An open-label, phase 2 study, evaluating the efficacy of treatment with Gilteritinib in measurable residual disease eradication in patients with FLT3-ITD mutated acute myeloid leukemia

Conditions

Acute myeloid leukaemia

Brief summary

The rate of FLT3-ITD MRD negativity after 2 cycles of treatment with Gilteritinib.

Detailed description

Frequency and type of resistance mutations detected at relapse, with particular emphasis on the, RAS/MAPK pathway ( such as NRAS, KRAS, PTPN11, CBL, NF1)., Time to development of resistance (e.g., median duration of treatment before resistance is observed)., Phenotypic changes (via flow cytometry) associated with resistance. More specifically, phenotypes such as the CD34+CD38-CD123+ or the CD47, TIM-3, CD96 expression and the appearence/re-appearence of antigenic characterists will be evaluated., Baseline biomarker profiles (gene expression, mutation profile, proteomics) correlated with treatment response. More specifically, the presence of co-mutations in DNMT3A, NPM1, TET2, ASXL1, TP53 and the expression of antiapoptotic genes (such as the BCL2 and the MCL1) will be evaluated., Correlation between immune microenvironment features and response to treatment. More specifically, the expression of immunoregulatory molecules, such as the PD-L1, LAG-3, TIM-3 and the percentage of CD8+, T-regulatory and NK cells will be evaluated., Predictive value of specific genomic or transcriptomic signatures for MRD negativity.

Related papers

No related papers found yet.

Interventions

DRUGXospata 40 mg film-coated tablets

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
The rate of FLT3-ITD MRD negativity after 2 cycles of treatment with Gilteritinib.	—

Secondary

Measure	Time frame
Frequency and type of resistance mutations detected at relapse, with particular emphasis on the, RAS/MAPK pathway ( such as NRAS, KRAS, PTPN11, CBL, NF1)., Time to development of resistance (e.g., median duration of treatment before resistance is observed)., Phenotypic changes (via flow cytometry) associated with resistance. More specifically, phenotypes such as the CD34+CD38-CD123+ or the CD47, TIM-3, CD96 expression and the appearence/re-appearence of antigenic characterists will be evaluated., Baseline biomarker profiles (gene expression, mutation profile, proteomics) correlated with treatment response. More specifically, the presence of co-mutations in DNMT3A, NPM1, TET2, ASXL1, TP53 and the expression of antiapoptotic genes (such as the BCL2 and the MCL1) will be evaluated., Correlation between immune microenvironment features and response to treatment. More specifically, the expression of immunoregulatory molecules, such as the PD-L1, LAG-3, TIM-3 and the percentage of CD8+, T-	—

Measure

Time frame

Frequency and type of resistance mutations detected at relapse, with particular emphasis on the, RAS/MAPK pathway ( such as NRAS, KRAS, PTPN11, CBL, NF1)., Time to development of resistance (e.g., median duration of treatment before resistance is observed)., Phenotypic changes (via flow cytometry) associated with resistance. More specifically, phenotypes such as the CD34+CD38-CD123+ or the CD47, TIM-3, CD96 expression and the appearence/re-appearence of antigenic characterists will be evaluated., Baseline biomarker profiles (gene expression, mutation profile, proteomics) correlated with treatment response. More specifically, the presence of co-mutations in DNMT3A, NPM1, TET2, ASXL1, TP53 and the expression of antiapoptotic genes (such as the BCL2 and the MCL1) will be evaluated., Correlation between immune microenvironment features and response to treatment. More specifically, the expression of immunoregulatory molecules, such as the PD-L1, LAG-3, TIM-3 and the percentage of CD8+, T-

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Countries

Greece

Outcome results

None listed