EZN-3042 Administered With Re-induction Chemotherapy in Children With Relapsed Acute Lymphoblastic Leukemia (ALL)

This is a phase I multi-site study of the new investigational agent EZN-3042, which is
highly effective at blocking survivin and inhibiting survivin protein expression. Survivin
plays pivotal roles in tumor formation by inhibiting cell death and regulating cell cycle
progression. The primary objective is to study EZN-3042 in children with relapsed acute
lymphoblastic leukemia (ALL). Patients will receive 2 doses of EZN-3042 prior to initiating
systemic therapy with vincristine, doxorubicin, prednisone and PEG-asparaginase. In
addition, blood and bone marrow specimens will be drawn to measure minimal residual disease
(MRD), pharmacokinetic levels of EZN-3042 and survivan expression. The study will follow a
standard 3+3 dose escalation design. We hypothesize that EZN-3042 will be safe, tolerable
and biologically active, when given both alone and in combination with standard re-induction
chemotherapy.