Open-label, dose escalation, multi-center, Phase I/II clinical trial to assess the safety/tolerability and determine the recommended Phase II Dose (RP2D) of ET140203 T-cells in pediatric subjects who are AFP-positive/HLA-A2-positive and have relapsed/refractory HB, HCN-NOS, or HCC.
Histologically confirmed HB, HCN-NOS, or HCC with serum AFP >100ng/mL at the time of screening and following the most recent line of therapy.
Disease recurrence after remission following initial standard-of care (SOC) treatment (i.e., relapse) or failure of response to SOC treatment (i.e., refractory).
Age ≥ 1 year and ≤ 21 years.
Molecular Human Leukocyte Antigen (HLA) class I allele typing that confirms subject carries at least one HLA-A2 allele.
Recurrent HB who are candidates for complete surgical resection (e.g., isolated pulmonary relapse amendable to pulmonary metastasectomy).
Pre-existing illness including heart failure, uncontrolled pulmonary disease not cancer-related, or psychiatric illness/social situation that would limit compliance with study requirements.
Active, uncontrolled systemic bacterial, fungal, or viral infection. Subjects with Human Immunodeficiency Virus (HIV), hepatitis B, or hepatitis C are eligible provided their infection is being treated and the viral load is controlled.
Any known active malignancy (other than HB, HCN-NOS, or HCC).
Single group
ET140203 T Cells
DrugBiological/Vaccine: ET140203 autologous T-cell product Autologous T cells transduced with lentivirus encoding an ET140203 expression construct
Incidence rates of adverse events (AEs) after infusion of ET140203 T cells
Safety of ET140203 T cells as assessed by the number of adverse events (AEs) after infusion
Severity rates of adverse events (AEs) after infusion of ET140203 T cells
Safety of ET140203 T cells as assessed by the severity of adverse events (AEs) after infusion.
Incidence rates of dose limiting toxicities (DLTs) after infusion of ET140203 T cells
Tolerability of ET140203 T cells after infusions assessed by committee review of dose limiting toxicities (DLTs)
The recommended phase 2 dose (RP2D) regimen of ET140203 T cell therapy primarily based on DLT
The RP2D will be determined by the study Dose Escalation Committee (DEC) and primarily based on DLTs.
Assess the efficacy of ET140203 T cells in pediatric subjects with relapsed/refractory HB, HCN-NOS, or HCC
Response rate will be assessed by radiographic scans and assessed according to RECIST criteria.
Determine the pharmacokinetics of ET140203 T cells after infusion.
Assess the expansion and persistence of ET140203 T cells circulating in blood over time.
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