NCT03967093

A Study of BXQ-350 in Children and Young Adults With Relapsed Solid Tumors

A Phase 1 Safety Study of BXQ-350 Administered as a Single Agent by Intravenous Infusion in Children and Young Adults With Relapsed Solid Tumors, Including Recurrent Malignant Brain Tumors

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Bexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 1 Year – 30 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Neoplasms

Interventions

Type	Name	Description
DRUG	BXQ-350	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). BXQ-350 is administered by intravenous (IV) infusion over a minimum of six 28-day cycles.

Timeline

• Start date: 2019-04-15
• Primary completion: 2020-01-03
• Completion: 2020-01-17
• First posted: 2019-05-30
• Last updated: 2021-07-22

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study