Therapy Detail
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| Therapy Name | Midostaurin |
| Synonyms | |
| Therapy Description |
Rydapt (midostaurin) is a multi-kinase inhibitor with activity against FLT3, KIT, PDGFRB, KDR (VEGFR2) and PKC, with higher selectivity for conventional PKC isoforms, which induces cell cycle arrest and apoptosis (PMID: 12124173, PMID: 23127174, PMID: 15914319). Rydapt (midostaurin) is FDA approved for FLT3-mutant AML in combination with chemotherapy, and is approved for use in aggressive systemic mastocytosis, systemic mastocytosis with associated hematological neoplasm, or mast cell leukemia as a single therapy (FDA.gov). |
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| Drug Name | Trade Name | Synonyms | Drug Classes | Drug Description |
|---|---|---|---|---|
| Midostaurin | Rydapt | PKC412|CGP 41251 | CSF1R Inhibitor 28 FLT3 Inhibitor 66 KIT Inhibitor 57 PKC alpha Inhibitor 6 PKC beta Inhibitor 6 PKC Inhibitor (Pan) 11 VEGFR2 Inhibitor 37 | Rydapt (midostaurin) is a multi-kinase inhibitor with activity against FLT3, KIT, PDGFRB, KDR (VEGFR2) and PKC, with higher selectivity for conventional PKC isoforms, which induces cell cycle arrest and apoptosis (PMID: 12124173, PMID: 23127174, PMID: 15914319). Rydapt (midostaurin) is FDA approved for FLT3-mutant AML in combination with chemotherapy, and is approved for use in aggressive systemic mastocytosis, systemic mastocytosis with associated hematological neoplasm, or mast cell leukemia as a single therapy (FDA.gov). |
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| Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|
| FLT3 exon 14 ins FLT3 F621L | acute myeloid leukemia | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, acute myeloid leukemia cells harboring a FLT3 internal tandem duplication (ITD) and expressing FLT3 F621L demonstrated sensitivity to Rydapt (midostaurin) in culture (Blood 2009 114:3776). | detail... |
| FLT3 V592G | Advanced Solid Tumor | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited FLT3-induced phosphorylation of ERK1/2 and reduced growth of transformed cells expressing FLT3 V592G in culture (PMID: 18068628). | 18068628 |
| CBL Y371H FLT3 pos | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited proliferation of cells expressing wild-type FLT3 and CBL Y371H in culture, and inhibited leukemic growth and prolonged survival in cell line xenograft models expressing FLT3 and CBL Y371H (PMID: 31943762). | 31943762 |
| FLT3 exon 14 ins FLT3 G697S | hematologic cancer | predicted - resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed hematologic cells expressing FLT3-ITD and FLT3 G697S were moderately resistant to Rydapt (midostaurin) treatment in culture (PMID: 35395091). | 35395091 |
| FLT3 exon19 | acute myeloid leukemia | sensitive | Midostaurin | Guideline | Actionable | Rydapt (midostaurin) is included in guidelines as maintenance therapy for patients with acute myeloid leukemia harboring a mutation in the FLT3 tyrosine kinase domain (exons 14-23) (NCCN.org). | detail... |
| FLT3 exon 14 ins IDH2 R140Q | acute myeloid leukemia | predicted - resistant | Midostaurin | Preclinical | Actionable | In a preclinical study, Rydapt (midostaurin) failed to inhibit growth of cells isolated from a transgenic mouse model of acute myeloid leukemia harboring a FLT3-ITD mutation and IDH2 R140Q in culture (PMID: 33268594). | 33268594 |
| FLT3 exon17 | acute myeloid leukemia | sensitive | Midostaurin | Guideline | Actionable | Rydapt (midostaurin) is included in guidelines as maintenance therapy for patients with acute myeloid leukemia harboring a mutation in the FLT3 tyrosine kinase domain (exons 14-23) (NCCN.org). | detail... |
| FLT3 D835A | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited Flt3 autophosphorylation and viability in transformed cells expressing FLT3 D835A in culture (PMID: 28077790). | 28077790 |
| FLT3 exon15 | acute myeloid leukemia | sensitive | Midostaurin | Guideline | Actionable | Rydapt (midostaurin) is included in guidelines as maintenance therapy for patients with acute myeloid leukemia harboring a mutation in the FLT3 tyrosine kinase domain (exons 14-23) (NCCN.org). | detail... |
