SIOPEN High-Risk Neuroblastoma Clinical Trial 2 to open in the UK in 2021
Neuroblastoma UK with Solving Kids’ Cancer has awarded a grant of £609,762.40 to enable the SIOPEN High-Risk Neuroblastoma Clinical Trial 2 (HR-NBL2) to open in the UK. The trial is anticipated to open in early 2021.
This Phase 3 clinical trial will offer children in the UK, with a diagnosis of high-risk neuroblastoma, to have the same opportunities as children across Europe and take part in this pan-European study, enabling a series of important randomised questions to be answered within the overall framework of the trial.
Tony Heddon, Chair of Neuroblastoma UK said, “We are delighted to join forces with Solving Kids Cancer in funding this crucial clinical research trial. As a charity, we receive no government funding - it is thanks to our generous supporters that we can provide funding of £175,000 to support this vital research, which offers real potential to develop more effective and kinder treatment for children with high-risk neuroblastoma in the UK.”
“We are committed to ensuring that research into neuroblastoma continues beyond the coronavirus pandemic. International collaboration to understand more about this rare disease is hugely important and can help to ensure that children in the UK can have equal access to new treatment. By funding this ten-year project, we can give children across the UK a chance to access treatment which could potentially save their lives.”
Principal Investigators: Dr Martin Elliott (Leeds Children’s Hospital), Dr Mark Gaze (University College Hospital, London), Professor Pamela Kearns (CRUK Clinical Trials Unit, University of Birmingham), Dr Juliet Gray (University of Southampton).
Total project award: £609,762.40
Funding Partners: Solving Kids’ Cancer (£434,762.40) and Neuroblastoma UK (£175,000)
Date of award: April 2020
The HR-NBL2 clinical trial is a 10-year study, which will enable children in the UK to access this Europe wide trial. The study is anticipated to open in early 2021.
This commitment of funding is absolutely crucial for children with high-risk neuroblastoma in the UK enabling them to have equal access to this pan-European study. The trial builds on the successes of the previous High-Risk Neuroblastoma Trial 1 (HR-NBL1) pan-European study developed by SIOPEN and provides a clinical trial that ensures equal access to the very best treatments and care across the UK, and a pathway for future innovative therapies to be safely administered and studied.
UK patients accounted for 20% of patients recruited to the SIOPEN HR-NBL1 and hence made a major contribution to international research. Opening of the SIOPEN HR-NBL2 trial in the UK will offer children newly diagnosed with high-risk neuroblastoma in the UK the same opportunity as children across Europe:
To take part in a clinical trial with the potential to significantly improve their chance of survival
To benefit from ongoing plans to incorporate clinical trials of targeted agents into upfront treatment for high-risk neuroblastoma, most notably by the addition an ALK inhibitor for patients whose tumours harbour aberations in the ALK gene.
To participate in research looking at how the presence and levels of tumour markers in blood, circulating tumour DNA (ctDNA), might be used at diagnosis and for ongoing disease response assessments instead of existing more invasive imaging and biopsy techniques.
To benefit from ongoing plans to study whether adding anti-GD2 monoclonal antibody therapy alongside induction chemotherapy will improve survival for children with high-risk neuroblastoma.
To contribute to further improvements in clinical outcomes in neuroblastoma by participation in and be able to benefit from the introduction of future innovations over the course of the next 5-year cycle
The trial is anticipated to open in the UK in early 2021 will open across the UK in January 2021, as part the wider SIOPEN pan-European trial. It is a ten-year longitudinal study that includes five years of clinical investigation and five years of corresponding data collection and research analysis.
The trial is currently open in France, the sponsor nation, and the UK will be joining in 2021. The University of Birmingham Cancer Research UK Clinical Trials Unit is the UK national coordinator and will have the responsibility for delivering the trial across UK sites.
“As the UK lead investigator of the new European High-Risk Neuroblastoma 2 trial, I am extremely grateful to Solving Kids’ Cancer and Neuroblastoma UK for funding the opening and running of the trial in the UK allowing UK patients and families to actively contribute to future improvements in treatment and survival of children with neuroblastoma through participation in high quality, collaborative international research and build on the advances in treatment and in survival of patients, resulting from the previous High-Risk Neuroblastoma 1 Trial, which have now been incorporated in to standard treatment in the NHS.” writes Dr Martin Elliot, Consultant Paediatric Oncologist.
“The funding will secure the ability for UK clinicians to offer high-risk neuroblastoma patients and their families access to a clinical trial, which I am certain will be highly appreciated by families recruited to the trial and even more so by patients and families that will benefit from the potential positive outcomes of this research, which will hopefully lead to further improvements in cure rates of children with this devastating cancer. As an investigator I am excited to work with doctors and research teams in all UK paediatric oncology centres, to enable this trial to be an option for newly diagnosed patients and families across the UK in the near future.”
Around 100 children a year in the UK are diagnosed with neuroblastoma, of which approximately half will be classified as high-risk. It is a rare and aggressive childhood cancer and the vast majority of children diagnosed are under 5, despite intensive multi-modal therapy long-term survival from the high-risk form of the disease remains around 40-50%.
