Leukemia Clinical Trials

• Age ≥ 2 year to 21 years

• ECOG performance status of ≤ 2.

• Relapsed/refractory: AML60, Mixed phenotype acute leukemia61 (MPAL), ALL61, Acute leukemia of ambiguous lineage (ALAL)62 patients with KMT2A-r, NPM1-m, NUP98-r, or HOX pathway mutation as detailed in background section

• a. ≥5% leukemic blasts in the bone marrow:

• WBC must be below 25 K/µL at time of enrollment. Participants may receive cytoreduction prior to enrollment.

• Baseline ejection fraction must be \> 40%.

• Adequate hepatic function (direct bilirubin \< 1.5x upper limit of normal (ULN) unless increase is due to Gilbert's disease or leukemic involvement, and AST and/or ALT \< 5x ULN unless considered due to leukemic involvement, in which case direct bilirubin \< 3x ULN or AST and/or ALT \< 5x ULN will be considered eligible).

• Adequate renal function (creatinine clearance ≥ 30 mL/min) unless related to disease. (Justification on page 11)

• In the absence of rapidly proliferative disease, the interval from prior treatment to time of initiation will be at least 14 days for cytotoxic or non-cytotoxic (immunotherapy agent(s), or an interval of 5 half-lives of the prior therapy (whichever is shorter). Oral hydroxyurea and/or cytarabine (up to 2 g/m2) for patients with rapidly proliferative disease is allowed before the start of study therapy, as needed, for clinical benefit and after discussion with the PI. Concurrent therapy for central nervous system (CNS) prophylaxis or continuation of therapy for controlled CNS disease is permitted.

• Unless surgically or biologically sterile: Women of childbearing potential must agree to adequate methods of contraception during the study and at least 3 months for males, and 6 months for females, after the last treatment.