| FGFR1 rearrange | myeloid and lymphoid neoplasms associated with FGFR1 abnormalities | sensitive | Midostaurin | Guideline | Actionable | Rydapt (midostaurin) is included in guidelines for patients with a myeloid/lymphoid neoplasm with eosinophilia harboring an FGFR1 rearrangement (NCCN.org). | detail... |
| FLT3 exon 14 ins FLT3 Y842H | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD and FLT3 Y842H were sensitive to Rydapt (midostaurin) treatment in culture (PMID: 34768286). | 34768286 |
| FLT3 I836T | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited Flt3 autophosphorylation and viability in transformed cells expressing FLT3 I836T in culture (PMID: 28077790). | 28077790 |
| FLT3 D835N | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited Flt3 autophosphorylation and viability in transformed cells expressing FLT3 D835N in culture (PMID: 28077790). | 28077790 |
| FLT3 R834Q | Advanced Solid Tumor | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited FLT3-induced phosphorylation of ERK1/2 and reduced growth of transformed cells expressing FLT3 R834Q in culture (PMID: 18068628). | 18068628 |
| FLT3 exon 14 ins FLT3 D835V | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD and FLT3 D835V were sensitive to Rydapt (midostaurin) treatment in culture (PMID: 34768286). | 34768286 |
| FLT3 exon 14 ins FLT3 D835V | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) treatment inhibited growth of transformed hematologic cells expressing FLT3-ITD and FLT3 D835V in culture (PMID: 35395091). | 35395091 |
| FLT3 exon 14 ins | acute myeloid leukemia | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, an acute myeloid leukemia cell line harboring a FLT3 internal tandem duplication (ITD) demonstrated sensitivity to Rydapt (midostaurin) treatment, but when co-cultured with human stromal cells, demonstrated resistance to Rydapt (midostaurin) treatment in culture (PMID: 31751472). | 31751472 |
| FLT3 exon 14 ins | acute myeloid leukemia | sensitive | 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 | acute myeloid leukemia | sensitive | Midostaurin | Guideline | Actionable | Rydapt (midostaurin) is included in guidelines as maintenance therapy for patients with acute myeloid leukemia harboring a FLT3 internal tandem duplication (FLT3-ITD) mutation (NCCN.org). | detail... |
| FLT3 exon14 | acute myeloid leukemia | sensitive | Midostaurin | Guideline | Actionable | Rydapt (midostaurin) is included in guidelines as maintenance therapy for patients with acute myeloid leukemia harboring a mutation in the FLT3 tyrosine kinase domain (exons 14-23) (NCCN.org). | detail... |
| KIT D816Y | mast cell neoplasm | predicted - sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited proliferation of mouse mastocytoma cells harboring KIT D814Y (corresponds to D816Y in human) in culture (PMID: 31085175). | 31085175 |
| FLT3 exon 14 ins FLT3 D839G | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD and FLT3 D839G were sensitive to Rydapt (midostaurin) treatment in culture (PMID: 34768286). | 34768286 |
| KIT exon 11 del | gastrointestinal stromal tumor | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited Kit phosphorylation and proliferation of gastrointestinal stromal tumor cells harboring KIT exon 11 deletion in culture (PMID: 31085175). | 31085175 |
| FLT3 exon 14 ins FLT3 Y842D | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited proliferation of transformed hematologic cells expressing FLT3-ITD with FLT3 Y842D in culture (PMID: 19318574). | 19318574 |
| FLT3 exon 14 ins FLT3 N676D | Advanced Solid Tumor | resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells over expressing FLT3 ITD and N676D were resistant to Rydapt (midostaurin) in culture (PMID: 23392356). | 23392356 |
| KIT S476I | mast-cell leukemia | predicted - sensitive | Midostaurin | Preclinical - Patient cell culture | Actionable | In a preclinical study, treatment with Rydapt (midostaurin) decreased proliferation of patient-derived neoplastic mast cells harboring KIT S476I in culture (PMID: 25209843). | 25209843 |
