Phase I Study of APX005M in Pediatric Central Nervous System Tumors

Summary

This phase I trial studies the side effects and best dose of APX005M in treating younger patients with primary malignant central nervous system tumor that is growing, spreading, or getting worse (progressive), or newly diagnosed diffuse intrinsic pontine glioma. APX005M can trigger activation of B cells, monocytes, and dendritic cells and stimulate cytokine release from lymphocytes and monocytes. APX005M can mediate a direct cytotoxic effect on CD40+ tumor cells.

Conditions

• Glioblastoma Multiforme
• High-grade Astrocytoma Not Otherwise Specified (NOS)
• CNS Primary Tumor, Not Otherwise Specified (NOS)
• Ependymoma, Not Otherwise Specified (NOS)
• Diffuse Intrinsic Pontine Gliomas (DIPG)
• Medulloblastoma

Interventions

BIOLOGICAL

APX005M treatment for recurrent or refractory primary malignant CNS tumor patients

APX005M dosing will begin at 0.1 mg/kg, the APX005M dose may be increased (0.3, 0.45, 0.6 mg/kg) or decreased (0.03 mg/kg) in subsequent cohorts until the maximum tolerated dose (MTD) is reached or until dose level 3 (0.6 mg/kg) is complete without the MTD being defined. APX005M will be administered at the assigned dose level every 21 days (3 weeks). Patients may continue to receive APX005M for 36 courses (approximately 2 years) or until disease progression, unacceptable toxicity or death, whichever occurs first.

BIOLOGICAL

APX005M treatment for newly diagnosed DIPG patients

The starting dose of APX005M for the DIPG patients will be one dose level below the recommended phase II dose (RP2D) determined in Stratum 1 patients. The dose may be decreased or increased to the RP2D established in Stratum 1. APX005M will be administered at the assigned dose level every 21 days (3 weeks). Patients may continue to receive APX005M for 36 courses (approximately 2 years) or until disease progression, unacceptable toxicity or death, whichever occurs first.

Sponsors & Collaborators

• American Lebanese Syrian Associated Charities

  collaborator OTHER

• Pyxis Oncology, Inc

  collaborator INDUSTRY

• Solving Kids' Cancer

  collaborator OTHER

• Ty Louis Campbell Foundation

  collaborator OTHER

• A Kids' Brain Tumor Cure Foundation

  collaborator OTHER

• National Cancer Institute (NCI)

  collaborator NIH

• Pediatric Brain Tumor Consortium

  lead NETWORK

Principal Investigators

• Ira Dunkel · Memorial Sloan Kettering Cancer Center

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

SEQUENTIAL

Eligibility

Min Age

1 Year

Max Age

21 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2018-03-01

Primary Completion

2023-09-30

Completion

2026-06-30

FDA Drug

Yes

Countries

• United States

Study Locations