NCT04771897

A Study of BXQ-350 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG) or Diffuse Midline Glioma (DMG)

A Phase 1 Open Label, Multi-Center Study to Evaluate the Safety and Tolerability of BXQ-350 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG) and Diffuse Midline Glioma (DMG)

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 10 (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 with newly diagnosed DIPG or DMG. All patients will receive BXQ-350 by intravenous (IV) infusion and radiation therapy. 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 enroll patients requiring a biopsy in order to assess BXQ-350 concentrations in the biopsied tumor.

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 cell membrane phospholipid (clinical formulation BXQ-350). Given via intravenous IV, data indicate that the agent exhibits the propensity to enter the body and brain, target cells in the tumor mass, and induce cell death. DIPG and DMG present a treatment dilemma due to resistance to standard therapy (radiotherapy) and aggressive clinical course. The use of BXQ-350 provides a novel approach to the treatment of cancer through interaction with the cancer cell membrane. This drug appears to be well tolerated in previous clinical trials, and in combination with the standard of care radiation therapy, may help improve overall survival in these patients. The study is divided into 2 parts: Part 1: Dose Escalation and Safety - Sequential cohorts of patients 1-30 years of age with newly diagnosed DIPG or DMG 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: BXQ-350 Tumor and Plasma Concentrations - Patients undergoing neurosurgical biopsy prior to receiving radiation therapy will be enrolled and receive BXQ-350 at the MTD determined in Part 1, or at the highest planned dose level, and radiation therapy. Excised tumor tissue will be evaluated for SapC levels and pharmacodynamic effects. This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.)

Conditions

• Diffuse Intrinsic Pontine Glioma
• Diffuse Midline Glioma, H3 K27M-Mutant

Interventions

Type	Name	Description
DRUG	BXQ-350 - Part 1 Dose Escalation: Safety and Tolerance	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). During Part 1, BXQ-350 will be administered by intravenous (IV) infusion over twelve months.
DRUG	BXQ-350 - Part 2 BXQ-350 Tumor and Plasma Concentrations	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). During Part 2, BXQ-350 will be administered by intravenous (IV) infusion over twelve months.

Timeline

• Start date: 2021-05-24
• Primary completion: 2024-10-04
• Completion: 2024-10-04
• First posted: 2021-02-25
• Last updated: 2024-12-20

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study