| FLT3 exon 14 ins FLT3 Y693N | hematologic cancer | predicted - resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed hematologic cells expressing FLT3-ITD and FLT3 Y693N were moderately resistant to Rydapt (midostaurin) treatment in culture (PMID: 35395091). | 35395091 |
| FLT3 I836S | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited Flt3 autophosphorylation and viability in transformed cells expressing FLT3 I836S in culture (PMID: 28077790). | 28077790 |
| KIT N655K KIT D816V | Advanced Solid Tumor | resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells co-expressing KIT D816V and KIT N655K demonstrated resistance to treatment with Rydapt (midostaurin) in culture compared to cells expressing KIT D816V alone (PMID: 31270078). | 31270078 |
| FLT3 exon 14 ins FLT3 A848P | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) treatment inhibited growth of cells expressing FLT3-ITD with FLT3 A848P in culture (PMID: 20520641). | 20520641 |
| FLT3 exon 14 ins FLT3 F691L | acute myeloid leukemia | decreased response | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, acute myeloid leukemia cells harboring a FLT3 internal tandem duplication and expressing FLT3 F691L demonstrated a decreased response to Rydapt (midostaurin) compared to treated cells harboring a FLT3 ITD and expressing FLT3 Y842C (Blood 2009 114:3776). | detail... |
| FLT3 exon 14 ins FLT3 N676K | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) treatment inhibited growth of transformed hematologic cells expressing FLT3-ITD and FLT3 N676K in culture (PMID: 35395091). | 35395091 |
| KIT V560G | mast cell neoplasm | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited proliferation of mast cells harboring KIT V560G in culture (PMID: 31085175). | 31085175 |
| FLT3 exon 14 ins FLT3 D835Y | acute myeloid leukemia | predicted - resistant | Midostaurin | Preclinical - Patient cell culture | Actionable | In a preclinical study, a patient-derived acute myeloid leukemia cell line harboring a FLT3 internal tandem duplication (ITD) and FLT3 D835Y demonstrated resistance to Rydapt (midostaurin) treatment in culture (PMID: 27908881). | 27908881 |
| FLT3 D835G | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited Flt3 autophosphorylation and viability in transformed cells expressing FLT3 D835G in culture (PMID: 28077790). | 28077790 |
| KIT V654A KIT D816V | Advanced Solid Tumor | resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells co-expressing KIT D816V and KIT V654A demonstrated resistance to treatment with Rydapt (midostaurin) in culture compared to cells expressing KIT D816V alone (PMID: 31270078). | 31270078 |
| FLT3 Q575del | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) decreased downstream signaling and proliferation in transformed cells expressing FLT3 Q575del in culture (PMID: 33914060). | 33914060 |
| FLT3 I836D FLT3 I836L | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited Flt3 autophosphorylation and viability in transformed cells expressing FLT3 I836D and I836L in culture (PMID: 28077790). | 28077790 |
| FLT3 exon 14 ins FLT3 K429E | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) treatment inhibited growth of transformed hematologic cells expressing FLT3-ITD and FLT3 K429E in culture (PMID: 35395091). | 35395091 |
| FLT3 wild-type | Advanced Solid Tumor | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited FLT3 phosphorylation in transformed cells expressing wild-type FLT3 in culture (PMID: 12124173). | 12124173 |
| FLT3 S451F | Advanced Solid Tumor | decreased response | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells expressing FLT3 S451F demonstrated decreased sensitivity to inhibition of FLT3 phosphorylation and growth by Rydapt (midostaurin) in culture, when compared to cells expressing other FLT3 activating mutations (PMID: 18068628). | 18068628 |
| FLT3 exon20 | acute myeloid leukemia | sensitive | Midostaurin | Guideline | Actionable | Rydapt (midostaurin) is included in guidelines as maintenance therapy for patients with acute myeloid leukemia harboring a mutation in the FLT3 tyrosine kinase domain (exons 14-23) (NCCN.org). | detail... |
