ANBL1821, A Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab with or without Eflornithine (DFMO) (IND# 141913) in Children with Relapsed, Refractory or Progressive Neuroblastoma
Study ID: STU-2019-1024
This will be a prospective, randomized phase 2 trial to determine whether the addition of DFMo to the dinutuximab/irinotecan/temozolomide backbone improves the response rate compared to the backbone alone. Patients will be randomized 1:1 to Regimen a (irinotecan/ temozolomide/dinutuxumab) or Regimen B (DFMo + irinotecan/temozolomide/ dinutuximab), stratified by disease category (measurable vs. evaluable), prior anti-GD2 therapy (prior exposure to anti-GD2 antibody vs. no prior exposure to anti-GD2 antibody), prior DFMo therapy (prior exposure to DFMo vs. no prior exposure to DFMo), and MYCn status (amplified vs. non-amplified vs. unknown).
Primary aim - To determine whether administration of eflornithine (DFMo) in combination with dinutuximab, irinotecan and temozolomide results in an improved response rate compared to dinutuximab, irinotecan and temozolomide in patients with relapsed or refractory neuroblastoma and therefore is a therapeutic regimen worthy of further testing in patients with newly-diagnosed high-risk neuroblastoma.