A precision medicine randomized trial for patients with relapsed or refractory T-cell Acute Lymphoblastic Leukemia based on a functional approach.

The aim of the ALL-TARGET project is to validate the added-value of a precision medicine strategy for patients with relapsed or refractory T-cell acute lymphoblastic leukemia (T-ALL) by running a randomized prospective pragmatic trial in 6 European countries. T-ALL patients (older than 15 years) are treated in this phase 2 clinical trial with a combination of drugs versus standard of care. The targeted therapeutic options (TTOs) are selected by using a functional companion drug testing assay. By using combinations of venetoclax plus tofacitinib, venetoclax plus temsirolimus and erwinase and venetoclax plus 5-azacytidine, the three most frequently altered signaling pathways in T-ALL are targeted.

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