High risk neuroblastoma starts in certain nerve cells and can sometimes return (relapse) or stop responding to standard therapies. BEACON2 is an international trial designed to find more effective drug combinations for children and young adults whose neuroblastoma has come back. Instead of testing just one drug, this “platform trial” allows researchers to test multiple combinations of treatments simultaneously. Patients are randomly assigned to different groups to see which new combinations are safest and most effective. By constantly updating the combinations being tested, the study aims to quickly identify better ways to improve survival and quality of life for children facing this aggressive cancer.
BEACON2 is an international, multi arm, randomised Phase I/II platform trial for paediatric patients with relapsed or refractory high risk neuroblastoma. The study utilises an adaptive design to evaluate the safety and efficacy of various novel therapeutic combinations. The trial has two Tiers; Tier 1 is the main randomisation for treatment arms (two initially with potential for more). Tier 2 includes a smaller dose expansion/confirmation cohortfor more novel experimental treatment combinations. The primary objective is to test novel treatments again current best avilable treatment in relapsed neuroblastoma, and the secondary objective is to evaluate the safety of the regimes, anti-tumour response, longer term outcome and quality of life.
