Table 3.

Examples of precision trials in pediatric oncology

Trial type	Examples of precision trials	Sponsor	ClinicalTrials ID
Basket in relapsed/refractory cancers across multiple diagnoses	NCI–COG Pediatric MATCHAcSé-ESMART	COG/NCIGustave Roussy	NCT03155620NCT02813135
Disease-specific umbrella in patients with progressive disease	Ruxolitinib or Dasatinib with Chemotherapy in Ph-Like ALLNEPENTHE (Neuroblastoma)	MD AndersonCHOP	NCT02420717NCT02780128
Single-agent targeted therapy in advanced cancers	Larotrectinib in NTRK Fusion Positive TumorsEZH2 Inhibitor Tazemetostat in INI-1 Negative tumorsCrizotinib for Tumors with an ALK, MET or ROS1 alterationLDK378 (Ceritinib) in ALK-activated Pediatric TumorsDabrafenib with Trametinib for BRAF V600 Positive TumorsAfatinib in Pediatric Tumors with ErbB Pathway Deregulation	LOXO OncologyEpizymeUNICANCERNovartisNovartisBoehringer Ingelheim	NCT02637687NCT02601937NCT02034981NCT01742286NCT02684058NCT02372006
Disease-specific trials in newly diagnosed patients	Total Therapy XVII JAK/STAT Mutations in ALL and LymphomaAddition of Dasatinib for ALL with TKI-targetable FusionsCombination Therapy Plus Dasatinib for Ph-Like B-ALLClinical and Molecular Risk-Directed Therapy (Medulloblastoma)BIOMEDE (DIPG)	St. JudeDFCICOG/NCISt. JudeGustave Roussy	NCT03117751NCT03020030NCT02883049NCT01878617NCT02233049

ALL, acute lymphoblastic leukemia; AcSé-ESMART, Secured Access Program of the French National Cancer Institute (INCa); COG, Children's Oncology Group; ESMART, European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors in Children; MATCH, Molecular Analysis for Therapy Choice; NCI, National Cancer Institute; NEPENTHE, Next Generation Personalized Neuroblastoma Therapy; Ph-like, Philadelphia chromosome-like.