- ICH GCP
- Registre américain des essais cliniques
- Essai clinique NCT07758673
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
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
- Médicament: Cyclophosphamide
- Médicament: Fludarabine
- Procédure: Leucaphérèse
- Procédure: Balayage d'acquisition multiportée
- Procédure: Tomographie par émission de positrons
- Procédure: Radiographie thoracique
- Procédure: Tomodensitométrie
- Procédure: Imagerie par résonance magnétique
- Procédure: Ponction de moelle osseuse
- Procédure: Biopsie de la moelle osseuse
- Biologique: Cellules CAR T autologues ciblant le BAFFR
- Procédure: Test d'échocardiographie
- Procédure: Biospecimen Collection
- Procédure: Bridge Therapy
- Procédure: Ultrasonographic Elastography
Description détaillée
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.
Type d'étude
Inscription (Estimé)
Phase
- La phase 1
Contacts et emplacements
Lieux d'étude
-
-
California
-
Duarte, California, États-Unis, 91010
- City of Hope Medical Center
-
Chercheur principal:
- Ibrahim Aldoss
-
Contact:
- Ibrahim Aldoss
- Numéro de téléphone: 626-218-2405
- E-mail: ialdoss@coh.org
-
Irvine, California, États-Unis, 92618
- City of Hope at Irvine Lennar
-
Chercheur principal:
- Ibrahim Aldoss
-
Contact:
- Ibrahim Aldoss
- Numéro de téléphone: 626-218-2405
- E-mail: ialdoss@coh.org
-
-
Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
- Adulte
- Adulte plus âgé
Accepte les volontaires sains
La description
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)
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: N / A
- Modèle interventionnel: Affectation à un seul groupe
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
|---|---|
|
Expérimental: 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.
|
Étant donné IV
Autres noms:
Étant donné IV
Autres noms:
Subir une leucaphérèse
Autres noms:
Subir MUGA
Autres noms:
Subir une TEP/TDM
Autres noms:
Passer une radiographie pulmonaire
Autres noms:
Subir une tomodensitométrie ou une TEP/TDM
Autres noms:
Passer une IRM cérébrale
Autres noms:
Subir une aspiration et une biopsie de la moelle osseuse
Subir une aspiration et une biopsie de la moelle osseuse
Autres noms:
Étant donné IV
Autres noms:
Subir l'écho
Autres noms:
Undergo blood and CSF specimen collection
Autres noms:
Receive bridging therapy
Autres noms:
Undergo liver ultrasonographic elastography
Autres noms:
|
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Incidence of adverse events
Délai: 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
Délai: 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
|
Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Disease response rate
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
|
Collaborateurs et enquêteurs
Parrainer
Collaborateurs
Les enquêteurs
- Chercheur principal: Ibrahim Aldoss, City of Hope Medical Center
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude (Estimé)
Achèvement primaire (Estimé)
Achèvement de l'étude (Estimé)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Réel)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
- Tumeurs
- Maladies du système immunitaire
- Infections
- Maladies virales
- Tumeurs par type histologique
- Maladies hématologiques
- Infections par le virus de l'ADN
- Maladies lymphatiques
- Troubles lymphoprolifératifs
- Troubles immunoprolifératifs
- Lymphome non hodgkinien
- Lymphome à cellules B
- Lymphome
- Leucémie, Lymphoïde
- Leucémie
- Infections par le virus Epstein-Barr
- Infections à Herpesviridae
- Infections virales tumorales
- Leucémie-lymphome lymphoblastique à cellules précurseurs
- Maladies hémiques et lymphatiques
- Lymphome de Burkitt
- Leucémie-lymphome lymphoblastique à précurseurs de cellules B
- Produits chimiques organiques
- Techniques d'investigation
- Thérapeutique
- Techniques de laboratoire clinique
- Techniques et procédures de diagnostic
- Diagnostic
- Procédures chirurgicales, opératoires
- Techniques cytologiques
- Cytodiagnostic
- Hydrocarbures
- Phénomènes physiques
- Techniques de diagnostic, chirurgicale
- Techniques de chimie, analytique
- Analyse du spectre
- Moutards phosphoramides
- Composés de moutarde d'azote
- Composés moutarde
- Hydrocarbures, halogénés
- Phosphoramides
- Composés organophosphores
- Phénomènes électromagnétiques
- Phénomènes magnétiques
- Thérapie biologique
- Cytaphérèse
- Élimination des composants sanguins
- Procédures de réduction des leucocytes
- Séparation des cellules
- Rayonnement électromagnétique
- Radiation
- Rayonnement, ionisant
- Cyclophosphamide
- Biopsie
- Manipulation des échantillons
- Spectroscopie de résonance magnétique
- fludarabine
- Leukaphérèse
- Rayons X
- Bridge Therapy
Autres numéros d'identification d'étude
- 260193 (Autre identifiant: City of Hope Medical Center)
- P30CA033572 (Subvention/contrat des NIH des États-Unis)
- NCI-2026-05498 (Identificateur de registre: CTRP (Clinical Trial Reporting Program))
Informations sur les médicaments et les dispositifs, documents d'étude
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