Safety and Efficacy of CAR T Cell Therapy in Patients with R/r B-ALL

Официальное название

A Phase I/II Single Arm Study, Safety and Efficacy Assessment of the CD19 CAR T Cell on Pediatric Patients with Relapsing or Refractory B Cell Acute Lymphoblastic Leukemia (r/r B-ALL)

Обзор

The goal of this clinical trial is to evaluate the safety and efficacy of CD19 CAR-T cells in pediatric patients of all genders, aged 2 to 18 years, with relapsing or refractory B cell acute lymphoblastic leukemia (r/r B-ALL). The main questions it aims to answer are as following: 1. What is the percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi)? 2. What is the rate of Event-free survival at first month and 2-3 months after intervention? 3. What is the rate of Overall survival at first month and at 3 months after the intervention?

Подробное описание

B-cell acute lymphoblastic leukemia (B-ALL), as the most common type of pediatric tumor, is identified by unregulated cell proliferation of immature lymphoid cells that can infiltrate the bone marrow and blood. Also, relapse and refractory B-ALL (R/R B-ALL) is the main reason of global mortality due to the constraints of combination chemotherapy.

Over the past few years, substantial advancements have been made in treatment of ALL, specifically in the R/R context. Chimeric antigen receptor T (CAR-T) cells are a type of cancer immunotherapy treatment that function through modification of patient T cells to express CAR antigen on their surface. CAR-T cells aimed at CD19 have demonstrated promising activity in treatment of r/r B-ALL. In this study we aim to evaluate safety and efficacy of Anti-CD19 CAR T cell therapy in children with R/R B-ALL.

Вмешательства

• Биопрепарат anti-CD19 CAR T cell therapy
  Anti-CD19 CAR-T cell therapy for R/R B-ALL pediatric patients. For patients 50 kg and less: 0.2 to 5 in ten to the power of six live CAR+ T cells per kilogram of body weight/ For patients over 50 kg: 0.1 to 2.5 in ten to the power of eight live CAR+ T cells (without considering weight).

Первичные конечные точки

• Percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi) [Срок оценки: First month and 2-3 months after intervention]
• Overall survival [Срок оценки: First month and 3 months after intervention]
• Incidence of cytokine release syndrome: grade 3 and 4 [Срок оценки: First month and 3 months after intervention]
• Incidence of Immune effector cell-associated neurotoxicity syndrome (ICANS): grade 3 and 4 [Срок оценки: First month and 3 months after intervention]
• Event-free survival [Срок оценки: First month and 2-3 months after intervention]

Вторичные конечные точки (9)

• Percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi) [Срок оценки: 6 months and 12 months after intervention]
• Investigation of Minimal residual disease in patient [Срок оценки: First month and 2-3 months after intervention]
• Incidence of cytokine release syndrome: grade 3 and 4 [Срок оценки: 6 months and 12 months after intervention]
• Incidence of Immune effector cell-associated neurotoxicity syndrome (ICANS): grade 3 and 4 [Срок оценки: 6 months and 12 months after intervention]
• Incidence of tumor lysis syndrome (TLS) [Срок оценки: Months 1, 3, 6, and 12 after the intervention]
• Incidence of leukopenia [Срок оценки: Months 1, 3, 6, and 12 after the intervention]
• Incidence of infection [Срок оценки: Months 1, 3, 6, and 12 after the intervention]
• Event-free survival [Срок оценки: 6 months and 12 months after intervention]
• Overall survival [Срок оценки: 6 months and 12 months after intervention]

Критерии участия

Критерии включения

• Ages 2 to 18 years with relapsed or refractory CD19+ B-ALL
• Presence of disease in the bone marrow
• Able to tolerate the apheresis process
• Life expectancy \> 12 weeks
• Lansky or Karnofsky score \> 50%
• At least 7 days passed since the last chemotherapy and the last treatment with corticosteroids
• Informed consent
• Having potential donor for stem cell transplantation

Критерии исключения

• Presence of active malignancy other than the disease under study
• Chloroma and leukemic infiltration on MRI or significant neurological symptoms
• Any CNS disorder
• Presence of active GVHD
• Radiation therapy within last 14 days
• History of Anti-CD19 or Anti-CD20 therapy
• Donor lymphocyte injection or other cell therapy methods within the last 30 days
• Presence of severe active infection
• Organ dysfunction

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

Иран · 1 центр

• Pediatric cell and gene therapy research center, Children medical center — Tehran

Идентификаторы

NCT: NCT06635330 · IR.NREC.1403.003

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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