A Phase I/Pilot Study Of Cpx-351 For Children, Adolescents And Young Adults With Recurrent Or Refractory Hematologic Malignancies.

10541Background: CPX-351, a liposomal formulation of cytarabine and daunorubicin in a 5:1 molar ratio, has demonstrated pre-clinical activity against AML and ALL cell lines and is currently under phase-III investigation in adults with AML and high risk features. A phase I trial evaluating this agent in children with relapsed or refractory hematologic malignancies was conducted. Methods: Children and young adults age 12 months to 21 years with relapsed or refractory leukemia were enrolled in the dose exploration phase of the study in which participants received a single course of CPX-351. Using a rolling 6 design, CPX-351 was administered once on days 1, 3, and 5. Enrollment was initiated at the recommended dose for study in adults (dose Level 1: 100 Units/m2/dose) with provisions to explore either a single dose escalation to 134 Units/m2/dose (dose level 2) or de-escalation to 67 Units/m2/dose. Results: Nine patients [median age 8 y (range 1-17y)] with AML (n = 6) or ALL (n = 3) were enrolled and evaluabl...