High Risk Neuroblastoma Study 1 (HR-NBL1)
Chief Investigator: Dr Ruth Ladenstein
Sponsor: St. Anna Kinderkrebsforschung, Vienna, Austria
Funders / Charity: SIOPEN
High Risk Neuroblastoma Study 1 (HR-NBL1) of SIOP-Europe (SIOPEN).
Open, multicentre randomised Phase III Trial; therapy optimisation study.
The initial aims was to test the hypothesis that myeloablative therapy (MAT) with busulphan and melphalan (BUMEL) in patients with high risk neuroblastoma (stage 4 disease older than one year, stages 2 and 3 disease with MycN amplification) will result in a superior, 3-year, event free survival (EFS) than MAT with continuous infusion carboplatin, etoposide and melphalan (CEM).
The secondary aim was to test the hypothesis that immunotherapy with chimeric 14.18 anti-GD2 antibody, following MAT, in addition to differentiation therapy with 13-cis retinoic acid, will improve 3-year EFS in patients with high-risk neuroblastoma.
Currently, with all substudies having randomisation questions closed, the main objective of the HR-NBL1 trial is to correlate clinical, therapeutic and molecular biological features with Event Free Survival (EFS).
As secondary objective, metastatic and primary tumour response and long - term Overall Survival (OS) and toxcicity or sequelae to be investigated.
Current study status
Recruitment:
First Pt. First Visit (FPFV) - 15.02.2002
Last pt. enrolled 14.06.2023
Recruitment stopped Jul 2023
Study to be closed Dec 2024.
Total patients recruited: 3.569 patients
Participating countries and sites
23 countries
188 active sites from the 209 planned
