PedAL BCU, LLC
The primary objective of the study 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).
Inclusion Criteria: * 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. * Eligible participants also must fulfill one of the following conditions: 1. Bone marrow relapse is defined as: 1. A single bone marrow sample showing ≥ 5% leukemic bla…
Oral capsule
Intravenous (IV) infusion
IV infusion