A Phase 1 Trial of the Menin Inhibitor Ziftomenib in Combination with Chemotherapy for Children with Relapsed/Refractory KMT2A-rearranged, NUP98-rearranged, or NPM1-mutant Acute Leukemia

Acute Myeloid Leukemia, Mixed Phenotype Acute Leukemia, Acute Lymphocytic Leukemia

The primary endpoint is determination of the RP2D based on: Dose-limiting toxicities (DLTs) assessed during the first cycle of treatment.; Pharmacokinetic parameters of ziftomenib: AUC.

Adverse events (AEs), as characterized by type, frequency, severity (as graded using CTCAE version 5.0), timing, seriousness, and relation to study therapy., AEs, as characterized by type, frequency, severity (as graded using CTCAE version, v5.0), timing, seriousness, and relation to study therapy during prolonged exposure, AEs, as characterized by type, frequency, severity (as graded using CTCAE version, v5.0), timing, seriousness, and relation to study therapy post HSCT, PK of ziftomenib in combination with FLA chemotherapy: PK parameters of ziftomenib including Cmax, Cmin, Tmax, AUC0-t, AUC0-∞, CL/F, Vz/F, and t½, The rate of those proceeding to subsequent hematopoietic stem cell transplantation as consolidation therapy is calculated as the number of patients who receive a hematopoietic stem cell infusion divided by the total number of patients enrolled and started treatment., The following parameters will be studied: Morphological ORR, defined as CR plus CRp and CRi (defined in Section 11.2.1); Flow-based overall response rate (ORR, defined in Section 11.2.1); Flow-based Measurable Residual Disease (MRD) negativity rate; Duration of response (DOR); Event free survival (EFS): 1 year after EOT of the last patient with ziftomenib; Overall survival (OS): 1 year after EOT of the last patient with ziftomenib; Cumulative incidence of relapse (CIR): 1 year after EOT

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