International Phase III Study Evaluating Sequential Chemotherapy and Radiotherapy Treatments for High-Risk Neuroblastoma in Patients Up to 21 Years Old

What this Trial Is About

Researchers are conducting an international, open-label, randomized phase III trial to evaluate the effectiveness of chemotherapy and radiotherapy treatments for patients with high-risk neuroblastoma. The study includes three sequential randomizations to compare different induction chemotherapy regimens, consolidation high-dose chemotherapy HDC approaches, and radiotherapy strategies. The goal is to improve event-free survival EFS over three years among children and young adults diagnosed with this aggressive cancer. The first randomization compares two induction chemotherapy regimens RAPID COJEC versus GPOH. The second randomization assesses single HDC with Busulfan-Melphalan Bu-Mel versus tandem HDC with Thiotepa followed by Bu-Mel. For patients with macroscopic residual disease after surgery and HDC, the third randomization compares standard radiotherapy 21.6 Gy to the tumor bed versus the same radiotherapy plus an additional boost 14.4 Gy. Patients without residual disease receive standard radiotherapy to the tumor bed. Treatments include various chemotherapy drugs, immunotherapy with dinutuximab beta, and radiotherapy delivered according to the assigned groups. Participants will undergo multiple assessments including disease evaluation after induction chemotherapy and surgery, blood tests, and monitoring of organ function. Researchers will measure event-free survival at one year after each randomization sequence. Patients are followed through the different phases of induction, consolidation, and radiotherapy, with adherence to protocol visits and procedures required. Safety and treatment responses are closely monitored throughout the study, which continues until the end date in November 2032.

High-Risk Neuroblastoma Study 2 of SIOP-Europa-Neuroblastoma (SIOPEN)