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Gilteritinib + Azacitidine + Venetoclax
This triple combination targets acute myeloid leukemia by inhibiting FLT3 signaling (gilteritinib), promoting DNA methylation-dependent differentiation (azacitidine), and blocking anti-apoptotic BCL-2 proteins (venetoclax).
This triple combination targets acute myeloid leukemia by inhibiting FLT3 signaling (gilteritinib), promoting DNA methylation-dependent differentiation (azacitidine), and blocking anti-apoptotic BCL-2 proteins (venetoclax). Used for Acute myeloid leukemia (AML), including FLT3-mutated disease.
At a glance
| Generic name | Gilteritinib + Azacitidine + Venetoclax |
|---|---|
| Sponsor | Institute of Hematology & Blood Diseases Hospital, China |
| Drug class | FLT3 inhibitor + hypomethylating agent + BCL-2 inhibitor combination |
| Target | FLT3, DNA methyltransferase, BCL-2 |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
Gilteritinib is a selective FLT3 inhibitor that blocks proliferative signaling in FLT3-mutated AML. Azacitidine is a hypomethylating agent that restores normal gene expression and induces differentiation. Venetoclax is a BCL-2 inhibitor that promotes apoptosis by disrupting mitochondrial survival pathways. The combination targets multiple pathways implicated in AML pathogenesis and chemoresistance.
Approved indications
- Acute myeloid leukemia (AML), including FLT3-mutated disease
Common side effects
- Myelosuppression/neutropenia
- Thrombocytopenia
- Anemia
- Infection
- Nausea/vomiting
- Diarrhea
- Fatigue
Key clinical trials
- Venetoclax and HMA Treatment of Older and Unfit Adults With FLT3 Mutated Acute Myeloid Leukemia (AML) (A MyeloMATCH Treatment Trial) (PHASE2)
- MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial) (PHASE2)
- VAG Versus Standard Chemotherapy With FLT3 Inhibitor in Adult Patients With FLT3-Mutated AML (PHASE3)
- A Phase 1/2 Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia (Horizen-1) (PHASE1, PHASE2)
- IMPACT-AML: A Randomized Pragmatic Clinical Trial for Relapsed or Refractory Acute Myeloid Leukemia. (PHASE3)
- A Study of Gilteritinib, Venetoclax and Azacitidine as a Combined Treatment for People Newly Diagnosed With Acute Myeloid Leukemia (PHASE1, PHASE2)
- FLT3-ITD Targeted Therapy in Fit AML Patients (PHASE2, PHASE3)
- Gilteritinib Plus VA Followed By Consolidation Chemotherapy in Newly Diagnosed FLT3-ITD+ AML (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
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