ANBL1531: A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or Crizotinib Added to Intensive Therapy for Children with Newly Diagnosed High-Risk Neuroblastoma (NBL) (IND# 134379)
ANBL1531
What is the goal of the study?
This is a complicated Phase 3 trial for newly diagnosed high risk NBL that builds off of the successful results of ANBL0532 and prior pilot studies in COG that demonstrated feasibility of delivering I131 MIBG within COG trial and feasibility of rapid genomic testing of diagnostic tissue. The trial will study the addition of 2 different targeted therapies; I131 MIBG for those with MIBG avid tumors and crizotinib for those with tumors harboring ALK aberrations. The rationale for use of I131-MIBG is strong, most active single agent studied in refractory/recurrent NBL with response rate of 30-40%. The study builds off of a COG pilot that determined feasibility of delivering I131 MIBG; moves the MIBG therapy sooner in induction to avoid long delays to Consolidation therapy. Dose of MIBG will be supported with PBSC rescue to ensure timeliness of subsequent induction chemotherapy. Patients with MIBG avid tumors will be randomized to one of 3 arms; standard tandem transplant, I131 MIBG with tandem transplant and I131 MIBG with BuMel (superior arm in recent SIOPEN trial). Subjects with non-MIBG avid tumors will be non-randomly assigned to standard induction and tandem to prospectively evaluate their outcome. Subjects with ALK aberrations will be nonrandomly assigned to crizotinib + standard therapy. Tissue at diagnosis required for standard biology/histology and now ALK testing performed at Biopath Central Lab. Correlative studies include circulating tumor cells, circulating GD2, biomarkers of MIBG response, immune profiling, metabolomic profiling related to Busulfan clearance, prospective evaluation of MIBG response and patterns of failure.
Who can participate in the study?
Please contact the study team listed below to learn more.