Neuroblastoma (NB) is a malignant tumor of the sympathetic nervous system.Chemotherapy and autologous hematopoietic stem cell transplantation are the main treatments for neuroblastoma, and the prognosis of patients with high-risk recurrence and refractory treatment is very poor. There is a large unmet medical need in patients with relapsed refractory neuroblastoma, and further research into new therapeutic approaches is needed for these patients.GD2 is a dissialic ganglioside expressed by neuroectodermal tumors. The proportion of GD2 expression in neuroblastoma is up to 100%, so GD2 is a specific target for neuroblastoma immunotherapy and an ideal target for CAR-T treatment of neuroblastoma.
All cases were diagnosed as neuroblastoma with positive expression of GD2 antigen in tumor cells. Informed consent of patient or guardian.
Diagnosis of recurrent/refractory neuroblastoma.
At least 2 weeks or 5 half-lives (whichever is shorter) from the beginning of preconditioning chemotherapy after prior systemic treatment.
Toxic reactions caused by previous antitumor therapy must be stabilized and restored to ≤ grade 1.
The subject has had other malignancies, non-melanoma skin tumors, carcinoma in situ (e.g. Cervix, bladder, breast), unless disease-free survival of at least 3 years.
There is an uncontrollable infection, including fungal, bacterial, viral or other.
Known human immunodeficiency virus (HIV) infection.
Known history of hepatitis B (HBsAg positive) or hepatitis C (HCV antibody positive). Subjects with latent or prehepatitis B infection (defined as HBcAb positive and HBsAg negative) can be enrolled only if PCR tests for HBV DNA are negative. In addition, these subjects were required to undergo a monthly PCR test for HBV DNA. Participants who are serologically positive for HCV antibodies can also be enrolled if their PCR test results for HCV RNA are negative.
Single group
GD2-CAR-T cell
Biological/VaccineSplit intravenous infusion of GD2-CAR-T cells \[dose escalating infusion of (1-100)x10\^6 GD2-CAR-T cells/kg\]
Number of Adverse Events
Adverse events are evaluated with CTCAE V4.03
Overall response rate (ORR)
ORR includes CR, PR,MR.SD,PD.Complete response (CR)#All components CR.Partial response (PR)#PR in at least one component and all other components CR,minimal disease (bone marrow), PR or not involved at baseline.Minor response (MR)#PR or CR in at least one component but at least one other component with SD; no component with PD.Stable disease (SD)#SD in one component with no better than SD or not involved at baseline in any other component, no component with PD.Progressive disease (PD)#Any component with PD.
Duration of overall response (DOR)
Duration of overall response will be assessed from the CAR-T cell infusion to progression, death or last follow-up.
Progression-free survival(PFS)
PFS will be assessed from the CAR-T cell infusion to progression, death or last follow-up.
Overall survival(OS)
OS will be assessed from the CAR-T cell infusion to death or last follow-up.
The duration of CAR T-cells in vivo
The time of CAR-T cells' persistence in blood and the copies of CAR-T cells
CAR-T related cytokine expression
CAR-T related cytokine expression.
Click on the lead sponsor to view all of their trials.
The General Hospital of Western Theater Command
Lead sponsor
Yake Biotechnology Ltd.
Collaborator