| FLT3 F590_D593delinsLY | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited Flt3 and Stat5 phosphorylation and viability in cells expressing FLT3 F590_D593delinsLY in culture (PMID: 26012842). | 26012842 |
| FLT3 exon23 | acute myeloid leukemia | sensitive | Midostaurin | Guideline | Actionable | Rydapt (midostaurin) is included in guidelines as maintenance therapy for patients with acute myeloid leukemia harboring a mutation in the FLT3 tyrosine kinase domain (exons 14-23) (NCCN.org). | detail... |
| KIT exon 11 del KIT T670I | gastrointestinal stromal tumor | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited proliferation of gastrointestinal stromal tumor cells harboring both KIT exon 11 deletion and KIT T670I in culture (PMID: 31085175). | 31085175 |
| FLT3 exon 14 ins JAK3 V722I | hematologic cancer | resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed hematologic cells expressing a FLT3-ITD mutation and JAK3 V722I were resistant to treatment with Rydapt (midostaurin) in culture (PMID: 33149267). | 33149267 |
| FLT3 exon 14 ins | Advanced Solid Tumor | sensitive | 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 FLT3 A627T | hematologic cancer | predicted - resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells expressing a FLT3-ITD mutation with FLT3 A627T demonstrated resistance to Rydapt (midostaurin) in culture (PMID: 17620426). | 17620426 |
| FLT3 exon 14 ins FLT3 A627T | hematologic cancer | predicted - resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells expressing a FLT3-ITD mutation with FLT3 A627T demonstrated moderate resistance to treatment with Rydapt (midostaurin) in culture (PMID: 15374944). | 15374944 |
| FLT3 exon 14 ins FLT3 D835Y | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) treatment inhibited growth of transformed hematologic cells expressing FLT3-ITD and FLT3 D835Y in culture (PMID: 35395091). | 35395091 |
| FLT3 exon 14 ins FLT3 D835Y | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells expressing FLT3-ITD and FLT3 D835Y were sensitive to Rydapt (midostaurin) treatment in culture (PMID: 34768286). | 34768286 |
| FLT3 exon 14 ins FLT3 D698N | hematologic cancer | predicted - resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed hematologic cells expressing FLT3-ITD and FLT3 D698N were moderately resistant to Rydapt (midostaurin) treatment in culture (PMID: 35395091). | 35395091 |
| FLT3 I836del | cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited phosphorylation of FLT3, ERK, and STAT5, and growth of transformed cells expressing FLT3 I836del in culture (PMID: 17827387). | 17827387 |
| FLT3 E573del | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) decreased proliferation in transformed cells expressing FLT3 E573del in culture (PMID: 33914060). | 33914060 |
| FLT3 exon 15 ins | acute myeloid leukemia | sensitive | Midostaurin | Guideline | Actionable | Rydapt (midostaurin) is included in guidelines as maintenance therapy for patients with acute myeloid leukemia harboring a FLT3 internal tandem duplication (FLT3-ITD) mutation (NCCN.org). | detail... |
| CBL Q365_E366insSK FLT3 pos | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited proliferation of cells expressing wild-type FLT3 and CBL Q365_E366insSK in culture, and inhibited leukemic growth and prolonged survival in cell line xenograft models expressing FLT3 and CBL Q365_E366insSK (PMID: 31943762). | 31943762 |
| FLT3 exon 14 ins FLT3 F691L | hematologic cancer | resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) did not inhibit growth of transformed hematologic cells expressing both FLT3 exon 14 insertions (ITD) and FLT3 F691L in culture (PMID: 31309543). | 31309543 |
| FLT3 exon 14 ins FLT3 F691L | hematologic cancer | resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed hematologic cells expressing FLT3-ITD and FLT3 F691L were moderately resistant to Rydapt (midostaurin) treatment in culture (PMID: 35395091). | 35395091 |
