BXQ-350 is a novel anti-neoplastic therapeutic agent configured from two components: Saposin C (SapC), an expressed (human) lysosomal protein, and the phospholipid dioleoylphosphatidyl-serine (DOPS), a phospholipid located on cell membranes (clinical formulation BXQ-350). Given via intravenous (IV) infusion, data indicate that the agent exhibits the propensity to enter the body and brain, target cells in the tumor mass, and induce cell death.~The study is divided into 2 parts:~Part 1: Dose Escalation and Safety - Sequential cohorts of patients 1-30 years of age with relapsed solid tumors, including recurrent malignant brain tumors will be treated with escalating doses of BXQ-350 until the MTD is established, or in the absence of a Maximum Administered Dose, the highest planned dose level is reached.~Part 2: Safety and Preliminary Anti-tumor Activity - Patients will be enrolled into one of four cohorts and administered BXQ-350 at the established MTD or at the highest planned dose level. The four cohorts will consist of: recurrent ependymoma, recurrent malignant brain tumor, recurrent Diffuse Intrinsic Pontine Glioma (DIPG), and relapsed non-central nervous system (non-CNS) solid tumor.
This study will evaluate the safety of BXQ-350 and determine the maximum tolerated dose (MTD) in children and young adults with relapsed solid tumors, including recurrent malignant brain tumors. All patients will receive BXQ-350 by intravenous (IV) infusion. The study is divided into two parts: Part 1 will enroll patients at increasing dose levels of BXQ-350 in order to determine the MTD. Part 2 will use the MTD to further assess the safety of BXQ-350 as well as preliminary anti-tumor activity.