR-CAR T Cells) for the Treatment of Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia and B-cell Lymphoblastic Lymphoma
A Phase 1 Study to Evaluate BAFFR-Targeting CAR T Cells for Patients With Relapsed or Refractory B-Cell Acute Lymphoblastic Leukemia
Studieoversigt
Status
Status
Betingelser
Betingelser
Intervention / Behandling
Intervention / Behandling
- Medicin: Cyclofosfamid
- Medicin: Fludarabin
- Procedure: Leukaferese
- Procedure: Multigated Acquisition Scan
- Procedure: Positron emissionstomografi
- Procedure: Røntgen af brystet
- Procedure: Computertomografi
- Procedure: MR scanning
- Procedure: Knoglemarvsaspiration
- Procedure: Knoglemarvsbiopsi
- Biologisk: Autologe BAFFR-målrettede CAR T-celler
- Procedure: Ekkokardiografitest
- Procedure: Biospecimen Collection
- Procedure: Bridge Therapy
- Procedure: Ultrasonographic Elastography
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
Undersøgelsestype
Tilmelding (Anslået)
Tilmelding
Fase
Fase
- Fase 1
Kontakter og lokationer
Studiesteder
-
-
California
-
Duarte, California, Forenede Stater, 91010
- City of Hope Medical Center
-
Ledende efterforsker:
- Ibrahim Aldoss
-
Kontakt:
- Ibrahim Aldoss
- Telefonnummer: 626-218-2405
- E-mail: ialdoss@coh.org
-
Irvine, California, Forenede Stater, 92618
- City of Hope at Irvine Lennar
-
Ledende efterforsker:
- Ibrahim Aldoss
-
Kontakt:
- Ibrahim Aldoss
- Telefonnummer: 626-218-2405
- E-mail: ialdoss@coh.org
-
-
Deltagelseskriterier
Berettigelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
- Voksen
- Ældre voksen
Tager imod sunde frivillige
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
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Antal våben
Våben og indgreb
Deltagergruppe / ArmDeltagergruppe / Arm |
Intervention / BehandlingIntervention / 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:
Givet IV
Andre navne:
Gennemgå leukaferese
Andre navne:
Gennemgå MUGA
Andre navne:
Gennemgå PET/CT
Andre navne:
Gennemgå røntgen af thorax
Andre navne:
Gennemgå CT eller PET/CT
Andre navne:
Gennemgå hjerne MR
Andre navne:
Gennemgå knoglemarvsaspiration og biopsi
Gennemgå knoglemarvsaspiration og biopsi
Andre navne:
Givet IV
Andre navne:
Gennemgå Echo
Andre navne:
Undergo blood and CSF specimen collection
Andre navne:
Receive bridging therapy
Andre navne:
Undergo liver ultrasonographic elastography
Andre navne:
|
Hvad måler undersøgelsen?
Primære resultatmål
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
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
Sponsor
Sponsor
Samarbejdspartnere
Samarbejdspartnere
Efterforskere
Efterforskere
- Ledende efterforsker: Ibrahim Aldoss, City of Hope Medical Center
Datoer for undersøgelser
Studer store datoer
Studiestart (Anslået)
Studiestart
Primær færdiggørelse (Anslået)
Primær færdiggørelse
Studieafslutning (Anslået)
Studieafslutning
Datoer for studieregistrering
Først indsendt
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Først opslået
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering sendt
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
- Neoplasmer
- Sygdomme i immunsystemet
- Infektioner
- Virussygdomme
- Neoplasmer efter histologisk type
- Hæmatologiske sygdomme
- DNA-virusinfektioner
- Lymfesygdomme
- Lymfoproliferative lidelser
- Immunproliferative lidelser
- Lymfom, Non-Hodgkin
- Lymfom, B-celle
- Lymfom
- Leukæmi, lymfoid
- Leukæmi
- Epstein-Barr-virusinfektioner
- Herpesviridae infektioner
- Tumorvirusinfektioner
- Precursorcelle lymfoblastisk leukæmi-lymfom
- Hemiske og lymfatiske sygdomme
- Burkitt lymfom
- Precursor B-celle lymfoblastisk leukæmi-lymfom
- Organiske kemikalier
- Undersøgelsesteknikker
- Terapeutik
- Kliniske laboratorieteknikker
- Diagnostiske teknikker og procedurer
- Diagnose
- Kirurgiske procedurer, operative
- Cytologiske teknikker
- Cytodiagnose
- Kulbrinter
- Fysiske fænomener
- Diagnostiske teknikker, kirurgisk
- Kemiteknikker, analytisk
- Spektrumanalyse
- Fosforamid -sennep
- Nitrogen sennepsforbindelser
- Sennepsforbindelser
- Kulbrinter, halogeneret
- Phosphoramider
- Organophosphorforbindelser
- Elektromagnetiske fænomener
- Magnetiske fænomener
- Biologisk terapi
- Cytaferese
- Fjernelse af blodkomponent
- Leukocytreduktionsprocedurer
- Celleseparation
- Elektromagnetisk stråling
- Stråling
- Stråling, ioniserende
- Cyclofosfamid
- Biopsi
- Håndtering af eksemplar
- Magnetisk resonansspektroskopi
- fludarabin
- Leukapherese
- Røntgenstråler
- Bridge Therapy
Andre undersøgelses-id-numre
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
Studerer et amerikansk FDA-reguleret enhedsprodukt
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