| FLT3 exon 14 ins FLT3 F691L | hematologic cancer | resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed hematologic cells expressing FLT3-ITD and FLT3 F691L demonstrated resistance to treatment with Rydapt (midostaurin) in culture (PMID: 33780043). | 33780043 |
| FLT3 exon 14 ins FLT3 F691L FLT3 D835I | acute myeloid leukemia | predicted - resistant | Midostaurin | Preclinical - Patient cell culture | Actionable | In a preclinical study, a patient-derived acute myeloid leukemia cell line harboring a FLT3 internal tandem duplication (ITD) with FLT3 F691L and FLT3 D835I demonstrated resistance to Rydapt (midostaurin) treatment in culture (PMID: 27908881). | 27908881 |
| KIT D677N KIT D816V | Advanced Solid Tumor | resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells co-expressing KIT D816V and KIT D677N demonstrated resistance to treatment with Rydapt (midostaurin) in culture compared to cells expressing KIT D816V alone (PMID: 31270078). | 31270078 |
| KIT T670V KIT D816V | Advanced Solid Tumor | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells co-expressing KIT D816V and KIT T670V were sensitive to treatment with Rydapt (midostaurin) in culture, demonstrating decreased cell proliferation (PMID: 31270078). | 31270078 |
| FLT3 exon21 | acute myeloid leukemia | sensitive | Midostaurin | Guideline | Actionable | Rydapt (midostaurin) is included in guidelines as maintenance therapy for patients with acute myeloid leukemia harboring a mutation in the FLT3 tyrosine kinase domain (exons 14-23) (NCCN.org). | detail... |
| FLT3 exon18 | acute myeloid leukemia | sensitive | Midostaurin | Guideline | Actionable | Rydapt (midostaurin) is included in guidelines as maintenance therapy for patients with acute myeloid leukemia harboring a mutation in the FLT3 tyrosine kinase domain (exons 14-23) (NCCN.org). | detail... |
| KIT D816V | acute myeloid leukemia | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited Kit phosphorylation and growth of erythroleukemia cells expressing KIT D816V in culture (PMID: 31309543). | 31309543 |
| FLT3 E598_Y599del | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited Flt3 and Stat5 phosphorylation and viability in cells expressing FLT3 E598_Y599del in culture (PMID: 26012842). | 26012842 |
| KIT N822K | acute myeloid leukemia | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited proliferation of acute myeloid leukemia cells harboring KIT N822K in culture (PMID: 31085175). | 31085175 |
| FLT3 exon 14 ins FLT3 D835E | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) treatment inhibited growth of transformed hematologic cells expressing FLT3-ITD and FLT3 D835E in culture (PMID: 35395091). | 35395091 |
| FLT3 exon 14 ins FLT3 D835G | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) treatment inhibited growth of transformed hematologic cells expressing FLT3-ITD and FLT3 D835G in culture (PMID: 35395091). | 35395091 |
| FLT3 rearrange | myeloid neoplasm | sensitive | Midostaurin | Guideline | Actionable | Rydapt (midostaurin) is included in guidelines for patients with a myeloid/lymphoid neoplasm with eosinophilia harboring a FLT3 rearrangement (NCCN.org). | detail... |
| FLT3 exon 14 ins FLT3 G697R | hematologic cancer | resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed hematologic cells expressing a FLT3-ITD mutation and FLT3 G697R were resistant to Rydapt (midostaurin) in culture (PMID: 15374944). | 15374944 |
| CBL Y371H FLT3 wild-type | hematologic cancer | sensitive | Midostaurin | Preclinical | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited growth of transformed hematologic cells expressing CBL Y371H and wild-type FLT3 in culture, reduced leukemia burden and prolonged survival in animal models (PMID: 31309543). | 31309543 |
| FLT3 exon 14 ins FLT3 Y693C | hematologic cancer | predicted - resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed hematologic cells expressing FLT3-ITD and FLT3 Y693C were moderately resistant to Rydapt (midostaurin) treatment in culture (PMID: 35395091). | 35395091 |
| FLT3 exon 14 ins FLT3 D835A | hematologic cancer | predicted - sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) treatment inhibited growth of transformed hematologic cells expressing FLT3-ITD and FLT3 D835A in culture (PMID: 35395091). | 35395091 |
