The primary objective of the trial is to determine the recommended phase 2 dose (RP2D) of ziftomenib in combination with chemotherapy (FLA) in children with relapsed or refractory KMT2A-r, NUP98-r, or NPM1-m acute leukemia based on safety and pharmacokinetics (PK).
Age: 0-21 years (and at least 5 kg body weight), with a minimum of 80% of participants under 18 years of age.
Diagnosis: KMT2A-r, NPM1-m, or NUP98-r acute leukemia in first or greater relapse or refractory to standard (re-) induction treatment (including HSCT). Please note that genetic alteration must be confirmed by the central laboratory, or the participant will discontinue protocol therapy.
Bone marrow relapse is defined as: a single bone marrow sample or bone biopsy showing ≥ 5% leukemic blasts by local morphology.
Participants with combined extramedullary and bone marrow relapse (defined as above), including central nervous system (CNS) disease, are eligible.
Participants who in the opinion of the investigator may not be able to comply with the trial requirements of the trial.
Participants with Down syndrome.
Participants with EMD are not eligible. EMD relapse is defined as biopsy proven extramedullary disease without bone marrow disease after documented CR following initial therapy.
Participants with isolated CNS relapse are not eligible, as well as symptomatic CNS3 disease.
Single group
Ziftomenib
DrugOral capsule
Cytarabine
DrugIntravenous (IV) infusion
Fludarabine
DrugIV infusion
Number of Participants Who Experience a Dose-limiting Toxicity (DLT)
Area Under the Plasma Concentration-time Curve (AUC) of Ziftomenib
Number of Participants Who Experience an Adverse Event (AE)
Including seriousness and relation to study therapy.
Number of AEs by Severity
As graded using CTCAE version 5.0.
Maximum Observed Plasma Concentration (Cmax) of Ziftomenib
Minimum Observed Plasma Concentration (Cmin) of Ziftomenib
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PedAL BCU, LLC
Lead sponsor
Kura Oncology
Collaborator
