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Profile Name FLT3 exon 14 ins
Gene Variant Detail

FLT3 exon 14 ins (gain of function - predicted)

Relevant Treatment Approaches FLT3 Inhibitor

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Molecular Profile Indication/Tumor Type Response Type Relevant Treatment Approaches Therapy Name Approval Status Evidence Type Efficacy Evidence References
FLT3 exon 14 ins acute myeloid leukemia sensitive CCT137690 + Palbociclib Preclinical - Cell culture Actionable In a preclinical study, the combination therapy of Ibrance (palbociclib) and CCT137690 resulted in a synergistic effect in acute myeloid leukemia cells harboring a FLT3-ITD, demonstrating a greater reduced cell viability compared to either agent alone (PMID: 30544932). 30544932
FLT3 exon 14 ins Advanced Solid Tumor sensitive FLT3 Inhibitor Midostaurin Preclinical - Cell culture Actionable In a preclinical study, Rydapt (midostaurin) demonstrated efficacy in inhibiting proliferation and viability of transformed cells expressing a FLT3-ITD mutation in culture (PMID: 12124173). 12124173
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor ENMD-2076 Phase I Actionable In a Phase I trial, an acute myeloid leukemia patient with a FLT3-ITD mutation demonstrated anti-leukemia activity when treated with ENMD-2076 (PMID: 27406088). 27406088
FLT3 exon 14 ins acute myeloid leukemia sensitive A-1210477 + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, A-1210477 and Venclexta (venetoclax) combination treatment synergistically inhibited viability of acute myeloid leukemia cell lines harboring FLT3 internal tandem duplication (ITD) in culture (PMID: 31751472). 31751472
FLT3 exon 14 ins acute myeloid leukemia sensitive Alisertib + Palbociclib Preclinical - Cell culture Actionable In a preclinical study, the combination therapy of Ibrance (palbociclib) and Alisertib (MLN8237) resulted in a synergistic effect in acute myeloid leukemia cells expressing a FLT3-ITD, demonstrating a greater reduced cell viability compared to either agent alone (PMID: 30544932). 30544932
FLT3 exon 14 ins acute myeloid leukemia sensitive Daunorubicin + LAM-003 Preclinical - Cell culture Actionable In a preclinical study, LAM-003 and Cerubidine (daunorubicin) combination treatment synergistically reduced viability of acute myeloid leukemia cell lines harboring FLT3 internal tandem duplication (ITD) in culture (PMID: 31751472). 31751472
FLT3 exon 14 ins acute myeloid leukemia predicted - sensitive FLT3 Inhibitor Cytarabine + Quizartinib Phase Ib/II Actionable In a Phase I/II trial, Quizartinib (AC220) in combination with Cytosar-U (cytarabine) resulted in a median overall survival of 6.7 months and a median progression-free survival of 3 months in acute myeloid leukemia patients harboring FLT3 ITD mutations (ASH 59th Annual Meeting and Exposition, Dec 2017, Abstract 723). detail...
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor HQP1351 Preclinical - Cell line xenograft Actionable In a preclinical study, GZD824 (HQP1351) reduced Flt3 and Stat5 phosphorylation, induced cell cycle arrest and apoptosis, and inhibited growth of acute myeloid leukemia cells harboring FLT3-ITD in culture, and suppressed tumor growth, decreased Flt3 activation, and induced apoptosis in cell line xenograft models (PMID: 32247263). 32247263
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3/CD3 Fabsc antibody Preclinical - Cell culture Actionable In a preclinical study, treatment of an acute myeloid leukemia (AML) cell lines or patient-derived xenograft (PDX)-derived AML cells harboring a heterozygous FLT3-ITD mutation with a Fabsc FLT3/CD3 bi-specific antibody resulted in near complete eradication of AML cells in culture (PMID: 28895560). 28895560
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Sorafenib + Trametinib Preclinical - Cell culture Actionable In a preclinical study, Nexavar (sorafenib) combined with Mekinist (trametinib) enhanced apoptosis in acute myeloid leukemia cell lines harboring FLT3 internal tandem duplication (ITD) mutations in culture (PMID: 28923853). 28923853
FLT3 exon 14 ins leukemia predicted - sensitive FLT3 Inhibitor GMI-1359 + Sorafenib Preclinical - Cell culture Actionable In a preclinical study, combination of GMI-1359 with Nexavar (sorafenib) enhanced apoptosis compared to Nexavar (sorafenib) alone (48.9% vs 36.6%) in leukemia cell lines harboring FLT3 internal tandem duplication (ITD) mutations (Blood 2015 126(23):3790). detail...
