{"entity":{"id":"hr-nbl1","kind":"trial","name":"SIOPEN HR-NBL1","aka":[],"tldr":"Europe's long-running high-risk neuroblastoma trial set busulfan-melphalan as the transplant regimen and showed that adding IL-2 to anti-GD2 therapy added toxicity but not benefit.","summary":"R1: BuMel superior to CEM (3-year EFS 50% vs 38%; Lancet Oncol 2017). R2: dinutuximab beta + IL-2 vs dinutuximab beta alone: 3-year EFS 56% vs 60%, no benefit, more toxicity (Lancet Oncol 2018). R3/R4 explored induction regimens (rapid COJEC vs modified N7; 2025 report). ClinicalTrials.gov lists an estimated 3,300 participants; enrolment opened in 2002.","status":"mixed","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT01704716","url":"https://clinicaltrials.gov/study/NCT01704716"}],"tags":[],"related":[],"cancers":["neuroblastoma","neuroblastoma-high-risk"],"sections":[],"technologies":["autologous-stem-cell-transplant"],"targets":[],"drugs":["dinutuximab","busulfan"],"companies":["cclg"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":["ruth-ladenstein"],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"nct":"NCT01704716","phase":"3","setting":"High-risk neuroblastoma (Europe): multiple randomisations, including busulfan-melphalan vs CEM conditioning and dinutuximab beta ± IL-2","sponsor":"SIOPEN / Children's Cancer and Leukaemia Group","result":"BuMel > CEM (EFS 50% vs 38%); IL-2 adds no benefit to anti-GD2.","started":"2002-02","yearReported":2017,"enrolled":3300,"enrolledBasis":"registry","outcomes":[{"endpoint":"Event-free survival at 3 years (R1 conditioning)","unit":"%","arms":[{"name":"Busulfan-melphalan","n":296,"value":50},{"name":"CEM","n":302,"value":38}],"p":"0.0005","source":"https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(17)30070-0/fulltext"},{"endpoint":"Event-free survival at 3 years (R2 immunotherapy)","unit":"%","arms":[{"name":"Dinutuximab beta + IL-2","n":200,"value":56},{"name":"Dinutuximab beta","n":206,"value":60}],"source":"https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(18)30578-3/fulltext"}]},"route":"/trials/hr-nbl1/","neighbours":{"cancer":[{"id":"neuroblastoma-high-risk","kind":"cancer","name":"High-risk neuroblastoma","route":"/cancers/neuroblastoma-high-risk/"},{"id":"neuroblastoma","kind":"cancer","name":"Neuroblastoma (paediatric)","route":"/cancers/neuroblastoma/"}],"technology":[{"id":"autologous-stem-cell-transplant","kind":"technology","name":"Autologous stem cell transplant (high-dose therapy)","route":"/technologies/autologous-stem-cell-transplant/"}],"drug":[{"id":"busulfan","kind":"drug","name":"Busulfan","route":"/drugs/busulfan/"},{"id":"dinutuximab","kind":"drug","name":"Dinutuximab (ch14.18) / dinutuximab beta","route":"/drugs/dinutuximab/"}],"company":[{"id":"cclg","kind":"company","name":"Children's Cancer and Leukaemia Group","route":"/companies/cclg/"}],"person":[{"id":"ruth-ladenstein","kind":"person","name":"Ruth Ladenstein","route":"/people/ruth-ladenstein/"}],"pairing":[{"id":"il2-anti-gd2-caution","kind":"pairing","name":"Caution: IL-2 added to anti-GD2 therapy","route":"/pairings/il2-anti-gd2-caution/"}],"bottleneck":[{"id":"b-rare-cancers","kind":"bottleneck","name":"Rare and paediatric cancers without markets","route":"/bottlenecks/b-rare-cancers/"}],"paper":[{"id":"paper-hr-nbl1-busulfan-melphalan-ladenstein-lancet-oncol-2017","kind":"paper","name":"HR-NBL1/SIOPEN: busulfan-melphalan versus carboplatin-etoposide-melphalan high-dose chemotherapy for high-risk neuroblastoma","route":"/key-papers/paper-hr-nbl1-busulfan-melphalan-ladenstein-lancet-oncol-2017/"}],"institution":[{"id":"siop-europe","kind":"institution","name":"SIOP Europe (European Society for Paediatric Oncology)","route":"/institutions/siop-europe/"}],"roadmap":[{"id":"paediatric-oncology-roadmap","kind":"roadmap","name":"Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first","route":"/roadmaps/paediatric-oncology-roadmap/"}]}}