Table 1.

Genomic targets and associated agents available commercially or through ongoing clinical trials

TargetGenetic alterationAvailable agentsAgents under investigationActive pediatric clinical trials
FLT3 FLT3-ITD Midostaurin
Sorafenib
Gilteritinib 
Gilteritninb
Crenolanib
Quizartinib 
NCT04293562
NCT03793478
NCT04240002 
FLT3-TKD Midostaurin
Gilteritinib 
Gilteritinib
Crenolanib 
NCT04293562
 
BCL2 — Venetoclax
 
Venetoclax
Navitoclax
Obatoclax
 
NCT03194932
NCT03826992
NCT03236857
NCT04898894
NCT05317403 
IDH IDH1 Ivosidenib — — 
IDH2 Enasidenib Enasidenib NCT04203316 
MEK NRAS
KRAS
PTPN11
NF1
CBL 
Trametinib Trametinib NCT03190915 
Menin KMT2Ar
NPM1
NUP98 
— SNDX-5613
KO-539
JNJ-75276617
DSP 5336
BMF-219 
NCT04065399
NCT05326516 
E-selectin E-selectin ligand expression — Uproleselan NCT05146739 
XPO1 — Selinexor Selinexor NCT04898894 
TargetGenetic alterationAvailable agentsAgents under investigationActive pediatric clinical trials
FLT3 FLT3-ITD Midostaurin
Sorafenib
Gilteritinib 
Gilteritninb
Crenolanib
Quizartinib 
NCT04293562
NCT03793478
NCT04240002 
FLT3-TKD Midostaurin
Gilteritinib 
Gilteritinib
Crenolanib 
NCT04293562
 
BCL2 — Venetoclax
 
Venetoclax
Navitoclax
Obatoclax
 
NCT03194932
NCT03826992
NCT03236857
NCT04898894
NCT05317403 
IDH IDH1 Ivosidenib — — 
IDH2 Enasidenib Enasidenib NCT04203316 
MEK NRAS
KRAS
PTPN11
NF1
CBL 
Trametinib Trametinib NCT03190915 
Menin KMT2Ar
NPM1
NUP98 
— SNDX-5613
KO-539
JNJ-75276617
DSP 5336
BMF-219 
NCT04065399
NCT05326516 
E-selectin E-selectin ligand expression — Uproleselan NCT05146739 
XPO1 — Selinexor Selinexor NCT04898894 
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