MedPath

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

Phase 1
Recruiting
Conditions
Relapse/Refractory B-cell Acute Lymphoblastic Leukemia
Interventions
Registration Number
NCT06635330
Lead Sponsor
Kara Yakhteh Tajhiz Azma Company
Brief Summary

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?

Detailed Description

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.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
5
Inclusion Criteria
  • 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
Exclusion Criteria
  • 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

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Anti-CD19 CAR-T treatment groupanti-CD19 CAR T cell therapy-
Primary Outcome Measures
NameTimeMethod
Percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi)First month and 2-3 months after intervention
Overall survivalFirst month and 3 months after intervention
Incidence of cytokine release syndrome: grade 3 and 4First month and 3 months after intervention
Incidence of Immune effector cell-associated neurotoxicity syndrome (ICANS): grade 3 and 4First month and 3 months after intervention
Event-free survivalFirst month and 2-3 months after intervention
Secondary Outcome Measures
NameTimeMethod
Percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi)6 months and 12 months after intervention
Investigation of Minimal residual disease in patientFirst month and 2-3 months after intervention
Incidence of cytokine release syndrome: grade 3 and 46 months and 12 months after intervention
Incidence of Immune effector cell-associated neurotoxicity syndrome (ICANS): grade 3 and 46 months and 12 months after intervention
Incidence of tumor lysis syndrome (TLS)Months 1, 3, 6, and 12 after the intervention
Incidence of leukopeniaMonths 1, 3, 6, and 12 after the intervention
Incidence of infectionMonths 1, 3, 6, and 12 after the intervention
Event-free survival6 months and 12 months after intervention
Overall survival6 months and 12 months after intervention

Trial Locations

Locations (1)

Pediatric cell and gene therapy research center, Children medical center

🇮🇷

Tehran, Iran, Islamic Republic of

© Copyright 2025. All Rights Reserved by MedPath