CAR-T for r/r Malignant Tumors in Children

Early Phase 1

• Conditions: Malignant Tumor; Child, Only; CAR-T
• Interventions: Drug: CAR-T

• Registration Number: NCT04691349
• Lead Sponsor: PersonGen BioTherapeutics (Suzhou) Co., Ltd.

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2020-09-27
• Study First Submitted: 2020-12-28
• Study First Submitted QC: 2020-12-28
• Study First Posted: 2020-12-31
• Status Verified: 2021-01
• Last Update Submitted: 2021-01-08
• Last Update Posted: 2021-01-11
• Primary Completion: 2021-01-26
• Study Completion: 2021-09-26

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 10

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Chimeric antigen receptor T cell	CAR-T	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

Primary Outcome Measures

Name	Time	Method
ORR3	Three months after CAR T cell infusion	3-month objective response rate

Trial Locations

Locations (1)

Chilren's Hospital of Soochow University; 🇨🇳 Suzhou, Jiangsu, China