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor UNC1666 Preclinical - Cell culture Actionable In a preclinical study, UNC1666 inhibited FLT3 phosphorylation and downstream signaling, induced apoptosis of acute myeloid leukemia cells harboring FLT3 exon 14 insertions (ITD) in culture (PMID: 25762638). 25762638
FLT3 exon 14 ins acute myeloid leukemia conflicting FLT3 Inhibitor Midostaurin Preclinical - Cell culture Actionable In a preclinical study, co-culturing human stromal cells with an acute myeloid cell line harboring a FLT3 internal tandem duplication (ITD) demonstrated resistance to Rydapt (midostaurin) treatment in culture (PMID: 31751472). 31751472
FLT3 exon 14 ins acute myeloid leukemia conflicting FLT3 Inhibitor Midostaurin Preclinical - Cell culture Actionable In a preclinical study, Rydapt (midostaurin) inhibited Flt3 phosphorylation and growth of acute myeloid leukemia cell lines harboring FLT3 exon 14 insertions (ITD) in culture (PMID: 31309543). 31309543
FLT3 exon 14 ins hematologic cancer sensitive FLT3 Inhibitor HQP1351 Preclinical - Cell culture Actionable In a preclinical study, GZD824 (HQP1351) inhibited growth of transformed cells expressing FLT3-ITD in culture (PMID: 32247263). 32247263
FLT3 exon 14 ins Advanced Solid Tumor sensitive FLT3 Inhibitor Brigatinib Preclinical - Cell culture Actionable In a preclinical study, Alunbrig (brigatinib) inhibited growth of transformed cells expressing FLT3 ITD in culture (PMID: 27780853). 27780853
FLT3 exon 14 ins hematologic cancer decreased response Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) inhibited growth of transformed hematologic cells expressing FLT3 exon 14 insertions (ITD) in culture with reduced potency compared to other kinase inhibitors (PMID: 31309543). 31309543
FLT3 exon 14 ins acute myeloid leukemia sensitive MK2206 + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, MK2206 and Venclexta (venetoclax) combination treatment synergistically induced cell death of acute myeloid leukemia cell lines harboring FLT3 internal tandem duplication (ITD) in culture (PMID: 31751472). 31751472
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor PD-0325901 + Sorafenib Preclinical - Patient cell culture Actionable In a preclinical study, Nexavar (sorafenib) and PD-0325901 synergistically induced apoptosis and inhibited proliferation of acute myeloid leukemia (AML) cell lines harboring FLT3 internal tandem duplication (ITD) mutations in culture, inhibited Erk phosphorylation in FLT3-ITD mutant AML patient cells, and reduced leukemic burden in a FLT3-ITD mutant AML cell line xenograft model (PMID: 28923853). 28923853
FLT3 exon 14 ins acute myeloid leukemia predicted - sensitive FLT3 Inhibitor Azacitidine + Quizartinib Phase Ib/II Actionable In a Phase I/II trial, Quizartinib (AC220) in combination with Vidaza (azacitidine) resulted in a median overall survival of 13.4 months and a median progression-free survival of 6.9 months in acute myeloid leukemia patients harboring FLT3 ITD mutations (ASH 59th Annual Meeting and Exposition, Dec 2017, Abstract 723). detail...
FLT3 exon 14 ins hematologic cancer sensitive FLT3 Inhibitor Crenolanib Preclinical - Cell culture Actionable In a preclinical study, Crenolanib inhibited growth of transformed hematologic cells expressing FLT3 exon 14 insertions (ITD) in culture (PMID: 31309543). 31309543
FLT3 exon 14 ins acute myeloid leukemia predicted - sensitive FLT3 Inhibitor Pacritinib Preclinical - Cell line xenograft Actionable In a preclinical study, Pacritinib (SB1518) induced apoptosis and inhibited proliferation of acute myeloid leukemia cells harboring a FLT3-ITD mutation in culture, and inhibited tumor growth and induced tumor regression in cell line xenograft models (PMID: 22829080). 22829080
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor MRX-2843 Preclinical - Cell line xenograft Actionable In a preclinical study, MRX-2843 inhibited Flt3 activation, resulted in growth inhibition in acute myeloid leukemia cell lines harboring FLT3 internal tandem duplication (ITD) in culture and in cell line xenograft models, and prolonged survival in patient-derived xenograft models (PMID: 27158668). 27158668
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor CG-806 Preclinical - Cell line xenograft Actionable In a preclinical study, CG-806 inhibited FLT3 downstream signaling, resulted in growth inhibition of acute myeloid leukemia cells harboring FLT3 exon 14 insertion (ITD) in culture and tumor inhibition in cell line xenograft models (Clin Cancer Res 2017; 23(24_Suppl):Abstract nr 25). detail... detail...
