Phase III Study of ET140203 T Cell Therapy for Children and Young Adults With Relapsed or Refractory Hepatoblastoma, HCN-NOS, or Hepatocellular Carcinoma

What this Trial Is About

Researchers are evaluating ET140203 T-cell therapy in pediatric patients aged 1 to 21 years who have relapsed or refractory hepatoblastoma HB, hepatocellular neoplasm-not otherwise specified HCN-NOS, or hepatocellular carcinoma HCC and are AFP-positive and HLA-A2-positive. This open-label, multi-center, Phase III trial aims to assess safety, tolerability, and determine the recommended Phase II dose RP2D through dose escalation. Participants receive intravenous ET140203 autologous T cells, which are genetically modified T cells designed to target cancer cells. The trial uses a traditional 33 dose escalation to find the RP2D, followed by an expansion phase where more subjects are treated at this dose. Tumor response and serum AFP levels are monitored before the first infusion and then at months 1, 3, 6, 9, 12, 18, and 24. Participants will be actively assessed for two years with radiographic imaging and blood tests to evaluate treatment and tumor response. Long-term follow-up for safety and overall survival will continue up to 15 years. The study measures adverse events within 28 days after infusion, dose-limiting toxicities, and pharmacokinetics of the T cells during this period.

ET140203 T Cells in Pediatric Subjects With Hepatoblastoma, HCN-NOS, or Hepatocellular Carcinoma