TRLS-10. ROVER: A PHASE 1/2 STUDY OF AVAPRITINIB IN PEDIATRIC PATIENTS WITH SOLID TUMORS DEPENDENT ON KIT OR PDGFRA SIGNALING

Abstract Pediatric patients with advanced relapsed/refractory (R/R) solid (including central nervous system [CNS]) tumors have poor prognoses. KIT alterations are common in germ cell tumors and high-grade glioma (HGG); platelet-derived growth factor receptor alpha (PDGFRA) alterations are common in...

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Published in:Neuro-oncology (Charlottesville, Va.) Va.), 2023-06, Vol.25 (Supplement_1), p.i81-i81
Main Authors: Koschmann, Carl, Hoffman, Lindsey M, Kramm, Christof M, Plant-Fox, Ashley, Abdelbaki, Mohamed S, Bui, Ashley, Casanova, Michela, Morgenstern, Daniel A, Swamy, Preethi, Shi, Hongliang, Hong, Janet, Rinne, Mikael L, Chi, Susan N
Format: Article
Language:English
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Final Category: Translational Therapeutics/Clinical Trials - TRLS
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Summary:Abstract Pediatric patients with advanced relapsed/refractory (R/R) solid (including central nervous system [CNS]) tumors have poor prognoses. KIT alterations are common in germ cell tumors and high-grade glioma (HGG); platelet-derived growth factor receptor alpha (PDGFRA) alterations are common in sarcoma and HGG. Diffuse midline gliomas with H3K27-altered (DMG-H3K27-altered) depend on PDGFRA signaling for tumor growth. However, no KIT-/PDGFRA-targeted therapies are currently approved for pediatric patients with R/R solid or CNS tumors, including DMG-H3K27-altered. The selective KIT and PDGFRA inhibitor avapritinib has demonstrated potent activity against KIT activation-loop (exon 17) and juxtamembrane (exon 11) mutants (IC50
ISSN:1522-8517
1523-5866
DOI:10.1093/neuonc/noad073.313