FLT3 exon 14 ins leukemia sensitive FLT3 Inhibitor E6201 Preclinical - Cell line xenograft Actionable In a preclinical study, E6201 induced growth inhibition and apoptosis in leukemia cell lines harboring FLT3 internal tandem duplications (ITD) and reduced tumor burden in xenograft models (PMID: 26822154). 26822154
FLT3 exon 14 ins leukemia sensitive FLT3 Inhibitor FN-1501 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with FN-1501 reduced phoshorylation of FLT3 and downstream STAT5, ERK, and AKT, induced apoptosis and cell-cycle arrest, and decreased proliferation in a human leukemia cell line harboring a FLT3-ITD mutation in culture, and induced tumor regression in xenograft models (PMID: 29357250). 29357250
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Selinexor + Sorafenib Phase Ib/II Actionable In a Phase I/II trial, combination of Selinexor and Nexavar (sorafenib) treatment resulted in complete remission in 29% (4/14) and more than 50% blast reduction in 14% (2/14) of patients with acute myeloid leukemia harboring FLT3 ITD and/or D835 mutations (ASH, 59th Annual Meeting and Exposition, Dec 2017, Abstract 1344; NCT01607892). detail...
FLT3 exon 14 ins acute myeloid leukemia predicted - sensitive FLT3 Inhibitor Cytarabine + Daunorubicin + Quizartinib Phase I Actionable In a Phase I trial (QuANTUM-First), the combination therapy, Quizartinib (AC220) with Cytosar-U (cytarabine) and Cerubidine (daunorubicin), resulted in 67% (6/9) of acute myeloid leukemia patients harboring a FLT3-ITD achieving a composite complete response and two patients achieving morphologic leukemia-free state (PMID: 29139135; NCT01390337). 29139135
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor FF-10101 Preclinical - Pdx & cell culture Actionable In a preclinical study, FF-10101 inhibited Flt3 autophosphorylation and cell growth of leukemic cells in culture and in AML-patient-derived xenograft models with FLT3-ITD mutations (PMID: 29187377). 29187377
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Cytarabine + G-749 Preclinical - Cell culture Actionable In a preclinical study, the combination of G-749 and Cytosar-U (cytarabine) resulted in a synergistic effect, demonstrating greater inhibition of proliferation compared to G-749 alone in acute myeloid leukemia cells harboring a FLT3 internal tandem duplication (PMID: 24532805). 24532805
FLT3 exon 14 ins acute myeloid leukemia sensitive GSK690693 + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, GSK690693 and Venclexta (venetoclax) combination treatment synergistically induced cell death of acute myeloid leukemia cell lines harboring FLT3 internal tandem duplication (ITD) in culture (PMID: 31751472). 31751472
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Azacitidine + Sorafenib Guideline Actionable Nexavar (sorafenib) in combination with Vidaza (azacitidine) is included in guidelines for patients with relapsed or refractory acute myeloid leukemia harboring a FLT3 internal tandem duplication (FLT3-ITD) mutation (NCCN.org). detail...
