CAR-T for r/r Malignant Tumors in Children

PersonGen BioTherapeutics

Status and phase: Unknown; Early Phase 1

Conditions: CAR-T; Child, Only; Malignant Tumor

Treatments: Drug: CAR-T

Study type: Interventional

Funder types: Industry

Identifiers

NCT04691349

PG-CART-01

Details and patient eligibility

About

This study is a clinical study of CAR-T treatment of patients with relapsed/refractory malignant tumors in children. The purpose is to evaluate the safety and effectiveness of chimeric antigen receptor T cells in the treatment of relapsed/refractory malignant tumors in children.

Enrollment: 10 estimated patients

Sex: All

Ages: 3 to 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Age 3-18
• Expected survival time ≥ 12weeks
• ECOG 0-2
• At least second-line or above chemotherapy failed
• Liver and kidney function, heart and lung function meet the following requirements:

Creatinine is within the normal range; Left ventricular ejection fraction ≥ 45%; Baseline blood oxygen saturation>91%; Total bilirubin≤1.5×ULN; ALT and AST≤2.5×ULN

• Understand the trial and have signed the informed consent

Exclusion criteria

• Those who have graft-versus-host disease (GVHD) or need to use immunosuppressive agents
• Hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) positive and peripheral blood HBV DNA titer test is not within the normal reference range; hepatitis C virus (HCV) antibody positive and peripheral blood HCV RNA positive; human immunodeficiency virus (HIV) Antibody positive; CMV DNA test positive; Syphilis test positive
• Severe heart disease
• Systemic diseases judged by the investigator to be unstable: including but not limited to severe liver, kidney or metabolic diseases that require medication
• Within 7 days before screening, there are active infections or uncontrollable infections that require systemic treatment (except for mild urogenital infections and upper respiratory tract infections)
• Those who have received CAR-T therapy or other genetically modified cell therapy before screening
• According to the researcher's judgment, it does not meet the situation of cell preparation
• Situations that other researchers think are not suitable for inclusion

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

Chimeric antigen receptor T cell

Experimental group

Description:

Chimeric antigen receptor T cells (car-t) is one of the most effective therapies for malignant tumors (especially hematological tumors). Like other immunotherapies, the basic principle is to use the patient's own immune cells to clear cancer cells. Chimeric antigen receptor (car) is the core component of car-t, which endows T cells with the ability to recognize tumor antigens in an independent manner, which enables car modified T cells to recognize a wider range of targets than natural T cell surface receptors (TCR). The basic design of car includes a tumor associated antigen binding region (usually derived from scFv segment of monoclonal antibody antigen binding region), transmembrane region and intracellular signal region. The selection of target antigen is a key determinant for the specificity and effectiveness of car and the safety of genetically modified T cells

Treatment:

Drug: CAR-T