| CBL Y371del FLT3 pos | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited proliferation of cells expressing wild-type FLT3 and CBL Y371del in culture (PMID: 31943762). | 31943762 |
| KIT T670I KIT D816V | Advanced Solid Tumor | decreased response | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells co-expressing KIT D816V and KIT T670I demonstrated a decreased response compared to cells expressing KIT D816V alone when treated with Rydapt (midostaurin) in culture (PMID: 31270078). | 31270078 |
| FLT3 exon 14 ins FLT3 Y842C | acute myeloid leukemia | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, acute myeloid leukemia cells harboring a FLT3 internal tandem duplication and expressing FLT3 Y842C demonstrated sensitivity to Rydapt (midostaurin) in culture (Blood 2009 114:3776). | detail... |
| FLT3 D835Y | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited growth of cells expressing FLT3 D835Y in culture (PMID: 38049555). | 38049555 |
| FLT3 D835Y | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited growth of transformed hematologic cells expressing FLT3 D835Y in culture (PMID: 31309543). | 31309543 |
| FLT3 exon 14 ins FLT3 D835N | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) treatment inhibited growth of transformed hematologic cells expressing FLT3-ITD and FLT3 D835N in culture (PMID: 35395091). | 35395091 |
| FLT3 exon 14 ins FLT3 D835F | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) treatment inhibited growth of transformed hematologic cells expressing FLT3-ITD and FLT3 D835F in culture (PMID: 35395091). | 35395091 |
| FLT3 D835Y | Advanced Solid Tumor | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with Rydapt (midostaurin) inhibited FLT3 phosphorylation and resulted in decreased proliferation and viability of transformed cells expressing FLT3 D835Y in culture (PMID: 12124173). | 12124173 |
| FLT3 Y572C | Advanced Solid Tumor | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited FLT3 phosphorylation of ERK1/2 and reduced cell growth in transformed cells expressing FLT3 Y572C in culture (PMID: 18068628). | 18068628 |
| FLT3 exon 14 ins FLT3 N676T | Advanced Solid Tumor | resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells co-expressing FLT3 ITD and FLT3 N676T were resistant to treatment with Rydapt (midostaurin) in culture (Blood (2019) 134 (Supplement_1): 2672). | detail... |
| FLT3 Y842C | acute myeloid leukemia | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) induced cell death and inhibited FLT3 activation and downstream STAT5 activation in primary acute myeloid leukemic blasts harboring FLT3 Y842C in culture (PMID: 15345593). | 15345593 |
| KIT T670A KIT D816V | Advanced Solid Tumor | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells co-expressing KIT D816V and KIT T670A were sensitive to treatment with Rydapt (midostaurin) in culture, demonstrating decreased cell proliferation (PMID: 31270078). | 31270078 |
| FLT3 S574del | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) decreased proliferation in transformed cells expressing FLT3 S574del in culture (PMID: 33914060). | 33914060 |
| FLT3 D835K FLT3 D835L | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited Flt3 autophosphorylation and viability in transformed cells expressing FLT3 D835L and D835K in culture (PMID: 28077790). | 28077790 |
| KIT V560G KIT D816V | mast cell neoplasm | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited proliferation of mast cells harboring KIT D816V and KIT V560G in culture (PMID: 31085175). | 31085175 |
| FLT3 exon 14 ins FLT3 D835H | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) treatment inhibited growth of transformed hematologic cells expressing FLT3-ITD and FLT3 D835H in culture (PMID: 35395091). | 35395091 |
| FLT3 G846D | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited growth of cells expressing FLT3 G846D in culture (PMID: 38049555). | 38049555 |