FLT3 exon 14 ins leukemia sensitive FLT3 Inhibitor Pexidartinib Preclinical - Cell line xenograft Actionable In a preclinical study, PLX3397 inhibited proliferation of human leukemia cells harboring FLT3-ITD in culture and in cell line xenograft models (PMID: 25847190). 25847190
FLT3 exon 14 ins acute myeloid leukemia predicted - sensitive Entospletinib Preclinical - Patient cell culture Actionable In a preclinical study, FLT3-ITD mutations correlated with sensitivity to Entospletinib in patient-derived acute myeloid leukemia samples in an ex vivo assay (PMID: 30333627). 30333627
FLT3 exon 14 ins acute myeloid leukemia sensitive Ipatasertib + Palbociclib Preclinical - Cell culture Actionable In a preclinical study, the combination therapy of Ibrance (palbociclib) and Alisertib (MLN8237) resulted in a synergistic effect in acute myeloid leukemia cells expressing a FLT3-ITD, demonstrating a greater reduced cell viability compared to either agent alone (PMID: 30544932). 30544932
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Sorafenib Preclinical - Patient cell culture Actionable In a preclinical study, FLT3-ITD mutations correlated with sensitivity to Nexavar (sorafenib) in patient-derived acute myeloid leukemia samples in an ex vivo assay (PMID: 30333627). 30333627
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Sorafenib Preclinical - Cell culture Actionable In a preclinical study, Nexavar (sorafenib) inhibited Flt3 phosphorylation and growth of acute myeloid leukemia cell lines harboring FLT3 exon 14 insertions (ITD) in culture (PMID: 31309543). 31309543
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Gilteritinib + LAM-003 Preclinical - Cell culture Actionable In a preclinical study, LAM-003 and Xospata (gilteritinib) combination treatment reduced viability of acute myeloid leukemia cell lines harboring FLT3 internal tandem duplication (ITD) in culture (PMID: 31751472). 31751472
FLT3 exon 14 ins acute myeloid leukemia resistant Avapritinib Preclinical - Cell culture Actionable In a preclinical study, Ayvakit (avapritinib) did not inhibit Flt3 phosphorylation or growth of acute myeloid leukemia cell lines harboring FLT3 exon 14 insertions (ITD) in culture (PMID: 31309543). 31309543
FLT3 exon 14 ins leukemia predicted - sensitive GMI-1359 Preclinical - Cell line xenograft Actionable In a preclinical study, GMI-1359 in combination with chemotherapy resulted in significant survival benefit compared to chemotherapy alone (67% vs 30%) in cell line xenograft models of FLT3-ITD leukemia (Blood 2015 126(23):3790). detail...
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Gilteritinib FDA approved - On Companion Diagnostic Actionable In a Phase III trial (ADMIRAL) that supported FDA approval, Xospata (gilteritinib) treatment resulted in complete remission (CR) or CR with partial hematologic recovery in 21% (29/138) of patients with relapsed or refractory acute myeloid leukemia harboring a FLT3 internal tandem duplication (ITD; exon 14 insertion), D835, or I836 mutation (FDA.gov; NCT02421939). detail... detail...
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Gilteritinib Guideline Actionable Xospata (gilteritinib) is included in the guidelines for patients with relapsed or refractory acute myeloid leukemia harboring a FLT3 internal tandem duplication (FLT3-ITD) mutation (NCCN.org). detail...
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Gilteritinib Phase Ib/II Actionable In a Phase I/II trial, treatment with Gilteritinib (ASP2215) at a dose of 80mg/day or higher resulted in an overall response rate of 55% (77/141) in patients with relapsed or refractory acute myeloid leukemia harboring FLT3 internal tandem duplication mutations (PMID: 28645776; NCT02014558). 28645776
FLT3 exon 14 ins B-cell acute lymphoblastic leukemia predicted - sensitive CD19/CD22 CAR T cells Case Reports/Case Series Actionable In a clinical case study, CD19/CD22 CAR T cell treatment resulted in rapid and durable suppression of leukemia cells in a patient with refractory acute B-cell lymphoblastic leukemia harboring a FLT3 internal tandem duplication (ITD) mutation (PMID: 32005917). 32005917
FLT3 exon 14 ins acute myeloid leukemia sensitive MK2206 + Palbociclib Preclinical - Cell culture Actionable In a preclinical study, the combination therapy of Ibrance (palbociclib) and MK2206 resulted in a synergistic effect in acute myeloid leukemia cells expressing a FLT3-ITD, demonstrating a greater reduced cell viability compared to either agent alone (PMID: 30544932). 30544932
