SIOPEN High Risk Neuroblastoma Study 2 (HR-2)
Chief Investigator: Dr Claudia Pasqualini
Sponsor: Institut Gustave Roussy, Paris
Funders / Charity: Institut National du Cancer, Solving Kids Cancer, Enfants Cancers Santé
International multicenter, open-label, randomized phase III trial including three sequential randomizations to assess the efficacy of induction and consolidation chemotherapies and radiotherapy for patients with high-risk neuroblastoma.
This study aims to improve the outcome for patients with newly diagnosed high-risk neuroblastoma.
The treatment of high-risk neuroblastoma is complex, and involves a number of phases:
Initial “induction” chemotherapy
Surgery to remove the primary tumour
"Consolidation" with high-dose chemotherapy followed by autologous stem cell rescue (ASCR)
Radiotherapy to the site of the primary tumour
Maintenance therapy with anti-GD2 immunotherapy and retinoic acid.
The SIOPEN HR-2 study aims to answer several questions:
To compare two different induction chemotherapy regimens: Rapid-COJEC and the GPOH.
To investigate the impact of giving more intensive consolidation: comparing standard single course of Busulfan-Melphalan (Bu-Mel) with two courses of high dose chemotherapy (Thiotepa followed by Bu-Mel), autologous stem each course being supported by ASCR
In patients with a macroscopic residue disease before radiotherapy, to evaluate the benefit of a radiotherapy boost to the residue ( total 36 Gy) in comparison to the standard 21 Gy dose delivered to the site of the primary tumour.
All patients enrolled in the trial must have newly diagnosed High Risk Neuroblastoma
The study opened in France in 2020, and aims to recruit 800 patients over 6 years. Other countries are in set up, with a plan to open in 26 countries, which includes a total of 270 sites: Germany, Austria, Belgium, Denmark, Greece, Ireland, Israel, Italy, The Netherlands, Poland, Spain, Switzerland, United Kingdom, Australia and New Zealand; Croatia; Finland; Hungary; Lithuania; Norway; Portugal; Czech Republic; Slovakia; Slovenia and Sweden. Currently, the study has already recruited 394 patients and is open in 122 sites.
There are no results available from the study yet.
Further details about the trial can be found at clinicaltrials.gov database: https://clinicaltrials.gov/ct2/show/NCT04221035
Patient information videos
Information introduction to HR Neuroblastoma
Information on Induction, Consolidation, and Maintenance treatments
Information on Stem Cell Harvest, Radiotherapy and Surgery.
What happens after treatment for HR Neuroblastoma?
