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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).
Cytarabine Cytosar-U Ara-C Cytosar-U (cytarabine) is a cytidine analog that, when incorporated into DNA, inhibits DNA synthesis and repair (PMID: 32524309).
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)
Gemtuzumab ozogamicin Mylotarg gemtuzumab|CDP-771|CMA-676|WAY-CMA-676 CD33 Antibody 11 Mylotarg (gemtuzumab ozogamicin) is an antibody-drug conjugate comprising an anti-CD33 antibody linked to the cytotoxic agent calicheamicin, which results in DNA damage in CD33-expressing tumor cells (PMID: 28607471). Mylotarg (gemtuzumab ozogamicin) is FDA approved for newly-diagnosed CD33-positive acute myeloid leukemia (AML) in adult and pediatric patients 1 month and older, and in relapsed or refractory CD33-positive AML in adult and pediatric patients 2 years and older (FDA.gov).
Venetoclax Venclexta ABT-199|RG7601|GDC-0199|ABT119|Venclyxto BCL2 inhibitor 25 Venclexta (venetoclax) is a BH3-mimetic that binds to and inhibits BCL2, resulting in increased tumor cell apoptosis (PMID: 26589495, PMID: 25048785). Venclexta (venetoclax) is FDA approved for use in patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), and in combination with chemotherapy in patients 75 years old or older with newly-diagnosed acute myeloid leukemia (FDA.gov).

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References

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Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
NCT05183035 Phase III Azacitidine + Cytarabine + Fludarabine + Gemtuzumab ozogamicin + Venetoclax Azacitidine + Cytarabine + Fludarabine + Gemtuzumab ozogamicin Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML) Recruiting USA 0


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