FLT3 exon 14 ins acute myeloid leukemia sensitive Altiratinib Preclinical - Cell culture Actionable In a preclinical study, Altiratinib (DCC-0701) inhibited proliferation of an acute myeloid leukemia cell harboring a FLT3-ITD mutation in culture (PMID: 26285778). 26285778
FLT3 exon 14 ins acute myeloid leukemia sensitive LAM-003 + Tazemetostat Preclinical - Cell culture Actionable In a preclinical study, LAM-003 and Tazemetostat (EPZ-6438) combination treatment synergistically inhibited viability of acute myeloid leukemia cell lines harboring FLT3 internal tandem duplication (ITD) in culture (PMID: 31751472). 31751472
FLT3 exon 14 ins Advanced Solid Tumor sensitive FLT3 Inhibitor Pexidartinib Preclinical Actionable In a preclinical study, PLX3397 inhibited proliferation of transformed cells expressing FLT3-ITD in culture (PMID: 25847190). 25847190
FLT3 exon 14 ins acute myeloid leukemia sensitive Cytarabine + LAM-003 Preclinical - Cell culture Actionable In a preclinical study, LAM-003 and Cytosar-U (cytarabine) combination treatment synergistically reduced viability of an acute myeloid leukemia cell line harboring FLT3 internal tandem duplication (ITD) in culture (PMID: 31751472). 31751472
FLT3 exon 14 ins hematologic cancer sensitive FLT3 Inhibitor Gilteritinib Preclinical - Cell culture Actionable In a preclinical study, Xospata (gilteritinib) inhibited growth of transformed hematologic cells expressing FLT3 exon 14 insertions (ITD) in culture (PMID: 31309543). 31309543
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Ponatinib Phase I Actionable In a Phase I trial, Iclusig (ponatinib) resulted in a 25% (3/12) overall response rate, indicated as partial remission or better, in acute myeloid leukemia patients harboring FLT3-ITD (PMID: 23691988). 23691988
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Ponatinib Preclinical - Patient cell culture Actionable In a preclinical study, Iclusig (ponatinib) inhibited viability of patient derived acute myeloid leukemia cells harboring FLT3 ITD mutations in culture, and inhibited growth of tumors in cell line xenograft models (PMID: 21482694). 21482694
FLT3 exon 14 ins hematologic cancer sensitive FLT3 Inhibitor GTP-14564 Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing a FLT3 internal tandem duplication were sensitive to GTP-14564 treatment in culture, demonstrating decreased Stat5 activity and growth inhibition (PMID: 12815052). 12815052
FLT3 exon 14 ins acute myeloid leukemia no benefit FLT3 Inhibitor Rebastinib Phase I Actionable In a Phase I trial, acute myeloid leukemia patients harboring a FLT3 internal tandem duplication did not benefit from treatment with Rebastinib (DCC-2036) (PMID: 27927766). 27927766
FLT3 exon 14 ins Advanced Solid Tumor sensitive LAM-003 Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing FLT3 internal tandem duplication (ITD) demonstrated sensitivity to LAM-003 treatment in culture (PMID: 31751472). 31751472
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor G-749 Preclinical - Cell line xenograft Actionable In a preclinical study, G-749 induced apoptosis in acute myeloid leukemia cells harboring a FLT3 internal tandem duplication in culture, and inhibited tumor growth and induced tumor regression in cell line xenograft models (PMID: 24532805). 24532805
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Semaxanib Preclinical - Cell culture Actionable In a preclinical study, acute myeloid leukemia cells harboring a FLT3 internal tandem duplication were sensitive to Semaxanib (SU5416) in culture, demonstrating inhibition of cell proliferation and inhibition of Flt3, Mapk, and Stat5 phosphorylation (PMID: 12351406). 12351406
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Crenolanib Preclinical - Cell culture Actionable In a preclinical study, Crenolanib inhibited Flt3 phosphorylation and growth of acute myeloid leukemia cell lines harboring FLT3 exon 14 insertions (ITD) in culture (PMID: 31309543). 31309543
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Crenolanib Phase I Actionable In a Phase I trial, Crenolanib treatment resulted in an overall survival (OS) of 238 days in AML patients harboring FLT3 exon 14 insertions (ITD) that received no prior therapy, and an OS of 158 days in those progressed on TKIs (J Clin Oncol 34, 2016 (suppl; abstr 7008)). detail...
