New title: Randomized, international and multicentric phase 3 study that evaluates and compares 2 treatment strategies in 3 therapeutic phases (induction, high-dose chemotherapy and radiotherapy) for patients with high-risk neuroblastoma and introduces chemoimmunotherapy for patients with insufficient metastatic response after induction chemotherapy. Old title: “Randomized, international and multicentric phase 3 study that evaluates and compares 2 treatment strategies in 3 therapeutic phases (induction, high-dose chemotherapy and radiotherapy) for patients with high-risk neuroblastoma”

Research summary

High-risk neuroblastoma (HR-NBL). remains one of the areas of unmet need in childhood cancer, as despite improvements in survival, current treatment strategies only cure 40% of patients. The SIOPEN High-Risk Neuroblastoma 2 (HR-NBL2) trial is a pan-European phase 3 randomised clinical trial that aims to improve the outcome of children with HR-NBL It will seek to improve the upfront treatment of this population of children by comparing i) 2 different induction chemotherapy regimens ii) intensifying high dose chemotherapy iii) increasing radiotherapy dose in children with residual disease following surgery. The HR-NBL2 trial has 3 primary aims: • Induction chemotherapy : The trial is looking to compare 2 well established induction chemotherapy regimens to find out if either provides better outcomes, including survival and to compare the side effect profile of the regimens. • Consolidation chemotherapy : The trial is looking to find out if two courses of high dose chemotherapy (HDC) each followed by a separate stem cell transplant results in better outcomes than the standard single course of high dose chemotherapy and single stem cell transplant, as consolidation therapy. • Radiotherapy: The trial will compare the standard dose of radiation to the standard dose plus an additional boost dose targeted to the residual tumour. Outcomes including survival will be compared. The study will recruit 800 patients with high-risk neuroblastoma internationally over a 5-year period with an estimated 206 from the UK. All eligible HR-NBL patients will be able to enter the trial and the will be offered to participate in the three randomised aspects of treatment. The primary endpoint will be 3-year event free survival (EFS) from the time of each randomisation. Secondary end points will include Overall Survival and treatment related toxicity and morbidity.

Principal Investigator

Dr Amy Mitchell

Contact us

Email: Paedhaem.oncresearch@ouh.nhs.uk

IRAS number

284657