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Therapy Name | Azacitidine + Midostaurin |
Synonyms | |
Therapy Description | |
Drug Name | Trade Name | Synonyms | Drug Classes | Drug Description |
---|---|---|---|---|
Azacitidine | Vidaza | azacytidine|CC-486|5-azacytidine|5-AC|U-18496|Onureg | DNMT inhibitor (Pan) 5 | Vidaza (azacitidine) is a cytidine analog that incorporates into DNA and RNA and binds to DNA methyltransferases (DNMTs), resulting in DNMT degradation and decreased DNA methylation, and leading to increased tumor cell death (PMID: 28159832, PMID: 28067760). Vidaza (azacitidine) is FDA-approved for use in patients with some subtypes of myelodysplastic syndrome and Onureg (azacitidine) is FDA approved for use in continued treatment of acute myeloid leukemia(FDA.gov). |
Midostaurin | Rydapt | PKC412|CGP 41251 | CSF1R Inhibitor 28 FLT3 Inhibitor 69 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). |
Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
---|---|---|---|---|---|---|---|
FLT3 mutant | acute myeloid leukemia | sensitive | Azacitidine + Midostaurin | Phase Ib/II | Actionable | In a Phase Ib/II trial, acute myeloid leukemia patients harboring a FLT3 mutation demonstrated an improved remission duration when treated with the combination therapy, Rydapt (midostaurin) and Vidaza (azacitidine) (PMID: 25530214). | 25530214 |
Clinical Trial | Phase | Therapies | Title | Recruitment Status | Covered Countries | Other Countries |
---|---|---|---|---|---|---|
NCT01093573 | Phase Ib/II | Azacitidine + Midostaurin | Midostaurin and Azacitidine in Treating Elderly Patients With Acute Myelogenous Leukemia | Completed | USA | 0 |
NCT03092674 | Phase II | Cytarabine + Decitabine Azacitidine + Midostaurin Decitabine Azacitidine + Nivolumab Azacitidine | Azacitidine With or Without Nivolumab or Midostaurin, or Decitabine and Cytarabine Alone in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome | Active, not recruiting | USA | 0 |