Safety and Efficacy of CAR-T Cell Therapy for Relapsed/refractory Neuroblastoma and Desmoplastic Small Round Cell Tumors: a Single-arm, Open-label Trial

What this Trial Is About

Researchers are evaluating the safety and effectiveness of GD2/B7H3 CAR-T cell therapy for patients with relapsed or refractory neuroblastoma and desmoplastic small round cell tumors. This open-label, single-arm trial aims to study how the CAR-T cells behave in the body, including their pharmacokinetics and pharmacodynamics, as well as how long they survive. The study is sponsored by Sun Yat-Sen University Cancer Center and focuses on these rare and challenging cancers. Participants will receive a lymphodepletion treatment with intravenous chemotherapy based on fludarabine and cyclophosphamide within 14 days before the CAR-T cell infusion. The CAR-T cells are then infused intravenously at doses ranging from 1.00 to 10.00 x 10^6 CAR-positive T cells per kilogram of body weight. After infusion, participants will be followed for one year or until adverse events resolve, disease progression occurs, or they move on to other treatments. During the study, participants will be monitored closely for any adverse events related to the CAR-T therapy, including their severity and duration. Researchers will also assess the pharmacokinetic and pharmacodynamic properties of the CAR-T cells and their survival in the body. Key outcomes include overall response rate, complete and partial responses, overall survival, progression-free survival, event-free survival, time to progression, and duration of response. Safety assessments will continue for one year after treatment.

Safety and Efficacy of CAR-T Cell Therapy for Relapsed/refractory Neuroblastoma and Desmoplastic Small Round Cell Tumors: a Single-arm, Open-label Trial.