This single-arm, multicenter clinical study will treat the patient who have relapsed or refractory neuroblastoma with an infusion of the patient's own T cells that have been genetically modified to express a chimeric antigen receptor(CAR)that will bind to tumour cells modified to express the GD2 protein on the cell surface. The study will determine if these modified T cells help the body's immune system eliminate tumour cells .The trial will also study the safety of treatment for CAR-T, how long CAR-T cells stay in the patient's body and the impact on this treatment for survival.
This is a single-arm, multicenter clinical study to evaluate efficacy and safety of chimeric antigen receptor T cell immunotherapy (CAR-T) in the treatment of relapsed or refractory neuroblastoma in children. The study will be conducted using a phaseⅠ/Ⅱdesign the study will have the following sequential phases: part A (screening, leukapheresis,cell product preparation and cytoreductive chemotherapy) and part B (treatment and follow-up). the follow-up period for each participant is approximately 35 months after the final CAR-T infusion. The total duration of the study are expected to be approximately 3 years. A total of 22 patients may be enrolled over a period of 3 years.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
22
This study have only one arm that is CAR-T experimental arm. Firstly all participators will be attended the screening, who passed the screening for the treatment of CAR-T cells, the CAR-GD2-modified T cells can recognize and kill tumor cells in the body,follow-up 35 months.
Nanjing Children's Hospital
Nanjing, Jiangsu, China
RECRUITINGChildren's Hospital of Fudan University
Shanghai, Shanghai Municipality, China
RECRUITINGThe overall efficiency of patients with neuroblastoma after autologous CAR-T cell therapy
The overall efficiency will be determined by the evaluation of CT/MRI scans and bone marrow biopsy. Assessment of tumor remission rate according to International Neuroblastoma Response Criteria. The overall efficiency = (complete remission (CR) number + the number of very good partial remission (VGPR) number + partial response (PR) number + mixed reaction (MR) number + no response (NR) number) / total number of cases receiving treatment.
Time frame: 28d,56d,90d
Progression free survival
From the test of the progression of disease progression or the interval between disease and death.
Time frame: 3 years
Overall survival
For all patients, overall survival refers to the period from being included in the test group to death caused by any reason
Time frame: 3 years
Patients-based Quality of Life Evaluation
According to EORTC quality of life measurement scale PedsQL4.0\_ children's quality of life of the core scale of the evaluation and comparison of physical condition before and after treatment.
Time frame: 3 years
3°or above incidence rate of serious adverse reaction related to treatment
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time frame: 3 years
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