The purpose of this study is to test the safety and efficacy of iC9-GD2-CAR T-cells, a third generation (4.1BB-CD28) CAR T cell treatment targeting GD2 in paediatric or young adult patients affected by relapsed/refractory malignant central nervous system (CNS) tumors. In order to improve the safety of the approach, the suicide gene inducible Caspase 9 (iC9) has been included.
Imaging assessments performed within 14 days of start of treatment
Age: 6months-30years
Measurable or evaluable disease on at least 2 dimensions on MRI at the time of treatment enrollment
Karnofsky/Lansky≥60
Pregnant or lactating women
Severe,uncontrolledactiveinfections
HIV or active HCV and/or HBV infection
Rapidly progressive disease with life expectancy < 6 weeks
Single group
GD2-CART01 (iC9-GD2-CAR T-cells)
Biological/VaccineFollowing a lymphodepleting treatment with conventional chemotherapy, patients will be treated with 1.0 to 6.0 x 10⁶/kg GD2 Chimeric Antigen Receptor (CAR) positive T cells, infused i.v. as a single dose
Safety and definition of the MTD/RD
To evaluate the safety of the infusion of iC9-GD2-CAR-T cells at different escalating/de-escalating doses and establish the dose limiting toxicity (DLT) and the maximum tolerated dose/recommended dose (MTD/RD) of the cellular product
In vivo expansion and persistence
To assess the in vivo persistence and expansion of the infused CAR T-cells in the peripheral blood (PB) and CSF using immunoassays and transgene detection (Droplet PCR), both for the whole population and the specific T cells subsets
Tumor infiltration
To evaluate the tumor infiltration of the infused T cells by immunohistochemistry (IHC), flow cytometry and/or transgene detection (Droplet PCR), whenever the tumor sample is available after the treatment
iC9-GD2-CAR-T cells clearance after AP1903 infusion
To assess the kinetic of iC9-GD2-CAR-T cells clearance after AP1903 infusion
Serum cytokine profiling
To define the serum and CSF cytokine profile and its correlation with CRS in order to identify a possible predictive profile
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