CAR-T for r/r Malignant Tumors in Children

Chimeric Antigen Receptor T Cells Therapy for r/r Malignant Tumors in Children

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.

Study Overview

• Status: Recruiting
• Conditions: Child, Only; Malignant Tumor; CAR-T
• Intervention / Treatment: Drug: CAR-T

• Study Type: Interventional
• Enrollment (Anticipated): 10
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: ShaoYan Hu; Phone Number: 137-7187-0462; Email: hushaoyan@suda.edu.cn
• Study Contact Backup: Name: HuiMin Meng; Phone Number: 188-9680-2149; Email: huimin.meng@persongen.com.cn

Study Locations

• China
  • Jiangsu
    • Suzhou, Jiangsu, China, 215000
      • Recruiting
      • Chilren's Hospital of Soochow University
      • Contact: ShaoYan Hu; Phone Number: 137-7187-0462; Email: hushaoyan@suda.edu.cn
      • Contact: HuiMin Meng; Phone Number: 188-9680-2149; Email: huimin.meng@persongen.com.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Chimeric antigen receptor T cell; 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

Drug: CAR-T; 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
ORR3	3-month objective response rate	Three months after CAR T cell infusion

Collaborators and Investigators

• Sponsor: PersonGen BioTherapeutics (Suzhou) Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): September 27, 2020
• Primary Completion (Anticipated): January 26, 2021
• Study Completion (Anticipated): September 26, 2021

Study Registration Dates

• First Submitted: December 28, 2020
• First Submitted That Met QC Criteria: December 28, 2020
• First Posted (Actual): December 31, 2020

Study Record Updates

• Last Update Posted (Actual): January 11, 2021
• Last Update Submitted That Met QC Criteria: January 8, 2021
• Last Verified: January 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms
• Other Study ID Numbers: PG-CART-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No