- ICH GCP
- Реестр клинических исследований США
- Клиническое испытание 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
Обзор исследования
Статус
Условия
Вмешательство/лечение
- Лекарство: Циклофосфамид
- Лекарство: Флударабин
- Процедура: Лейкаферез
- Процедура: Мультигейтное сканирование захвата
- Процедура: Позитронно-эмиссионная томография
- Процедура: Рентгенография грудной клетки
- Процедура: Компьютерная томография
- Процедура: Магнитно-резонансная томография
- Процедура: Аспирация костного мозга
- Процедура: Биопсия костного мозга
- Биологический: Аутологичные CAR T-клетки, нацеленные на BAFFR
- Процедура: Эхокардиографический тест
- Процедура: Biospecimen Collection
- Процедура: Bridge Therapy
- Процедура: Ultrasonographic Elastography
Подробное описание
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.
Тип исследования
Регистрация (Оцененный)
Фаза
- Фаза 1
Контакты и местонахождение
Места учебы
-
-
California
-
Duarte, California, Соединенные Штаты, 91010
- City of Hope Medical Center
-
Главный следователь:
- Ibrahim Aldoss
-
Контакт:
- Ibrahim Aldoss
- Номер телефона: 626-218-2405
- Электронная почта: ialdoss@coh.org
-
Irvine, California, Соединенные Штаты, 92618
- City of Hope at Irvine Lennar
-
Главный следователь:
- Ibrahim Aldoss
-
Контакт:
- Ibrahim Aldoss
- Номер телефона: 626-218-2405
- Электронная почта: ialdoss@coh.org
-
-
Критерии участия
Критерии приемлемости
Возраст, подходящий для обучения
- Взрослый
- Пожилой взрослый
Принимает здоровых добровольцев
Описание
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)
Учебный план
Как устроено исследование?
Детали дизайна
- Основная цель: Уход
- Распределение: Н/Д
- Интервенционная модель: Одногрупповое задание
- Маскировка: Нет (открытая этикетка)
Оружие и интервенции
Группа участников / Армия |
Вмешательство/лечение |
|---|---|
|
Экспериментальный: 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.
|
Учитывая IV
Другие имена:
Учитывая IV
Другие имена:
Пройти лейкаферез
Другие имена:
Пройти МУГА
Другие имена:
Пройти ПЭТ/КТ
Другие имена:
Пройти рентген грудной клетки
Другие имена:
Пройти КТ или ПЭТ/КТ
Другие имена:
Пройти МРТ головного мозга
Другие имена:
Пройти аспирацию костного мозга и биопсию
Пройти аспирацию костного мозга и биопсию
Другие имена:
Учитывая IV
Другие имена:
Пройти эхо
Другие имена:
Undergo blood and CSF specimen collection
Другие имена:
Receive bridging therapy
Другие имена:
Undergo liver ultrasonographic elastography
Другие имена:
|
Что измеряет исследование?
Первичные показатели результатов
Мера результата |
Мера Описание |
Временное ограничение |
|---|---|---|
|
Incidence of adverse events
Временное ограничение: 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
Временное ограничение: 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
|
Вторичные показатели результатов
Мера результата |
Мера Описание |
Временное ограничение |
|---|---|---|
|
Disease response rate
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
|
Соавторы и исследователи
Спонсор
Соавторы
Следователи
- Главный следователь: Ibrahim Aldoss, City of Hope Medical Center
Даты записи исследования
Изучение основных дат
Начало исследования (Оцененный)
Первичное завершение (Оцененный)
Завершение исследования (Оцененный)
Даты регистрации исследования
Первый отправленный
Впервые представлено, что соответствует критериям контроля качества
Первый опубликованный (Действительный)
Обновления учебных записей
Последнее опубликованное обновление (Действительный)
Последнее отправленное обновление, отвечающее критериям контроля качества
Последняя проверка
Дополнительная информация
Термины, связанные с этим исследованием
Дополнительные соответствующие термины MeSH
- Новообразования
- Заболевания иммунной системы
- Инфекции
- Вирусные заболевания
- Новообразования по гистологическому типу
- Гематологические заболевания
- ДНК-вирусные инфекции
- Лимфатические заболевания
- Лимфопролиферативные заболевания
- Иммунопролиферативные заболевания
- Лимфома, неходжкинская
- Лимфома, В-клеточная
- Лимфома
- Лейкемия, лимфоидная
- Лейкемия
- Вирусные инфекции Эпштейна-Барра
- Герпесвирусные инфекции
- Опухолевые вирусные инфекции
- Клетки-предшественники лимфобластный лейкоз-лимфома
- Гемики и лимфатические заболевания
- Лимфома Беркитта
- Предшественник В-клеточного лимфобластного лейкоза-лимфомы
- Органические химические вещества
- Следственные методы
- Терапия
- Клинические лабораторные методы
- Диагностические методы и процедуры
- Диагноз
- Хирургические процедуры, оперативные
- Цитологические методы
- Цитодиагностика
- Углеводороды
- Физические явления
- Диагностические методы, хирургический
- Химические методы, аналитические
- Анализ спектра
- Фосфорамид горчицы
- Азотные соединения горчицы
- Горчичные соединения
- Углеводороды, галогенированные
- Фосфорамиды
- Соединения органофосфора
- Электромагнитные явления
- Магнитные явления
- Биологическая терапия
- Цитаферез
- Удаление компонента крови
- Процедуры сокращения лейкоцитов
- Разделение клеток
- Электромагнитное излучение
- Излучение
- Радиация, ионизация
- Циклофосфамид
- Биопсия
- Обработка образца
- Магнитно -резонансная спектроскопия
- флударабин
- Лейкаферез
- Рентген
- Bridge Therapy
Другие идентификационные номера исследования
- 260193 (Другой идентификатор: City of Hope Medical Center)
- P30CA033572 (Грант/контракт NIH США)
- NCI-2026-05498 (Идентификатор реестра: CTRP (Clinical Trial Reporting Program))
Информация о лекарствах и устройствах, исследовательские документы
Изучает лекарственный продукт, регулируемый FDA США.
Изучает продукт устройства, регулируемый Управлением по санитарному надзору за качеством пищевых продуктов и медикаментов США.
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