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?
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
5
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).
Pediatric cell and gene therapy research center, Children medical center
Tehran, Tehran Province, Iran
RECRUITINGPercentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi)
Time frame: First month and 2-3 months after intervention
Overall survival
Time frame: First month and 3 months after intervention
Incidence of cytokine release syndrome: grade 3 and 4
Time frame: First month and 3 months after intervention
Incidence of Immune effector cell-associated neurotoxicity syndrome (ICANS): grade 3 and 4
Time frame: First month and 3 months after intervention
Event-free survival
Time frame: First month and 2-3 months after intervention
Percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi)
Time frame: 6 months and 12 months after intervention
Investigation of Minimal residual disease in patient
Time frame: First month and 2-3 months after intervention
Incidence of cytokine release syndrome: grade 3 and 4
Time frame: 6 months and 12 months after intervention
Incidence of Immune effector cell-associated neurotoxicity syndrome (ICANS): grade 3 and 4
Time frame: 6 months and 12 months after intervention
Incidence of tumor lysis syndrome (TLS)
Time frame: Months 1, 3, 6, and 12 after the intervention
Incidence of leukopenia
Time frame: Months 1, 3, 6, and 12 after the intervention
Incidence of infection
Time frame: Months 1, 3, 6, and 12 after the intervention
Event-free survival
Time frame: 6 months and 12 months after intervention
Overall survival
Time frame: 6 months and 12 months after intervention
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.