Main
aggressive extracranial solid tumor, toxicities, Neuroblastoma, ALK-driven
NEUROBLASTOMA, NEUROBLASTOMA, SECONDARY
Neuroblastoma is an aggressive extracranial solid tumor of childhood that is responsible for a disproportionate number of pediatric cancer-related deathsLorlatinib, a third-generation macrocyclic inhibitor of ALK and ROS1, was developed to maintain potency across resistant Lorlatinib demonstrates high potency across acquired Here we present the safety, tolerability and anti-tumor activity of a first-in-child New Approaches to Neuroblastoma Therapy (NANT) Consortium phase 1 study (NANT2015-02) of lorlatinib in children, adolescents and adults with ALK-driven refractory or relapsed neuroblastoma. Primary aims were to determine the toxicities, pharmacokinetics and RP2D of lorlatinib administered both as monotherapy and in combination with topotecan/cyclophosphamide. A secondary aim was to evaluate the anti-tumor activity by determining the RR (complete response (CR) and partial response (PR)) and a modified RR (CR/PR/minor responses (MRs)) (as defined in NANT version 2.0 response criteria