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|Therapy Name||Busulfan + CPX-351 + Fludarabine + Melphalan + Midostaurin|
|Drug Name||Trade Name||Synonyms||Drug Classes||Drug Description|
|Busulfan||Busulfex||Busulphan||Busulfex (busulfan) inhibits DNA replication and RNA transcription, likely through induction of DNA alkylation (PMID: 27481448). Busulfex (busulfan) in combination with cyclophosphamide is FDA approved as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for patients with chronic myelogenous leukemia (FDA.gov).|
|CPX-351||Vyxeos||Liposome-encapsulated Daunorubicin-Cytarabine||Vyxeos (CPX-351) is a liposomal formulation of cytarabine and daunorubicin with anti-tumor activities (PMID: 25223583). Vyxeos (CPX-351) is FDA approved for use in patients with newly-diagnosed therapy-related acute myeloid leukemia (AML) or AML with myelodysplasia-related changes (FDA.gov).|
|Fludarabine||Fludara||FAMP|Fludarabine phosphate||Flurdara (fludarabine) is converted to 2-fluoro-ara-ATP intracellularly, which potentially inhibits DNA polymerase alpha, ribonucleotide reductase and DNA primase, leading to decreased DNA synthesis and reduced tumor growth (NCI Drug Dictionary)|
|Melphalan||Alkeran||Chemotherapy - Alkylating 16||Alkeran (melphalan) is an antineoplastic alkylating agent, which cross-links DNA and induces cell toxicity and is FDA approved for multiple myeloma and epithelial ovarian carcinoma (FDA.gov).|
|Midostaurin||Rydapt||PKC412|CGP 41251||CSF1R Inhibitor 27 FLT3 Inhibitor 61 KIT Inhibitor 55 PKC alpha Inhibitor 6 PKC beta Inhibitor 6 PKC Inhibitor (Pan) 11 VEGFR2 Inhibitor 36||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|
|Clinical Trial||Phase||Therapies||Title||Recruitment Status||Covered Countries||Other Countries|
|NCT04982354||Phase Ib/II||Busulfan + CPX-351 + Fludarabine + Melphalan + Midostaurin||Induction Therapy for Patients With FLT3 Mutated Acute Myeloid Leukemia||Recruiting||USA||0|