Europe-wide frontline high-risk neuroblastoma trial

Study name SIOPEN High-risk neuroblastoma 2
Study type Phase III clinical trial
Principal investigator(s) Dr Martin Elliott, Dr Mark Gaze, Prof Pamela Kearns, Dr Juliet Gray
Institutions Trial will be open at 20 Principal Treatment Centres across the UK
Partners Neuroblastoma UK
Total awarded £609,762.40
Solving Kids’ Cancer UK contribution £434,762


The treatment protocol for high-risk neuroblastoma is one of the most intense and toxic of any cancer. It involves surgery, chemotherapy, radiation, stem-cell transplant and immunotherapy. Even with these many steps that involve harsh treating agents, there are still many cases where the treatment is not effective, or the cancer returns which leads to little options being left. The SIOPEN high-risk neuroblastoma 2 trial is investigating the effectiveness of current standard therapies across Europe as well as trialling new approaches in induction chemotherapy, consolidation chemotherapy and radiotherapy.

The three randomisations to find the most effective treatment are:

  • Comparing two induction chemotherapies- RAPID COJEC (commonly used in the UK) and GPOH (used widely across Europe).
  • Comparing single high-dose chemotherapy (standard use in UK) with tandem high-dose chemotherapy (uses an additional cycle of HDC- used in some European countries and standard in the US).
  • Comparing standard radiotherapy (21.6 Gy) with standard plus a sequential boost (21.6 Gy + 14.4 Gy) in cases where there is a large amount of residual disease present


The outcome of this trial could lead to changes in standard protocols across Europe and an alignment in the approach taken by different countries. The predecessor to this trial, SIOPEN High-Risk Neuroblastoma 1, led to significant changes in treatment across Europe including the addition of immunotherapy, which we know has led to improved survival. The hope is that gathering data on this scale can lead to similar impact on the trial's completion.

"The international community is exceptionally grateful to Solving Kids’ Cancer and Neuroblastoma UK for funding SIOPEN High-risk neuroblastoma 2 trial in the United Kingdom. The participation of the United Kingdom in this trial is absolutely crucial for children with high-risk neuroblastoma in the UK and ultimately the inclusion of the United Kingdom in this study will have a very positive benefit for children in Europe and globally."

Prof Andy Pearson, Chair of SAB Solving Kids’ Cancer