Study of Autologous GD2-CAR T Cells for Relapsed or Refractory Neuroblastoma Evaluating Safety and Response with Dose Escalation in Children

What this Trial Is About

Neuroblastoma is a cancer of the sympathetic nervous system that can be difficult to treat, especially in cases that have returned or do not respond well to standard therapies. This research is focused on testing a new treatment using GD2-CAR-T cells, which target a specific marker found on nearly all neuroblastoma tumors. The study is an early phase 1 clinical trial aimed at finding safe and effective ways to use these immune cells for patients with relapsed or refractory neuroblastoma. Participants receive increasing doses of GD2-CAR-T cells through intravenous infusions, starting at a low dose and escalating to the highest dose tolerated without severe side effects. The treatment is carefully monitored for up to 24 months to track adverse events like neurological or blood-related effects, infections, and secondary tumors. The study also measures tumor responses three months after treatment and follows patients for up to five years to observe recurrence, progression, and overall survival. Throughout the study, participants undergo various tests and evaluations to assess organ function, side effects, and treatment response. Researchers collect data on the presence and persistence of GD2-CAR-T cells in the body and monitor safety over the long term. The total duration of follow-up includes up to 24 months for adverse events and extended tracking of survival and disease status for several years, ensuring comprehensive assessment of this investigational therapy.

A Study on the Efficacy of GD2-CAR T Cells in the Treatment of Neuroblastoma