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Crenolanib Preclinical - Patient cell culture Actionable In a preclinical study, FLT3-ITD mutations correlated with sensitivity to Crenolanib in patient-derived acute myeloid leukemia samples in an ex vivo assay (PMID: 30333627). 30333627
FLT3 exon 14 ins acute myeloid leukemia sensitive LAM-003 + Venetoclax Preclinical - Patient cell culture Actionable In a preclinical study, LAM-003 and Venclexta (venetoclax) combination treatment synergistically induced cell death, and inhibited viability of acute myeloid leukemia cell lines and patient-derived cells harboring FLT3 internal tandem duplication (ITD) in culture, and increased survival in a cell line xenograft model (PMID: 31751472). 31751472
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Ki23819 Preclinical - Cell culture Actionable In a preclinical study, acute myeloid leukemia cells harboring a FLT3 internal tandem duplication were sensitive to Ki23819 in culture, demonstrating inhibition of autophosphorylation and downstream signaling, and reduced cell proliferation (PMID: 15815726). 15815726
FLT3 exon 14 ins acute myeloid leukemia sensitive LAM-003 Preclinical - Patient cell culture Actionable In a preclinical study, LAM-003 treatment inhibited colony formation, and reduced viability of acute myeloid cell lines harboring FLT3 internal tandem duplication (ITD) in the presence of stromal factors that confer resistance to Xospata (gilteritinib) and Crenolanib, and induced apoptosis in patient-derived cells in culture, and induced tumor regression, and increased survival in cell line xenograft models (PMID: 31751472). 31751472
FLT3 exon 14 ins hematologic cancer sensitive FLT3 Inhibitor Quizartinib Preclinical - Cell culture Actionable In a preclinical study, Quizartinib (AC220) inhibited growth of transformed hematologic cells expressing FLT3 exon 14 insertions (ITD) in culture (PMID: 31309543). 31309543
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor E6201 Preclinical - Cell culture Actionable In a preclinical study, E6201 induced apoptosis in blast samples derived from acute myeloid leukemia patients harboring FLT3 internal tandem duplications (ITD) in culture (PMID: 26822154). 26822154
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Cytarabine + Daunorubicin + Midostaurin FDA approved - On Companion Diagnostic Actionable In a Phase III trial that supported FDA approval, treatment with Rydapt (midostaurin), combined with Cytosar-U (cytarabine) and Daunorubicin, improved overall survival (74.7 mo vs 25.6 mo) in patients with FLT3-mutant (D835X and I836X) or FLT3-ITD (exon 14 insertions) acute myeloid leukemia compared to Cytosar-U (cytarabine) and Daunorubicin with placebo (PMID: 28644114). 28644114 detail... detail...
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Decitabine + Sorafenib Guideline Actionable Nexavar (sorafenib) in combination with Dacogen (decitabine) is included in guidelines for patients with relapsed or refractory acute myeloid leukemia harboring a FLT3 internal tandem duplication (FLT3-ITD) mutation (NCCN.org). detail...
FLT3 exon 14 ins acute myeloid leukemia predicted - sensitive FLT3 Inhibitor KW-2449 Preclinical - Patient cell culture Actionable In a preclinical study, FLT3-ITD mutations correlated with sensitivity to KW-2449 in patient-derived acute myeloid leukemia samples in an ex vivo assay (PMID: 30333627). 30333627
FLT3 exon 14 ins acute myeloid leukemia not applicable N/A Guideline Prognostic FLT3 internal tandem duplication (FLT3-ITD) mutations are associated with inferior prognosis in acute myeloid leukemia patients with normal karyotype (NCCN.org). detail...
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Quizartinib Preclinical - Cell culture Actionable In a preclinical study, Quizartinib (AC220) inhibited proliferation of several acute myeloid leukemia cell lines harboring different FLT3-ITD mutations in culture (PMID: 28895560). 28895560
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Quizartinib Phase I Actionable In a Phase I trial, acute myeloid leukemia (AML) pediatric patients harboring a FLT3-ITD mutation demonstrated a greater sensitivity to treatment with Quizartinib (AC220) when compared to AML patients with wild-type FLT3, resulting in three complete responses, four with stable disease, and a lower bone marrow blast cell count (PMID: 26920889). 26920889
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Quizartinib Phase II Actionable In a Phase II trial, Quizartinib (AC220) treatment resulted in a composite complete remission (CCR) in 56% (63/112; 3 complete remission (CR)) of FLT3-ITD-positive patients vs. 36% (16/44; 1 CR) of FLT3-ITD-negative patients with relapsed/refractory acute myeloid leukemia (AML) after first-line therapy, and CCR in 46% (62/136; 5 CR) of FLT3-ITD-positive vs. 30% (12/40; 1 CR) in FLT3-ITD-negative patients with relapsed/refractory AML after salvage chemotherapy or transplant (PMID: 29859851; NCT00989261). 29859851
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Quizartinib Preclinical - Cell culture Actionable In a preclinical study, Quizartinib (AC220) inhibited Flt3 phosphorylation and growth of acute myeloid leukemia cell lines harboring FLT3_OT exon 14 insertions (ITD) in culture (PMID: 31309543). 31309543
FLT3 exon 14 ins acute myeloid leukemia predicted - sensitive SHP099 Preclinical - Pdx Actionable In a preclinical study, treatment with SHP099 resulted in reduction of CD45-positive leukemic cells and decreased splenomegaly in a human primary tumor-derived xenograft model of acute myeloid leukemia harboring a FLT3-ITD mutation (PMID: 27362227). 27362227
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor Tandutinib Preclinical - Cell culture Actionable In a preclinical study, Tandutinib (CT53518) treatment inhibited viability of an acute myeloid leukemia cell line harboring FLT3 internal tandem duplication (ITD) in culture (PMID: 31751472). 31751472
FLT3 exon 14 ins myelodysplastic syndrome not applicable N/A Guideline Prognostic FLT3 internal tandem duplication (FLT3-ITD) mutations are associated with poor prognosis in patients with myelodysplastic syndrome (NCCN.org). detail...
