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. Author manuscript; available in PMC: 2022 Feb 1.
Published in final edited form as: Curr Opin Pediatr. 2020 Dec 29;33(1):49–58. doi: 10.1097/MOP.0000000000000981

Table 2.

Clinical trials in pediatric hematologic malignancies testing RAS pathway inhibitors.

Trial identifier Study name Disease Targeted agent Class

Hematologic malignancy
NCT03190915 Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia JMML Trametinib MEK inhibitor
NCT03705507 International Trial of Selumetinib in Combination With Dexamethasone for the Treatment of Acute Lymphoblastic Leukaemia (SeluDex) ALL Selumetinib MEK inhibitor
NCT03585686 A Combination of Vemurafenib, Cytarabine and 2-chlorodeoxyadenosine in Children With LCH and BRAF V600E Mutation LCH Vemurafenib BRAF inhibitor
Pediatric MATCH

NCT04284774 Tipifarnib for the Treatment of Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With HRAS Gene Alterations, a Pediatric MATCH Treatment Trial Solid Tumors, non-Hodgkin lymphoma, or histiocytic Disorders with HRAS mutations Tipifarnib Farnesyltransferase inhibitor
NCT03220035 Vemurafenib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With BRAF V600 Mutations (A Pediatric MATCH Treatment Trial) Solid Tumors, non-Hodgkin lymphoma, or histiocytic Disorders with BRAF V600E mutations Vemurafenib BRAF inhibitor

NCT03213691 Selumetinib Sulfate in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Activating MAPK Pathway Mutations (A Pediatric MATCH Treatment Trial) Solid Tumors, non-Hodgkin lymphoma, or histiocytic Disorders with MAPK pathway mutations Selumetinib MEK inhibitor
NCT03698994 Ulixertinib in Treating Patients With Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With MAPK Pathway Mutations (A Pediatric MATCH Treatment Trial) Solid Tumors, non-Hodgkin lymphoma, or histiocytic Disorders with MAPK pathway mutations Ulixertinib ERK inhibitor