This Phase 1, open-label, non-randomized study will enroll pediatric and young adult subjects with relapsed or refractory non-central nervous system (CNS) malignant solid tumors expressing glypican-3 (GPC3) to examine the safety, feasibility, and efficacy of administering T cell products derived from peripheral blood mononuclear cells (PBMC) that have been genetically modified to co-express a GPC3-specific chimeric antigen receptor (CAR), interleukin (IL)-15 and IL-21 as well as the inducible caspase 9 (iC9) suicide gene (SC-CAR.GPC3xIL15.21 T cells).
A child or young adult meeting all eligibility criteria and meeting none of the exclusion criteria will have a blood sample collected, which will be used to bioengineer the CAR T cells targeting their tumor.
Procurement Eligibility
Diagnosis of a solid tumor expressing GPC3
Lansky or Karnofsky score of >=60%
Life expectancy of >16 weeks
History of hypersensitivity reactions to murine protein-containing products OR presence of human anti-mouse antibody (HAMA) prior to enrollment for patients who have received prior therapy with murine antibodies.
History of organ transplantation
Known HIV positivity
Active bacterial, fungal, or viral infection (except Hepatitis B or Hepatitis C virus infections)
Single group
SC-CAR.GPC3xIL15.21 CAR T cells
Biological/VaccineAutologous SC-CAR.GPC3xIL15.21 T cell products infusion
The number of successfully manufactured SC-CAR.GPC3xIL15.21 T cell products will be assessed
The number of successfully manufactured products will be measured
Establish the safety, defined by adverse events of SC-CAR.GPC3xIL15.21 T cells.
Type, frequency, severity, and duration of adverse events will be tabulated and summarized
Estimate the maximum tolerated dose (MTD) or biologically effective dose and dose limiting toxicities (DLT), and describe the full toxicity profile of SC-CAR.GPC3xIL15.21 T cells.
Type, frequency, severity, and duration of adverse events will be tabulated and summarized
To estimate anti-tumor responses by measuring changes in tumor burden using disease-specific evaluations following SC-CAR.GPC3xIL15.21 T cells.
Standard imaging and blood pathology will be used to determine disease response
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