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

Kara Yakhteh Tajhiz Azma Company

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Relapse/Refractory B-cell Acute Lymphoblastic Leukemia

Treatments: Biological: anti-CD19 CAR T cell therapy

Study type: Interventional

Funder types: NETWORK

Identifiers

NCT06635330

IR.NREC.1403.003

Details and patient eligibility

About

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?

Full 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.

Enrollment: 5 estimated patients

Sex: All

Ages: 2 to 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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5 participants in 1 patient group

Anti-CD19 CAR-T treatment group

Experimental group

Treatment:

Biological: anti-CD19 CAR T cell therapy