A Phase 1 Study of Anitocabtagene Autoleucel for the Treatment of Subjects With Non-oncology Plasma Cell-related Diseases
Ориентир для пациента и семьи
Простыми словами
Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.
- Что изучают
- В протоколе указаны: anito-cel, Standard Lymphodepletion regimen.
- Кому может быть актуально
- Состояния в реестре: Muscular Diseases, Neuromuscular Manifestations, Autoimmune, Autoimmune Diseases. Базовые параметры: от 18 лет · Все.
- Что важно проверить
- Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
- Где проводится
- США
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Обзор
A Phase 1 dose-escalation study designed to evaluate the safety, tolerability, and preliminary efficacy of anito-cel in subjects with generalized myasthenia gravis (GMG). Anitocabtagene autoleucel (anito-cel) is a BCMA-directed CAR-T cell therapy.
Подробное описание
This is a Phase 1 open-label, multi-center safety and dose-escalation study of anito-cel\* in adult subjects with GMG (MGFA Grade 2 to 4a), in whom immunosuppressive therapy is clinically indicated in the judgement of the treating neurologist. The primary objective of this study is to assess the safety profile, including any DLT and identification of a MTD (if applicable), to support selection of the RP2D of anito-cel when administered to subjects with GMG.
The study will have the following sequential phases: screening, enrollment (leukapheresis), pretreatment with lymphodepletion (LD) chemotherapy, treatment with anito-cel and follow-up. Optional bridging therapy is allowed at investigator discretion while anito-cel is being manufactured.
Following a single infusion of anito-cel both safety and efficacy data will be assessed. The DLTs will be assessed in the first 28 days following anito-cel administration, and safety data will be collected throughout the study.
\*Anitocabtagene autoleucel (anito-cel) drug product consists of autologous T cells that have been genetically modified ex vivo to express a D-domain Chimeric Antigen Receptor (CAR), followed by a cluster of differentiation 8 (CD8) hinge and transmembrane region that is fused to the intracellular signaling domains for 4-1BB and CD3ξ, that specifically recognizes B-cell maturation antigen (BCMA). The active substance of anitocabtagene autoleucel is CAR+ CD3+ T cells that have undergone ex vivo T-cell activation, gene transfer by replication-deficient lentiviral vector, and expansion.
Вмешательства
- Биопрепарат anito-cel
Anitocabtagene autoleucel BCMA directed CAR T-cell therapy using a novel, synthetic binding domain, called a D-Domain - Препарат Standard Lymphodepletion regimen
Standard lymphodepletion regimen subject receive 5 days prior to CAR T infusion
Первичные конечные точки
- Assess safety profile, including any DLT and MTD (if applicable) [Срок оценки: 24 months]
- Selection of RP2D [Срок оценки: 24 months]
Вторичные конечные точки (11)
- Quantify Clinical Effect of Anito-cel in the Myasthenia Gravis Activities of Daily Living (MG ADL) score [Срок оценки: 24 months]
- Quantify Clinical Effect of Anito-cel in the Quantitative Myasthenia Gravis (QMG) score [Срок оценки: 24 months]
- Quantify Clinical Effect of Anito-cel in the Myasthenia Gravis Composite (MGC) scale. [Срок оценки: 24 months]
- Mean change in QMG score [Срок оценки: 24 months]
- Mean change in MG-ADL score [Срок оценки: 24 months]
- Mean change in MCG score [Срок оценки: 24 months]
- Mean change in MG-QoL15R score [Срок оценки: 24 months]
- Change in titer of myasthenia specific autoantibodies [Срок оценки: 24 months]
- PK Parameter for anito-cel: Cmax [Срок оценки: Day 1 up to 24 months]
- PK Parameter for anito-cel: Tmax [Срок оценки: Day 1 up to 24 months]
- PK Parameter for anito-cel: Area under the curve (AUC) [Срок оценки: Day 1 up to 24 months]
Критерии участия
Критерии включения
- Subject must be 18 years of age or older
- Must have MGFA clinical classification Grades 2-4A at time of screening
- Subject must have clinically active disease and requiring ongoing therapy for GMG
- MG-ADL score 6 and QMG score >10 at screening
- GMG specific autoantibodies must be above the reference laboratory ULN
Критерии исключения
- Subject is pregnant or breastfeeding
- Treatment with Anti-CD20 agents, calcineurin inhibitors, FcRN inhibitors, azathioprine, mycophenolate mofetil, methotrexate, or cyclophosphamide within the specified time frame prior to leukapheresis or prior to anito-cel infusion
- Previous treatment with any gene therapy, chimeric antigen receptor therapy or T cell engager
- Previous thymectomy within 6 months of screening
- Major chronic illness that is not well managed at the time of study entry and in the opinion of the investigator
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Распределение
- Не применимо
- Модель
- Одна группа
- Маскирование
- Открытое
- Основная цель
- Лечение
Центры проведения
США · 13 центров
- UCLA Medical Center — Los Angeles
- University of California, Irvine — Orange
- Stanford Hospital — Palo Alto
- University of South Florida - Carol and Frank Morsani Center of Advanced Healthcare — Tampa
- Karmanos Cancer Institute — Detroit
- University of Minnesota Delaware Clinical Research Unit — Minneapolis
- Mayo Clinic — Rochester
- Columbia University Irving Medical Center — New York
- … и ещё 5 центров
Идентификаторы
NCT: NCT06626919 · ARC-311