FLT3 exon 14 ins hematologic cancer sensitive Palbociclib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing FLT3 ITD were sensitive to treatment with Ibrance (palbociclib), demonstrating inhibition of cell growth in culture (PMID: 30544932). 30544932
FLT3 exon 14 ins Advanced Solid Tumor predicted - sensitive FLT3 Inhibitor Cabozantinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing a FLT3-ITD mutation were sensitive to Cometriq (cabozantinib) in culture (PMID: 21926191). 21926191
FLT3 exon 14 ins acute myeloid leukemia predicted - sensitive Ibrutinib Preclinical - Patient cell culture Actionable In a preclinical study, FLT3-ITD mutations correlated with sensitivity to Imbruvica (ibrutinib) in patient-derived acute myeloid leukemia samples in an ex vivo assay (PMID: 30333627). 30333627
FLT3 exon 14 ins Advanced Solid Tumor sensitive FLT3 Inhibitor Crenolanib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing FLT3 internal tandem duplication (ITD) demonstrated sensitivity to Crenolanib treatment in culture (PMID: 31751472). 31751472
FLT3 exon 14 ins hematologic cancer sensitive FLT3 Inhibitor Midostaurin Preclinical - Cell culture Actionable In a preclinical study, Rydapt (midostaurin) inhibited growth of transformed hematologic cells expressing FLT3 exon 14 insertions (ITD) in culture (PMID: 31309543). 31309543
FLT3 exon 14 ins hematologic cancer sensitive FLT3 Inhibitor Sorafenib Preclinical - Cell culture Actionable In a preclinical study, Nexavar (sorafenib) inhibited growth of transformed hematologic cells expressing FLT3 exon 14 insertions (ITD) in culture (PMID: 31309543). 31309543
FLT3 exon 14 ins acute myeloid leukemia sensitive FLT3 Inhibitor GTP-14564 Preclinical - Cell culture Actionable In a preclinical study, acute myeloid leukemia cells harboring a FLT3 internal tandem duplication were sensitive to GTP-14564 treatment in culture, demonstrating growth inhibition (PMID: 12815052). 12815052
Clinical Trial Phase Therapies Title Recruitment Status
NCT01892371 Phase Ib/II Azacitidine + Quizartinib Cytarabine + Quizartinib Quizartinib With Azacitidine or Cytarabine in Treating Participants With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome Active, not recruiting
NCT03250338 Phase III Cytarabine + Filgrastim + Fludarabine + Idarubicin + Mitoxantrone Crenolanib + Cytarabine + Filgrastim + Fludarabine + Idarubicin + Mitoxantrone Study Investigating the Efficacy of Crenolanib With Chemotherapy vs Chemotherapy Alone in R/R FLT3 Mutated AML Recruiting
NCT03552029 Phase I DS-3032b + Quizartinib Milademetan Plus Quizartinib Combination Study in FLT3-ITD Mutant Acute Myeloid Leukemia (AML) Recruiting
NCT04209725 Phase II CPX-351 + Quizartinib A Study of CPX-351 (Vyxeos) With Quizartinib for the Treatment of FLT3-ITD Mutation-Positive Acute Myeloid Leukemia Recruiting
NCT03836209 Phase II Cytarabine + Daunorubicin + Gilteritinib Cytarabine + Daunorubicin + Midostaurin Cytarabine Midostaurin Randomized Trial of Gilteritinib vs Midostaurin in FLT3 Mutated Acute Myeloid Leukemia Recruiting
NCT03674411 Phase II Busulfan + Filgrastim + Fludarabine + Melphalan + MGTA-456 + Mycophenolate mofetil + Tacrolimus Cyclophosphamide + Filgrastim + Fludarabine + MGTA-456 + Mycophenolate mofetil + Tacrolimus Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy Recruiting
NCT02543879 Phase I FT-1101 Azacitidine + FT-1101 Study of a Novel BET Inhibitor FT-1101 in Patients With Relapsed or Refractory Hematologic Malignancies Completed
