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

ANBL1821 A Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab with or without Eflornithine (DFMO) (IND# 141913) in Children with Relapsed, Refractory or Progressive Neurobl astoma GOALS AND OBJECTIVES (SCIENTIFIC AIMS) 1.1 Primary Aims 1.1.1 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. 1.2 Secondary Aims 1.2.1 To compare progression-free survival and overall survival between patients receiving dinutuximab, irinotecan and temozolomide with and without the addition of DFMO. 1.2.2 To define the toxicity profile of DFMO administered with dinutuximab, irinotecan and temozolomide. 1.3 Exploratory Aims 1.3.1 To characterize the immune and cytokine profiles of patients treated with DFMO/chemotherapy/dinutuximab combination and correlate with response to therapy. 1.3.2 To evaluate GD2 levels in tumor cells from patient bone marrow samples and correlate with response to therapy. 1.3.3 To explore whether the addition of DFMO to the dinutuximab and chemotherapy backbone affects pain as determined by patient report and opiate usage.