| FLT3 exon22 | acute myeloid leukemia | sensitive | Midostaurin | Guideline | Actionable | Rydapt (midostaurin) is included in guidelines as maintenance therapy for patients with acute myeloid leukemia harboring a mutation in the FLT3 tyrosine kinase domain (exons 14-23) (NCCN.org). | detail... |
| FLT3 exon 14 ins FLT3 D835I | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) treatment inhibited growth of transformed hematologic cells expressing FLT3-ITD and FLT3 D835I in culture (PMID: 35395091). | 35395091 |
| KIT D816V | Advanced Solid Tumor | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells expressing KIT D816V were sensitive to treatment with Rydapt (midostaurin) in culture, demonstrating decreased cell proliferation (PMID: 31270078). | 31270078 |
| FLT3 exon 14 ins FLT3 F691I | Advanced Solid Tumor | resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells over expressing FLT3 ITD and F691I were resistant to Rydapt (midostaurin) in culture (PMID: 23392356). | 23392356 |
| FLT3 exon 14 ins FLT3 N676K | acute myeloid leukemia | predicted - resistant | Midostaurin | Case Reports/Case Series | Actionable | In a clinical case study, a patient with acute myeloid leukemia harboring a FLT3-ITD mutation progressed on Rydapt (midostaurin) after 288 days and was found to have acquired a FLT3 N676K mutation, and resistance was also confirmed in patient-derived cells in culture (PMID: 16150941). | 16150941 |
| KIT Y672C KIT D816V | Advanced Solid Tumor | decreased response | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed cells co-expressing KIT D816V and KIT Y672C demonstrated a decreased response compared to cells expressing KIT D816V alone when treated with Rydapt (midostaurin) in culture (PMID: 31270078). | 31270078 |
| FLT3 exon 14 ins JAK2 V617F | hematologic cancer | resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed hematologic cells expressing a FLT3-ITD mutation and JAK2 V617F were resistant to treatment with Rydapt (midostaurin) in culture (PMID: 33149267). | 33149267 |
| FLT3 exon 14 ins FLT3 A627P | hematologic cancer | resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed hematologic cells co-expressing FLT3 ITD and FLT3 A627P were resistant to treatment with Rydapt (midostaurin) in culture (PMID: 22858906). | 22858906 |
| FLT3 exon 14 ins FLT3 D835del | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) treatment inhibited growth of transformed hematologic cells expressing FLT3-ITD and FLT3 D835del in culture (PMID: 35395091). | 35395091 |
| FLT3 exon 14 ins JAK3 R953* | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited growth of transformed hematologic cells expressing a FLT3-ITD mutation and JAK3 R953* in culture (PMID: 33149267). | 33149267 |
| FLT3 exon 14 ins FLT3 N701K | hematologic cancer | resistant | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, transformed hematologic cells expressing FLT3-ITD and FLT3 N701K demonstrated resistance to treatment with Rydapt (midostaurin) in culture (PMID: 33780043). | 33780043 |
| KIT D816V | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited Kit phosphorylation and growth of transformed hematologic cells expressing KIT D816V in culture (PMID: 31309543). | 31309543 |
| CBL Q365_E366insSK FLT3 wild-type | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited growth of transformed hematologic cells expressing CBL Q365_E366insSK and wild-type FLT3 in culture (PMID: 31309543). | 31309543 |
| KIT K509I | systemic mastocytosis | predicted - sensitive | Midostaurin | Preclinical - Patient cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) treatment inhibited growth but did not induce apoptosis in primary bone marrow mononuclear cells derived from patients with aggressive systemic mastocytosis harboring KIT K509I in culture (PMID: 25139846). | 25139846 |
| KIT exon 11 del KIT D816E | gastrointestinal stromal tumor | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) inhibited proliferation of gastrointestinal stromal tumor cells harboring both KIT exon 11 deletion and KIT D816E in culture (PMID: 31085175). | 31085175 |
| FLT3 exon 14 ins | hematologic cancer | sensitive | 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 | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) treatment inhibited growth of transformed hematologic cells expressing a FLT3-ITD mutation in culture (PMID: 35395091). | 35395091 |