NCT03365661 Phase II ALT-803 QUILT-3.034: Non-Myeloablative TCRa/b Deplete Haplo HSCT With Post ALT-803 for AML Withdrawn
NCT02997202 Phase III Gilteritinib A Trial of the FMS-like Tyrosine Kinase 3 (FLT3) Inhibitor Gilteritinib Administered as Maintenance Therapy Following Allogeneic Transplant for Patients With FLT3/Internal Tandem Duplication (ITD) Acute Myeloid Leukemia (AML) Recruiting
NCT02722668 Phase II Cyclophosphamide + Fludarabine + Mycophenolate mofetil Sirolimus anti-thymocyte globulin UCB Transplant for Hematological DIseases Using a Non Myeloablative Prep Recruiting
NCT02927262 Phase III Gilteritinib A Study of ASP2215 (Gilteritinib), Administered as Maintenance Therapy Following Induction/Consolidation Therapy for Subjects With FMS-like Tyrosine Kinase 3 (FLT3/ITD) Acute Myeloid Leukemia (AML) in First Complete Remission Active, not recruiting
NCT04336982 Phase Ib/II Azacitidine + CC-90009 + Venetoclax CC-90009 + Gilteritinib A Safety and Efficacy Study of CC-90009 Combinations in Subjects With Acute Myeloid Leukemia Not yet recruiting
NCT04140487 Phase Ib/II Azacitidine + Gilteritinib + Venetoclax Azacitidine, Venetoclax, and Gilteritinib in Treating Patients With Recurrent/Refractory FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome Recruiting
NCT03513484 Phase I Azacitidine + Nintedanib Nintedanib and Azacitidine in Treating Participants With HOX Gene Overexpression Relapsed or Refractory Acute Myeloid Leukemia Recruiting
NCT04240002 Phase Ib/II Cytarabine + Filgrastim + Fludarabine + Gilteritinib A Study of Gilteritinib (ASP2215) Combined With Chemotherapy in Children, Adolescents and Young Adults With FMS-like Tyrosine Kinase 3 (FLT3)/Internal Tandem Duplication (ITD) Positive Relapsed or Refractory Acute Myeloid Leukemia (AML) Active, not recruiting
NCT02283177 Phase II Crenolanib + Cytarabine + Daunorubicin A Safety and Tolerability Study of Crenolanib in Combination With Chemotherapy in Newly Diagnosed Acute Myeloid Leukemia Patients With FLT3 Mutations Active, not recruiting
NCT02400255 Phase II Crenolanib Crenolanib Maintenance Following Allogeneic Stem Cell Transplantation in FLT3-positive Acute Myeloid Leukemia Patients Recruiting
NCT03793478 Phase Ib/II Etoposide Cytarabine + Fludarabine + Methotrexate + Prednisolone + Quizartinib Cytarabine + Daunorubicin + Fludarabine + Methotrexate + Prednisolone + Quizartinib Cytarabine + Daunorubicin + Fludarabine + Hydrocortisone + Methotrexate + Quizartinib Cytarabine + Fludarabine + Methotrexate + Prednisolone Quizartinib Safety and Effectiveness of Quizartinib in Children and Young Adults With Acute Myeloid Leukemia (AML), a Cancer of the Blood Recruiting
NCT03735875 Phase Ib/II Quizartinib + Venetoclax Venetoclax and Quizartinib in Treating Patients With FLT3-mutated Recurrent or Refractory Acute Myeloid Leukemia Recruiting
NCT02752035 Phase II Gilteritinib Azacitidine + Gilteritinib Azacitidine A Study of ASP2215 (Gilteritinib), Combination of ASP2215 Plus Azacitidine and Azacitidine Alone in the Treatment of Newly Diagnosed Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) Mutation in Patients Not Eligible for Intensive Induction Chemotherapy Recruiting
NCT02270463 Phase Ib/II tagraxofusp-erzs SL-401 as Consolidation Therapy in Patients With Adverse Risk Acute Myeloid Leukemia in First Complete Remission Completed
NCT02756962 Phase II Midostaurin Cytarabine Improving Risk Assessment of AML With a Precision Genomic Strategy to Assess Mutation Clearance Recruiting