ONC206 for Treatment of Newly Diagnosed, Recurrent Diffuse Midline Gliomas, and Other Recurrent Malignant CNS Tumors

Summary

This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas that is newly diagnosed or has come back (recurrent) or other recurrent primary malignant CNS tumors. ONC206 is a recently discovered compound that may stop cancer cells from growing. This drug has been shown in laboratory experiments to kill brain tumor cells by causing a so called "stress response" in tumor cells. This stress response causes cancer cells to die, but without affecting normal cells. ONC206 alone or in combination with radiation therapy may be effective in treating newly diagnosed or recurrent diffuse midline gliomas and other recurrent primary malignant CNS tumors.

Conditions

• Diffuse Midline Glioma (DMG)
• Glioblastoma
• Recurrent Ependymoma
• Recurrent Malignant Central Nervous System Neoplasm
• Spinal Cord Glioma
• World Health Organization (WHO) Grade III Glioma
• CNS Tumor
• Central Nervous System Tumor

Interventions

DRUG

ONC206

Given orally (PO)

RADIATION

Standard of Care Radiation Therapy

Undergo RT

PROCEDURE

Optional Proton (1H) MR spectroscopy (MRS)

Optional imaging procedure

Sponsors & Collaborators

• Jazz Pharmaceuticals

  collaborator INDUSTRY

• Mithil Prasad Foundation

  collaborator UNKNOWN

• Storm the Heavens Fund

  collaborator UNKNOWN

• The ChadTough Defeat DIPG Foundation

  collaborator UNKNOWN

• National Cancer Institute (NCI)

  collaborator NIH

• Dana-Farber Cancer Institute

  collaborator OTHER

• Sabine Mueller, MD, PhD

  lead OTHER

Principal Investigators

• Sabine Mueller, MD, PhD · University of California, San Francisco

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Min Age

2 Years

Max Age

21 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2021-08-23

Primary Completion

2027-05-31

Completion

2027-07-31

FDA Drug

Yes

Countries

• United States
• Netherlands
• Switzerland

Study Locations