| FLT3 Y572del | hematologic cancer | sensitive | Midostaurin | Preclinical - Cell culture | Actionable | In a preclinical study, Rydapt (midostaurin) decreased proliferation in transformed cells expressing FLT3 Y572del in culture (PMID: 33914060). | 33914060 |
| FLT3 exon16 | acute myeloid leukemia | sensitive | Midostaurin | Guideline | Actionable | Rydapt (midostaurin) is included in guidelines as maintenance therapy for patients with acute myeloid leukemia harboring a mutation in the FLT3 tyrosine kinase domain (exons 14-23) (NCCN.org). | detail... |
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| Clinical Trial | Phase | Therapies | Title | Recruitment Status | Covered Countries | Other Countries |
|---|---|---|---|---|---|---|
| NCT02624570 | Phase I | Midostaurin | Midostaurin Access Program for Newly Diagnosed FLT3 (ITD or TKD) Mutated AML Adult Patients Eligible for Standard Induction and Consolidation Chemotherapy. | No longer available | USA | 0 |
| NCT03114228 | Expanded access | Midostaurin | An Expanded Treatment Protocol (ETP) of Midostaurin (PKC412) in Patients 18 Years of Age or Older With Newly-diagnosed FLT3-mutated Acute Myeloid Leukemia (AML) | No longer available | CAN | 0 |
| NCT00866281 | Phase Ib/II | Midostaurin | A Study of the Safety and Preliminary Efficacy of Oral Midostaurin (PKC412) in Relapsed or Refractory Pediatric Leukemia | Terminated | USA | NLD | ITA | FRA | 1 |
| NCT01883362 | Phase II | Midostaurin | Standard of Care +/- Midostaurin to Prevent Relapse Post Stem Cell Transplant in Patients With FLT3-ITD Mutated AML | Completed | USA | CAN | 0 |
| NCT04027309 | Phase III | Midostaurin Gilteritinib | A Study of Gilteritinib Versus Midostaurin in Combination With Induction and Consolidation Therapy Followed by One-year Maintenance in Patients With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndromes With Excess Blasts-2 With FLT3 Mutations Eligible for Intensive Chemotherapy (HOVON 156 AML) | Active, not recruiting | NLD | FRA | ESP | DEU | BEL | AUT | AUS | 7 |
| NCT02756962 | Phase II | Midostaurin Cytarabine | Improving Risk Assessment of AML With a Precision Genomic Strategy to Assess Mutation Clearance | Active, not recruiting | USA | 0 |
| NCT02115295 | Phase II | Cladribine + Cytarabine + Idarubicin Midostaurin | Cladribine Plus Idarubicin Plus Cytarabine (ARAC) in Patients With Acute Myeloid Leukemia (AML), High Risk Myelodysplastic Syndrome (HR MDS) or Myeloid Blast Phase of Chronic Myeloid Leukemia (CML) | Recruiting | USA | 0 |
| NCT02723435 | Phase II | Midostaurin | Midostaurin in Treating Older Patients With Mutated Acute Myeloid Leukemia Post-Transplant | Withdrawn | 0 | |
| NCT01282502 | Phase I | Midostaurin | Midostaurin (PKC412) for Locally Advanced Rectal Cancer | Completed | USA | 0 |
| NCT03591510 | Phase II | Cytarabine Cytarabine + Etoposide Cytarabine + Mitoxantrone Cytarabine + Fludarabine + Idarubicin Cytarabine + Daunorubicin + Fludarabine Cytarabine + Daunoxome + Fludarabine Midostaurin | A Global Study of Midostaurin in Combination With Chemotherapy to Evaluate Safety, Efficacy and Pharmacokinetics in Newly Diagnosed Pediatric Patients With FLT3 Mutated AML | Recruiting | USA | ITA | DEU | AUT | 9 |
| NCT03900949 | Phase I | Midostaurin Cytarabine Cytarabine + Gemtuzumab ozogamicin Cytarabine + Daunorubicin + Gemtuzumab ozogamicin | Gentuzumab Ozogamicin and Midostaurin Combination With Standard Cytarabine and Danunorubi Midostaurin as a Novel Approach to Treating Patients With Newly Diagnosed FLT-3 Mutated Acute Myeloid Leukemia | Recruiting | USA | 0 |
| NCT03836209 | Phase II | Cytarabine + Daunorubicin + Gilteritinib Cytarabine + Daunorubicin + Midostaurin Cytarabine Midostaurin | Gilteritinib vs Midostaurin in FLT3 Mutated Acute Myeloid Leukemia | Active, not recruiting | USA | 0 |
| NCT03686345 | Phase II | Midostaurin | Midostaurin Associated With Standard Chemotherapy in Patients With Core-binding Factor Leukemia (AML FLT3) | Recruiting | ITA | 0 |
CKB BOOST